The National Cancer Institute

The Nation’s

Investment in

Cancer Research

A Plan and Budget Proposal

for Fiscal Year 2002

Prepared by the Director

National Cancer Institute 

National Institutes of Health
ON THE CUTTING EDGE

NCI’s Intramural Program will soon 

make tissue arrays on single slides 

available to researchers who need to

study large numbers of tissue samples

side by side. See page 17.

EXTRAORDINARY OPPORTUNITIES FOR INVESTMENT

New, intensive efforts and increased resources in six research areas offer the potential to yield profound insights that could 

lead to vast improvements in our ability to prevent, detect, diagnose, and treat cancer. Descriptions of these opportunities and 

our specific plans and resource requirements for addressing them begin on page 63. 

HIGHLIGHTS OF PROGRESS

Every day, our nationwide programs for supporting cancer research yield new 

discoveries, new prevention and treatment approaches, and greater hope for people

with cancer and those at risk. See page 10 for highlights of recent scientific and

technological progress.

FISCAL YEAR 2002 BUDGET REQUEST

A core budget will sustain our established research programs. Additional funding 

will allow us to continue building a research infrastructure for the future (“NCI’s 

challenge”) and to more rapidly expand on past discovery by taking advantage of 

“extraordinary opportunities.” See page 9 for a summary of our FY 2002 budget request.

SPOTLIGHTS ON RESEARCH

Exciting progress has been made in harnessing the immune system to prevent and

treat cancer, in developing new detection tools, and in using new technologies to

improve understanding of specific cancers. Turn to pages 37, 76, and 82 to read 

special spotlights on these advances. 

HOW IT ALL COMES TOGETHER

The efforts we describe in this plan and budget proposal all come together to 

make a difference for patients, families, and physicians struggling to find better

answers to perplexing questions about cancer. See page 100 to learn what this 

means for lung cancer, as an example.

NCI’S CHALLENGE: BUILDING OUR

CAPACITY FOR FUTURE DISCOVERY 

To ensure continued progress in the

future, we need to focus on several

aspects of our research infrastructure

resources, technologies, organization, and

personnel. For descriptions, plans, and

funding requirements in each of these

essential areas, go to page 24.

PLANNING NATIONAL AGENDAS IN

DISEASE-SPECIFIC RESEARCH

Progress Review Groups, a unique 

mechanism through which we work with 

the research and advocacy communities 

to chart the course of research for 

specific cancers, have become an integral

component of our planning process. 

Learn more starting on page 96.

HOW WE WORK

NCI conducts, coordinates, and funds

cancer research and provides vision 

and leadership for the cancer research

community in several ways. We describe

our activities and core programs 

beginning on page 14.

PEOPLE’S STORIES

Dealing with cancer, and the threat of

cancer, is an intensely personal journey

filled with uncertainties, fears, and

hopes. On pages 22 and 61, two 

individuals share their stories. 

What’s Inside

Each year, as mandated by the National Cancer Act of 1971 (P.L. 92-218), the National Cancer Institute (NCI) prepares a

plan for building on research successes, supporting the cancer research workforce with the technologies and resources it needs,

and ensuring that research discoveries are applied to improve human health. This annual plan and budget proposal is 

provided directly to the President of the United States for formulating the budget request to Congress. This document 

also is used by NCI staff; the researcher community; professional organizations; advisory groups; cancer information, 

education, and advocacy organizations; and public and private policy makers. It is our hope that this document will 

inspire all who read it to join the fight against cancer. Here’s what you will find inside:

OUR ROLE IN CANCER RESEARCH

NCI’s goal is to stimulate and support 

scientific discovery and its application 

to achieve a future when all cancers are

uncommon and easily treatable. Turn 

to page 8 to learn about our approach 

to providing vision and leadership for

accomplishing this goal.
Director’s Message 1

Director’s 

Message

A

s the 21st century dawns, scientific discovery

is occurring at a pace that would astound our

forebears. Nearly every day some new

advance, some new insight, brings us closer to

answering one of the many questions that have

long confounded scientists. Nowhere is the excitement generated by this new atmosphere of discovery more evident than in cancer research. The pace

of discovery is continuing to accelerate, fueled by

progress in three evolving areas:

The explosion of information on the fundamental

nature of cancer. Over the past decade, scientists

have gained stunning new insights into the fundamental mechanisms of the cancer cell. These

remarkable discoveries hold promise for improving

our ability to target prevention measures, to detect

cancers early while they are most curable, and to

develop treatments that are individually targeted

for maximum results with the fewest side effects. 

The development of new tools and approaches for

conducting clinical research to test these insights in

patients. Novel imaging technologies allow us to view

activity at the molecular level. High-throughput

tests that enable us to rapidly scan a person’s

genome for cancer-related abnormalities may soon

allow us to more accurately assess individual risk or

detect cancer earlier. Researchers and clinicians are

now using workstations with high resolution medical image displays for real-time medical consultation from multiple locations around the world.

Information management tools allow investigators

to collect and analyze patient data in a more efficient fashion. All of these advances are enabling us

to answer important clinical questions more rapidly,

accurately, and effectively.

The growing sophistication in behavioral and population research that allows us to understand and address

the burden of cancer. By identifying the forces and

factors that determine the burden of cancer – studying patterns of cancer, searching for causes, more

fully comprehending the behaviors of individuals

and communities, and understanding the impact of

public health and medical systems – we are beginning to create the tools to effectively reduce that

burden. We are gaining in our understanding of

why some groups of Americans are disproportionately affected by cancer. We are using tools that

give us clear and precise information on cancer

trends among specific population groups and help

us formulate hypotheses about why the trends

occur. And we are better able to target our communications about cancer because we more fully

understand our audiences and their needs.

Underlying these three areas of progress are several key changes in the overall research enterprise.

First, we are witnessing and facilitating a fundamental change in the way scientific discovery is

accomplished. Advances in our understanding of

cancer at the molecular level, coupled with technologies drawn from the chemical, physical, and

material sciences are enabling us to approach our

research with a greater sense of predictability and

precision. We are now able to move from empiric,

fact-finding studies to “designed” research in which

we identify a range of compelling options for prevention, detection, diagnosis, and treatment; verify

their efficacy using models and electronic tools; and

validate them through extensive testing and clinical trials. This fundamental change in how we

approach research maximizes the effectiveness of all

areas of scientific discovery, from broad-based science with potential to benefit all types of cancer to

“disease-based” studies that focus on specific types

of cancer. 

Second, we are seeing an increasing amount of

research that brings together and melds different
2 The Nation’s Investment in Cancer Research

scientific perspectives and the tools of multiple disciplines. For example, by integrating the work of

epidemiologists, geneticists, and population scientists, we hope to better understand the potential

interplay among inherited susceptibility, lifestyle,

and exposure to environmental pollutants and

infectious agents in cancer causation. By combining

the efforts of engineering technology and biomedical science, we have been able to devise highly

sophisticated imaging tools for detecting, diagnosing,

and even aiding in the treatment of cancer patients.

Third, our ability to do research on a large scale

and with greater complexity has increased significantly. For instance, the development of common

forms, terminology, and reporting requirements for

use in cancer clinical trials is increasing the speed,

efficiency, and accuracy of results reporting and

analysis. We are also able to store and disseminate

data on thousands of tumor tissue samples, making

the data available to researchers around the world.

These areas of progress and the changes that

underlie them are driving an evolution and

rethinking of NCI’s activities. Over the past few

years, literally dozens of opportunities have been

created – for example, the Phased Innovation

Award, the Unconventional Innovation Program,

and the Institute’s functional imaging initiatives –

to enable researchers to develop, access, and use

new technologies. In addition, initiatives like the

Cancer Genetics Network and the Early Detection

Research Network bring together researcher communities and their collective resources, allowing

them to ask questions on a larger scale. These new

programs are accelerating the pace of discovery and

speeding our efforts to unravel cancer’s intricacies.

As the depth of our knowledge of cancer grows, we

will be able to bring the benefits of increasingly

sophisticated scientific discovery to the American

people more quickly.

Progress in our understanding of cancer and our

ability to detect and treat it have led to a real and

continuing decline in the cancer incidence and

death rates. However, our excitement over important scientific progress and the very real human

gains that result is tempered by the knowledge that

far too many Americans continue to suffer and die

from cancer each day. Incidence of some cancers

continues to rise. For example, the incidence of

melanoma, a deadly skin cancer, has risen on average of 4 percent per year since 1973. Moreover, all

groups of people are not benefiting equally from our

advances against cancer. Rates of colon cancer, for

instance, are declining overall, but not among

African Americans. Too many Americans, for a

host of reasons, lack access to high quality, cuttingedge cancer treatment and care. And, as more cancer patients are successfully treated, we recognize

that we know far too little about the long-term

needs of cancer survivors.

As we look forward to the next decade of progress,

much remains to be done. This document, our plan

and budget proposal for Fiscal Year 2002, describes in

detail how we intend to proceed. Over the past five

years, this document has come to be viewed as a

national plan for cancer research and has been

adopted by cancer researchers around the Nation. As

such, it is no longer just a statement of the NCI’s

goals and priorities, but increasingly of the goals and

priorities of the National Cancer Program.

The plans outlined in this document are ambitious – in some ways daunting. But the returns on

our investment promise to be beyond price – no less

than the prevention and cure of cancer. By continuing our investment in research and discovery, we

will move ever closer to the long-awaited day when

cancer is finally conquered.

Richard D. Klausner, M.D.

Director

National Cancer Institute
Directors Message ’ . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 1

Executive Summary . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 4

Our Role in Cancer Research . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 8

2002 Budget Request. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 9

Highlights of Progress . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 10

How We Work . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 14

NCI’s Challenge: Building Our Capacity for the Future . . . . . . . . . . . . . . . . . . . . . . 24

Investigator-Initiated Research . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 25

Centers, Networks, and Consortia . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 29

National Clinical Trials Program . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 33

Studying Emerging Trends in Cancer . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 38

Quality of Cancer Care . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 43

Reducing Cancer-Related Health Disparities. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 48

Informatics and Information Flow . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 53

Training, Education, and Career Development . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 57

Extraordinary Opportunities for Investment . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 63

Genes and the Environment . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 64

Cancer Imaging. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 69

Defining the Signatures of Cancer Cells . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 74

Molecular Targets of Prevention and Treatment . . . . . . . . . . . . . . . . . . . . . . . . . . . . 80

Research on Tobacco and Tobacco-Related Cancers . . . . . . . . . . . . . . . . . . . . . . . . . 86

Cancer Communications. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 91

Planning National Agendas in Disease-Specific Research. . . . . . . . . . . . . . . . . . . . . . 96

How It All Comes Together. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 100

On the Cutting Edge 

Developing Tissue Microarrays . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 17

Spotlights on Research

Harnessing the Immune System to Prevent and Treat Cancer. . . . . . . . . . . . . . . . . . 37

A New Detection Tool – Looking for DNA Mutations . . . . . . . . . . . . . . . . . . . . . . . 76

Diffuse Large B-Cell Lymphoma: A Disease Within a Disease . . . . . . . . . . . . . . . . . . 82

People’s Stories

Adult and Pediatric Brain Tumors. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 22

Ovarian Cancer. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 61 

Table of Contents 3

Table of 

Contents

Table of Contents
Executive 

Summary

4 The Nation’s Investment in Cancer Research

T

he National Cancer Institute’s goal is to stimulate and support scientific discovery and its

application to achieve a future when all cancers are uncommon and easily treated. NCI works

toward this goal by providing vision to the Nation’s

cancer research effort and leadership for thousands

of NCI-funded researchers across the United States

and around the world. We:

■ Conduct, coordinate, and support cutting-edge

research and its application. 

■ Build upon past discoveries and promote creativity and innovation. 

■ Support development of, access to, and use of

new technologies.

■ Disseminate cancer information.

■ Support training and career development for

cancer researchers. 

■ Facilitate the movement of research findings into

clinical practice. 

■ Maintain support mechanisms and collaborative

environments to link scientists with their colleagues and with critical technological and

information resources. 

■ Develop strategies to define, improve, measure,

and monitor the quality of cancer prevention

and care and reduce disparities in outcomes.

For all of our work, we depend on a superb workforce both inside and outside the Institute. We rely

on the insight and advice of a broad spectrum of

experts and advocates who serve on our standing

advisory boards and committees and who assist with

planning and assessment.

Our total Fiscal Year 2002 Budget Request is

$5,030,000,000. This represents an increase of

$1,524,928,000 over the Fiscal Year 2001

President’s Budget. Of this increase, $203,667,000

will be provided to continue NCI commitments

into 2002 (Core Budget). An additional

$810,711,000 will be used for NCI’s Challenge and

$510,550,000 will support Extraordinary

Opportunities for Investment. 

We will continue our work to sustain proven,

productive research programs, build capacity for the

future, and seize extraordinary scientific opportunities that build upon past discovery. 

Sustain Proven, Productive 

Research Programs

Science cannot thrive without the resources that

support an enterprise of discovery. NCI conducts,

coordinates, and funds research through two key

programs. Our Extramural Research Program supports individual basic, clinical, and population scientists, cancer centers, collaborative research

teams, a comprehensive cancer control program,

and training, education, and career development

activities. Nearly three-quarters of our budget 

supports studies by thousands of investigators

nationwide.

NCI also supports cancer research through our

Intramural Research Program, which encompasses

the work of over 400 principal investigators working in NCI laboratories and clinics. Approximately

16 percent of the Institute’s budget is invested in

this work. The Intramural Research Program complements ongoing research in universities and

industry and has been involved in pivotal discoveries in cancer research, such as the first successful

treatments for childhood leukemias, establishing

the foundations for tumor vaccines, and identifying

genetic causes of familial cancers.
Executive Summary 5

Build Our Capacity for the Future 

(NCI’s Challenge)

Major breakthroughs in cancer research depend on

building and sustaining the strong research infrastructures and interdisciplinary collaborations that

will enable us to apply rapidly evolving discoveries in

cancer genetics, cancer biology, molecular-based

technologies, imaging, and targeted therapeutic

interventions. Our challenge is to provide the vision,

creative environments, and diverse resources needed

to ensure a smooth flow between advances in cancer

research knowledge and their application in cancer

prevention and treatment.

Investigator-Initiated Research

Investigator-initiated research – research conceived

and conducted by scientists in laboratories and

clinics across the country and at NCI – is the wellspring of scientific discovery. Our goal for Fiscal

Year 2002 is to speed the rate of discovery and

accelerate the application of those discoveries to

the population by expanding and facilitating

researchers’ access to resources and new technologies. In understanding the basic processes of cancer

and translating this research into clinical practice, we

must link researchers with the resources and technologies they need, ensure collaboration, and encourage involvement of researchers across disciplines. 

Centers, Networks, and Consortia 

NCI will continue to create and sustain research

infrastructures for collaboration, technology support and development, and access to resources that

enable multiple scientific disciplines to address

large problems in cancer that could not be solved

by individual investigators. We will achieve this by

expanding our nationwide infrastructure of cancer

centers, centers of research excellence, networks,

and consortia in ways that promote and facilitate

complex scientific interactions and the sharing of

information and resources.

National Clinical Trials Program 

We will continue our efforts to ensure that the clinical trials program addresses the most important

medical and scientific questions in cancer treatment

and prevention quickly and effectively through

state-of-the-art clinical trials that are broadly accessible to cancer patients, populations at risk for cancer, and the physicians who care for them. As the

research investment of the past decade has led to

major advances in our understanding of tumor biology and potential molecular targets for cancer prevention and treatment, our capacity to apply and

test these findings in clinical settings has not kept

pace. The NCI will invest more resources in developing and testing new therapies and increasing

access to and participation in clinical trials. 

Studying Emerging Trends In Cancer

We will expand surveillance data systems, methods,

communications, and training to improve capacity

for monitoring progress in cancer control and for

exploring potential causes of cancer nationally and

among diverse populations. Recent changes in

health care financing, information and computer

technologies, and the social and cultural diversity

of our country present new challenges and opportunities in surveillance research. In addition to traditional incidence and mortality data, we need data

on patterns of care, patient-centered measures such

as quality of life, and sociodemographic and economic population characteristics. New investments

also are required to support and adopt new methodologic and informatics tools, including innovative

approaches to modeling trends, more refined cancer

maps, and information systems that will allow

investigators to analyze individuals and potential

environmental exposures by location. 

Quality of Cancer Care

Our goal is to enhance the state of the science for

defining, monitoring, and improving the quality of

cancer care and inform Federal decision making on

cancer care delivery, coverage, and regulation. NCI

is launching research to improve the quality of cancer care by strengthening the information base for

cancer care decision making. Researchers seek to

better understand what constitutes quality cancer

care, with an emphasis on the patient’s perspective;

identify geographic, racial/ ethnic, and other disparities in who receives quality care; and strengthen

the scientific basis for selecting appropriate interventions. 

Executive Summary
6 The Nation’s Investment in Cancer Research

Reducing Cancer-Related 

Health Disparities 

NCI is embracing the challenge of understanding

the causes of health disparities in cancer and developing effective interventions to reduce them.

Advances in biomedical science have contributed

to increased longevity and improved quality of life

for many Americans, but the burden of disease is

not borne equally by all population groups in the

United States. Plans call for increasing fundamental research into the social causes of health disparities, the psychosocial factors that mediate them,

and the biologic pathways that can explain their

impact. In addition, we will expand our cancer control intervention and population research on disparities, better define and monitor cancer-related

health disparities, and strengthen training and education in this research area. 

Informatics and Information Flow

NCI has taken on the challenge to create a cancer

informatics infrastructure that enables cancer

research by enhancing information and resource

exchange among researchers, clinicians, and the

public and reduces the barriers experienced by individuals seeking information about cancer prevention, diagnosis, and treatment. One of our first steps

is to create a framework to increase the speed with

which we carry out clinical trials by creating common forms, terminology, and reporting requirements.

We will expand this infrastructure to enable integration among research initiatives of all types and

improve information access for all user communities. 

Training, Education, and 

Career Development

NCI will continue to expand its efforts to design and

implement opportunities for scientists at all career levels to meet the challenge of building a stable, diverse

cadre of basic, clinical, behavioral, and population scientists trained to work together effectively and use the

most advanced technologies. We must more adequately prepare basic scientists, reverse the migration

of physicians from research to practice, increase the

numbers and stabilize the careers of population, behavioral, and public health scientists, create a research

workforce that is ethnically and racially diverse, and

attract and integrate a wide array of technical and

informatics disciplines into cancer research.

Seize Extraordinary Scientific

Opportunities (Extraordinary

Opportunities for Investment)

Six areas of extraordinary scientific opportunity in

cancer research identified through formal input

from cancer scientists, educators, advocates, and

community leaders will be continued from Fiscal

Year 2001 into 2002.

Genes and the Environment

Our goal in this area is to discover genetic, environmental, and lifestyle factors, and their interactions that define cancer risk and use this information to inform cancer prevention, early detection,

and treatment strategies. We will achieve this goal

through research on environmental risk factors,

susceptibility genes, and their interactions; development of risk models to integrate genetic and

environmental determinants of cancer; and clinical

studies to address the clinical, behavioral, and societal issues associated with cancer susceptibility. 

Cancer Imaging

To ensure continued exploration of the benefits of

imaging technology in cancer, NCI will accelerate

the discovery and development of imaging methods

and use these new technologies to identify biological and molecular properties of precancerous and

cancerous cells in order to predict clinical course

and response to interventions. These developments

will lead to a whole range of improved cancer prevention, diagnostic, and treatment options. 

Defining the Signatures of Cancer Cells:

Detection, Diagnosis, and Therapy 

All cells have unique, identifiable “signatures”– a

combination of active and inactive genes, and the

proteins and other cellular products being manufactured by the cell. By reading cellular signatures

accurately, we may be able to detect and diagnose
Executive Summary 7

very early cancers before they invade nearby tissues.

NCI’s goal in this area of extraordinary opportunity

is to generate a complete catalog of distinguishing

molecular profiles of normal, precancerous, and

cancer cells at all stages and in all tissues, and use

the catalog to develop diagnostic techniques for the

earliest detection of precancerous lesions and cancers; develop signature-based therapies; and identify subsets of patients with different prognoses to

predict therapeutic response. 

Molecular Targets of Prevention 

and Treatment

Many existing anti-cancer compounds inhibit the

growth of cancer cells successfully, but they also

affect healthy cells, causing short- and long-term

toxic effects that can themselves endanger patients’

health and quality of life. But advances in cancer

biology, chemistry, and technology are providing

the knowledge and tools to develop a whole new

generation of cancer treatments and preventives

that attack essential tumor cell processes with

greatly reduced side effects. Our goal is to accelerate the discovery, development, and testing of prevention and treatment agents that target the

molecular changes underlying various stages of cancer initiation and progression. 

Research on Tobacco and 

Tobacco-Related Cancers 

Over the past 30 years, we have learned much

about the enormous burden tobacco use places on

our Nation’s public health, the cancer-causing

agents in tobacco smoke, and the molecular

changes that follow exposure to tobacco.

Investigators also have learned a great deal about

the psychosocial, biobehavioral, and biological

determinants of tobacco use and addiction. NCI

will build upon this knowledge base and devote

increased resources to further understand the specific causes of tobacco use, addiction, and tobaccorelated cancers and apply this knowledge to their

prevention and treatment, particularly in high-risk

individuals and groups.

Cancer Communications

The current communications revolution, particularly

the power and potential of the Internet, is unprecedented in human history. At no other time has it

been so easy for so many people to access such a vast

wealth of information. But major gaps remain in our

knowledge and understanding of how consumers use

health information and how to improve access to it.

Both the public and the physician community have

a great need for timely, accurate cancer-related information on topics ranging from primary prevention to

survivorship and end-of-life issues. NCI’s goal is to

understand and apply the most effective communications approaches to maximize access to and use of

cancer information by the public, consumers,

patients, survivors, and health professionals with a

special focus on diverse populations. 

The Nation’s Investment in Cancer Research

NCI Budget Request for Fiscal Year 2002

(dollars in thousands)

Base of FY 2001 President’s Budget $3,505,072

Core Increase 203,667

NCI’s Challenge Increase

Investigator-Initiated Research 124,561

Centers, Networks, and Consortia 93,500

National Clinical Trials Program 328,000

Studying Emerging Trends in Cancer 47,300

Quality of Cancer Care 21,500

Reducing Cancer-Related Health Disparities 50,600

Informatics and Information Flow 77,250

Training, Education, and 

Career Development 68,000

Subtotal Challenge 810,711

Extraordinary Opportunities Increase

Genes and the Environment 68,250

Cancer Imaging 90,550

Defining the Signatures of Cancer Cells 110,750

Molecular Targets of Prevention 

and Treatment 146,500

Research on Tobacco and 

Tobacco-Related Cancers 67,000

Cancer Communications 27,500

Subtotal Opportunities 510,550

Total FY 2002 Budget Request $5,030,000

Executive Summary
Our Role in 

Cancer Research

8 The Nation’s Investment in Cancer Research

NCI works toward this goal by providing vision

to the Nation and leadership for thousands of

NCI-funded researchers across the United

States and around the world and working to assure

that the results of research are translated into reducing the burden of all cancers for all people. We:

■ Conduct, coordinate, and support cutting-edge

research and its application. 

■ Build upon past discoveries and promote creativity and innovation.

■ Support development of, access to, and use of

new technologies.

■ Disseminate cancer information to our many

communities.

■ Support training and career development for

cancer researchers. 

■ Facilitate the movement of research findings

into clinical practice. 

■ Maintain support mechanisms and collaborative

environments to link scientists with their colleagues and with critical technological and

information resources. 

■ Develop strategies to define, improve measure,

and monitor the quality of cancer prevention

and care and reduce disparities in outcomes.

Our work is driven by careful planning and priority setting. This includes:

■ Defining and building a research infrastructure. We

give priority to developing the technological and

personnel resources needed to support changing

scientific and resource needs and the translation

of new knowledge and emerging technologies

into clinical practice. 

■ Identifying extraordinary scientific opportunities.

We seek opportunities that promise to provide

profound insights into cancer – those opportunities that hold greatest potential to lead to major

improvements in our ability to prevent, control,

detect, diagnose, and treat cancer. 

■ Planning national agendas for disease-specific

research. We continually assess our portfolios and

plan for the research needed to uncover the biological characteristics that are unique to specific

forms of cancer.

Our success in these endeavors depends on:

■ A superb workforce both inside and outside the

Institute. The competence, hard work, and dedication of our researchers and clinicians and

those who support their efforts each day drive

our success in discovery and innovation.

■ Insight and advice from those who know and care

most about cancer. We look to representatives

from a broad spectrum of scientific, medical, and

advocacy communities in several ways:

■ Members of standing and special advisory

groups assist us in planning for and evaluating

cancer research from conceptual beginnings

through clinical application.

■ Many people aid us in identifying, planning

for, and implementing our “extraordinary

opportunities for investment.”

■ People with diverse perspectives help us plan

national agendas in disease-specific research

through our Progress Review Groups. 

The National Cancer Institute’s goal is to stimulate and support scientific discovery and

its application to achieve a future when all cancers are uncommon and easily treated. 
NCI Budget Request 9

2002 Budget Request

Core, 

2000 2001 Challenge, and 

Operating President’s Core and Extraordinary

Budget Budget Core Challenge Opportunities

Research Project Grants (RPGs): 

Ongoing $1,103,744 $1,245,664 $1,401,840 $1,401,840 $1,401,840

New and Renewal 420,514 404,811 404,811 588,072 841,072

Subtotal RPGs 1,524,258 1,650,475 1,806,651 1,989,912 2,242,912

Small Business Innovation Research 67,046 71,337 73,121 86,121 100,121

Total RPGs 1,591,304 1,721,812 1,879,772 2,076,033 2,343,033

Intramural Research 511,786 532,002 545,302 584,802 586,802

Cancer Centers 173,609 182,216 186,771 239,771 269,771

Specialized Programs of 

Research Excellence 70,591 60,916 62,439 94,439 112,689

Clinical Trials Infrastructure: 

Cooperative Clinical Research 145,503 154,763 158,632 377,132 389,382

Community Clinical Oncology Program 62,173 69,232 70,963 138,963 170,963

Subtotal Clinical Trials 207,676 223,995 229,595 516,095 560,345

Training and Education Grants 121,564 133,971 138,446 202,546 207,046

Research Support Contracts 357,266 367,286 376,468 478,068 600,918

Cancer Control Operations 104,033 105,052 107,678 117,678 119,678

Research Management and Support 117,643 120,600 123,615 151,365 171,065

Other Grants 55,609 57,222 58,653 58,653 58,653

Total NCI 3,311,081 3,505,072 3,708,739 4,519,450 5,030,000

Cancer Control included above 365,837 375,367 383,668 541,768 681,018

Requested Increases for Fiscal Year 2002

Extraordinary 

Core Challenge Opportunities Total

Research Project Grants (RPGs): 

Ongoing $156,176 $156,176

New and Renewal $183,261 $253,000 436,261

Subtotal RPGs 156,176 183,261 253,000 592,437

Small Business Innovation Research 1,784 13,000 14,000 28,784

Total RPGs 157,960 196,261 267,000 621,221

Intramural Research 13,300 39,500 2,000 54,800

Cancer Centers 4,555 53,000 30,000 87,555

Specialized Programs of Research Excellence 1,523 32,000 18,250 51,773

Clinical Trials Infrastructure: 

Cooperative Clinical Research 3,869 218,500 12,250 234,619

Community Clinical Oncology Program 1,731 68,000 32,000 101,731

Subtotal Clinical Trials 5,600 286,500 44,250 336,350

Training and Education Grants 4,475 64,100 4,500 73,075

Research Support Contracts 9,182 101,600 122,850 233,632

Cancer Control Operations 2,626 10,000 2,000 14,626

Research Management and Support 3,015 27,750 19,700 50,465

Other Grants 1,431 0 0 1,431

Total Requested Increases 203,667 810,711 510,550 1,524,928

NCI Budget Request for Fiscal Year 2002

(dollars in thousands)

Budget Request
10 The Nation’s Investment in Cancer Research

Highlights of Progress

Though cancer continues to be a real threat to

human life and health, we are encouraged by the

decline in overall incidence and mortality rates for

cancer over the past few years. The rates of new

cases and deaths for all cancers combined declined

in the United States between 1990 and 1997.1

Death rates for the four most common cancer sites –

lung, colorectal, breast, and prostate – continue to

drop, albeit slowly. And for the first time, between

1996 and 1997, the total number of cancer deaths

did not rise, despite a growing and aging population. 

That is the good news. The bad news is that

death rates are still increasing for some forms of cancer including liver, non-Hodgkin’s lymphoma,

esophagus, multiple myeloma, and kidney cancers.

The recent declines in the incidence of cancer stemming from the dramatic reductions in adult tobacco

use may well be reversed if teen smoking continues

to rise. Moreover, the overall number of Americans

who develop cancer is expected to increase as our

population ages and “baby boomers” enter the time

of life when cancer is most common. An increase in

the number of people with cancer will, in turn, place

mounting pressure on our Nation’s capacity to

respond. With the expected growth and aging of the

U.S. population, expenditures for cancer treatment

are predicted to nearly double over the next decade,

rising to just under $100 billion. The costs of screening more people will add another five to ten percent

to this bill. Keenly felt, but less easily calculated, are

the productivity and contributions lost to society

when people are afflicted with cancer. 

Growing knowledge about the nature of cancer,

newly developed tools and approaches for conducting clinical research, and increasingly sophisticated

behavioral and population research techniques

continue to provide ever improving options for preventing, detecting, diagnosing, and treating cancer.

Still, the time required for discovery and breakthroughs can seem painstakingly slow. Each

endeavor takes a different course. Some things we

can predict. Many we cannot. Some results come

quickly while others take years. Learning what

doesn’t work is sometimes as important as learning

what does. The following snapshot of recent

progress in science and technology describes results

from our past research investments and shows how

these can fuel continued efforts in the areas

described in this document. 

Preventing and Controlling Cancer

■ NCI is collaborating with two pharmaceutical

companies to study the effectiveness of the drug

celecoxib in preventing several precancerous

and cancerous conditions. An earlier NCI study

determined that celecoxib helped reduce the

number of colon polyps in patients with familial

adenomatous polyposis (FAP), a condition that

is often a precursor to cancer. Based on this finding, the FDA approved celecoxib as an adjunct

to usual care for patients with FAP. Through the

National Clinical Trials Program, NCI and its

partners hope to determine whether celecoxib is

effective in preventing sporadic polyps, hereditary nonpolyposis colon cancer syndrome,

Barrett’s esophagus, bladder dysplasia, actinic

keratoses of the skin, and the progression of FAP

to colorectal cancer.

■ The recently published U.S. Public Health

Service Report, Treating Tobacco Use and

Dependence: A Clinical Practice Guideline,

provides specific, evidence-based recommendations for brief and intensive tobacco cessation

interventions and changes in the systems

designed to promote tobacco use assessment and

treatment. The guidelines outlined in the report,

developed by a consortium of seven non-profit

and government institutions including NCI, are

based on an analysis of nearly 6,000 articles and

abstracts published between 1975 and 1999. 

1 Annual Report to the Nation on the Status of Cancer, 1973-1997, prepared jointly by the National Cancer Institute, the American Cancer Society, the National

Center for Health Statistics/Centers for Disease Control and Prevention, and the North American Association of Central Cancer Registries.
Highlights of Progress 11

■ Recently, researchers have found evidence of

nicotine dependence and withdrawal among

young tobacco smokers before they become daily

smokers. NCI-supported researchers found that

63% of 7th-grade students who smoked one or

more cigarettes a month reported experiencing

one or more symptoms of nicotine dependence,

such as cravings, withdrawal, and loss of control

over the amount and duration of tobacco use.

This and other recent studies indicate that – contrary to past assumptions – adolescents who have

never smoked daily may encounter significant difficulty in trying to give up smoking, making it all

the more important to develop new strategies to

prevent young people from starting to smoke and

to intervene early to help young smokers quit.

■ NCI-supported researchers recently compared

the outcomes of black and white patients with

colon cancer enrolled in five clinical trials. They

found, as have other analyses of NCI clinical trials, that equal treatment yields equal outcome

and that race is not a factor in cancer-specific

survival when there is equal treatment. Data

from NCI’s Black-White Study of Cancer

Survival showed that black and poor women

with breast cancer were less likely to receive

appropriate treatment after diagnosis. They also

are more often diagnosed at a later stage of disease and are less likely to have a regular source of

health care. Similar disparities in appropriate

treatment have been demonstrated among various population groups with prostate, lung, and

cervical cancers. NCI studies through the new

NCI Challenge for Reducing Cancer-Related

Health Disparities will focus on determining the

reasons for the differences in care. 

Detecting Cancer

■ Preliminary research findings have shown that

testing for the human papillomavirus (HPV),

the primary cause of 90 percent of cervical

cancers, is a highly sensitive way to detect the

underlying abnormalities that may progress to

cervical cancer. Investigators questioned

whether HPV testing might help physicians and

patients decide how to handle mildly abnormal

PAP test results. Although most of these mild

abnormalities will go away without treatment,

physicians have had no way to distinguish the

less serious abnormalities from those that will

progress to precancerous conditions or cancer.

Researchers concluded that the HPV test effectively detects abnormal lesions that need immediate attention. Through the Extraordinary

Opportunity for Signatures of Cancer Cells, scientists will explore ways to improve the specificity of this test.

■ Although early detection improves the likelihood that treatment will be successful, current

non-invasive approaches for detecting small

tumors have limited utility because they cannot

distinguish small areas of abnormal cells from

larger surrounding areas of normal tissue.

Recently, however, scientists developed a new

imaging method to more precisely view tumor

cells, an important step toward non-invasive

detection of small, and more treatable, tumors.

The scientists injected tumor-bearing mice with

a unique imaging agent composed of nearinfrared fluorescence imaging probes coupled

with a novel substance that moves effectively

into tumor cells. When the agent was taken up

by the tumors, cellular enzymes “activated” the

agent causing it to fluoresce and allowing noninvasive detection. The agent was not activated

in non-tumor cells. Using this technique, the

investigators were able to image tumors smaller

than three-tenths of a millimeter in diameter.

Although this technique is still in the early

stages of development, it illustrates the promise

of new initiatives in the Extraordinary

Opportunity for Cancer Imaging.

of Progress

Highlights 
Treating Cancer

■ Many cancer researchers have believed for some

time that immunotoxins hold great promise for

cancer treatment. The idea underlying this

approach is elegantly simple: bioengineer small

tumor-specific antibodies, link them to a powerful toxin, and directly target and deliver deadly

poisons to tumor cells. Yet generating clinically

active immunotoxins has proven to be a difficult

challenge. A team of NCI scientists recently

reported, however, that all patients included in a

small trial on hairy cell leukemia, an unusual

cancer of immune B cells, responded to a recombinant immunotoxin called LMB-2. One

patient has been in complete remission for nearly two years, and the other patients had partial

responses with significant reduction in the number of circulating malignant cells. None of the

patients had responded to standard treatments.

As many as 25 percent of hairy cell leukemia

patients develop a resistance to conventional

treatment, making this and other potential alternative treatments being tested through our

Extraordinary Opportunity for Molecular Targets

all the more promising.

■ For many years, hormone therapy (androgen

deprivation) was the only useful systemic treatment for advanced prostate cancers. It was never

curative, however, and advanced cancers usually

became resistant in one to two years. More

recently, clinical researchers began to ask

whether hormone therapy would be more beneficial if it were used before cancers ever become

advanced. Patients with high-risk presentations

of prostate cancer were randomized either to

have early androgen deprivation (generally by

injections every several months) along with initial radiation therapy or prostatectomy or not to

receive androgen deprivation unless and until

there was evidence of disease recurrence. All of

the studies demonstrated a benefit to earlier hormone therapy. In one study, after a median of 6.8

years of follow-up, 85 percent of men in the

adjuvant hormone arm were alive, compared to

65 percent in the group receiving hormone therapy only after their cancer recurred. A number of

studies conducted through NCI’s National

Clinical Trials Program are now building on

these results.

■ The monoclonal antibody Trastuzumab (commonly known as HerceptinTM) was the first targeted therapy for advanced breast cancer to

receive FDA approval. Trastuzumab targets

breast cancer cells with the protein Her-2, a molecule that straddles the cancer cell membrane,

picking up signals outside the cell and relaying

them inside to promote tumor growth. Blockage

of the Her-2 receptor by the antibody reversed

malignant transformation of cells. Human trials

also confirmed the positive effects of

Trastuzumab and demonstrated that this agent,

when combined with chemotherapy, can prolong median survival by 24 percent in women

whose tumors have high amounts of Her-2.

Currently, NCI is sponsoring two major studies

through the National Clinical Trials Program to

test whether adding this agent to standard

chemotherapy in earlier stages of breast cancer

can enhance survival.

■ Bisphosphonates, widely used to prevent osteoporosis, also have been shown to prevent bone

breakdown due to malignancy. Because bone is

the most common site of distant metastases in

breast cancer, this disease has been a logical

choice in which to test this approach. Several

trials conducted with patients with bone metastases have shown that bisphosphonates can

decrease the subsequent rate of fractures and

other complications. NCI is now sponsoring two

national trials to test whether bisphosphonates

can prevent the development of bone metastases

in breast cancer altogether. Findings from preliminary studies conducted in other countries

have been equivocal regarding this effect, and

larger studies are needed to provide a definitive

answer.

■ Development of the new drug STI571 by

Novartis Pharmaceuticals illustrates how NCIfunded molecular targets research can be translated into a powerful new cancer treatment. In

nearly all patients with chronic myeloid

leukemia (CML), leukemia cells express BcrAbl, an abnormal protein that alone is sufficient

to cause CML. Because of this feature, CML

12 The Nation’s Investment in Cancer Research
offered an ideal setting for testing the concept

that an agent targeted against a tumor-specific

abnormality can be an effective treatment.

Researchers developing STI571 found that this

new drug targets leukemia cells by selectively

inhibiting cells that express the Bcr-Abl protein,

making STI571 a promising candidate for treating CML and other Bcr-Abl positive leukemias.

Initial results from a Phase I study demonstrated

that virtually all patients treated with STI571

had a complete hematologic remission. 

■ Thalidomide, a drug that has been available only

for limited use since it was discovered to cause

birth defects in the children of pregnant women,

has shown effectiveness against Kaposi’s sarcoma (KS), an AIDS-related cancer. NCI

researchers have shown in a Phase II clinical

trial that thalidomide induced partial responses

in 40 percent of the patients treated. The positive outcome of this trial warrants additional

study of the drug, which inhibits angiogenesis.

Thalidomide has also been shown, in early trials,

to be active against multiple myeloma, a bone

marrow cancer.

■ Recent results from several clinical trials conducted to test the viability of bone marrow

transplants in combination with high-dose

chemotherapy for treating metastatic breast

cancer exemplify how negative research findings

are often as important as positive ones. In this

case, the rigorous and costly regimens did not

prove superior to standard-dose chemotherapy. 

Understanding Cancer and its Causes

■ NCI-supported scientists have identified a

genetic mutation that may be responsible for

more than 11,000 of the 129,000 cases of colorectal cancer diagnosed annually in this country. The TR-I(6A) defect, a mutant form of the

Transforming Growth Factor Beta (TGF-ß)

receptor, may increase a person’s risk for cancer.

Comparing blood samples from cancer patients

and healthy volunteers, scientists have observed

that the TR-I(6A) mutation seems to occur

more commonly in people with cancer than in

healthy people. People who inherited a copy of

the mutation from both parents, and thus carried

two mutant copies of the gene, had the most pronounced risk for developing cancer. The scientists suggest that this mutation may contribute to

cancer development, particularly colon cancer,

by hampering a cell’s normal efforts to control its

growth. This and other findings resulting from

new efforts through the Genes and the

Environment Extraordinary Opportunity may

one day help physicians identify at-risk patients

who should be monitored closely for early signs

of disease. 

■ NCI researchers have found that women who

use combined estrogen-progestin replacement

therapy have a greater risk for developing

breast cancer than those who use estrogen

alone. Using 15 years of follow-up data from

46,000 women who participated in the Breast

Cancer Detection Demonstration Project, a

nationwide breast cancer screening program, scientists found that compared to non-users, the

relative risk for breast cancer increased by eight

percent per year for the estrogen-progestin therapy compared to one percent for estrogen therapy alone in women who had used hormones during the previous four years. There was no

increase in risk among women who had stopped

either therapy for more than four years. The

increase in risk is still low for women taking both

hormones, but this finding further complicates

the decision women face about whether to take

hormone replacement therapy.

■ Using powerful new DNA microarray technology, a team of scientists recently determined that

the most common form of non-Hodgkins lymphoma (NHL) – diffuse large B-cell lymphoma

(DLBCL) – is actually two distinct diseases.

The scientists discovered that DLBCL showed

two distinct patterns of gene expression, suggesting that there are at least two subtypes of NHL,

only one of which responds to current treatment.

This finding underscores the importance of our

Extraordinary Opportunity for Defining the

Signatures of Cancer Cells as a path to more

accurate diagnosis and targeted treatments. (For

more information, see page 82.)

Highlights of Progress 13

of Progress

Highlights 
How We Work

14 The Nation’s Investment in Cancer Research

NCI conducts, coordinates, and funds cancer

research and provides vision and leadership

for the cancer research community by:

■ Planning and prioritizing all aspects of the

research we support.

■ Conducting ongoing assessments to ensure a

comprehensive and balanced research portfolio.

■ Seeking advice regularly from our stakeholders.

■ Supporting core extramural and intramural programs.

■ Maintaining a strong infrastructure to support

cancer research. 

PLANNING AND PRIORITY SETTING 

Research planning is an integral part of all NCI

activities. It involves identifying needs and opportunities, setting priorities, and developing strategies

for communicating and implementing decisions

and recommendations. We set our funding priorities to ensure that we:

■ Support the full range of research activities necessary to address the unanswered questions about

cancer. 

■ Give attention to the spectrum of cancers and the

various populations that experience them. 

■ Link all components of the cancer research

enterprise through translational research. 

■ Identify new opportunities, gaps, and barriers to

progress; create new programs; and improve

existing ones.

ASSESSMENT 

Assessment efforts ensure that our research portfolio is balanced and our support structure is strong.

To implement recommendations arising from these

assessments, we convene groups of outside scientists

and NCI staff to redesign programs and develop

new initiatives. We track implementation and regularly report on our progress. 

Progress Reviews

We seek the advice of experts through Progress

Review Groups (PRGs) to assess the scientific needs

and opportunities that are critical to advancing

research on specific forms of cancer. PRGs are composed of prominent members of the scientific, medical, and advocacy communities who work together

to define, develop, and prioritize national research

agendas. Go to planning.cancer.gov for more information.

Program Reviews

We use external reviews to guide us in strengthening our major research support programs. In the

past few years, we have completed in-depth reviews

of several programs including Cancer Centers,

Cancer Control, Clinical Trials, Cancer

Prevention, Developmental Therapeutics in

Cancer, and Developmental Therapeutics in AIDS

(deainfo.nci.nih.gov/advisory/bsa/bsa_program/pog

programfo.htm). In addition, implementation

groups were formed and plans completed for

advancing research in the areas of surveillance,

tobacco, and nutrition (dccps.nci.nih.gov/

DCCPS/SIG, dccps.nci.nih.gov/tcrb/TRIP, and

dcp.nci.nih.gov/reports/nutrition/3intro.htm).
How We Work

How We Work 15

ADVISORY ACTIVITIES

To ensure the wise use of resources to meet the goals

of the National Cancer Program, NCI actively seeks

the expertise and perspective of a variety of advisory

bodies both within and outside the Institute. 

(deainfo.nci.nih.gov/ADVISORY/boards.htm)

National Cancer Advisory Board 

NCI’s principal advisory body is the Presidentially

appointed National Cancer Advisory Board

(NCAB). Scientific experts and advocates on the

NCAB advise NCI’s Director on issues related to all

aspects of the National Cancer Program and provide a second level of review for NCI grant 

applications. 

President’s Cancer Panel

The President’s Cancer Panel, which consists of

three persons appointed by the President, monitors

the development and execution of National Cancer

Program activities. 

Board of Scientific Counselors

For advice on the progress, performance, and productivity of the Intramural Research Program, NCI

leadership looks to the outside scientific experts

and consumer advocates on the Board of Scientific

Counselors. Members evaluate scientific programs

through periodic site visits to intramural laboratories and advise NCI intramural divisions on future

direction for their programs. 

Board of Scientific Advisors 

For advice on the progress and future direction of

our Extramural Research Program, NCI leadership

looks to the distinguished scientists and consumer

advocates from outside NCI who serve on the

Board of Scientific Advisors. Members evaluate

Institute-awarded grants, cooperative agreements,

and contracts and review ideas for new research

solicitations to ensure that the concepts are meritorious and consistent with NCI’s goals. 

Advisory Committee to the Director

Members of the Advisory Committee to the

Director make recommendations to the Director for

the oversight and integration of various planning

and advisory group activities. The Committee serves

as the official channel through which the findings

and recommendations emerging from these groups

are submitted. Members of this Committee include

the chairs of all NCI standing advisory groups and

the Institute’s senior leadership. 

NCI Executive Committee 

The NCI Executive Committee, which includes

NCI division directors and other key advisors to the

Director, meets regularly to make major policy and

operating decisions for the Institute. 

Director’s Consumer Liaison Group

This all-consumer advocate advisory committee

serves as a primary forum for discussing issues and

concerns and exchanging viewpoints important to

the broad development of NCI program and

research priorities. The committee also helps develop and establish processes, mechanisms, and criteria for identifying appropriate consumer advocates

to serve on NCI program and policy committees. 

HOW WE SPEND OUR BUDGET 

About 89% of the budget supports basic and clinical studies conducted through our core research

programs. The remainder is used for preparing and

developing researchers and clinicians at various

stages in their careers, communicating cancer information to all who need it, and administration and

management (nci.nih.gov/admin/fmb/index.html).

Extramural

Research 

73%

Intramural

Research

16%

Training,

Education,

and Career

Development

4%

Communications

3%

Administration

and Management

4%

How We Work
CORE RESEARCH PROGRAMS 

NCI conducts, coordinates, and funds cancer

research through two key programs: the Extramural

Research Program (ERP), which links the NCI to

investigators, teaching hospitals, and other sites

throughout the country and the Intramural Research

Program (IRP), which encompasses the work of over

400 principal investigators employed by NCI.

For information on each of NCI’s divisions, go to

cancer.gov/aboutnci. 

Extramural Research Program

The ERP supports scientists; cancer centers; collaborative research teams; a comprehensive cancer

control program; and training, education, and

career development activities. Through the

Divisions of Cancer Biology (DCB), Cancer

Treatment and Diagnosis (DCTD), Cancer

Prevention (DCP), and Cancer Control and

Population Sciences (DCCPS), we support individual investigators and research teams from universities, private industry, and other Federal agencies. 

DCB supports basic and applied research on cancer cell biology, including research on carcinogenesis; cancer immunology; and the role of biological,

chemical, and physical agents in cancer initiation,

promotion, and inhibition. DCTD supports

research in five program areas: biomedical imaging,

cancer diagnosis, cancer therapy evaluation, developmental therapeutics, and radiation research.

Through DCP, we support an extensive research

portfolio in cancer prevention including chemoprevention; nutritional sciences; genetic and infectious agents; and early detection through biomarker development, validation, and biometry. Through

DCCPS, we conduct and support an integrated

research program in cancer genetics; epidemiology;

and behavioral, social, and surveillance research. 

ERP program staff provide the essential scientific expertise and national focus needed to work with

NCI-funded scientists. They synthesize the state of

the science in their areas, identify priorities for new

research directions, and foster collaborations

among scientists. Program staff monitor the

progress of extramural grants through contact with

individual investigators and review of annual

research progress reports. Results of these NCIfunded projects are communicated to the scientific

community and the public through peer-reviewed

scientific journals, scientific meetings, workshops,

and symposia, and through our other cancer communication outlets, such as the Cancer Information

Service, the Physician Data Query database, our

press office, and our Web site (cancer.gov). 

Research Project Grants (RPGs) are the main

pool of funds expended by NCI for awards to extramural scientists. These funds foster the creativity of

talented scientists by providing them with the support needed to pursue the best ideas that will yield

progress against cancer. 

RPGs are awarded to institutions for individual

principal investigators while Program Project

Grants go to groups of scientists involved in related

research projects. In Fiscal Year 2001, NCI anticipates supporting 4,700 separate research grants,

expending more than $1.5 billion. More than 1,250

of these awards will be new or competing renewal

projects. The individual investigator grant payline

rose from the 15th percentile in Fiscal Year 1995 to

the 22nd percentile in Fiscal Year 2000. 

NCI also has special mechanisms to fund exceptionally innovative, exploratory, and developmental

research activities, to allow investigators to embark

on projects of unusual scientific potential, and to

support research and development ideas that are

likely to lead to a commercial product or service.

Intramural Research Program

The IRP is uniquely structured to address cancer

research problems requiring long-term commitments and research activities not always conducive

to extramural funding. The Program provides an

environment conducive to cooperation between

laboratory-based scientists and clinical investiga16 The Nation’s Investment in Cancer Research

Research Project Grants (RPGs)

(dollars in thousands)

2000 2001 2002 Core

Operating President’s Budget

Budget Budget

Ongoing $1,103,744 $1,245,664 $1,401,840

New 420,514 404,811 404,811

Subtotal 1,524,258 1,650,475 1,806,651

Small Business 67,046 71,337 73,121

Innovation 

Research

TOTAL 1,591,304 1,721,812 1,879,772
How We Work

tors to facilitate rapid translation of new basic

research discoveries into early clinical trials. The

IRP complements ongoing research in universities

and industry and has been involved in pivotal discoveries in cancer research, such as the first successful treatments for childhood leukemias, establishing the foundations for tumor vaccines, and

identifying genetic causes of familial cancers.

Within the IRP, the Division of Cancer

Epidemiology and Genetics leads a national program of population-based studies to identify environmental and genetic determinants of cancer. The

Division of Basic Sciences (DBS) plans, directs,

coordinates, and evaluates the Institute’s intramural program in basic science relating to cellular,

molecular, genetic, biochemical, and immunologic

mechanisms relevant to understanding, diagnosing

and treating cancer. The Division of Clinical

Sciences provides the opportunity for patients from

across the country to be treated through groundbreaking research protocols at NIH’s Clinical

Center. Because of its national stature and unique

structure, the IRP is also a center for basic, clinical,

and population-based oncology training for

researchers from around the world. NCI plays a

major role in fostering the education and careers of

a growing number of nurses, doctors, and physician

assistants.

KEY INFRASTRUCTURE COMPONENTS

AND AREAS OF EMPHASIS

In addition to its core research funding programs,

NCI provides support for research infrastructure

and areas of emphasis including clinical research

and cancer centers.

How We Work 17

Intramural Research

(dollars in thousands)

2000 2001 2002 Core

Operating President’s Budget

Budget Budget

Basic $180,965 $188,114 $192,817

Clinical 131,965 137,178 140,607

Epidemiology 52,973 55,065 56,442

& Genetics

NIH Central 145,883 151,645 155,436

Services

TOTAL 511,786 532,002 545,302

I

magine how long it would take to determine if a

new diagnostic test works by analyzing hundreds 

of tissue samples one by one. Cancer researchers

nationwide may soon be able to dramatically shorten

that step using a new tissue analysis technology. The

new technology makes it possible to store hundreds 

of actual tissue samples on a single laboratory slide,

in a “tissue microarray.” Unlike traditional tissue

analysis tools that allow scientists to examine only 

a few tissue samples at a time or other types of

microarrays that contain only DNA, tissue microarrays

permit scientists to examine hundreds of tumor 

samples at once.

Tissue microarrays may include samples from many

different types of tumors for screening purposes or

may contain tissues from various stages of a single

kind of tumor for comparison studies. This new technology has astounding potential to accelerate

research by hastening the process of identifying the

unique characteristics (“molecular signatures”) of 

cancer cells, information vital to continued advances

in detection, diagnosis, and development of targeted

cancer therapies. 

The new Tissue Array Research Program (TARP) is a

collaborative effort between the National Human

Genome Research Institute and NCI. As one component of the Program, NCI will begin, in the Fall of

2000, to make tissue microarrays available to investigators across the country. The first to be offered will

be screening microarrays with tissue samples from a

variety of cancers such as colon, prostate, and breast. 

For more information on TARP activities and services,

go to cancer.gov/tarp. 

ON THE CUTTING EDGE

Developing Tissue Microarrays
Clinical Research 

Clinical research is a cornerstone of the National

Cancer Program. Every treatment we commonly

use today, every widely recommended preventive

measure, and every innovative detection strategy

has been, at one time, tested in cancer patients or

in people at risk for the disease. Participants in clinical research are often the unrecognized heroes of

the fight against cancer who allow us to amass the

body of information we are building upon every day.

Trials provide participants access to cutting-edge

interventions and provide researchers with information critical to continued advancement. 

To test potential treatment advances more rapidly NCI maintains the Cooperative Group

Clinical Trials program. The sheer number of different types of cancer and their biological complexity make the process of efficiently identifying and

evaluating new treatments or other anti-cancer

strategies extremely challenging. This national

network consists of 12 consortia (Cooperative

Groups) that collaborate regularly on clinical trials

for a variety of common cancers, and they frequently work together when the clinical question

requires such a large number of study participants

that one group working alone would be unable to

accrue enough people to conduct a meaningful

study. Each year, 1,700 institutions throughout the

United States and Canada and approximately

8,000 investigators in these institutions participate

in these trials. This kind of cooperation makes it

possible to centralize administration and data collection for trials taking place at a large number of

sites around the world. Approximately 20,000

patients participate in Cooperative Group clinical

trials each year, principally in large Phase III trials

that help establish the state of the art for cancer

therapy and prevention. Approximately 200 investigational agents or treatment strategies, ranging from

new chemotherapy drugs and cancer vaccines to

agents that prevent tumor blood vessel development,

are currently being studied.

The Community Clinical Oncology Program

(CCOP) is a network of 51 central offices in 32

states that provides the infrastructure to link more

than 2,700 community cancer specialists and primary care physicians with clinical Cooperative

Groups and cancer centers. This network enables

individuals to participate in state-of-the-art clinical

research trials at over 350 community hospitals without the burden of traveling to a distant site. Eight

minority-based CCOPs increase the participation of

minority and underserved individuals in clinical trials research. Each year, over 700 patients enter clinical trials through these specialized CCOPs. In addition, CCOPs support scientific development and the

implementation of ongoing cancer treatment, prevention, and control clinical trials among community Cooperative Group members and cancer centers. 

NCI continues to take steps to address issues

that threaten to compromise our ability to conduct

clinical trials. These issues center around ongoing

problems related to patient recruitment, changes in

the health care system, insurance coverage, and

recent public and media concerns about patient

safety. To address these issues, NCI is:

■ Developing ways to improve access and participation through the National Clinical Trials

Program.

■ Offering Established Investigator Awards to

ensure that physicians have the time to conduct

clinical trials (cancertraining.nci.nih.gov/

research/clinical/k24.html).

■ Working to assure insurance coverage for individuals participating in trials through agree18 The Nation’s Investment in Cancer Research

Clinical Trials Infrastructure

(dollars in thousands)

2000 2001 2002 Core

Operating President’s Budget

Budget Budget

TREATMENT:

Clinical $145,503 $154,763 $158,632

Cooperative 

Research

Community 8,078 8,275 8,482

Clinical Oncology 

Program

Subtotal 153,581 163,038 167,114

Treatment

PREVENTION:

Community 54,095 60,957 62,481

Clinical Oncology 

Program

TOTAL 207,676 223,995 229,595
ments with private insurers and other government agencies. (For extensive information about

initiatives related to coverage for clinical trials,

go to cancertrials.nci.nih.gov/understanding/

indepth/insurance/index.html.)

■ Working with patient advocate groups and professional societies, local communities, and nonprofit organizations to increase understanding

about clinical trials through education programs. 

■ Expanding initiatives to assure the protection of

individuals participating in clinical research

through increased oversight of studies, development of education programs for researchers, and

grant support in the area of research ethics.

Initiatives involving other agencies and organizations are providing greater access and coverage

for clinical trials. For example: 

■ NCI has established interagency agreements to

secure coverage for Department of Defense and

Veteran’s Administration health plan beneficiaries

to participate in NCI-sponsored clinical trials. 

■ The United Health Care Corporation has agreed

to provide coverage of patient care costs associated with cancer treatment trials conducted by

the NCI-supported Cooperative Groups. 

■ A recent Presidential memorandum directs the

Medicare program to reimburse providers for the

cost of routine patient care in clinical trials and

provides for additional actions to promote the

participation of Medicare beneficiaries in clinical studies. 

■ Several states have enacted legislation designed

to provide greater access and coverage for clinical trials.

Specific information about national and state

initiatives is kept current and available in a legislation and insurance coverage section on NCI’s

cancerTrials Web site. (cancertrials.nci.nih.gov/

news/coverage/index.html) 

Cancer Centers 

Throughout the country, NCI has over 60 key partners in its efforts to speed the process of discovery

and bring the benefits of cancer research directly to

people. These research-oriented institutions, formally referred to as NCI-designated Cancer

Centers in recognition of their scientific excellence, are hubs of cutting-edge research, high quality cancer care, and outreach and education for

both health care professionals and the public. 

Support for the cancer centers helps ensure a

close association between state-of-the-art research

and state-of-the-art clinical care within the institution. Moreover, it allows each center to develop key

collaborations with industrial, community, and

state health organizations, and to link the research

capabilities and expertise of scientists within the

institution to problems of cancer incidence and

mortality in their communities and regions. 

There are three types of centers: Cancer Centers

have specific research foci, such as epidemiologic or

basic research; Clinical Cancer Centers concentrate on research activities in clinical oncology; and

Comprehensive Cancer Centers demonstrate both

significant scientific strength in laboratory, clinical,

and population studies and strong interdisciplinary

collaboration. Comprehensive Cancer Centers also

must have in place effective cancer information,

education, and outreach activities for the regions

and communities they serve. 

Traditionally, cancer centers have had broad scientific bases, and most have been developed within a

single institution. Changes in the program, however,

are enabling the planning of new consortia of institutions, often linking free-standing clinical and academic centers with community hospitals to form networks. In addition, more focused scientific concepts

are being developed for cancer centers. For example,

some centers are focusing on population sciences and

others are concentrating on translational research

opportunities within a specific scientific discipline,

such as immunology. Go to cancer.gov/cancercenters

for more information.

How We Work 19

How We Work

Cancer Centers/ SPOREs

(dollars in thousands)

2000 2001 2002 Core

Operating President’s Budget

Budget Budget

Basic/Clinical/ $173,609 $182,216 $186,771

Comprehensive

Specialized 70,591 60,916 62,439

Programs of 

Research Excellence

TOTAL 244,200 243,132 249,210
Specialized Programs of 

Research Excellence

To move discoveries more rapidly from the laboratory to patient and population settings, NCI created Specialized Programs of Research Excellence

(SPOREs). Through the SPORE mechanism, we

can award sizable amounts of flexible funding to

enable teams of researchers to address emerging scientific opportunities in disease-specific research.

Since 1992, NCI has supported SPOREs in breast,

prostate, lung, gastrointestinal, and ovarian cancers. Plans are underway to support lymphoma,

head and neck, genitourinary tract, brain, and skin

cancer SPOREs over the next several years. The

SPORE concept is being broadened beyond a disease-specific focus to include support of Centers of

Research Excellence – interdisciplinary research

teams focused on a disease modality, biologic

process, or scientific area of particular interest.

These include the Tobacco Use Research Centers

and Interdisciplinary Research Teams for Molecular

Target Assessment.

Cancer Control 

Cancer control encompasses a full spectrum of

research in the behavioral, social, epidemiologic,

and population sciences aimed at creating or

enhancing interventions that, by themselves or in

combination with biomedical approaches, reduce

cancer risk, incidence, morbidity, and mortality,

and improve quality of life. For example, a cancer

control study might investigate the use of a medical

intervention, such as a nicotine patch, in combination with a behavioral intervention, such as a counseling program to help smokers quit. Interventions

may be directed at patients, physicians, and/or

other health care providers. Cancer control

research seeks to improve interventions across the

human lifespan and over the entire cancer continuum, and to move research findings into clinical

and public health practice. The foundation of cancer control research is epidemiology. Surveillance

and outcomes research are the fundamental mechanisms for assessing progress.

NCI maintains a firm commitment to cancer

control research through its Divisions of Cancer

Control and Population Sciences and Cancer

Epidemiology and Genetics. Our wide-ranging

efforts include research in epidemiology and genetics, tobacco, tailored communications, and theoretical models for studies of human behavior and

behavior change. 

AIDS Research

Malignancies occur in more than 30 percent of

patients living with AIDS and contribute greatly to

health consequences and death. Research into the

fundamental biology of HIV and AIDS, AIDS treatment, and particularly AIDS-related malignancies

takes place throughout NCI. The IRP is an internationally recognized center for research in HIV and

AIDS, housing the HIV Drug Resistance Program,

the HIV and Malignancies Branch, and the NIH

Vaccine Research Center, a joint project with the

National Institute of Allergy and Infectious Diseases

(rex.nci.nih.gov/ RESEARCH/basic/dbslabs.htm).

The ERP also has been a vital and innovative force

in this area of research. Among its programs are the

AIDS Malignancy Consortium, the AIDS

Malignancy Bank, the AIDS Oncology Clinical

Scientist Training Program, and an annual international forum on AIDS malignancies. NCI, in coordination with other NIH Institutes and the NIH

Office of AIDS Research, continues its commitment to AIDS research and is working to ensure

that NCI-supported AIDS and AIDS-related

research is integrated with national AIDS strategies. Go to ctep.info.nih.gov/AIDSOncoResources

for more information.

20 The Nation’s Investment in Cancer Research

Cancer Control*

(dollars in thousands)

2000 2001 2002 Core

Operating President’s Budget

Budget Budget

Grants $152,256 $163,777 $166,789

Contracts 109,548 106,538 109,201

Cancer Control 104,033 105,052 107,678

Operations

TOTAL 365,837 375,367 383,668

* Cancer Control grant and contract funds are distributed

among appropriate extramural mechanisms.
Training and Education 

NCI is implementing a long-range plan for extramural training, education, and career development

designed to prepare and sustain the national cadre

of trained cancer researchers critical to current and

future endeavors. This plan focuses on attracting

young scientists into cancer research, providing stability and protected research time for researchers in

disciplines critical to translational research, creating more opportunities for scientists from underserved ethnic and minority groups, and encouraging

research program diversification. In pursuit of these

objectives, NCI has implemented a number of new

training and career development programs in basic,

clinical, population, and diversified sciences as well

as for ethnic and minority groups who are underrepresented in the research workforce.

Other Research Support 

In addition to its many types of grants and awards,

NCI employs a variety of other research support

mechanisms. These mechanisms include:

■ Contracts to provide support for research, information dissemination, and management

■ Conference grants to fund meetings, conferences, and workshops

■ Resource-related awards to foster partnerships

and meet needs for shared resources such as specialized databases and instruments

■ Scientific evaluation awards to support the scientific review of grant and contract proposals

■ Construction grants and contracts to provide

partial support for modernizing or developing

cancer research facilities throughout the Nation 

NCI also sustains, guides, and monitors the

extramural and intramural activities of the Institute

through its research management and support

activities. These activities include overall scientific

program direction and administration by grant and

contract science managers and finance, human

resources, legislation, science program direction

and assessment, and technology transfer staff. The

review and oversight activities of the National

Cancer Advisory Board and President’s Cancer

Panel are also included. This part of the budget also

supports a share of central NIH facilities and operations, and extramural program staff salaries.

(Intramural staff salaries and intramural facilities

maintenance are included under the intramural

research budget.)

How We Work 21

How We Work

Training and Education 

(dollars in thousands)

2000 2001 2002 Core

Operating President’s Budget

Budget Budget

National $58,654 $62,445 $64,006

Research 

Service Awards

Research 40,424 45,525 46,663

Career Program

Cancer Education 18,886 21,978 22,527

Program

Minority 3,600 4,023 5,250

Biomedical 

Research Support

TOTAL 121,564 133,971 138,446

Other Research Support

(dollars in thousands)

2000 2001 2002 Core

Operating President’s Budget

Budget Budget

Research $357,266 $367,286 $376,468

Support Contracts

Scientific 4,981 6,342 6,501

Evaluation Grants

Research 45,905 46,263 47,420

Resource Grants 

Construction 3,500 3,000 3,075

Conference 1,223 1,617 1,657

Grants

Research 117,643 120,600 123,615

Management 

& Support

TOTAL 530,518 545,108 558,736
I was diagnosed with a brain tumor

six months ago. As frightening as it

can be to have a brain tumor, I know

I’m lucky that mine is one of the

less aggressive types. 

There are more than 100 types of

central nervous system (CNS) tumors.

Some are highly aggressive and rapidly fatal, while others grow slowly

and may be present for years before

the patient feels any effect. People

with very slow-growing tumors may

live with them for decades and

eventually die of an unrelated cause.

CNS tumors differ from other solid

tumors because those originating in

the brain or spinal cord rarely spread

to other parts of the body. More

than 35,000 of these primary tumors

are diagnosed each year,1 16,500 of

which are cancerous.2 It has been

estimated that as many as 170,000

people develop metastatic tumors

each year from cancer cells that

have migrated to the central nervous

system from a primary tumor elsewhere in the body. CNS tumors in

children often form in different

areas and come from different cell

types than those in adults, and children’s tumors may have a different

prognosis and treatment. CNS tumors

of all types cause approximately

13,000 deaths each year.

The outlook for a person with a brain

or CNS tumor can depend almost as

much on the tumor’s location as its

type. For example, a slow-growing

(“benign”) tumor can become lifethreatening if it begins to crowd a

crucial area of the brain and cannot

be removed or treated, while a more

aggressive tumor that is in a less

critical location

or more easily

treated may

pose less threat.

My brain tumor

was discovered

after I had a

seizure, but I’ve

learned people

can have a variety of symptoms. 

Symptoms vary

markedly

depending on

the tumor’s type

and location.

Headaches (particularly morning headaches),

seizures, and mental changes such

as memory, speech, and communication problems are symptoms that

may signal a CNS tumor, or may be

related to other medical conditions.

Similarly, some people with tumors

that are causing excess pressure in

the skull may experience nausea or

vomiting, blurred or double vision,

hearing disturbances, or problems

with muscle control or coordination.

These symptoms, too, may be related

to other medical problems.

The doctor was able to remove most

of my tumor, and we’re treating the

remaining part with radiation. I’m

also going to try a new type of

chemotherapy, but I know it may not

get rid of my tumor forever.

Continuing advances in imaging

technologies are improving our 

ability to diagnose CNS tumors and

remove them more safely. Currently,

magnetic resonance imaging (MRI)

is the preferred method for diagnosing many types of CNS tumors, but

computed tomography (CT) is often

used because it is more widely available and less expensive. Positron

emission tomography (PET) is one of

several other scans now used to

augment MRI or CT, but these other

scans require the use of scarce

equipment and are costly.

Researchers are evaluating new, noninvasive modalities for tumor assessment, such as magnetic resonance

spectroscopy (MRS), which is available with routine MRI equipment.

Image-guided surgery has improved

the surgeon’s ability to remove

tumor tissue without interfering

with crucial brain functions. New

techniques such as functional MRI

People ’ s Story

Adult and Pediatric Brain Tumors

22 The Nation’s Investment in Cancer Research
People’s Story 23

that shows brain areas involved in

motor control, sensory function, 

language, and other functions, 

are improving surgical precision

even further.

After surgery, many patients are

treated with radiation. Several types

of radiation therapy are available,

including conventional external

beam radiation, and both stereotactic radiotherapy and intensity modulated radiation therapy (IMRT),

which are used to treat very small,

multiple, and hard to reach tumors.

The gamma knife is a radiotherapy

that may be used to treat certain

tumors that are inoperable with conventional surgery.

Chemotherapy for CNS tumors has

been hampered by problems in getting anti-cancer drugs across the

blood-brain barrier, a network of

vessels and cells that filter blood

going to the brain. Researchers are

developing new drug delivery techniques and new agents that can

cross the blood-brain barrier. For

example, agents have been developed that temporarily open the

blood-brain barrier while chemotherapy is administered through a vein

or artery, and biodegradable wafers

filled with chemotherapy drugs can

be placed directly into the tumor.

Chemotherapy drugs now include

those designed to kill tumor cells,

those that inhibit the development

of blood vessels that feed the

tumor, agents that cause tumor cells

to behave more like normal cells,

drugs that keep tumors from invading normal tissue, and agents that

control cell growth and enzyme 

production or action. Scientists also

are working on gene therapies for

CNS tumors.

With my type of tumor – a lowgrade astrocytoma – my doctor

says my outlook is pretty good.

I’m hoping that in the next few

years they’ll have even better

treatments for my cancer. 

Research is underway on all types of

brain and CNS tumors. In addition to

developing new imaging techniques

and treatments, scientists are working intensively to better understand

the molecular and genetic changes

that occur in CNS tumors. These 

discoveries will accelerate the development of better preventive and

therapeutic interventions, and will

improve our ability to detect CNS

tumors and monitor their response

to treatment.

I can’t help wondering what

caused my brain tumor, but mostly

I’m just trying to make the most

of my life with my wonderful wife

and our little girl.

The causes of CNS tumors are not

well understood. Six inherited

genetic syndromes have been associated with CNS tumors, but inherited

genetic alterations are not believed

to account for a large proportion of

these tumors. Gene alterations that

may lead to a CNS tumor can also be

caused by environmental factors, but

the evidence on most factors studied

to date is inconsistent. One exception is that high-dose radiation used

to treat brain tumors has been 

related to second CNS tumors in survivors who have undergone this type

of treatment. However, low-dose

radiation from diagnostic x-rays and

workplace exposures have not clearly

been found to increase risk of CNS

tumors, nor has exposure to low

energy electromagnetic fields (EMFs)

or the microwave frequencies associated with cellular telephones. It

appears that regular exposure to

some categories of chemical agents

(e.g., N-nitroso compounds, some

pesticides) may increase the risk of

CNS tumors, but more research is

needed in this area. Similarly,

researchers are exploring whether

exposure to certain viruses may

affect CNS tumor risk. But the

majority of people with CNS tumors

do not have any of these possible

risk factors.

Most people living with CNS tumors

have symptoms that may change

over the course of their disease. An

important area for further research

is to learn how to help people cope

most effectively with the physical

and psychological impact of these

diseases, and the possible shortand long-term side effects of their

treatment.

This year, NCI convened a Progress

Review Group on adult and childhood brain tumors to assess the 

current state of knowledge and care

for this group of diseases, and identify research priorities for the

future.

Note: This vignette is a composite of experiences.

1 Central Brain Tumor Registry of the U.S.

2 NCI Surveillance, Epidemiology, and 

End Results (SEER) cancer registry program.

People’s Story
NCI’s Challenge: 

Building Our Capacity for the Future

We are entering the 21st century with everexpanding knowledge and an array of

sophisticated tools for continuing the fight

against cancer. The challenge before us is to build

and continually enhance an infrastructure that will

allow the scientific community to apply new discoveries and emerging technologies. We need

mechanisms that will promote and reward innovative thinking, the cross-fertilization of ideas among

disparate scientific disciplines, and enhanced collaborations among government, academia, and

industry. We must develop and maintain a cadre of

trained scientists from a variety of disciplines. And

we must address special societal concerns that

impact our Nation’s ability to provide the best possible treatment to cancer patients and to ensure

equal access to information, to care, and to research

opportunity. 

NCI must provide the vision, creative environments, and diverse resources needed to ensure a

smooth flow between the increasing number of discoveries and advances in cancer research and the

scientific community’s ability to apply these findings. If the pace of discovery is like an eight-lane

highway, our current ability to translate those discoveries into clinical application is still much like a

country road. Where the two meet, a bottleneck

still prevents a tremendous number of good ideas

from moving forward. Our challenge is to continue

to expand the country road and to move discoveries to their application in interventions across the

cancer care continuum. To respond to this challenge, we identified six key areas for investment in

our 2001 proposal and will continue these in 2002. 

As we work to address this challenge, other barriers limit our success. Two issues of special concern

are the quality of cancer care and disparities in

access to information, patient care, and research

opportunities and careers. We are adding two new

challenge areas for 2002 to address these needs. 

24 The Nation’s Investment in Cancer Research

Budget Increase Request for 2002

(dollars in thousands)

Investigator-Initiated Research $124,561

Centers, Networks, and Consortia 93,500

National Clinical Trials Program 328,000

Studying Emerging Trends in Cancer 47,300

Quality of Cancer Care 21,500

Reducing Cancer-Related 

Health Disparities 50,600

Informatics and Information Flow 77,250

Training, Education, and 

Career Development 68,000

Total $810,711 National Clinical

Trials Program

40.5%

InvestigatorInitiated 

Research

15.4%

Centers, 

Networks, and

Consortia

11.5%

Studying 

Emerging Trends

in Cancer

5.8%

Quality of 

Cancer Care

2.7%

Reducing CancerRelated Health

Disparities

6.2%

Informatics and

Information Flow

9.5%

Training,

Education, 

and Career

Development

8.4%
NCI’s Challenge

NCI’s Challenge: Building Our Capacity for the Future

NCI’s Challenge 25

THE CHALLENGE

I

nvestigator-initiated research – research conceived and conducted by scientists in laboratories and clinics across the country and at NCI – is

the wellspring of scientific discovery. Funded and

sustained by a variety of NCI grant mechanisms,

investigator-initiated research is continually yielding discoveries and insights into the mechanisms

and causes of cancer and its prevention, detection,

diagnosis, treatment, control, and survival.

Because of the steadfast national commitment to

cancer research, scientists are beginning to understand how cancer develops and progresses at the molecular level and

how to use this knowledge to effectively prevent, diagnose, and treat

cancer. We now must not only maintain our current momentum but must

push forward as quickly as possible

with critical research and the application of findings to prevention and

treatment interventions. The pace

and absolute number of discoveries

and the speed with which we can

bring them to bear to benefit people,

are directly linked to the resources

available to support the exploration of new leads

across the cancer research continuum. We must:

■ Expand our research portfolio to include a

greater number of proposals that may be somewhat speculative or that pursue novel paths. 

■ Continue to expand the translational research

that converts basic science discoveries into practical, affordable, and effective ways of restoring

cancer patients to health and preventing cancer

throughout our population. 

■ Create mechanisms that link basic, clinical, and

population-based research with state-of-the-art

resources and technologies; promote collaborations among researchers inside and outside of

cancer research; and draw into cancer-related

research scientists from allied fields such as

chemistry, biology, physics, engineering, and

mathematics to galvanize their complementary

knowledge and most quickly answer the crucial

questions in basic, clinical, population, and

translational research.

The Nation waits eagerly for the day when cancer

is no longer a threat to health and life, when most

cancers are prevented, and when those that occur are

cured or controlled rapidly and successfully. Meeting

this challenge requires increased investment in this

essential aspect of the research infrastructure, to span

the gap between discovery and application and transform the processes by which we bring

discoveries to the benefit of people.

PROGRESS TOWARD

MEETING THE CHALLENGE

Over the past four years, NCI has

increased substantially the number

of investigator-initiated grant applications funded. In 1995, the overall success rate for grants funded from the

Research Project Grant (RPG) pool was

23 percent. In 2000, we anticipate funding approximately 1,175 new and competing grant

applications from the RPG pool, for an overall success

rate of 30 percent. This increase has resulted in and

will continue to yield substantial research rewards, but

this improved funding level is still insufficient to support the wealth of innovative, high quality proposals

received each year. As we achieve success in attracting

new researchers to the cancer problem, the number of

excellent applications is expected to grow. 

To encourage the development of new technologies and innovative approaches to areas with special

needs, NCI has implemented several new or expanded

mechanisms. For example, the Quick-Trials program promises to speed the translation of ideas developed in the laboratory to early stage clinical trials by

simplifying the grant application process and providInvestigator-Initiated Research

GOAL

Accelerate discoveries 

and their application 

by expanding 

and facilitating 

researcher access to

resources and new 

technologies.
26 The Nation’s Investment in Cancer Research

THE PLAN – INVESTIGATOR-INITIATED RESEARCH

Goal

Speed the rate of discovery and accelerate the application of those discoveries to the population by

expanding and facilitating researchers’ access to resources and new technologies.

Objectives and Milestones for Fiscal Year 2002

1. Increase investment in research to ensure a higher and sustainable success rate for

competing grant applications. 

■ Fund 40 percent of competing applications, including those that: (1) have the

highest scientific merit, (2) may have low probability of success but may yield

potentially greater reward if they do succeed, (3) are unconventional but hold

unique promise, (4) are in areas of extraordinary need in specific fields of investigation or model systems, or (5) encourage new investigators.

2. Encourage investigators to commit to careers in cancer research and to propose

more innovative and higher reward projects.

■ Continue to allocate the first 80 to 90 percent of available RPG funds to competing applications that fall within conventional merit rank order paylines.

■ Ensure that applications that are particularly innovative and high reward have a

reasonable funding rate by identifying such research and using exceptions funding

and special competitions as warranted to maintain a comparable overall success rate.

3. Enhance the pace of high priority targeted research objectives by searching for outstanding applications that fall just beyond established paylines.

■ Ensure that applications from new investigators have a success rate equal to grants

from more established investigators.

■ Allocate up to 10 percent of competing RPG funds for meritorious applications

outside conventional paylines. 

■ Continue AER to fund single project applications in patient-oriented and basic

research that are near the payline and for which reviewer criticisms can be

addressed rapidly. Provide parallel expedited peer-review approaches for Program

Project Grants.

4. Invest in areas that NCI consensus planning processes (e.g., progress review

groups, extraordinary opportunity working groups, advisory committees) and staff

have identified as presenting special opportunity or need. 

■ Set aside 10 to 15 percent of funds for Requests for Applications (RFAs), Program

Announcements (PAs), and other applications that target identified gaps and/or

emergent opportunities. Monitor investigator-initiated research applications to assess

whether these projects alone are meeting programmatic objectives, including those

focused on special needs identified in specific disease areas. Use regular, novel, and

special award mechanisms to encourage investigation in priority areas. 
NCI’s Challenge 27

NCI’s Challenge

■ Provide sufficient staff, other resources, and enhanced electronic communications

to improve information dissemination, enhance coordination within and among 

initiatives, and increase direct contact with applicants and grantees.

5. Optimize each award to accelerate the pace of discovery. 

■ Provide funding at full peer-reviewed recommended budget levels for up to five

years of support. (See the NCI funding plan at cancer.gov/admin/fmb.)

■ Ensure that investigators have access to NCI-sponsored resources, infrastructure,

and technologies that facilitate discovery, both to expedite progress and to create

breakthrough opportunities.

■ Triple the amount of funding set aside for administrative supplements to current

awardees whose research is going better than expected, or who are poised to test

new ideas.

■ Expand R03, R21, R33, P20 and other award mechanisms that provide seed funds

and resources to pilot promising leads. (Go to deainfo.nci.nih.gov/flash/

awards.htm#menu for information on NCI funding mechanisms.)

6. Facilitate rapid movement from discovery to application by encouraging 

transdisciplinary and collaborative approaches.

■ Use established mechanisms and create novel and special awards to encourage 

collaborative and translational research.

■ Expand administrative supplements to encourage new collaborations that bring

together basic and clinical scientists and promote additional interdisciplinary 

collaborations and access to central resources such as databases, tissue banks, and

animal models. Expand access to resources and technologies that promote interdisciplinary research and collaborations through centers, networks, and consortia.

(See pp. 30-31, Objective 2.)

■ Expand cooperative resource programs (e.g., RAID, RAPID, CGAP, EDRN) that

give investigators access to technologies and expertise needed to move their discoveries to application. (See pp. 30-31, Objective 2; pp. 78-79, Objectives 2 and 3;

and p. 84, Objective 2.)

■ Encourage the development of information technology tools to foster and enhance

interdisciplinary communication and collaboration.

■ Double the funding for collaborative research awards such as Program Project

Grants and cooperative agreements for networks in cancer genetics, imaging, early

detection, and other areas.

■ Expand use of exploratory grants to encourage patient- and population-based research.

Total

$124.6 M
ing a rapid turnaround from application to funding.

Initially developed as a pilot program in prostate

cancer, this successful initiative has been expanded

to address all cancer sites and provide funding to test

and develop novel cancer therapies. 

The Phased Innovation Awards program, developed initially to support novel technology development for molecular analyses of cancer, will be

broadened in scope in 2001 to fund projects to

advance imaging technology. In addition, the

Institute has expanded the Award to include a 

follow-on Phased Technology Application Award

to ensure subsequent development towards an

applied result. Seven grants were funded through

this program in Fiscal Year 2000. 

In 1999, NCI used the Administrative

Supplements for Advanced Technologies awards

to fund over 20 centers to develop DNA array technology facilities. These grants promise to make this

exciting technology available to hundreds of investigators at least a year earlier than otherwise would

have been possible.

Through a special exceptions process, NCI

ensures that new investigators share the same success rate as more established applicants. In addition,

the Accelerated Executive Review (AER) process

ensures rapid reconsideration of applications

ranked within a few percentile points of the payline, especially those involving patient-oriented

research. In Fiscal Year 2000, over $35 million has

been spent on exceptions and more than 100 investigators benefited from these initiatives.

Budget Increase Request for 2002

Investigator-Initiated Research

1. Ensure a higher and sustainable success rate for 

competing grant applications.

2. Encourage careers in cancer research and more 

innovative and high reward projects.

3. Enhance the pace of high priority targeted research

beyond established paylines.

4. Invest in areas identified as presenting special 

opportunity or need.

5. Optimize each research award to accelerate the 

pace of discovery.

6. Encourage transdisciplinary and collaborative approaches.

Total $124.6 M

28 The Nation’s Investment in Cancer Research

Investigator-Initiated

Research

Unsolicited

Each year NCI receives over 8,000 applications for

research funding covering virtually all areas of science and medicine relevant to cancer. The vast

majority of these applications are unsolicited and

come from researchers representing a wide range of

disciplines driven by the synergism present at their

medical schools, hospitals, universities, research

centers, and corporations all across our nation and

around the world. Proposals selected for funding by

peer reviewers are those that (1) ask important

questions, (2) present novel and technically

advanced approaches, and (3) hold promise for

advancing the state of knowledge in a particular

area. The sheer number of applications and a stringent review process together generally result in a

fairly balanced and comprehensive research portfolio focusing on all aspects of cancer-related science and technology.

Solicited

In addition, NCI’s ongoing planning and evaluation

processes serve to further identify imbalances,

gaps, or opportunities. Set-aside funds are established to channel research support into areas not

adequately covered through unsolicited proposals,

and Requests for Applications and Program

Announcements are issued to solicit proposals in

these areas. Judicious use of funding solicitations

can cut years off the time needed to develop a

critical mass of research in an emerging field and

may be used to jump start new initiatives, such as

those developed to implement the “Extraordinary

Opportunities” outlined in this document. And

funding solicitations permit NCI to coordinate and

facilitate collaboration across institutional boundaries. Set asides also serve to assure investigators

that NCI will commit support for an area, even

though a problem may not have fared well previously in peer review or may seem intractable

because progress has been difficult to achieve.
NCI’s Challenge

NCI’s Challenge: Building Our Capacity for the Future

NCI’s Challenge 29

THE CHALLENGE

NCI’s mission to translate scientific knowledge

into more effective cancer interventions is challenged by the conventional ways research is conducted. Basic scientists, clinical scientists, population scientists, and behavioral scientists develop

their skills in distinctly different ways and in varied

environments with far too little emphasis on communicating and working with each other except in

very select environments. However, the rapid pace

of scientific and technological discovery is creating

enormous opportunities that require the close interaction and collaboration of clinical

and laboratory scientists from across

the research community. The challenge for NCI is to create integrated

research environments that foster the

complex multidisciplinary interactions needed to address the “big picture” problems in cancer research.

These integrated research environments must functionally link

basic, clinical, population, and

behavioral scientists to each other and

to newly developing, diverse fields of

science and technology. These scientists must have easy access to many different

patients and at-risk populations, tissue banks, new

technologies, and state-of-the-art informatics.

They must be able to work together with the same

ease and flexibility in multi-institutional research

settings as in the same institution.

To meet this challenge, NCI continues to create

and nurture a new overarching structure for research

composed of NCI-designated Cancer Centers,

Centers of Research Excellence, networks, and consortia. These infrastructures are enhancing the traditional research enterprise in ways that promote

and facilitate complex scientific interactions, provide the critical resources essential for the research,

and encourage the easy exchange of information

and ideas through new communications linkages. 

NCI-Designated Cancer Centers

NCI-designated Cancer Centers organize and integrate multidisciplinary research across departments

and schools within a single institution. Cancer centers provide scientists access to the most advanced

technologies and new research opportunities and

bring the benefits of their research directly to the

public. They link state-of-the art research and clinical care activities within the institution and form

key partnerships with industrial, community, and

state health organizations outside of the institution.

For example, the disease-specific Specialized

Programs of Research Excellence (SPOREs),

designed to move discoveries from the

laboratory into patient and population research settings, had their origins in cancer centers. The new

Special Populations Networks for

Cancer Awareness Research and

Training are designed to link local,

community, and regional problems of

cancer in underserved populations to

the broad-based research capabilities

of NCI-designated Cancer Centers.

Centers are critical in a new NCI initiative to incorporate Minority

Serving Institutions (MSIs) into

NCI’s cancer research, education, training, and

outreach activities. The Cancer Genetics Network

(CGN) sites are headquartered in centers. Nearly

all of the participants in the Mouse Models of

Human Cancer Consortium (MMHCC) are in

NCI-designated Cancer Centers. Centers have

worked closely with industry in developing new

cancer therapeutic agents and are rapidly becoming

significant partners with industry for new technology development. 

NCI will create Regional Enhancement Cancer

Centers to facilitate partnerships between smaller

institutions and the large, existing NCI-designated

Comprehensive Cancer Centers. These partnerships will provide patients and populations with

much improved access to the newest clinical, 

Centers, Networks, and Consortia

GOAL

Create and sustain 

research infrastructures 

for collaboration, 

technology support 

and development, 

and access 

to resources.
30 The Nation’s Investment in Cancer Research

THE PLAN – CENTERS, NETWORKS, AND CONSORTIA

While the NCI’s goal for collaborative research applies to all NCI-designated Cancer Centers, 

networks, and consortia, this plan focuses primarily on the objectives and resources for the centers and

SPOREs. Many networks and consortia activities are budgeted and discussed in the Extraordinary

Opportunities and NCI Challenge sections throughout this document. 

Goal

Create and sustain research infrastructures for collaboration, technology support and development,

and access to resources that enable multiple scientific disciplines to address large problems in 

cancer that could not be solved by individual investigators.

Objectives and Milestones for Fiscal Year 2002

1. Increase the number and broaden the geographic distribution of NCI-designated

Cancer Centers.

■ Designate 2 new cancer centers. $2.50 M

■ Award 2 new Cancer Center Planning Grants. $0.50 M

■ Establish 3 Regional Enhancement Cancer Centers that will collaborate with 

NCI-designated Comprehensive Cancer Centers to expand the base of patients

and populations available for early detection, prevention, and therapeutic research

studies. $2.00 M

■ Award 5 Cancer Center Supplements to encourage inter-center research 

collaborations when patient, community, and regional responsibilities are in 

competition or when combining resources can address important questions more

effectively. $3.00 M

■ Establish formal affiliations between cancer centers and MSIs in the form of 2 comprehensive partnerships, one planning grant for a comprehensive partnership, and

10 planning grants to increase the number of minorities engaged in cancer research,

enhance research capabilities of MSIs, and improve the effectiveness of cancer 

centers in serving minority communities. (See p. 59, Objective 4 for the training

component of these partnerships.) $7.00 M

2. Expand the capacity of cancer centers to engage in newly developing areas of

research and technology and to act as platforms for translating discoveries into

interventions.

■ Establish 10 Advanced Technology Programs in cancer centers to enable work with

industry to develop, access, and export the newest technologies for solving important problems in cancer research. $7.50 M

$15.0 M

$36.0 M

prevention, and control trials in early detection,

prevention, and therapeutic research. NCI anticipates that the centers also will play a key role in

integrating and coordinating NCI-supported centers of excellence, networks, and consortia into one

overarching, unified research framework. Go to

cancer.gov/cancercenters for more information.

Centers of Research Excellence

Centers of Research Excellence bring together

interdisciplinary and translational research teams

focused on a specific disease, modality, biologic

process, or scientific area. They are awarded sizeable amounts of flexible funding to enable them to

rapidly address emerging scientific opportunities. 
NCI’s Challenge

NCI’s Challenge 31

■ Increase funding to all cancer centers to encourage scientists in centers to develop

new technologies and methodologies for entirely new approaches to answering

important cancer research questions. $6.00 M

■ Establish 10 Informatics Planning Activities in cancer centers to build, in partnership with NCI, critical informatics capabilities in data acquisition, analysis, integration, and coordination. $7.50 M

■ Provide additional funding to build the clinical research and population research

infrastructure of cancer centers. Fund databases that conform to NCI’s clinical

informatics infrastructure, support population studies, and provide more core staff

to conduct innovative translational therapeutic and prevention trials. $15.00 M

3. Expand and enhance the research of Specialized Programs of Research 

Excellence (SPOREs).

■ Expand the SPORE program scope to include one in breast cancer and one in

prostate cancer. Add the following SPOREs: 2 in head and neck cancer, one in

brain cancer, and one in lymphoma. Continue implementing the transition plan to

include all cancer sites by Fiscal Year 2005. $25.00 M

■ Provide supplements to SPOREs for planning and developing complex inter-SPORE

research projects and for collaborative projects with other NCI centers of research

excellence, networks, and consortia. $5.00 M

■ Support development of an Internet platform and research database to enable

SPOREs to exchange research results and to foster communications for sharing

resources and developing collaborative inter-SPORE research projects. $1.00 M

4. Develop a system for linking and managing the entire research framework of 

centers, centers of research excellence, consortia, and networks.

■ Create a flexible management system for planning, initiating, and completing 

complex collaborative research projects that provides for specialized short-term

resources and research support. $5.00 M

■ Develop a communications network to identify areas of common interest, share

research information and resources, develop a continuing research dialogue, and

identify areas of potential collaboration. $5.00 M

Management and Support

Total

$31.0 M

$10.0 M

$1.5 M

$93.5 M

The first of these centers, the SPOREs, were created in 1992 and focused on specific cancers. They

serve as highly effective hubs for translational

research, moving discoveries back and forth among

laboratory, clinic, and population research settings.

To date, SPOREs have been established in breast,

prostate, lung, gastrointestinal, and ovarian cancer.

The SPOREs blueprint has been used to establish similar programs in other extraordinary opportunity research areas including Transdisciplinary

Tobacco Use Research Centers, In Vivo Cellular

and Molecular Imaging Centers, and

Interdisciplinary Research Teams for Molecular

Target Assessment. All of these support interactive,
32 The Nation’s Investment in Cancer Research

multidisciplinary research and provide resources,

flexible exploratory funds, training, and career

development. NCI also will collaborate with the

NIH Center for Complementary and Alternative

Medicine to establish centers of excellence. 

Networks and Consortia

Networks and consortia link the expertise and

innovation of scientists from different disciplines

and diverse research backgrounds to address important questions and issues about cancer. The CGN

addresses the issue of inherited predisposition to

cancer and is linking its goals and objectives to

those of SPOREs and NCI-designated Cancer

Centers. The newly established MMHCC will work

closely with SPOREs to develop mouse models that

reflect various precancerous and cancerous stages of

human cancer. The Diagnostic Imaging Network, a

multi-institutional team of scientists, is evaluating

and developing a new generation of imaging concepts and tools with device manufacturers and

other technology developers. The Early Detection

Research Network, which facilitates the discovery,

development, and initial steps in clinical validation

of molecular markers and assays that detect early

signs of cancer, is already interacting with SPOREs

and other interdisciplinary teams of scientists. The

SPNs will involve underrepresented racial, ethnic,

and minority communities in establishing research

priorities and conducting research that will benefit

these populations. The Pediatric Brain Tumor

Consortium, a network of 10 medical centers, is

evaluating promising treatments for children with

brain malignancies.

Networks and consortia interact with NCIdesignated Cancer Centers and Centers of

Research Excellence in a seamless way to advance

our understanding of cancer and to improve cancer

prevention, early detection, diagnosis, and treatment. 

PROGRESS TOWARD MEETING 

THE CHALLENGE

NCI is completing the foundation for overarching research frameworks that will bring diverse

scientific disciplines together across institutional

boundaries. NCI-designated Cancer Centers continue to evolve as key strategic partners of NCI. In

2000, NCI added a center in Iowa and will fund a

new planning grant for developing a center in New

Mexico. In addition, the number of cancer centers

with the “Comprehensive” designation increased in

2000 to 37. Finally, NCI has been working with

institutions in Georgia, New York, Louisiana,

Rhode Island, Florida, West Virginia, Kentucky,

Arkansas, South Carolina, and Michigan to develop

cancer centers or become Regional Enhancement

Cancer Centers. 

In 2000, the SPORE program added a new cancer site and funded four ovarian cancer SPOREs.

These research teams already are establishing

research links and preparing to conduct inter-institutional research that cannot be accomplished

through any other venue. In addition, the SPORE

program has become open to all types of cancer

through a transition plan that will gradually add

new cancers over the next five years. By the end of

Fiscal Year 2001, NCI expects to support SPOREs

in breast, prostate, lung, ovarian, genitourinary

tract, gastrointestinal tract (pancreatic and colorectal), and skin cancers. 

NCI recently launched the Minority Institution/

Cancer Center Partnerships (MICCPs) to engage

minority institutions in NCI’s research activities,

including training, education, and community outreach. The program will engage five major institutions with medical schools (Drew, Meharry,

Howard, Morehouse, and the University of Puerto

Rico) and also will provide opportunities for over

300 other smaller institutions throughout the Nation

to partner with NCI-designated Cancer Centers. 

Budget Increase Request for 2002

Centers, Networks, and Consortia

1. Increase the number and broaden the 

geographic distribution of NCI-designated 

Cancer Centers. $15.0 M

2. Expand the capacity of cancer centers to 

engage in newly developing areas. 36.0 M

3. Expand and enhance SPOREs. 31.0 M

4. Develop system for linking and managing 

entire research framework. 10.0 M

Management and Support 1.5 M

Total $93.5 M
NCI’s Challenge

NCI’s Challenge: Building Our Capacity for the Future

NCI’s Challenge 33

THE CHALLENGE

During the 1990s, the number of new cancer preventive, diagnostic, and therapeutic agents tested

in clinical trials increased dramatically. New cancer

interventions in development by industry increased

threefold to over 350. In 2000, NCI added to existing trials on 80 promising interventions for treatment and 35 for prevention, 30 more agents identified through its own drug discovery programs or in

collaboration with academic institutions or industry. 

But our present clinical trials system cannot

keep pace with the growing number of agents that

merit testing. As potential prevention and treatment strategies emerge

from early clinical testing, NCI is

faced with a backlog of agents that

need to enter large Phase III trials,

the final crucial step in translating

new discoveries into effective new

treatments and prevention strategies

for patients. NCI and its grantees are

able to initiate only about 30 Phase

III treatment trials each year.

Compounding this problem is the

fact that fewer than three percent of

patients with cancer participate in

the clinical trials that might define effective new

treatment approaches. For a variety of reasons

including limited access, lack of insurance coverage,

patient-physician communication issues, and therapy choice, only about 20,000 patients enter Phase

III trials each year. This means that it can take over

four years just to recruit enough patients for the

average Phase III treatment trial. Nearly 90 Phase II

and III prevention trials also have been initiated,

but progress in this area is limited by lack of methods such as the use of biomarkers to determine the

effect of a preventive over a short period of time,

lack of technologies to improve precision in characterizing precancerous lesions, and the growing

number and complexity of trials needed to determine the roles of a new generation of agents. 

To more efficiently convert recent scientific discoveries into effective interventions, eliminate the

backlog of agents for testing, and increase access for

patients and individuals at risk, we must invest in,

restructure, and increase the capacity of our national

clinical trials program. While the time from initiation

of clinical trials to Food and Drug Administration

(FDA) approval has improved, the clinical development required to establish the best uses of new agents

still occurs over a much longer period. 

Changes to NCI’s clinical trials system will

increase the number of trials and the

number of individuals who enroll in trials by easing the way for physicians to

communicate with patients and enroll

them in trials. Results of laboratory

studies to determine why particular

drugs are effective in some individuals

and not in others will help tailor

treatments for cancer patients and

interventions for people at risk for

cancer. These enhancements will

speed the development of new agents

and alleviate the backlog of treatments awaiting evaluation.

National Clinical Trials Program

GOAL

Ensure that clinical 

trials address the most

important questions 

in prevention and 

treatment and 

are broadly 

accessible.

Budget Increase Request for 2002

National Clinical Trials Program

1. Address compelling clinical questions. $ 16.5 M

2. Increase development and clinical testing 

of promising new agents. 279.5 M

3. Increase participation in clinical trials by 

patients, individuals at risk for cancer, 

and their physicians. 22.5 M

4. Reduce outcome disparities in special 

populations by increasing trials access. 4.0 M

Management and Support 5.5 M

Total $328.0 M
34 The Nation’s Investment in Cancer Research

THE PLAN – NATIONAL CLINICAL TRIALS PROGRAM

Goal

Ensure that the clinical trials program is poised to address the most important medical and 

scientific questions in cancer prevention and treatment quickly and effectively through 

state-of-the-art clinical trials that are broadly accessible to cancer patients, populations at 

risk for cancer, and the physicians who care for them.

Objectives and Milestones for Fiscal Year 2002

1. Identify and address compelling clinical questions confronting physicians and their

patients struggling with cancer. 

■ Conduct State of the Science meetings in gastrointestinal, lung, genitourinary, and

prostate cancers and leukemia to identify important research questions and define a

scientific research agenda to address them. $1.00 M

■ Expand Concept Evaluation Panels beyond initial lung and genitourinary 

cancer pilots to include peer review of all concepts for Phase III trials sponsored 

by NCI. $0.50 M

■ Provide scientific leadership funds to support researchers who chair studies and

statisticians responsible for writing, monitoring, and analyzing NCI-sponsored,

high-priority Phase III trials. $3.00 M

■ Provide translational research funds for clinical correlative studies to uncover 

the mechanisms of action, response, and resistance underlying new treatments 

and preventive strategies and to translate basic biology from the laboratory to 

clinical practice. $8.00 M

■ Support a national tissue resource system that includes normal, precancerous, and

cancer tissues to facilitate rapid evaluation of new assays and relevant clinical 

correlations as new targets are identified. (See p. 78, Objective 2.)

■ Fund tissue and specimen banks to store material from cancer patients 

undergoing treatment and those at risk of developing cancer to allow later studies

of drug effectiveness, molecular abnormalities, and clues to tumor initiation and

progression. $4.00 M

■ Develop and make widely available through Centers of Research Excellence molecular assays required to characterize/classify tumors. (See pp. 30-31, Objective 2.)

■ Better integrate treatment and prevention research into the clinical trials 

infrastructure. 

■ Incorporate behavioral, epidemiologic, outcomes and other relevant research, to

effectively address cancer in specific tumor types and patient populations. 

■ Incorporate the evaluation of relevant biomarkers into clinical trials. 

2. Increase the pace of development and clinical testing for promising new 

therapeutic and preventive agents. Over 2-3 years, triple the number of promising

agents entering NCI-sponsored clinical trials, triple annual patient accrual to early

clinical trials of promising agents, quadruple accrual to pivotal or proof-of-principle

early clinical trials, and double accrual to Phase III clinical trials. 

■ Expand resources for the RAID and RAPID programs. (See p. 84, Objective 2.)

■ Increase funding for early therapeutics development contracts to fund multiinstitutional consortia that are able to accrue many patients rapidly and perform

sophisticated correlative laboratory studies. $13.00 M

$16.5 M

$279.5 M
NCI’s Challenge 35

NCI’s Challenge

■ Fund Interdisciplinary Research Teams in Interventions Directed at Molecular

Targets to develop the necessary assays, tools, and approaches to assess the effects

of promising new agents on their molecular targets. $6.50 M

■ Fund Cooperative Groups and practice sites to support data management and allow

physician participation in clinical trials at double to triple the current rate through

the CTSU. $245.00 M

■ Support a rapid grant review process for mechanism-based clinical trials. 

■ Support the infrastructure needs of the intramural clinical trials program at the

NIH Clinical Center by increasing the numbers of data managers, research nurses,

biostatisticians, and clinicians required to support a critical mass of clinical investigators; continue to develop the net-Trials database system to link all NCI intramural clinical investigations and as a prototype that other institutions can use; and

continue the Tissue Array Research Program (TARP) to identify key molecular

alterations in cancers. $15.00 M

3. Substantially increase participation in clinical trials by patients, individuals at risk

of developing cancer, and the physicians who care for them.

■ Use the EPP to fund new participating physicians, including new minority physicians, to hire the necessary research nurses and data managers for effective trials

participation. $2.50 M

■ Expand the CTSU to consolidate all administrative tasks associated with clinical 

trials. $20.00 M

■ Provide extensive information about prevention and treatment options and 

clinical trials to enable patients and physicians to make informed medical choices.

(See pp. 54-55, Objective 2.)

■ Develop uniform data reporting mechanisms and informatics support to facilitate

clinical trials participation. (See p. 55, Objective 3.)

4. Reduce outcome disparities in special populations by increasing access to state-ofthe-art clinical trials in cancer prevention and treatment. 

■ Create a Clinical Trials Outreach Program to increase participation by 

underrepresented populations; establish clinical trials units at historically black

medical institutions; strengthen clinical trials units at minority-based community

oncology sites. $3.00 M

■ Increase clinical trials participation by minority physicians and health professionals by

implementing an NCI fellowship training program in clinical trials for minority physicians and forums for minority scientists’ input into developing clinical trials that

address issues of special importance for minority and special populations. $1.00 M

Management and Support

Total

$22.5 M

$4.0 M

$5.5 M

$328.0 M
36 The Nation’s Investment in Cancer Research

PROGRESS TOWARD MEETING 

THE CHALLENGE

We are finding better ways to generate new

ideas. State of the Science Meetings identify

disease-specific research gaps and opportunities,

advance findings into clinical evaluation, and stimulate integrated translational research. Pilot

Concept Evaluation Panels evaluate Phase III clinical trial proposals. The Quick-Trials program simplifies procedures and provides rapid turnaround for

the funding of clinical trials on new agents and

therapeutic approaches. The Clinical Oncology

Special Emphasis Panel provides the clinical oncology community with its own NIH study section for

reviewing clinical trials and translational research.

Clinical correlative science studies will deepen our

understanding of the relationship between tumor

characteristics and patient outcomes. 

We are broadening access to trials. Funding for

Cooperative Groups, through which most patients

enter clinical trials, has increased over 50 percent

since 1999. The Expanded Participation Project

(EPP) extends access to NCI-sponsored Cooperative

Group studies to oncologists who are not Group

members. Special Populations Networks for Cancer

Awareness Research and Training have raised the

proportion of minorities participating in treatment

trials to nearly 20 percent. A Physician

Communication Module developed with the

Howard University Cancer Center will provide

tools and technology for physicians to enroll and

follow patients on clinical treatment protocols.

NCI’s payer and provider partnerships with the

Department of Defense TRICARE/CHAMPUS

health care system and the Department of Veterans

Affairs provide coverage for trials participation by

military personnel and veterans. Agreements with

health plans such as Aetna-U.S. Health Care and

United Health Care Corporation cover costs for

patient care in trials. Medicare beneficiaries are now

guaranteed coverage for participation in clinical trials as a result of a presidential directive in June 2000.

We are communicating and educating people

about trials. The Cancer Clinical Trials Education

Program provides training to nurses and social

workers to educate patients and their families about

clinical trials. We are partnering with professional

societies and health plan organizations to develop

Web-based and self-paced modules about clinical trials participation for physicians and nurses. In collaboration with other NIH institutes and national

ethics groups, NCI is developing a Web-based

Human Subjects Protection Education program for

investigators. Two Web sites provide extensive information on clinical trials to physicians and patients.

CancerNet (cancernet.nci.nih.gov) hosts NCI’s

searchable clinical trials database containing information on over 1,800 active clinical trials, and

cancerTrials (cancertrials.nci.nih.gov) is NCI’s

online gateway to clinical trials resources. The latter

integrates information about clinical trials participation (including human subjects’ protection), treatment advances, and related resources for patients

and physicians. The Cancer Trials . . . Because Lives

Depend On It project is testing a new approach to

participant accrual where partner organizations

recruit “campaign ambassadors” to training programs

and encourage their communities to participate in

trials. New Minority Clinical Oncology Awards provide underrepresented minority health professionals

with the opportunity to gain expertise in research.

We are streamlining procedures and automating

data systems. The Cancer Trials Support Unit

(CTSU) streamlines and centralizes administrative,

financial, and data collection tasks. The submission

and development of ideas for protocols are made easier through electronic forms, templates, and databases. A Central Institutional Review Board (IRB) is

available to review protocols for multi-center trials,

reducing the administrative burden on individual

institutions. An informed consent template improves

patient understanding of cancer trials participation.

The Clinical Trials Monitoring Branch Audit

Information System permits online submission of

data collected during quality assurance audits. The

Clinical Data Update System will standardize and

streamline data reporting and reduce administration.

Common Data Elements and Common Toxicity

Criteria promote a common vocabulary among cancer research organizations. The Adverse Event

Expedited Reporting System is a Web-based system

for reporting to NCI serious or unexpected events. 

We continue new initiatives through our

Intramural Clinical Program including: a new program in neurooncology/brain tumor research in collaboration with the National Institute on

Neurological Disorders and Stroke, expansion of

the cDNA Microarray Facility to support clinical

research for finding new biomarkers, the Tumor
NCI’s Challenge

NCI’s Challenge 37

Vaccine Program through which NCI is initiating

clinical trials to test the efficacy of anti-lymphoma

and anti-melanoma vaccines, and the Radiation

Oncology Sciences Program for linking intramural

and extramural initiatives in improving radiation

treatment. Intramural researchers also are exploring

new imaging approaches to cancer. 

For more information on the cancer trials revitalization efforts described here, go to cancertrials.nci.nih.gov and select “New Trials System.”

Over the past century we have gained considerable

knowledge about how the immune system fights off foreign invaders and how cancer eludes this defense system. The application of this knowledge to creating

immunotherapies, such as immunotoxins and vaccines

that harness the immune system, is yielding promising

results for both treating and preventing cancer.

Recent research shows that an immunotoxin (LMB-2), a

type of treatment created by linking a monoclonal antibody to a deadly toxin, may be effective against hairy

cell leukemia (HCL), a rare cancer of immune system B

cells. When given to a patient, the antibody portion of

LMB-2 homes in on a cancer cell, attaches to it, and

then delivers its poison directly to the cell, thus killing

it. In a phase I clinical trial of LMB-2, NCI researchers

saw anti-tumor activity against a variety of malignancies

including chronic lymphocytic leukemia, Hodgkin’s disease, cutaneous T-cell lymphoma, and most strikingly,

HCL. Four out of four HCL patients who received LMB-2

responded to the treatment. One patient had a complete

remission that has lasted almost two years and the

other three patients had a reduction of 98 percent or

more in malignant cells circulating in the blood. Phase

II trials of LMB-2 will begin soon.

Vaccines are another type of immunotherapy. Prevention

vaccines, like the polio vaccine or childhood immunizations, evoke an immune response before infection

strikes, creating antibodies that will later ward off a

challenge. Prevention vaccines for cancer are similarly

designed to target cancer-causing agents, such as the

sexually transmitted human papillomavirus (HPV) which

is linked to over 90 percent of cervical cancer cases

worldwide. HPV is now believed to cause some types of

oral cancers, too, such as tumors found in the tonsils.

Researchers are hoping that inoculation with inactivated

HPV particles will enable the immune system to recognize and establish a defense against the virus.

Worldwide, at least six vaccines with potential to prevent HPV infection are being developed and tested. NCI

has launched early clinical trials of a vaccine that creates a protective effect by employing virus-like particles

from HPV-16, the form of the virus involved in over 50

percent of cervical cancer cases. Preliminary results

show that a low-dose injection of the vaccine induces

high levels of protective antibodies against HPV. Future

clinical trials may include oral cancer patients. In addition, NCI-funded scientists are working on creating a

vaccine that contains virus-like particles from the four

major strains of HPV that together contribute to more

than 80 percent of all cervical cancers. 

Unlike the HPV vaccine, most cancer vaccines are

designed to treat existing cancers by inducing an

immune response that leads to a direct attack on tumor

cells. For example, researchers have developed and are

testing a B-cell lymphoma vaccine that primes a

patient’s immune system to seek out and destroy tumor

cells. To create this vaccine, scientists removed tumor

cells from a patient’s lymph nodes and selected and 

isolated a protein present only on the cancer cells. The

isolated protein was then joined to a carrier protein

that could transport it into the body and aid in creating

an immune response. Finally, an immune system boosting drug was added to the vaccine combination for maximum effect. Of the 20 patients injected with custommade vaccines as part of a small Phase II trial, 18

remained in complete remission an average of four

years. A large-scale trial of the vaccine involving 390

patients has now been launched. 

Immunotherapy research has proven that the immune

system can be primed to recognize and destroy tumor

cells with minimal toxicity compared with chemotherapy

and other treatments. Thus, immunotoxins and vaccines

are a positive addition to our arsenal of anti-cancer

therapies, but additional clinical trials are needed to

help us maximize their potential.

SPOTLIGHT ON RESEARCH

Harnessing the Immune System to 

Prevent and Treat Cancer
38 The Nation’s Investment in Cancer Research

NCI’s Challenge: Building Our Capacity for the Future

THE CHALLENGE

I

dentifying and tracking rates and trends in cancer

and monitoring the factors that influence these

changes are crucial underpinnings of efforts to prevent and control cancer. Reduction in the cancer

burden is a critical measure of the progress we are

making against cancer. Since 1990, new cancer

cases and death rates have fallen for all cancers

combined and for most of the top ten cancer sites,

reversing a decades-long trend of rising cancer incidence and death rates in the United States. These

decreases support the wisdom of the Nation’s

investment in cancer research.

Appropriate decision making in

science and in public health depends

on accurate, reliable information

about the incidence and impact of

disease. NCI’s Surveillance,

Epidemiology, and End Results

(SEER) cancer registry program provides data to identify and study variations in cancer rates, assess trends,

track the impact of cancer on the

general population, and provide

information that researchers need to

ask critical questions about why certain populations are affected by cancer more severely than others. SEER data have enabled us to identify environmental carcinogens, monitor cancerrelated effects of tobacco, identify geographic areas

with higher than average cancer rates, study patterns and outcomes of cancer care, and identify risk

groups for research and public health intervention

programs. 

Recent changes in health care financing and

delivery, the revolution in informatics and computer

programming technology, and the social and cultural

diversity of our country present new challenges and

opportunities in surveillance research. Currently,

our understanding about how risk factors, screening, and treatment may affect trends in the cancer

burden is beginning to unfold, and advancing

knowledge in these areas will require new data

sources and statistical methods. In addition to the

incidence and mortality data now collected,

research is needed to improve methods for measuring quality of life, quality of care, health status,

morbidity, family history, cancer risk behaviors,

screening, and treatment. Better methods and models also are needed for relating these measures,

quantifying their impact on current and future cancer rates, and predicting outcomes. 

New investments are required to support the

adoption of tools that will improve the precision

and expand the reach of our cancer surveillance

efforts. These include geographic

information systems that allow data

linkage and statistical analysis of

individuals and potential environmental exposures by location, new

approaches to modeling trends, and

more refined cancer maps and spatial

statistical techniques for assembling,

analyzing, and disseminating surveillance data. 

NCI’s surveillance efforts should

be expanded to cover the spectrum of

racial, ethnic, socioeconomic, and

cultural diversity. New investments

in research on specific population groups will allow

us to connect information on prevention, risk factors, screening, treatments, and patterns of care

with outcomes such as incidence, quality of life,

and mortality. 

Greater efforts are needed to disseminate the

results of NCI’s surveillance research to scientists,

the general public, and policy makers. As the field

of surveillance research expands and takes on these

new challenges, it is clear that scientists with skills

encompassing the disciplines of epidemiology, statistics, disease registration, geographic information

systems, and informatics are in short supply. NCI

must create the training opportunities necessary to

prepare the next generation of surveillance

researchers. Enhancing our investment in surveilStudying Emerging Trends in Cancer

GOAL

Expand cancer 

surveillance to improve 

monitoring of progress 

in cancer control and 

explain potential 

causes of cancer across 

all populations.
NCI’s Challenge

NCI’s Challenge 39

lance research will ensure that NCI continues to

play an active and visible national leadership role

in developing a comprehensive national surveillance program. 

PROGRESS TOWARD MEETING 

THE CHALLENGE

T

he Cancer Surveillance Series, developed by

NCI scientists and the editorial staff of the

Journal of the National Cancer Institute, makes information about factors contributing to cancer more

accessible to researchers and the public. These published research articles address emerging patterns of

cancer in various population groups and explore the

elements affecting these patterns at the national

and regional level. Begun in June 1999, the series

provides a forum for disseminating the latest analysis and evaluation of U.S. cancer statistics, with

special emphasis on data from population-based

SEER cancer registries. Similarly, NCI recently

published the Atlas of Cancer Mortality in the

United States, 1950-1994, showing geographic

patterns of cancer death rates in over 3,000 counties across the country. Available on the Web (cancer.gov/atlas), the 254 color-coded maps make it

easy for researchers, public health professionals, and

health departments to more precisely identify

places with high or low cancer rates, and uncover

cancer patterns. 

To better understand the burden of cancer in

various populations, NCI is expanding its cancer

surveillance program to cover a broader spectrum

of the racial, ethnic, socioeconomic, and cultural

diversity. In 1999, coverage of Alaska Natives was

added to the SEER program, and steps were taken

to improve the quality of data on American

Indians. NCI now is expanding SEER to enhance

coverage of rural whites and blacks, non-Mexican

Hispanics, American Indians, and states with high

cancer death rates. Collaborative studies such as

the National Longitudinal Mortality Study

involving the NIH, the Census Bureau, and the

National Center for Health Statistics (NCHS)

were launched in 1999 to improve cancer mortality

data for racial, ethnic, immigrant, and socially disadvantaged populations. These studies will make it

possible to connect important socioeconomic and

demographic information with prevention, risk factors, screening, treatment, and patterns of care in

special populations. 

Computer software known as SEER Stat, developed by NCI, makes it easier for people to use SEER

data. The software is provided free of charge on

SEER public use files, more than 1,500 of which are

being distributed each year. A database management system for SEER cancer registries is being

developed to support registry operations and promote more uniform standards and consistency

among U.S. population-based cancer registries. 

A Cancer Control Topical Module is being

fielded in collaboration with NCHS as part of the

National Health Interview Survey (NHIS). This

module provides critical national data for tracking

progress in major cancer control health practices.

Similarly, NCI is partnering with the California

State Department of Public Health to initiate a

new state-level survey similar to the NHIS, the

California Health Interview Survey (wwwdccps.ims.nci.nih.gov/ARP/RiskFactor/chis.html).

A decade of data related to monitoring progress in

tobacco control in all states and the District of

Columbia has been made available as a public use

file with the Tobacco Use Supplement to the

Current Populations Survey (www-dccps.ims.

nci.nih. gov/ARP/RiskFactor/tobacco.html). Seven

centers and a statistical coordinating center were

funded this year to continue support of the Breast

Cancer Surveillance Consortium (BCSC – wwwdccps.ims.nci.nih.gov/ARP/breastcancer.htm). The

BCSC, established to enhance our understanding

Budget Increase Request for 2002

Studying Emerging Trends in Cancer

1. Improve cancer registry data. $11.8 M

2. Expand systems and methods to enhance 

the quality of cancer control data. 25.0 M

3. Expand systems and methods to enhance 

capacity for exploring causes of cancer, 

generating new hypotheses, and identifying 

new opportunities. 5.0 M

4. Improve and expand training in surveillance 

research and improve dissemination 

of information. 3.5 M

Management and Support 2.0 M

Total $47.3 M
40 The Nation’s Investment in Cancer Research

THE PLAN – STUDYING EMERGING TRENDS IN CANCER

Goal

Expand surveillance data systems, methods, communications, and training to improve capacity for

monitoring progress in cancer control and for explaining potential causes of cancer nationally and

among diverse populations.

Objectives and Milestones for Fiscal Year 2002

1. Improve cancer registry data by expanding SEER coverage, improving the quality of

all population-based cancer registries, and enhancing SEER as a research resource.

■ Support 2 to 5 new SEER registries to improve coverage of key populations: 

non-Mexican Hispanics, residents of Appalachia and other rural areas (especially

those of lower socioeconomic classes), rural African Americans, American Indians,

and populations with high cancer mortality rates. $6.00 M

■ Provide funding for technical assistance, quality control, and special studies to

improve operational efficiency and data comparability among population-based 

cancer registries. Support field and analytic audits; training and fellowship programs;

methodology development; linkage with other health-related information sources;

and development of more efficient technology-based information systems. $2.00 M

■ Expand support for applied innovations in SEER population-based cancer registry

data systems, including portable modules to facilitate cost-effectiveness, improve

methodology, and advance research in newly diagnosed patients. $0.80 M

■ Develop a biospecimen resource for archived tissue for population-based 

surveillance and epidemiologic research. Support on-staff pathologists, tissue 

accession managers, and administrative personnel to coordinate response to

approved requests for specimens. $3.00 M

2. Expand systems and methods to enhance the quality of cancer control data on 

risk, health and behaviors, and screening practices linked to high quality data on

cancer outcomes.

■ Build a surveillance system to monitor progress in tobacco control. (See p. 88,

Objective 1.) 

■ Enhance national and regional data systems to measure health disparities. in cancerrelated health behaviors and screening practices. Expand support for cancer control

supplements to national and regional surveys and enhance data on socioeconomic

and other demographic factors associated with disparate cancer outcomes. $8.00 M

■ Fund surveillance screening consortia to assess cancer screening performance at the

community level. Continue efforts to examine mammography screening quality

through the BCSC, begin a comparable research consortium in colorectal cancer

screening, and examine the need to establish comparable consortia to evaluate

screening quality for cervical and prostate cancers. $10.00 M

■ Supplement existing surveillance screening consortia and population research networks, such as the Cancer Research Network, to examine emerging issues in cancer

control including obtaining data on barriers to entry into cancer clinical trials,

expanding cancer screening surveillance in specific ethnic or at-risk populations, and

examining the effect of tobacco and food-related policy and legislation. $2.00 M

$11.8 M

$25.0 M
NCI’s Challenge

NCI’s Challenge 41

■ Fund data collection and research on prognostic risk factors, health behaviors,

quality of life, and cancer care processes. (See p. 46, Objective 2.)

■ Conduct SEER special studies to obtain similar cancer control and quality of care

data within SEER registries. Explore the feasibility of obtaining specific cancer care

data currently not collected as part of routine cancer registration. $1.00 M

■ Develop new data linkages and expand support for analysis of linked data sets, such

as the SEER-Medicare database, managed care and other sources of claims data, or

other regional or national health surveillance data. $2.00 M

■ Support statistical and methodological studies to improve accuracy and reliability

of data on socioeconomic determinants of cancer rates and risk, health behaviors,

and screening. (See p. 50, Objective 3.)

■ Develop statistical and graphical methods, software applications, and other 

technologies relevant to geospatial and mapping research. $2.00 M

3. Expand systems and methods to enhance capacity for exploring causes of cancer,

generating new hypotheses on risk, and identifying new opportunities for cancer

control interventions. 

■ Fund epidemiologic research studies utilizing the NCI Atlas of Cancer Mortality in

the United States, 1950-1994 and other population-based data systems on cancer

incidence, vital statistics, and vital records to identify potential new leads in 

cancer causation. $2.50 M

■ Support research initiatives using geospatial, mapping, and other analytic methods

applied to existing population-based systems of environmental, sociocultural, and

other relevant risk factors to develop hypotheses for more in-depth studies of 

cancer causation. $2.50 M

■ Support studies that use biospecimen resources from population-based cancer 

registries. (See p. 66, Objective 1.)

4. Improve and expand training in surveillance research and improve dissemination of

information to researchers, public health professionals, the public, policy makers,

advocates, and legislators. 

■ Fund existing surveillance and applied research networks and consortia to conduct

intensive training programs, provide sabbatical opportunities for research professionals, and initiate and develop academic curricula on surveillance research. $2.00 M

■ Improve surveillance data dissemination using emerging information technologies

and the Internet for information exchange. Expand innovative dissemination, 

particularly visual representation of data, to improve its comprehension by 

non-technical audiences. $1.00 M

■ Continue to produce the annual NCI National Cancer Progress Report to improve

dissemination of data on the cancer burden and progress in cancer control. $0.50 M

Management and Support

Total

$5.0 M

$3.5 M

$2.0 M

$47.3 M
42 The Nation’s Investment in Cancer Research

of the relationship between screening practices and

breast cancer mortality, includes data on nearly

three million screening mammograms. Key expansion activities include: tracking digital mammography performance as it enters clinical practice,

obtaining more detailed risk factor data, and

expanding the diversity of the populations represented in the BCSC. Plans for establishing a

Colorectal Cancer Surveillance Consortium are

underway. 

The SEER-Medicare linked data (wwwdccps.ims.nci.nih.gov/ARP/seermedicare.html) is a

collaborative NCI/Health Care Financing

Administration (HCFA) effort initiated in 1987 to

link clinical data collected by the SEER registries

with claims for health services collected by

Medicare. SEER-Medicare data are being used for a

broad array of studies, including those assessing patterns of care, costs of cancer treatment, and use of

screening technologies. The data are recognized as

a major national research resource for examining

dissemination and quality of cancer care. Building

on ten years of data from SEER-based studies, NCI

is now analyzing, presenting, and publishing data

on trends in cancer care. An example is the publication of data from the Prostate Cancer Outcomes

Study (PCOS), the first longitudinal study on the

quality of life of men living with prostate cancer

(www-dccps.ims.nci.nih.gov/ARP/PCOS). These

data, drawn from a cohort of 3,600 men, reveal significant differences in urinary, bowel, and sexual

function over time and between different types of

prostate cancer treatment. They will provide

patients and their physicians a much better sense of

patient-centered outcomes following prostate cancer treatment – a critical element for informed clinical decision making. A major new NCI initiative,

Cancer Care Outcomes Research and

Surveillance (CanCORS), will build on PCOS,

supporting research to determine the relationship

between various measures of quality of care to

important cancer outcomes in patients with lung

and colorectal cancer. 

NCI is working to improve its understanding of

how physicians in the community translate and

apply new knowledge about cancer risk, screening,

and treatment in the clinical setting (wwwdccps.ims.nci.nih.gov/ARP/physician.html). For

example, current colorectal cancer screening rates

are much too low to have a large impact on colorectal cancer mortality. The Survey of Colorectal

Cancer Screening Practices in Health Care

Organizations is assessing current, nationally representative data on the physician and health system

factors that may influence the use of screening and

diagnostic follow-up for colorectal cancer detection. In the Physician Survey on Cancer

Susceptibility Testing, national data are being collected on physicians’ knowledge of available tests

for specific cancer susceptibility genes and on their

attitudes toward genetic testing. 

While the Cancer Atlas shows differences in the

geographic patterns of cancer by disease site and

over time, Geographic Information Systems (GIS)

provide new tools for exploring such patterns and

generating hypotheses for etiologic research. In

1999, NCI funded a prototype GIS system for breast

cancer research that was piloted as part of the Long

Island Breast Cancer Study, applying this emerging

technology to the study of possible environmental

causes of breast cancer. Through a new initiative,

NCI will support research aimed at moving the use

of GIS beyond database storage and mapping to

become an analytic tool for cancer research.

NCI is supporting a program called the Cancer

Intervention and Surveillance Modeling

Network, the goals of which are to answer the

“why” questions in the analysis of cancer incidence

and mortality; determine if recommended interventions are having their expected population impact;

predict the impact of new interventions on national trends; and study optimal cancer control strategies. In Fiscal Year 2000, nine projects were funded,

seven in breast cancer and one each in colorectal

and prostate cancer. 

A number of national efforts are ongoing in cancer control surveillance. NCI has worked for many

years with other Federal agencies, such as the

Centers for Disease Control and Prevention

(CDC), the Food and Drug Administration, and

HCFA to expand the Nation’s capacity to track

cancer control progress and provide data resources

for exploring new leads in cancer etiology. In

February 2000, NCI and CDC, established a

Memorandum of Understanding to collaborate in

forming a comprehensive, federally integrated

cancer surveillance and cancer control research

system.

For more information on SEER, go to 

www-seer.cancer.gov.
NCI’s Challenge

NCI’s Challenge: Building Our Capacity for the Future

NCI’s Challenge 43

THE CHALLENGE

About nine million Americans living today have

had a diagnosis of cancer, and another 1.2 million people will be diagnosed in 2000. Of these, a

large percentage are undergoing active treatment for

their disease, and all require life-long quality care to

detect and treat recurrences, new cancers, and treatment side effects, as well as to meet their supportive

care needs. This year, direct medical care costs

attributable to cancer will exceed $50 billion. The

toll in human pain, suffering, and fear cannot be

captured in dollars, but will be keenly felt by the

millions of people with cancer, their

families, and community caregivers.

The National Cancer Institute, long

at the forefront of basic and clinical

research on cancer and the translation of findings into more effective

interventions, is now launching

research to improve the quality of

cancer care by strengthening the

information base for cancer care decision making. 

NCI will seek to better understand

what constitutes quality cancer care,

with an emphasis on the patient’s

perspective; to identify geographic, racial/ethnic,

and other disparities in who receives quality care;

and to strengthen the scientific basis for selecting

appropriate interventions along the entire continuum of care. All decision makers, public and private,

will benefit from this expanded information base.

Special emphasis will be placed on ensuring that

Federal agencies that pay for, directly deliver, or

regulate cancer care have the information they

need to make quality-enhancing policy choices. 

The quality of cancer care is a major national

concern that has been underscored by Ensuring the

Quality of Cancer Care, the report from the Institute

of Medicine’s National Cancer Policy Board; the

1999 report of the President’s Cancer Panel on cancer care quality; and initiatives undertaken recently

by a number of organizations (e.g., the American

Society of Clinical Oncology). These various analyses and proposals for change all point to an emerging

consensus about the critical elements of a research

agenda to improve the quality of cancer care.

First, we need a core set of cancer care measures

that are patient-centered and acceptable to

providers and payers, span the continuum of care,

and meet the highest technical standards of validity, reliability, and sensitivity to change. These endpoint measures will greatly enhance our ability to

compare interventions across studies and over time.

Equally important, we need a comprehensive, critical evaluation of existing practice guidelines to develop a

baseline understanding of what

providers and payers currently consider to be “quality” cancer care. 

Second, we need a stronger data and

methods “infrastructure” for conducting

quality of care analyses. This includes

studies to determine which interventions improve patient-valued outcomes (thereby enhancing the quality

of care); to identify geographic or

racial/ethnic variations in receipt of

quality care; and to monitor quality over time, at

both the individual and population levels. Indeed,

the National Cancer Policy Board has recommended

the development of a national cancer data system to

support such studies on an ongoing basis. Developing

this system will require close cooperation among a

number of public and private entities, including

community- and hospital-based tumor registries, professional associations, and advocacy groups. Such

collaboration among public agencies, physician

groups, and private organizations that also are working on quality of care initiatives will be essential to

improve both the community representativeness of

the data and the rigor and relevance of the analyses. 

Quality of Cancer Care

GOAL

Enhance the state 

of the science on the 

quality of cancer care 

and inform Federal 

decision making on care

delivery, coverage, 

and regulation.
44 The Nation’s Investment in Cancer Research

Third, it is critically important to determine whether

therapies shown to be efficacious in clinical trials have

been incorporated successfully into community practice.

If so, what were the key factors that made it happen? If not, what explains the failure to turn good

science into good medicine? In addition, there

should be a critical examination of whether, when,

and how NCI-sponsored trials, which have been the

backbone of the clinical discovery process in cancer,

should give greater emphasis to patient-centered

outcomes in addition to the important traditional

endpoints of survival and tumor progression. 

Finally, the quality of cancer communications – for

example, between patient and provider, the news

media and the patient, and the patient’s family and

the third-party payer – is a central determinant of

cancer care quality. We must have a better understanding of the information needs of patients, families, and other decision makers involved in the

choice of cancer interventions. Based on that

understanding, we must develop practical toolkits

and other innovative approaches to enhancing

access to information relevant to cancer care choices.

This program of research responds directly to the

challenges posed in this emerging consensus about

how to improve the quality of cancer care. In presenting an initial design of the research program to

Department of Health and Human Services

Secretary Donna Shalala, NCI urged that cancer be

made a “working model” for quality of care research

and application. The Secretary responded by

approving the creation of the Quality of Cancer

Care Committee (QCCC), a trans-agency task

force with representatives from Federal agencies

involved in cancer care delivery (e.g., the

Department of Veterans Affairs), coverage (e.g.,

the Health Care Financing Administration), and

regulation (e.g. the Food and Drug

Administration). In addition, the NCI quality of

care initiative now operates organizationally within

the Secretary’s larger Quality Improvement

Initiative, in close coordination with the Agency

for Healthcare Research and Quality. In all of these

efforts, NCI will draw on its experience in supporting, and participating directly in, a number of quality-related projects over the past decade.

NCI understands the importance of ensuring

that scientific advances in preventing, detecting,

treating, and curing cancer are translated into

interventions that extend and improve quality of

life for the millions of individuals and families who

not only bear the burden but carry the hope. For

this to occur, sustained collaboration will be essential not only among Federal researchers and policy

makers, but among public agencies and the full

spectrum of private entities involved in cancer care

– providers and third-party payers, professional

associations, patient advocacy groups, and organizations that measure and monitor quality of care.

NCI’s quality initiative will be an important early

step in support of such sustained collaboration.

Moreover, while this research program is clearly

cancer-focused, it should deepen our understanding

of how to define, monitor, and improve the quality

of health care overall.

PROGRESS TOWARD MEETING 

THE CHALLENGE

NCI’s research programs are providing much of

the evidence base for the national agenda to

improve health care quality. The Cancer Care

Outcomes Research and Surveillance Consortium

(CanCORS), launched in 1999, is a major initiative to study the impact of cutting-edge interventions on patient-centered outcomes, investigate the

dissemination of state-of-the-science therapies into

community practice, and analyze disparities in the

delivery of quality cancer care. CanCORS multicenter teams will collaborate on large observational cohort studies of newly diagnosed cancer

patients. Initial projects are focusing on lung and

colorectal cancer, although expansion to other

high-prevalence cancer sites is anticipated. These

analyses will support development of an expanded

set of core quality and outcome measures that may

be collected routinely by tumor registries in support

of a national data system to monitor cancer care

quality. CanCORS teams also are examining major

methodological issues in outcomes research conducted in community settings.
NCI’s Challenge

NCI’s Challenge 45

Because results from clinical trials are critical to

defining quality cancer care, we are working to

ensure the optimal performance of these studies.

For example, NCI’s Cancer Therapy Evaluation

Program is collaborating with the Outcomes

Research Branch (see below) in evaluating the

Cancer Trials Support Unit, a new administrative

mechanism for streamlining trials-related data collection and other administrative functions. Part of

this evaluation will assess efforts to strengthen the

quality of communications about clinical trials to

patients and physicians. In addition, the

cancerTrials Web site highlights important clinical

trials results, providing accurate and timely information to both patients and health care providers.

NCI has long had in place the programmatic and

organizational mechanisms to pursue important

research questions related to quality of care. Since

1973, the Surveillance, Epidemiology, and End

Results (SEER) program (www-seer.cancer.gov)

has monitored the national cancer burden and provided an indispensable data resource for assessing

the impact of research advances on cancer outcomes. For example, SEER patterns-of-care studies have shown that, between 1987 and 1995,

breast-conserving surgery for early stage breast cancer increased among women of all ages, but that

older women were less likely to receive the recommended post-surgical radiation therapy. Studies

linking SEER and Medicare data have shown that

for patients with early stage lung cancer, rates of

surgery and survival were lower for black patients

than for white patients. The Prostate Cancer

Outcomes Study, initiated in 1994, is using data

from six SEER registries to conduct the first systematic evaluation of the impact of treatments for

primary prostate cancer on quality of life for men 

living with this disease (www-dccps.ims.nci.

nih.gov/ ARP/PCOS/index.html).

NCI’s Applied Research Program (ARP)

(www-dccps.ims.nci.nih.gov/ARP/index.html) is

an established locus of key NCI research on quality

issues. In addition to its involvement in the SEERrelated efforts described above, the ARP recently

launched the HMO Cancer Research Network

(www-dccps.ims.nci.nih.gov/ARP/hmo.html),

which promotes collaborative cancer research

among managed care systems. Supported by the

first award under this program, a consortium of

researchers affiliated with 10 major not-for-profit

HMOs is conducting studies of late stage breast and

invasive cervical cancer cases to identify patient,

provider, and system factors that could have prevented advanced disease. They also will study the

quality and impact of smoking cessation programs

delivered in HMOs. To provide a focal point for

this emerging research area, in 1999, NCI established an Outcomes Research Branch within the

ARP (www-dccps.ims.nci.nih.gov/ARP/outcomes.

html).

Other NCI quality of care initiatives include the

Breast Cancer Surveillance Consortium, which

examines the accuracy, cost, and quality of screening mammography programs in eight community

settings; the newly developed Colorectal Cancer

Surveillance Consortium; and the Black-White

Cancer Survival Study. 

Budget Increase Request for 2002

Quality of Cancer Care

1. Develop core process and outcome measures 

for assessing the quality of cancer care. $ 1.0 M

2. Strengthen the methodological and 

empirical foundations of quality of 

care assessment in cancer. 15.0 M

3. Enhance quality of care research within the 

restructured NCI clinical trials program. 2.0 M

4. Improve the quality of cancer care by 

strengthening the quality of cancer 

communications. 1.0 M

5. Ensure that Federal decision making on 

cancer care is informed by the best available 

scientific evidence about quality. 2.0 M

Management and Support 0.5 M

Total $21.5 M
46 The Nation’s Investment in Cancer Research

THE PLAN – QUALITY OF CANCER CARE

Goal

Enhance the state of the science for defining, monitoring, and improving the quality of cancer care

and inform Federal-level decision making on cancer care delivery, coverage, and regulation.

Objectives and Milestones for Fiscal Year 2002 

1. Develop core process and outcome measures for assessing the quality of 

cancer care. 

■ Establish a national panel of experts in cancer outcomes measurement theory and

practice to evaluate existing endpoint measures and instrumentation and formulate

alternative strategies for valid, reliable, sensitive, and feasible clinical and patientcentered endpoint measures for use in quality of cancer care studies. $0.50M 

■ Evaluate published practice guidelines and care maps from major professional

organizations, medical care providers, third-party payers, and researchers that

describe “quality” cancer care for the most prevalent cancers and across the full

continuum of care. $0.50 M

2. Strengthen the methodological and empirical foundations of quality of cancer 

care assessment.

■ Sustain support for CanCORS studies of the impact of targeted interventions on

patient-centered outcomes, dissemination of state-of-the-science therapies into

community practice, the influence of modifiable risk factors, and disparities in the

delivery of quality cancer care. $8.00 M

■ Increase support for analyses of the linked SEER-Medicare database to investigate

the diffusion and outcomes of selected cancer interventions, with special emphasis

on whether differential application of interventions contributes to cancer outcome

disparities among individuals aged 65 and older. $1.00 M

■ Support the creation of databases that link tumor registry information with private

payer administrative data to expand capacity to investigate whether cancer interventions are reaching and improving the health of individuals under age 65. $2.00 M

■ Sponsor innovative economic and health services research studies on the impact of

cancer prevention, control, and screening interventions on patient-valued outcomes and economic costs of cancer to improve cost-effectiveness and outcomes

analysis in cancer prevention and screening trials and in community cancer prevention and control interventions. $2.50 M

■ Sponsor new investigator-initiated studies to strengthen the methodological foundations of outcomes research and quality of care assessment. $1.50 M

3. Enhance quality of care research within the restructured NCI clinical trials program.

■ Sponsor a symposium and follow-on workshops to bring together leading researchers,

patient advocates, and the relevant Federal agencies to assess the current state of

the art, identify key research questions, and develop a decision strategy for encouraging comprehensive assessments of patient outcomes in clinical trials. $0.30 M

$1.0 M

$15.0 M

$2.0 M
NCI’s Challenge

NCI’s Challenge 47

■ Expand support for studies of diffusion patterns and the overall diffusion rates of

important clinical trial findings into community practice. Focus on factors that

accelerate or impede the use of new therapies and geographic or population group

differences in their availability or adoption. Building on study findings, develop a

process for incorporating successful diffusion techniques into clinical trials design

and communicating trial results to the public and medical community. $1.70 M

4. Improve the quality of cancer care by strengthening the quality of cancer 

communications.

■ Gather nationally representative data to assess the current status of cancer 

communications and better understand the amount and quality of information 

now available and being used in cancer care decision making. (See p. 92,

Objective 1.) 

■ Within the Centers for Excellence in Cancer Communications Research, support

projects to evaluate and enhance cancer communications’ effectiveness in 

helping patients understand the risks and benefits of interventions and in 

improving overall quality of care. (See p. 92, Objective 2.)

■ Provide supplemental funding to Community Clinical Oncology Programs for 

pilot projects to identify information needs of cancer patients, survivors, and their

families and develop new communications strategies to improve cancer care decision making, particularly in vulnerable populations. $1.00 M

■ Create new communications products and tools for cancer patients and their 

caregivers, individuals at elevated risk, advocacy groups, health care professionals,

third-party payers, and public agencies to improve the accuracy, clarity, and timeliness of cancer care decision making. (See pp. 92-93, Objective 3.)

5. Ensure that Federal decision making on cancer care is informed by the best 

available scientific evidence about quality measures and assessment. Share new

knowledge on ways to translate research into practice at the Federal level with 

private partners through the National Cancer Policy Board, private associations,

and health care systems.

■ To identify and meet Federal agency needs for cancer care information, continue to

convene the QCCC. Support and participate in interagency collaborative demonstration projects on quality measures and assessment. $2.00 M

Management and Support

Total

$1.0 M

$2.0 M

$0.5 M

$21.5 M
48 The Nation’s Investment in Cancer Research

NCI’s Challenge: Building Our Capacity for the Future

THE CHALLENGE

Despite progress in biomedical science over the

past several decades that has increased longevity

and improved quality of life for many Americans,

the burden of disease is not borne equally by all population groups in the United States. For example,

the death rate from prostate cancer among African

American men is almost twice that of white men,

and stomach cancer mortality is substantially higher among Asian-Pacific Islanders, including Native

Hawaiians, than other populations. Cervical cancer

incidence in Hispanic women has been consistently

higher at all ages than for other

women, and African American

women have the highest death rate

from cervical cancer. Persons of low

socioeconomic status have higher

death rates for most cancers than persons of higher socioeconomic status.

Overall, men are about 50 percent

more likely than women to die from

cancer, and among all women,

Alaskan Natives are about 30 percent

more likely to die from cancer.

Disease always occurs within a

context of human life circumstances. Social position, economic status, culture, and environment are

critical determinants of who is born healthy, who

grows up healthy, who sustains health throughout

their life span, who survives disease, and who maintains a good quality of life after diagnosis and treatment. In particular, social injustice has to a large

extent created the health disparities that currently

exist in the United States. The unequal burden of

disease in our society is a challenge to science and

a moral and ethical dilemma for our Nation.

If we are to reduce these disparities, the relative

importance of social causes to their development

and their relation to factors that result in unequal

access to high quality cancer diagnosis and treatment must be explained. To do this, we must increase

fundamental research into the social causes of

health disparities, the psychosocial factors that

mediate them, and the biologic pathways that can

explain their impact. 

The relative importance of different determinants also can be expected to vary depending where

in the disease process a disparity occurs. A key question is: How can we best measure and monitor cancer-related health disparities across the spectrum of

cancer incidence, stage of disease at diagnosis, disease recurrence, quality of life, and cancer mortality?

Another key question is the extent to which prevention, early detection, treatment, and communication

interventions can effectively reduce cancer-related

health disparities. 

The National Cancer Institute is

strongly committed to a research program that will address cancer health

disparities across the cancer control

continuum from prevention to end of

life care, consistent with recommendations in the Institute of Medicine’s

report, The Unequal Burden of Cancer,

and reflected in the Healthy People

2010 goal to eliminate racial and ethnic health disparities. 

NCI has developed and will pursue

a research framework that builds upon the growing

evidence that socioeconomic, cultural, health care

provider, institutional, and environmental factors

contribute substantially to cancer-related health

disparities. The elements that influence health disparities are complex, and their interactions are

largely unknown. While health disparities have

been framed historically in the context of racial and

ethnic disease differences, racial and ethnic classifications have always been socially and politically

determined with no basis in biological science. The

power of scientific discovery must be used to elucidate the meaning and effect of the human circumstances in which differential disease burdens occur.

Finally, in our national effort against cancer,

there is a critical disconnect between scientific discovery and cancer care delivery, and this disconnect

Reducing Cancer-Related Health Disparities

GOAL

Understand the 

causes of cancer 

health disparities 

and develop 

effective interventions 

to reduce them.
NCI’s Challenge

NCI’s Challenge 49

is itself a key determinant of the unequal burden of

cancer in our society. Barriers that prevent the

benefits of research from reaching all populations,

particularly those who bear the greatest disease

burden, must be identified and removed. In

response to this challenge, we will develop new

intergovernmental and public/private partnerships

to improve the dissemination and diffusion of evidence-based interventions and encourage the

development of health care policies for underserved

communities.

PROGRESS TOWARD MEETING 

THE CHALLENGE 

T o direct implementation of NCI’s Strategic

Plan to Reduce Health Disparities (ospr.nci.

nih.gov/healthdisprpt.pdf) and provide an organizational locus for critical tasks in translating discovery into delivery, NCI has created a Center to

Reduce Cancer Health Disparities. NCI also has

established a variety of infrastructures and initiatives to improve understanding of disparities and

develop strategies and interventions to overcome

them. These activities provide a firm foundation

from which to expand and intensify our efforts. 

For example, NCI’s SEER cancer registry program (www-seer.cancer.gov) has been expanded to

cover more of the racial, ethnic, and socioeconomic

diversity of the United States, allowing for better

description and tracking of trends in health disparities. Methodologic studies are seeking better ways to

measure socioeconomic factors and determine their

relationship to cancer incidence, survival, and mortality. In addition, NCI supports a growing body of

research to examine the environmental, sociocultural, behavioral, and genetic causes of cancer in different populations and apply these discoveries

through interventions in clinical and community

settings. These interventions cover topics such as

tobacco control, dietary modification, and adherence to screening practices. Recognizing the broad

relevance of this research to other disease outcomes,

NCI collaborates with other Federal agencies in

supporting important research initiatives, including

co-funded research with the Agency for Healthcare

Research and Quality (AHRQ) under its initiative,

“Understanding and Eliminating Minority Health

Disparities.”

NCI’s Office of Special Populations Research

(OSPR) has been a focal point for leadership and

coordination on research addressing the cancer-related concerns of underserved and other vulnerable populations. OSPR administers a variety of outreach and

other programs targeting specific special populations

(ospr.nci.nih.gov). Its newest initiative is the Special

Populations Networks for Cancer Awareness

Research and Training (SPN), a network of 17 institutions that will create and implement cancer control, prevention, research, and training programs in

minority and underserved communities. 

The Comprehensive Minority Biomedical

Program (CMBP) aims to increase the number of

minority scientists in biomedical research and

enhance the careers of those already in the field.

CMBP programs include NCI’s newest training initiative for underserved minorities, the Continuing

Umbrella of Research Experiences (CURE), and

a host of other training opportunities targeting high

school students through established researchers.

Increasing the representation of ethnic and minority

individuals in research and clinical care is crucial to

ensure that important research questions about disparities are investigated and that discoveries are

translated into community practice. (See p. 57 for a

more detailed description of NCI training initiatives

targeting special populations.)

Greater participation of minority health professionals in clinical trials is essential to our efforts to

explore research questions about cancer-related

Budget Increase Request for 2002

Reducing Cancer-Related Health Disparities

1. Create a plan to organize, coordinate, and 

monitor NCI activities in health disparities. $ 2.0 M

2. Improve capacity and accelerate knowledge 

through research. 12.0 M

3. Expand our ability to define and monitor 

health disparities. 9.0 M

4. Expand control intervention research. 17.5 M

5. Expand the channels for research 

dissemination and diffusion. 7.0 M

6. Strengthen training and education in 

health disparities research. 1.6 M

Management and Support 1.5 M

Total $50.6 M
50 The Nation’s Investment in Cancer Research

THE PLAN – REDUCING CANCER-RELATED HEALTH DISPARITIES

Goal

Understand the causes of health disparities in cancer and develop effective interventions 

aimed at reducing these disparities.

Objectives and Milestones for Fiscal Year 2002

1. Create a new and comprehensive plan to organize, coordinate, and monitor 

NCI activities in health disparities research, education, training, and health 

services support.

■ Maintain support for the NCI Center to Reduce Cancer Health Disparities

(CRCHD). $1.50 M

■ Refine and implement an agenda for reducing health disparities. Identify and 

support successful research activities, facilitate dissemination, and monitor 

success. $0.50 M

■ Develop an integrated low literacy education program.

2. Improve capacity and accelerate knowledge through fundamental cancer control

and population research.

■ Create 4 Centers for Population Health to (1) expand understanding of the social

and environmental causes of cancer-related health disparities and the psychosocial,

behavioral, and biological factors that mediate them, (2) develop hypotheses for

cancer control research at individual, social, institutional, and policy levels, and

(3) develop, apply, evaluate, and disseminate interventions to improve cancer outcomes and reduce outcome disparities. $8.00 M

■ Expand ongoing epidemiologic investigations to explore racial/ethnic cancer disparities with a focus on cancers for which these disparities are greatest (e.g., breast,

cervix, kidney, prostate). Conduct new methodologic studies to evaluate factors

influencing recruitment and participation of underserved populations in cancer

epidemiology studies. $4.00 M

3. Expand our ability to define and monitor cancer-related health disparities.

■ Support 2 to 5 new SEER registries to improve coverage of key populations: nonMexican Hispanics, residents of Appalachia and other rural areas (especially those

of lower socioeconomic classes), rural African Americans, American Indians, and

populations with high cancer mortality rates. (See p. 40, Objective 1.) 

■ Enhance national and regional data systems to measure disparities in cancer-related

health behaviors and screening practices. Expand support for supplements to

national and regional surveys to enhance data on socioeconomic and other demographic factors associated with disparate cancer outcomes. (See p. 40, Objective 2.) 

■ Support statistical and methodological studies to improve accuracy and reliability

of data on socioeconomic determinants of cancer rates and risk, health behaviors,

and screening in national and regional populations. Expand use of modeling and

geographic analyses to examine the unequal burden of cancer. $9.00 M 

■ Conduct national tobacco control surveys on activities that impact tobacco control,

including diverse populations. (See p. 88, Objective 1.)

$2.0 M

$12.0 M

$9.0 M
NCI’s Challenge

NCI’s Challenge 51

4. Expand cancer control intervention research in prevention, early detection, 

treatment, and communications.

■ Fund an additional 4 to 6 SPN sites to enhance research infrastructure and training

in underserved communities. Partner with academic cancer centers to continue

developing and testing community-based, participatory cancer control interventions addressing disparities. Provide additional funds for pilot cancer control

research projects within SPNs. $6.00 M

■ Provide supplemental funding to Transdisciplinary Tobacco Use Research Centers

(TTURCs) to study differential tobacco use and quitting patterns among underserved populations and support development of more effective interventions to

reduce the burdens associated with tobacco use. $1.00 M

■ Expand colorectal cancer screening use and follow-up studies. Conduct new 

intervention research to identify and overcome sociocultural and health care system barriers to the continuing under-use of fecal occult blood testing and flexible

sigmoidoscopy, and to address co-morbid illness and other barriers to appropriate

clinical follow-up of abnormal findings. $3.00 M

■ Expand research on breast and cervical cancer screening for women who have

never been screened and those who are not screened regularly. Support new intervention research to identify barriers to screening for women who underuse or never

use breast and cervical screening and address sociocultural determinants in planning, implementing, and evaluating these interventions. $3.50 M

■ Strengthen the methodologic and empirical foundation of quality of care assessment, to improve understanding of unequal access and treatment differences compared to other broader social determinants of these cancer outcomes. (See p. 46,

Objective 2.)

■ Provide supplements to investigators and cancer centers to expand research on disparities in survivorship, including ethnic, cultural, socioeconomic, and institutional factors affecting the quality and length of cancer survivorship in underserved

communities and strategies to help cancer patients and their families make the

transition to extended and long-term survivorship. $2.00 M

■ Establish formal affiliations between NCI Cancer Centers and minority-serving

institutions. (See p. 30, Objective 1.)

■ Provide up to 3 years of supplemental funding to cancer centers to stimulate 

disparity research, particularly those located in or near underserved communities

that experience the heaviest burden of cancer. High priority areas will include 

disparities in risk factor exposures and access to prevention interventions 

(e.g., smoking cessation, dietary change, physical activity), quality cancer care, 

and clinical trials. $2.00 M

5. Expand the channels for research dissemination and diffusion.

■ Work with the Centers for Disease Control and Prevention (CDC) and the

American Cancer Society (ACS) to model and monitor the impact of research dissemination and diffusion efforts on DHHS Year 2010 health promotion objectives

generally and on cancer-related health disparities in particular. $1.80 M

■ Facilitate adoption of evidence-based cancer control interventions through: collaborative (NCI, CDC, ACS) review of cancer health disparities objectives; consen-

$17.5 M

$7.0 M
52 The Nation’s Investment in Cancer Research

sus on indices of dissemination and diffusion program impact; identification of

the most useful formats for presenting intervention research evidence; annual

reviews of published evidence on best practices to reduce disparities; and publishing/posting intervention evidence reviews and best practices on NCI’s

Dynamic Evidence in Cancer Control Web site (dccps.nci.nih.gov/DECC/

default.html). $2.20 M

■ Fund Pilot Projects to Overcome the Digital Divide. (See p. 92, Objective 1.)

■ Build special education/dissemination and diffusion partnership programs to

reach underserved communities, test these programs’ value in reducing health 

disparities in underserved communities, and make successful pilot programs

available for use by local, regional, and national organizations concerned with

cancer and public health. $1.00 M

■ Provide one-year competitive supplements to investigator-initiated intervention

research grants. Create a supplemental funding mechanism to develop and implement a dissemination and diffusion plan for interventions proven by the original

intervention research to be effective in reducing health disparities. $2.00 M

6. Strengthen training and education in health disparities research.

■ Develop a new track in the Cancer Prevention Fellowship Program to increase

the number of scientists studying health disparities. Recruit 2 fellows per year to

focus on health disparities research within OSPR and the Division of Cancer

Control and Population Sciences. $0.50 M

■ Expand community-based, cancer control research training within underserved

communities. Encourage cancer centers to partner with community organizations and health care institutions in underserved areas to apply for training

grant support for community-based clinical and cancer control research training

in health disparities research. $1.10 M

■ Expand programs to recruit, train, and sustain underserved racial and ethnic 

minority individuals in cancer research and provide partnership opportunities

for Minority-Serving Institutions with NCI Cancer Centers. (See p. 59,

Objective 4.)

Management and Support

Total

$1.6 M

$1.5 M

$50.6 M

health disparities. The Minority-based Community

Clinical Oncology Programs have for many years

sought to address clinical research questions relevant

to the disproportionate cancer burden experienced

by specific populations. NCI also has established

collaborations with key minority professional

organizations to increase participation of physicians from underserved populations in cancer treatment and prevention trials. 

NCI’s Office of Liaison Activities was established to provide a consistent point of contact with

diverse consumer constituencies, to ensure that the

Institute is aware of and responsive to cancer-related issues affecting specific population groups. In

addition, NCI has convened a Special Populations

Working Group to bring to bear the expertise of

individuals in the community on the evolving

health disparities research agenda.

All of NCI’s efforts to explain cancer-related

health disparities are closely related to our initiatives to improve the quality of care for people with

cancer and those at risk for the disease. (See p. 43

for discussion of our progress in quality of care

research and interventions.)
NCI’s Challenge

NCI’s Challenge: Building Our Capacity for the Future

NCI’s Challenge 53

THE CHALLENGE

Around the globe, patients and cancer

researchers alike are benefiting from the explosive growth of the World Wide Web and advances

in computing. But vast amounts of existing knowledge go unused because material from diverse

sources cannot be accessed and organized effectively. Therefore, we have asked ourselves, “How can

we best collect, manage, and share this information?” To answer this question, NCI is designing a

standards-based knowledge management framework – a cancer informatics infrastructure – and a

set of tools that will enable us to capture, analyze, apply, and reuse knowledge. This framework will create

electronic interfaces among cancer

research communities – basic, translational, clinical, and populationbased – and with consumers and individuals who deliver cancer care

and/or require information. The new

framework will unify the research

and cancer practice communities by

enabling easy access to cancer knowledge, drastically reducing the time

and effort needed to generate and use

information, and facilitating the rapid translation

of research observations into clinical and public

health interventions. 

As a first step, we are using the cancer informatics infrastructure to increase the speed with which

we carry out clinical trials. We have revised our criteria and standards for reporting data collected during a clinical trial and are developing common

forms, terminology, and reporting requirements

across all types of clinical trials. This uniformity

will increase the speed, efficiency, and accuracy of

results reporting. Special databases of medical and

scientific terminology will help researchers, clinicians, and other users of NCI information systems

find and understand the information they seek.

NCI’s leadership in this area will enable us to establish a national clinical trials effort that takes full

advantage of the information revolution to increase

patient accrual, establish common reporting practices, and optimize our ability to share knowledge

with the medical and research communities and the

public. (Go to cancertrials.nci.nih.gov and select

“New Trials System.”)

The new cancer informatics infrastructure is

complemented by an NCI program that aims to

integrate the informatics elements of the research

programs outlined in our extraordinary opportunity

areas. An extension of the informatics efforts developed to support the Cancer Genome Anatomy

Project (CGAP), this program will

provide an essential mechanism to

bridge the traditional barriers that

block communication of research

information among differing scientific

disciplines. New integration and tools

developed through these efforts will

also support the initiatives of the

Extraordinary Opportunity for Cancer

Communications. The NCI informatics infrastructure will help us address

the issue of computer system compatibility, help us manage the burgeoning

body of information about fundamental discoveries, and help alleviate the current 

bottleneck that exists between discovery and its

application. By moving quickly to implement the

framework and to develop general tools, we can

speed discovery, lower costs for all participants, and

capture data electronically in a way that makes

information accessible to all participants in the

cancer community. 

PROGRESS TOWARD MEETING 

THE CHALLENGE

T

o reduce the paperwork burden of researchers

participating in clinical trials and help them

more efficiently develop protocols and test

Informatics and Information Flow

GOAL

Create an informatics 

infrastructure that 

enhances information 

and resource exchange

among researchers, 

clinicians, 

and the public.
54 The Nation’s Investment in Cancer Research

THE PLAN – INFORMATICS AND INFORMATION FLOW

Goal

Create a cancer informatics infrastructure that enables cancer research by enhancing information

and resource exchange among researchers, clinicians, and the public and reduces the barriers experienced by individuals seeking information about cancer prevention, diagnosis, and treatment.

Objectives and Milestones for Fiscal Year 2002

1. Expand NCI’s informatics infrastructure to enable integration and interface within

and among basic, clinical, translational, and population research initiatives.

■ Establish a Center for Bioinformatics that supports the integration of data and

tools generated through the NCI’s Extraordinary Opportunities and facilitates

information exchange within and between NCI-supported research initiatives.

$20.00 M

■ Establish a toolbox of open source informatics applications and services based on a

common set of operating principles and standards that support diverse cancer

research activities. $5.00 M

■ Develop research tools that exploit and institutionalize the use of common data

elements and a common vocabulary to further the exchange of all types of cancer

information and data among the cancer community. $10.00 M

■ Create a standing review panel for NCI information standards that formalizes

NCI’s role in the national standards development process and incorporates these

standards into the development of informatics research tools. $0.25 M

2. Make information on cancer research, diagnosis, treatment, screening, and 

prevention more readily accessible to the entire cancer community, including

physicians, patients, survivors, family members, and persons at risk.

■ Equip the NCI’s cancer information system with powerful facilities for creating,

integrating, and accessing a wide range of cancer information resources, including

richly formatted and modular text documents and multimedia assets such as

images, sound, and video. This technical infrastructure is essential to improve

access to relevant and easily understandable cancer information. $3.00 M

■ Restructure and index current NCI information products, including approximately

300 cancer information summaries and 1,800 ongoing clinical trials descriptions in

CancerNet/PDQ. Take full advantage of the NCI’s restructured cancer information

system to enable all types of users to easily identify and interpret information relevant to their concerns and move seamlessly between different layers of information

to find the level most appropriate to their needs. Once complete, users will be able

$35.3 M

$21.0 M

advances in clinical oncology, NCI is creating the

Clinical Trials Informatics System. This innovative

system links all phases of the clinical trial life cycle,

from protocol development, distribution of information to assist patient recruitment, screening, and

protocol implementation to publication of clinical

trials results. Several components of the system

were launched in Fiscal Year 2000, including: 

■ A document management tool designed to

streamline clinical trials’ data collection relevant to NCI’s scientific, regulatory, administrative, and communication needs. This tool will

automate protocol development allowing investigators and NCI staff to concentrate on scientific issues rather than on administrative details.

It also will provide administrative information
NCI’s Challenge

NCI’s Challenge 55

to identify clinical trials based on specific eligibility criteria. $7.50 M

■ Develop Web-based search tools and user interfaces to extend the NCI’s comprehensive electronic cancer information system, making research results and clinical

trials information more easily accessible to the public. Extensive cancer vocabulary

support will enable creation of both simple and expert search options, including

interactive interfaces that guide users to information specific to their needs. $5.00 M

■ Continue evaluating the feasibility of using concept-based searching and natural

language processing. Perform pilot tests on existing NCI cancer information 

systems such as CancerNet/PDQ to ensure that, as the technology advances, 

NCI is positioned to take advantage of the value these techniques offer. $1.50 M

■ Develop an outreach plan that encourages voluntary electronic submission to

CancerNet/PDQ of information about non-NCI sponsored clinical trials, such 

as trials conducted by pharmaceutical companies and European clinical trials

organizations. $1.00 M

■ Modify the processes for writing and reviewing all clinical trials so that 

development and review can be completed in less than three months. $3.00 M 

3. Make informatics tools easily accessible and use them to integrate and disseminate

information from different cancer research communities. 

■ Expand the capacity to maintain and distribute an inventory of tools and 

technologies developed by NCI. $0.50 M

■ Develop an easily accessible and comprehensive electronic clinical trials system 

to provide uniform and easy access, enhance clinical trials recruitment, and 

promote scientific knowledge exchange among all participants. $8.00 M

■ Further develop information portals such as the CGAP Web site as broad 

information access points that provide scientific and clinical information to the

research community. $5.00 M 

■ Expand systems and tools that facilitate the grant/contract review and award

process and simplify management of NCI’s cancer research portfolio. $3.00 M

■ Develop and provide methods for non-NCI Web sites and information services 

to directly search and retrieve information from the NCI’s comprehensive 

cancer information system. $1.50 M

Management and Support

Total

$18.0 M

$3.0 M

$77.3 M

for updating and maintaining NCI’s clinical trials on the CancerNet Web site. 

■ A real-time drug inventory developed to reduce

drug waste and automate the procurement and

distribution of agents used in clinical trials.

■ A primary clinical data reporting mechanism for

NCI-sponsored trials that will simplify, standardize, and streamline clinical data reporting to

NCI to facilitate drug development and clinical

research.

■ A system using common data elements, the clinical trial enterprise models, and the

CancerNet/PDQ clinical trials registry already in

use at NCI to offer oncologists a large portfolio

of clinical trials and easy-to-use Web-based

patient and enrollment data forms. 
56 The Nation’s Investm ent in Cancer Resear ch

■ A Web-based clinical trials prototype for electronic submission of clinical trials information to

CancerNet/PDQ. 

In Fiscal Year 2000, we also created common

data elements for breast, lung, and colon cancer

treatment trials to help simplify and standardize

data collection. In 2001, we will expand our efforts

to include treatment trials focused on five additional disease sites as well as prevention, early detection, and screening trials. Through collaboration

among all NCI programs involved in informatics

activities, we are able to take full advantage of

newly developed systems to enhance existing ones. 

NCI has made substantial progress in creating a

new XML/SGML-based information infrastructure to support its comprehensive cancer information products and services, including PDQ and

CancerNet. XML-SGML is a non-proprietary standard for storing and managing information. NCI’s

use of this standard will permit information to be

used in a greater variety of information and

retrieval environments. A recently developed

search and retrieval engine has made possible the

much-improved functionality of the redesigned

CancerNet Web site. In addition, work is underway

to create a powerful new data repository that will

permit construction, integration, and maintenance

of comprehensive cancer-related information. This

new system will support a broad range of content

received from a variety of sources, including multimedia assets.

NCI is continuing development of The Science

Place (SP) (ciiserver2.nci.nih.gov/tsp/index.html),

a knowledge management tool created to enable

scientists, analysts, and managers to search and

retrieve information from many sources, to organize

and interrelate it to reflect their personal interests,

and to share their information with others.

Launched in 2000 after several years of development, the SP enables users to automatically analyze

Web-based and other documents for information of

interest based on semantic mappings from online

vocabulary systems. It also serves as a doorway, or

portal, to other NCI tools, such as the Grant

Retrieval Information Technology System.

NCI has developed the Common Scientific

Outline (CSO), a tool with which we can categorize our research in both a broadly scientific and

disease-related manner. By providing the means to

analyze a national/international research portfolio,

the CSO can have a profound effect on shaping

cancer-related research planning. Using this instrument, NCI and its fellow cancer research funding

agencies will have an enhanced foundation for

informed discussions and planning. 

NCI’s award-winning Web-based directory, NCI

Research Resources (cancer.gov/resources), directs

researchers to resources that are generally accessible

without extensive negotiation or intellectual property issues and includes descriptions of each

resource as well as contact information. 

Using the rich and diverse collection of data

generated through the various components of the

Cancer Genome Anatomy Project (CGAP)

(ncbi.nlm.nih.gov/CGAP), NCI has created an

integrated model of cancer-related genomic data

and developed a Web portal that permits

researchers to explore CGAP data for information

such as gene expression patterns in different tissue

types or gene variants in the population.

NCI has developed and implemented a modular

computer program that assists in the rapid development, deployment, integration, and maintenance of NCI initiative-specific Web sites. This

program has been used to deploy the prototype Web

sites for the CGAP portal, the Mouse Models of

Human Cancer Consortium, and the Director’s

Challenge. In support of the CGAP Web portal,

NCI also has developed a prototype for an open

source-based informatics architecture that facilitates the retrieval and integration of data distributed among multiple, independent data sources.

The open source-based code allows users to modify

it at a fundamental level. 

Budget Increase Request for 2002

Informatics and Information Flow

1. Expan d NCI’s informatics infr astructure 

to enable in tegration and interface. $35.3 M

2. Make information more readily accessible 

to the entire cancer comm unity. 21.0 M

3. Make informatics tools easily accessible and 

use them to in tegrate and dissemin ate 

information. 18.0 M

Management and Support 3.0 M

Total $77.3 M
NCI’s Challenge

NCI’s Challenge: Building Our Capacity for the Future

NCI’s Challenge 57

THE CHALLENGE

T

raining, education, and career development for

the next generation of scientists remains one of

our most important challenges. The scientists of

the future will need to be more versatile in their use

of new technologies; able to work in teams to

understand the complicated environmental,

lifestyle, genetic, and molecular variables contributing to human cancers; and better prepared to

translate discoveries into public benefit. We need

to implement and sustain multiple long-term

strategies to attract the most talented individuals to

cancer research. We need to create a

stable cadre of well-trained technical,

biological, behavioral, medical, and

public health scientists dedicated to

the cancer research enterprise. And

we need to ensure that scientists can

and will work together effectively to

solve problems. Scientists in various

disciplines can no longer operate in

isolation. The interdisciplinary environment is becoming a way of life for

research. Our success will depend

upon our ability to move beyond traditional educational and research

cultures, overcome health financing

constraints, and address socioeconomic inequities

that have proven to be barriers to progress in the

past. The theme for the future is to train scientists

to work on problems as integrated, multidisciplinary teams. 

To meet this challenge, we must continue to

implement training, education, and career development strategies to address five crucial issues. We must:

■ More adequately prepare basic scientists by

providing them with the background to conduct

research directly related to human cancer and

preparing them to collaborate with clinical and

population scientists and function in team

research settings. Ultimately, all of our discoveries in model systems must be confirmed in

human systems, and basic scientists must be prepared to make these critical contributions to

cancer research. 

■ Reverse the migration of talented and creative

physicians from research to practice. This is the

single most threatening consequence to cancer

research of the shifting economics of the health

care system. We must use more effective ways to

train clinical investigators and ensure they have

protected time to conduct the patient-oriented

research that ultimately will translate basic discoveries into better methods for cancer prevention, diagnosis, and treatment. 

■ Increase the numbers and stabilize the careers of cancer prevention,

control, population, behavioral, and

public health scientists. The discoveries of scientists dedicated to prevention, early detection, behavior modification, and risk factor analysis will have

a major impact on reducing future cancer incidence and mortality. We must

develop better ways to train these scientists to function in interdisciplinary

research settings and work effectively

with patient-oriented and basic scientists. We also must provide them with

protected time to do research. 

■ Create a research establishment that is ethnically and racially diverse. We need scientists

who are sensitive to the factors that lead to disproportionate cancer incidence and mortality in

underserved populations. All scientists must be

better trained and prepared to conduct research

that will help overcome the cultural and socioeconomic barriers responsible for the unequal

burden of cancer. 

■ Attract and integrate technical and informatics

experts into cancer research. Specialists in these

disciplines are likely to provide a critical driving

force for future progress.

Training, Education, and Career Development

GOAL

Prepare a stable, diverse

cadre of scientists to 

work together and use 

technologies for 

building knowledge and

translating discoveries 

into application.
58 The Nation’s Investment in Cancer Research

THE PLAN – TRAINING, EDUCATION, AND CAREER DEVELOPMENT

Goal

Build a stable, racially and ethnically diverse cadre of basic, clinical, behavioral, and population 

scientists trained to work together effectively and use the most advanced technologies in building

our knowledge base and in translating discoveries into more effective cancer prevention, 

detection, diagnosis, and treatment strategies.

Objectives and Milestones for Fiscal Year 2002

1. Continue to provide training, career development opportunities, and protected

research time to developing and established cancer scientists. 

■ Maintain a stable NRSA program to train predoctoral and postdoctoral basic 

scientists through traditional institutional and individual awards. $3.00 M

■ Continue to increase the participation of clinically trained individuals in basic

research and in patient-oriented research by funding 20 new individual 

mentored awards, 30 new transition awards, and 15 new established investigator

awards. $10.00 M

■ Continue to expand the number of well-trained basic, population, behavioral, and

public health scientists in cancer research through the NRSA Program. In addition, fund 20 new mentored awards, 20 transition awards for junior independent

scientists, and 10 investigator awards to established scientists. $7.00 

■ Expand the existing NCI Scholars Program by funding 10 additional scholars who

will develop their research on the NCI campus and transition to an extramural

institution and by initiating 2 new experimental training and career development

programs to link the resources of the NCI Intramural Program with extramural

institutions. The latter will be used to develop a critical mass of mentors and combined facilities where few institutions have the full range of required capabilities

(e.g., prevention, radiation oncology). $4.00 M

■ Maintain Internet information services that provide full access, descriptions, 

and instructions for all training and career development opportunities offered 

by NCI. $1.00 M

2. Continue to provide and refine special training and career development 

opportunities that prepare new and established scientists to function in 

collaborative, team research settings.

■ Increase the number of basic scientists conducting human cancer research by 

funding 30 new special bridging awards that provide both a mentored training

experience and transition funding for establishing an independent research 

program. $4.00 M

■ Fund 5 new Institutional Clinical Oncology Career Development Programs to 

prepare clinically trained individuals to become expert in clinical trials design and

implementation and lead translational research projects involving teams of clinical

investigators and basic scientists. $3.00 M

$25.0 M

$17.0 M
NCI’s Challenge

NCI’s Challenge 59

■ Implement 10 new Institutional Career Development Programs for cancer prevention, control, population, and behavioral scientists. $5.00 M

■ Expand and initiate career development opportunities in highly specialized interactive, translational, and research consortia and networks (e.g., Specialized Programs

of Research Excellence, Imaging Centers, Tobacco and Tobacco-Related Centers)

that are accessible to new and established investigators. $5.00 M

3. Continue to integrate new technical and research disciplines into the cancer

research enterprise.

■ Support new specialized institutional education and career development programs

that integrate traditional biomedical researchers with other non-traditional sciences (e.g., physics, engineering, informatics) and technology developers. $6.00 M

■ Support 5 new individual Diversified Sciences Career Development Awards to

attract other disciplines into multidisciplinary cancer research settings. $1.00 M

4. Expand programs to recruit, train, and sustain underserved racial and ethnic

minority individuals in cancer research and provide partnership opportunities for

Minority-Serving Institutions (MSIs) with NCI Cancer Centers.

■ Expand the CURE Program by increasing the number of trainee positions on institutional NSRAs by 50; providing new supplemental funding to 10 cancer centers

for high school and undergraduate student research experience; funding 10 new

minority training positions in Clinical Oncology Career Development Programs;

funding 10 new positions for Cancer Education and Career Development Programs

in the population sciences; funding 50 new Minority Investigator Supplements to

NCI research project grants; and funding 20 new mentored career development

awards for basic scientists and clinically trained scientists. $11.50 M

■ Develop and add new features to the CURE Program by funding 10 new Career

Transition Awards for basic, clinical, and population minority scientists in their

first junior faculty positions and 20 NCI Cancer Centers to “broker” connections

between minority individuals seeking research experiences and the cancer center

scientists. $2.50 M

■ Expand the number of planning and partnership grants to MSIs and NCI Cancer

Centers by funding 10 collaborative planning grants focused on training and 

education programs, one comprehensive partnership planning grant, and 2 

comprehensive partnerships. (See p. 30, Objective 1 for the research aspect of

these partnerships.) $2.00 M

■ Increase minority access to training and career development opportunities by

improving NCI Internet information services and linking to public and private

agencies that provide related services. $1.00 M

Management and Support

Total

$7.0 M

$17.0 M

$2.0 M

$68.0 M
60 The Nation’s Investment in Cancer Research

PROGRESS TOWARD MEETING 

THE CHALLENGE

Avariety of individual and institutional training

and career development awards are being

employed to meet the needs of new and established

investigators and NCI’s anticipated research priorities. Special programs have focused increased

resources on career tracks for M.D.s in cancer

research, behavioral and population scientists,

minority scientists, and scientists in highly technical fields important to the future of cancer research. 

Individual Awards

■ Individual mentored five-year awards provide

opportunities for M.D.s in basic or clinical

research and individuals pursuing cancer prevention, control, behavioral, and population science.

■ Bridging awards encourage basic scientists and

minority scientists to pursue careers in cancer

research. These awards provide a mentored period

followed by an unmentored period and protected

time for developing first independent research

programs.

■ Transition awards provide for three years of protected time following mentored postdoctoral

training or for new investigators to initiate successful research programs. These awards are now

in place for NCI’s two most critical areas of need,

medically trained doctors in basic and clinical

research and population scientists. A transition

award for minority scientists will be established

in 2000.

■ Established investigator awards provide seasoned

investigators in the clinical sciences and in cancer

prevention, control, behavioral, and population

sciences protected time to conduct research and

mentor new scientists. NCI believes that this

award for clinical scientists will successfully curtail their migration from research to patient care.

This award for cancer prevention, control, behavioral, and population scientists is new in 2000.

■ New diversified sciences career development

awards attract technology developers and scientists in disciplines not traditionally associated with

cancer research but clearly needed for the future.

Institutional Awards

Institutional awards are for five years, for developing

and conducting training and career development

programs. These awards achieve special goals by

establishing specific requirements and assembling

mentors whose skills support program objectives.

■ National Research Service Awards (NRSA),

NCI’s mainstay for training basic scientists, now

includes provisions for curriculum and research

environments that orient all trainees to cancerrelated opportunities and approaches of the future.

■ Institutional Clinical Oncology Career

Development Programs prepare the next generation of clinical scientists to design and implement hypothesis-based clinical trials and to collaborate with basic scientists.

■ Institutional Education and Career Development

Programs, initiated in 2000, prepare participants

for collaborative, multidisciplinary team

research settings.

■ The Continuing Umbrella of Research

Experiences (CURE) Program engages minority high school and undergraduate students and

encourages and assists them to become independent investigators. 

■ Minority Institution/Cancer Center Partnerships link over 300 MSIs with NCI Cancer

Centers to increase the number of minorities

engaged in cancer research; strengthen the

research capabilities of minority institutions;

and support Centers in reducing cancer incidence and mortality in minority populations. 

For more information on these programs and

career tracks, go to cancertraining.nci.nih.gov.

Budget Increase Request for 2002

Training, Education, and Career

Development

1. Provide training, career opportunities, and 

protected time for developing and 

established scientists. $25.0 M 

2. Provide and refine special opportunities in 

collaborative, team research settings. 17.0 M

3. Integrate new technical and research 

disciplines into cancer research. 7.0 M 

4. Expand programs for underserved racial and 

ethnic minorities. 17.0 M 

Management and Support 2.0 M

Total $68.0 M
My mother died from ovarian cancer

when I was 17. By the time she

went to the doctor about the leg

pains and bloating, the cancer was

already advanced. 

Symptoms of ovarian cancer are

often vague and may be ignored or

mistaken for other illnesses. A

woman may feel bloated or have

general discomfort in the lower

abdomen. She may have a loss of

appetite or a feeling of fullness,

even after a light meal. She also

may experience frequent indigestion,

gas, or nausea. A tumor that presses

on the colon or bladder may cause

diarrhea, constipation, or frequent

urination. In some cases, fluid

buildup around the lungs can cause

shortness of breath. Contrary to

common belief, unusual vaginal

bleeding is rarely a symptom of

ovarian cancer.

This year, an estimated 23,100 new

cases of ovarian cancer will be diagnosed in this country, and nearly

14,000 women will lose their lives

to the disease. Ovarian cancer is the

sixth most common cancer in

women, and the fifth most common

cause of cancer death among

women. It has the highest mortality

rate of all female reproductive system cancers.

We got a computer this year, and

I’ve found information on the

Internet about ovarian cancer. The

trouble is, there are screening tests,

but they’re not very effective. It’s

over 50 miles from our farm to the

hospital where the gynecologist is,

but I’d make that trip any day of the

week for a test that could tell for

sure if I have cancer or not. 

The lack of a reliable screening test

has been major barrier to improving

ovarian cancer detection and

decreasing the chance of dying from

ovarian cancer. Routine pelvic examination, the standard screening

method, misses one-third of even

relatively large ovarian tumors. Two

other screening tests for ovarian

cancer are sometimes used. One is a

blood test that measures levels of a

protein called CA-125 that is produced by some ovarian cancer cells,

and the other is transvaginal ultrasound imaging. However, because

both tests have important limitations, they usually are performed

only if a woman has symptoms or is

known to have an inherited risk for

the disease. NCI’s Prostate, Lung,

Colorectal, and Ovarian (PLCO)

Screening Trial (dcp.nci.nih.gov/

plco) is currently exploring whether

or not routinely using these two

tests together might be more accurate and ultimately decrease the

number of ovarian cancer deaths.

(continued on next page)

People ’ s Story

Ovarian Cancer

People’s Story 61

People’s Story
Recognizing the urgent need for better ovarian cancer detection methods, NCI has several exciting new

activities underway. Intensive

research is being conducted to find

more accurate, simple marker tests

for ovarian cancer. For example,

some studies suggest that ovarian

tumors may have unique secretions

that, when detected, could signal

the earliest stages of cancer, or even

pre-cancer. Several centers and laboratories funded under NCI’s Early

Detection Research Network

(edrn.nci.nih.gov) are devoted to

identifying such biological indicators

of ovarian cancer that could be

detected quickly, easily, and 

accurately.

In addition, because it is so difficult

to obtain very early stage ovarian

cancer tissue for study, researchers

are working on designing mouse

models to learn how this cancer

develops. Recently, several mouse

models for early ovarian tumors have

been developed that show promise

for uncovering the earliest cellular

and related molecular changes that

give rise to ovarian cancer. Work in

this area has been greatly 

accelerated by the funding of four

Special Programs of Research

Excellence (SPOREs) dedicated to

ovarian cancer research, and by collaborations among researchers in

NCI’s Mouse Models of Human

Cancers Consortium, In Vivo Cellular

and Molecular Imaging Centers,

Cancer Genetics Network, and the

Divisions of Basic Sciences and

Clinical Sciences.

There are better treatments for ovarian cancer than there were when my

mother was sick, but they still need

to be improved.

Following surgery, women with 

ovarian cancer usually receive a

combination chemotherapy that

includes a drug containing platinum

and a taxane such as paclitaxel

(Taxol®). Recurrences may be treated

with additional doses of these

agents or with other anti-cancer

drugs. New or improved treatments

for ovarian cancer are being tested

in clinical trials. For example, a

recently reported randomized trial

compared a combination of paclitaxel

and cisplatin for treating advanced

ovarian cancer with a combination

of cyclophosphamide and cisplatin.

After nearly four years of follow-up,

the paclitaxel/cisplatin patients

showed improved survival and remission. Other new therapies under study

include high-dose chemotherapy

with bone marrow or stem cell 

transplant, other new chemotherapy

combinations, and gene and immune

system therapies, such as vaccines

that help the immune system recognize cancer cells. In addition, 

laboratory and animal studies are

underway to determine if lowering

certain hormone levels in women

treated for ovarian cancer will prevent the disease from recurring.

Since my mother had ovarian cancer,

my risk for the disease is higher

than average. My doctor says that if

my mother, sister, or even my

daughter has ovarian cancer, I am

nearly three times more likely than

other women to get it. So I’m going

to be careful – I don’t want my kids

to lose their mother the way I did.

Five to 10 percent of ovarian cancers are believed to be caused by

inherited mutations in the BRCA 1 or

BRCA 2 genes, which also increase

breast cancer risk. NCI is sponsoring

further research into inherited

genetic ovarian cancer risk through

its Cancer Genetics Network,

Cooperative Family Registries, and

Clinical Epidemiologic Studies in

Hereditary Breast/Ovarian Cancer. 

NCI is committed to improving ovarian cancer prevention, detection,

and treatment, and fully expects

that the new ovarian cancer SPORES,

which support innovative, multidisciplinary research with the potential

to have an immediate impact on

cancer care and prevention, will be

important hubs of progress against

this disease. In 2001, NCI will bring

together leaders in basic, clinical,

and population research and the

consumer community in a Progress

Review Group to assess the state of

knowledge and identify research priorities to hasten progress in understanding, preventing, and treating

ovarian cancer and other reproductive system cancers in women. 

Up-to-date information on ovarian

cancer is always available on NCI’s

Web site, CancerNet

(cancernet.nci.nih.gov) to help

women understand this disease and

learn about advances in screening,

risk assessment, and treatment.

Note: This vignette is a composite of experiences.

62 The Nation’s Investment in Cancer Research
NCI’s “extraordinary opportunities for investment” are scientific areas of cancer research

in which focused efforts and increased

resources can build on past successes to stimulate

dramatic progress toward reducing the cancer burden. If pursued, these doors to continued discovery

hold great promise to provide profound insights

into how cancer develops and to lead to vastly

improved techniques for preventing, detecting,

diagnosing, and treating various forms of cancer.

Although the needed resources are not trivial, our

failure to respond quickly with investment in all of

these areas will slow the pace of cancer research at

all levels and will impair our ability to better care

for those whose lives have been touched by cancer. 

NCI seeks formal input from a broad spectrum of

perspectives from the cancer research community,

members of advisory groups, and advocates to identify these areas of exceptional promise. Every three

years, we ask these scientific, professional, and lay

experts in the cancer field to revisit the “extraordinary opportunities” and select emerging investment

research areas for the next three-year cycle. We

assess these responses extensively, blend related

ideas, and, with our advisors, select new investment

areas. The current set of six extraordinary opportunities were first outlined in NCI’s Fiscal Year 2001

budget proposal and are continued in this proposal

for Fiscal Year 2002. 

To qualify as extraordinary opportunities,

research areas must: 

■ Respond to important recent developments in

knowledge and technology.

■ Offer approaches to cancer research beyond the

size, scope, and funding of our current research

activities.

■ Be implemented with specific, defined investments.

■ Be described in terms of achievable milestones.

■ Promise advances for making progress against 

all cancers.

These investment areas result in new research

awards, new or expanded programs, and collaborations.

Extraordinary Opportunities for Investment

Budget Increase Request for 2002

(dollars in thousands)

Extraordinary Opportunities 63

Opportunities

Extraordinary

Extraordinary Opportunities 

for Investment

Cancer Communications

5%

Genes 

and the

Environment

13%

Cancer

Imaging

18%

Defining the

Signatures of 

Cancer Cells

22%

Molecular Targets

for Prevention 

and Treatment

29%

Research on

Tobacco and

Tobacco Related

Cancers Amount 13%

Genes and the Environment $68,250

Cancer Imaging 90,550

Defining the Signatures of 

Cancer Cells 110,750

Molecular Targets for Prevention 

and Treatment 146,500

Research on Tobacco and 

Tobacco-Related Cancers 67,000

Cancer Communications 27,500

Total $510,550
THE OPPORTUNITY 

C onceptual and technical breakthroughs and the

often breathtaking pace of scientific discovery

have engendered among cancer researchers a

tremendous sense of optimism that new avenues

will be found to prevent, detect, diagnose, and treat

cancer. Nowhere is this sense of promise greater or

the potential implications more profound than at

the interface of epidemiology and genetics. By marrying the study of the distribution and causes of

cancer in human populations with cutting-edge

genetic and related molecular technologies, we will

over time be able to:

■ Identify genes that predispose people to cancer and point to previously unsuspected environmental

carcinogens.

■ Study genetically susceptible subgroups to predict the increased risk

that may result from their exposure to certain types of substances

in the environment.

■ Quantify the cancer risks associated with specific environmental

and genetic factors and their interactions, enabling us to guide individual and public health strategies

aimed at preventing and controlling cancer. 

■ Design new approaches to health and cancer

care based on an understanding of how genes

modify and interact with environmental 

exposures.

The pivotal role of lifestyle and other environmental exposures as causes of cancer is reflected in

the substantial variation in cancer incidence

around the world and in the changes in risk

observed among groups that migrate and settle in a

new country. Furthermore, epidemiologic research

has identified a wide range of cancer-causing exposures including tobacco use, dietary components,

sunlight, ionizing radiation, environmental chemicals, and infectious agents as well as risk factors

such as obesity, exercise, hormones, reproductive

factors, and sedentary lifestyle. 

Cancer susceptibility is another critical piece of

the puzzle. For example, why does one person with

a cancer-causing exposure (smoking or infection

with human papillomavirus, for example) develop

cancer, while another does not? Intensive study of

rare cancer-prone families has provided insights

into this apparent paradox. We know that disruption of fundamental cellular processes contributes

to the development and progression of the more

common, non-hereditary forms of

cancer. Yet even among individuals

who have inherited cancer-predisposing genes, the risk of developing cancer appears to be modified by other

genetic and environmental factors.

There is mounting evidence that a

person’s genetic make-up may influence susceptibility or even resistance

to cancer-causing exposures. Opportunities now exist to determine how

variations in these genes combine

with environmental and other factors

to induce cancer in the general 

population. 

The exciting opportunities of this

emerging field are accompanied by enormous challenges. Population studies sufficiently powerful to

examine the complex interactions of multiple

genetic and environmental factors will require

unprecedented numbers of participants and will

demand new research infrastructures and strategies

for interdisciplinary collaboration. As we gain in

our understanding of individual-specific risk factors, we must strive to ensure that this knowledge is

used appropriately and is not harmful to the individual. Psychosocial, legal, ethical, and clinical

issues must be addressed, and individuals, families,

and health care providers must be able to take

appropriate action without repercussions. 

64 The Nation’s Investment in Cancer Research

1

Extraordinary Opportunities for Investment

Genes and the Environment

GOAL

Discover genetic, 

environmental, and 

lifestyle factors 

and their interactions 

that define cancer 

risk and inform 

strategies for 

cancer control.
PROGRESS IN PURSUIT 

OF OUR GOAL

NCI’s earlier investments into the study of

genetics have yielded enormous dividends.

For example, NCI’s Cancer Genome

Anatomy Project (CGAP) has resulted in the discovery of over 44,000 new genes. New technologies

have permitted scientists to determine which genes

are active in normal or in cancerous tissues. There

has been an exponential increase in the pace of

identifying genes that maintain the integrity of our

genetic material, regulate cell growth and development, and determine our response to hormones and

other chemicals produced by the body or in the

environment. Related discoveries have enabled us

to characterize the function of hundreds of new

genes and pathways. Vast public databases contain

millions of entries describing gene sequences and

their location in the human genome.

Establishing significant and valid evidence for

gene-environment interactions requires studies of

large populations over long periods of time. In

cohort studies, information on exposures to factors

that might affect cancer risk and biologic samples

are collected from individuals in large population

subgroups. By systematically following these people

over time to determine who develops cancer and

who remains cancer free, scientists can understand

the risk of developing cancer for those with specified exposures and genetic profiles. In this way,

early detection can be directed to those at greatest

risk and diagnosis and treatment can be tailored to

individual needs. NCI is establishing a Cohort

Consortium of investigators from around the world

to facilitate the pooling of data on very large numbers of people, foster collaborative links among

resources, and organize collaborative studies. Casecontrol studies retrospectively examine exposure histories and genetic profiles of people who already

have cancer (cases) and compare them with those

of people who have not developed cancer (controls). NCI is assembling a Case-Control

Consortium to support large-scale studies of geneenvironment interactions for less common cancers.

NCI has expanded the tools available to the

cancer genetics community through the World

Wide Web. Through the Genetic Annotation

Initiative (GAI) of CGAP, scientists have identified more than 20,000 genetic variations, and they

expect to expand that number to nearly 500,000 by

2002. Researchers are using sophisticated computer

programs to identify variations in specific genes in

people with cancer to determine which variants are

associated with certain types of cancer and whether

some variants occur more often in some populations. New technology development through the

Innovative Molecular Analysis Technologies

Program is also improving our ability to effectively

analyze the large volumes of samples and data in

these population-based studies. 

Members of the Mouse Models of Human

Cancers Consortium (MMHCC) are developing

and validating mouse models – mice with cancers

similar to the major human cancers that can be

inherited. These models will be made available to

scientists for research. Composed of 20 groups of

investigators from institutions across the country,

the MMHCC uses Web-based discussion forums

and other communication tools to integrate emerging knowledge about cancer susceptibility from animal models with studies on human populations.

The MMHCC also supports a repository for models

of key cancers caused by specific gene variants. 

We have gained tremendous insight into risks for

cancer by examining the personal and medical histories of high-risk families and investigating how

Extraordinary Opportunities 65

Opportunities

Extraordinary

Budget Increase Request for 2002 

Genes and the Environment

1. Identify environmental risk factors and 

susceptibility genes. Determine their 

interactions in cancer causation. $22.0 M

2. Develop new ways of assessing and 

measuring environmental exposures. 10.5 M

3. Identify and characterize 

gene variations. 4.0 M

4. Develop new experimental models. 5.0 M

5. Identify cancer-predisposing genes in 

high-risk families. 8.0 M

6. Refine cancer risk prediction 

methods/models. 6.8 M

7. Expand enrollment of genetically high-risk 

individuals into clinical protocols and 

conduct cancer susceptibility studies. 9.0 M

Management and Support 3.0 M

Total $68.3 M
66 The Nation’s Investment in Cancer Research

THE PLAN – GENES AND THE ENVIRONMENT

Goal 

Discover genetic, environmental, and lifestyle factors and their interactions that define cancer risk

and can inform the development of new strategies for prevention, early detection, and treatment.

Objectives and Milestones for Fiscal Year 2002

1. Identify new environmental risk factors and susceptibility genes and determine

their interactions in cancer causation. 

■ Expand the biospecimen collections of the Cohort Consortium to increase the

number of individuals and diversity of the populations covered and to allow development of studies of gene expression and other molecular profiles. $6.00 M

■ Fund and conduct 2 large gene-environment, case-control studies of less frequently

occurring cancers, one in a population-based cooperative group and the other in a

hospital-based group. Cancer types selected will be based on increased incidence,

ethnic disparities, and geographic patterns. $10.00 M

■ Begin development of 2 regional centers for state-of-the-art biospecimen processing

and storage and for high throughput genotyping to support molecular 

epidemiology and other interdisciplinary studies of human cancer. $4.00 M

■ Continue development of informatics systems to collect, store, analyze, and integrate the vast amounts of epidemiologic, clinical, and laboratory data. $2.00M

2. Develop new ways to assess and measure environmental exposures for use in 

population studies.

■ Continue developing new epidemiologic methods for assessing complex exposures

over a lifetime. Coordinate the use of these methods for gene-environment 

interaction studies. $2.00 M

■ Further expand NCI’s Innovative Molecular Analysis Technologies Program to

develop new non-invasive techniques for collecting and measuring DNA and proteins in very small amounts of biologic material. Apply these techniques to largescale studies of gene-environment interactions. $2.00 M

■ Supplement ongoing research programs to develop and validate measures of the

cumulative cellular, genetic, and molecular effects of exposure to environmental

carcinogens in non-tumor tissue. $1.00 M

■ Continue to expand and supplement research programs to develop and 

validate molecular approaches to define mutation patterns that implicate 

specific carcinogens. $1.00 M

■ Use emerging technologies to develop molecular and immunologic techniques that

will enable screening of large numbers of biologic samples to identify infectious

agents relevant to human cancer. $4.00 M

■ Continue to work with academic centers, cancer centers, and schools of public

health to develop model training programs in molecular epidemiology. $0.50 M

3. Identify and characterize gene variations involved in molecular pathways 

important in cancer.

■ Extend CGAP’s GAI efforts to identify new gene variants relevant to cancer in

clinically- and epidemiologically-defined populations. $2.00 M

$22.0 M

$10.5 M

$4.0 M
Extraordinary Opportunities 67

■ Expand the GAI’s efforts to define key molecular pathways by performing comprehensive genetic variation characterizations on an extended set of gene and protein

expression profiles. $2.00 M

4. Develop new experimental models that parallel human cancer-related genes, 

pathways, and processes.

■ Augment the MMHCC to:

■ Derive and refine mouse models of human hereditary cancer genes for which

models do not exist. $1.00 M

■ Generate valid mouse models based on epidemiologic observations of genetic

and environmental modifiers of cancer risk. $1.00 M

■ Evaluate within the collection of appropriate models the potential for environmental factors to modify cancer development. $1.00 M

■ Discover biomarkers and in vivo approaches for early detection of cancer and to

develop and test new prevention strategies. $1.00 M

■ Stimulate development of new non-mammalian models of complex interactions

among the pathways and processes that contribute to cancer etiology. $1.00 M

5. Identify cancer-predisposing genes in high-risk families and investigate how 

expression of these genes is modified by other genes and environmental factors.

■ Fund 2 new consortia of investigators to identify the genetic basis of familial 

aggregations of cancers not currently linked to genes. $2.00 M

■ Expand the network of cooperative registries to enhance recruitment of 

high-risk families and foster development of shared informatics and other 

research tools. $3.00 M

■ Initiate large collaborative interdisciplinary studies of high-risk families to quantify 

cancer risk among carriers of major genes and investigate the genetic and environmental modifiers of risk. $1.00 M

■ Continue to expand the capacity and use of the NIH Center for Inherited 

Disease Research and other activities to accelerate cancer gene 

identification. $1.00 M

■ Initiate collection of fresh-frozen tumor tissue and other biospecimens from 

genetically cancer-prone individuals and families for microarray-based analyses of

molecular signatures of cancer. $1.00 M

6. Refine cancer risk prediction methods/models to integrate genetic and 

environmental determinants of cancer.

■ Augment clinical trials and observational studies of breast cancer to validate 

current risk prediction models and formulate new ones. Expand development of

risk models that predict the occurrence of other major cancers. $2.00 M

■ Expand risk prediction model application to target interventions aimed at cancer 

prevention, detection, diagnosis, or treatment. $1.50 M

■ Extend risk models to account for both cancer and non-cancer risks and to fit 

ethnic and minority populations. $1.00 M

$5.0 M

$8.0 M

$6.8 M

Opportunities

Extraordinary
cancer-predisposing genes are modified by other

genes and environmental factors in these families.

For example, through the Cooperative Family

Registries for breast/ovarian and colorectal cancers, we have collected clinical, epidemiological,

and pathological data as well as biospecimens for

over 8,000 high-risk families. Analysis of this information may lead to targeted approaches for the prevention, detection, and diagnosis of cancer.

New insights into genetic susceptibility, environmental carcinogens, and their potential interactions can be incorporated into cancer risk 

prediction models that can in turn be used to estimate individual risk. Breast cancer risk models

have been employed in two large cancer prevention trials and were found to be good predictors of

cancer risk. We now want to develop risk models

for other cancers.

Clinical trials involving genetically high-risk

individuals can increase our understanding of the

clinical, behavioral, and societal issues associated

with cancer susceptibility. One project in this area

has been a feasibility study of the comparative value

of early screening and diagnostic tools in breast

cancer gene mutation carriers. Another study is a

pilot program for screening and chemoprevention

in men who carry susceptibility genes for breast or

ovarian cancer, which have been shown to increase

prostate cancer risk. To gain further insights into

early detection, NCI has begun a follow-up study of

women prone to breast cancer who gave cell samples that were used to search for molecular fingerprints of pre-malignant cells.

NCI has established a state-of-the-art bioprocessing and biorepository facility and a genotyping facility to handle large volumes of specimens

and data to support NCI efforts in molecular epidemiology. These facilities are prototypes for a

regional network to support molecular epidemiology studies.

For more information on NCI’s Genes 

and the Environment initiatives, go to 

cancer.gov/initiatives.

68 The Nation’s Investment in Cancer Research

■ Devise and test risk communication strategies best suited to patients, 

professionals, and policy makers. $2.00 M

■ Create a risk prediction model prototype that accommodates gene-environment 

interactions for selected cancers. $0.25 M

7. Expand enrollment of genetically high-risk individuals into clinical protocols and 

conduct studies to address the clinical, behavioral, and societal issues associated

with cancer susceptibility. 

■ Recruit genetically high-risk individuals into clinical studies to assess the efficacy

of preventive, early detection, diagnostic, and treatment interventions. $2.50 M

■ Continue support for behavioral research within NCI-funded networks and other

settings to assess the impact of predictive genetic testing and cancer risk assessment on high-risk individuals and their families and to develop and evaluate

interventions that mitigate the negative consequences of genetic testing. $2.50 M

■ Support research in cancer survivorship to enable long-term follow-up of cancer

patients on the late physical and psychosocial effects of treatment and to 

evaluate the role of genetic susceptibility in these outcomes. $2.50 M

■ Continue and expand development and evaluation of educational tools to 

facilitate informed decision making about participation in cancer genetics

research, genetic testing, and cancer risk reduction. $1.50 M

Management and Support

Total

$9.0 M

$3.0 M

$68.3 M
THE OPPORTUNITY

As recently as 25 years ago, a physician or surgeon

who suspected the presence of a tumor in a

patient had few options: order x-ray studies to find

the tumor’s exact location, schedule the patient for

surgery, excise a portion of the unhealthy tissue for

biopsy, remove the tumor, and explore surrounding

tissues to determine if the cancer had spread. Over

the last quarter century, imaging technology refinements have substantially broadened the range of

medical options. Imaging tests now provide much

clearer and more detailed pictures of organs and tissues. New imaging technology allows

us to do more than simply view

anatomical structures such as bones,

organs, and tumors. Functional imaging – the visualization of physiological, cellular, or molecular processes in

living tissue – enables us to observe

activity such as blood flow, oxygen

consumption, or glucose metabolism

in real time. 

As we understand more fully cancer’s fundamental nature, our capacity to use imaging tools to detect the

molecular changes associated with a

tumor cell promises to vastly improve

our ability to detect and stage tumors, select treatments, monitor the effectiveness of a treatment,

and determine prognosis. 

Detection and Diagnosis

Imaging technology already has had lifesaving

effects on our ability to detect cancer early and

more accurately diagnose the disease. For example:

■ X-ray mammography has revealed the presence

of very small cancers in thousands of women

before the tumors could be detected by physical

examination.

■ Computed tomography (CT) and ultrasound permit physicians to guide long, thin needles deep 

within the body to biopsy organs, often eliminating the need for an open surgical procedure. 

■ CT can show if a tumor has invaded vital tissue,

grown around blood vessels, or spread to distant

organs. 

As the science advances, seeing how the processes and pathways inside a cell change as the cell

transforms from normal to cancerous will allow us

to detect changes in people earlier, and eventually

we expect to be able to visualize the actual molecular signatures of a cancer. We will be able to tell, in

the radiology suite of a hospital, which genes are

being expressed in a patient’s cells,

and we will be able to translate this

information directly into better management of the disease. 

Treatment

In addition to using CT and other

imaging technologies to guide treatment choices, combining precise imaging techniques with radiation sources

and high performance computing is

improving our ability to shape radiation

treatments to the tumor’s three-dimensional contours. Using today’s technology, we can identify the kinds of molecular structures/receptors that cover the surface of a

tumor and use this information to predict how it may

respond to certain treatments. With a picture of how

glucose is being used in tumor cells, we can tell –

without the need for a biopsy – how a tumor is

responding to a recently administered treatment.

In principle, imaging techniques can be interfaced with other tumor killing approaches – toxic

chemicals, gene therapy, heat, and cold – to more

precisely guide tissue destruction at the tumor site.

Being able to distinguish between cancerous and

normal tissue and deliver treatments only to diseased tissues in a minimally invasive way has the

potential to minimize surgical trauma, shorten

recovery time, and reduce health costs. 

Extraordinary Opportunities 69

2

Extraordinary Opportunities for Investment

Cancer Imaging 

GOAL

Accelerate discovery 

and development 

of imaging methods 

that will predict 

clinical course 

and response to 

interventions.

Opportunities

Extraordinary
PROGRESS IN PURSUIT 

OF OUR GOAL

We have made important strides, but much

remains to be done before the full promise of

the imaging sciences is realized for cancer research

and care. Having laid the groundwork, we are working toward greater improvements in cancer detection, diagnosis, and treatment that will provide real

benefits for people with cancer and those at risk.

Several new initiatives illustrate our progress. 

To foster multidisciplinary research on cellular

and molecular imaging, NCI has established three

In Vivo Cellular and Molecular Imaging Centers

(ICMIC) and awarded ten planning grants for additional Centers. These Centers will narrow the gap

between the discovery of new cancer genes and

intracellular pathways and the translation of these

discoveries into clinically useful, minimally invasive imaging approaches to expanding our understanding of cancer. 

NCI is funding projects to assist investigators

and small businesses in developing digital mammography display and workstation technology.

Digital mammography is one of the most promising

technologies for use in large-scale screening programs to improve breast cancer detection. 

To speed the development of new imaging methods, NCI has created the Small Animal Imaging

Resource Program (SAIRP). Our five SAIRP centers are developing and applying a wide variety of

functional, quantitative imaging modalities. For

example, an SAIRP-developed magnetic resonance

(MR) technique can detect prostate tumors less

than one millimeter in size in a mouse model. An

MR technique that measures the growth of brain

tumors in rats is being used to look for very early

recurrence of brain tumors in children.

Quantitating imaging data for small animals will

lead the way to methods that can be applied in

humans. The SAIRP’s progress in technology

development and its usefulness as a resource to cancer researchers, particularly Mouse Models of

Human Cancer Consortium (MMHCC)

researchers, has been greater than anticipated.

NCI’s Preclinical Models Imaging Forum will link

experts in the MMHCC and the SAIRP, and information from the forum will help in developing and

validating new preclinical models and in designing

and testing imaging techniques to detect human

cancers.

NCI has launched the Diagnostic Imaging

Network in partnership with the American

College of Radiology. The Network brings together

imaging experts to perform a broad spectrum of

multi-institutional clinical trials on new and

refined imaging tools. A number of such trials have

been launched or are in preparation, including a

comparison of MRI and CT in diagnosing gynecologic cancers, the use of positron emission tomography (PET) to monitor response to chemotherapy,

assessment of spiral CT for detecting lung malignancies, a comparative study of digital versus conventional mammography, and a comparison of MRI

versus CT for staging pediatric malignancies.

The Development of Clinical Imaging Drugs

and Enhancers (DCIDE) program will foster and

speed the development of promising imaging

enhancers (contrast agents) and molecular probes

and their translation from laboratory synthesis to

Investigational New Drug (IND) application. NCI

will make its preclinical development resources

available to competitively selected developers of a

promising diagnostic agent or probe in order to

remove a recognized barrier between laboratory discoveries and their entry into the clinic. To further

aid the development of promising imaging agents,

NCI is launching a DCIDE-like program to fund

early trials of novel imaging probes.

To improve imaging technologies for prostate

cancer detection that could enable clinicians to

more accurately localize and stage a tumor and

select an optimal therapy, NCI has established the

Phased Innovation Award for Diagnostic Imaging

and Guided Therapy in Prostate Cancer. In 2001,

several awards will be made for research to improve

image-guided surgery and radiotherapy. 

The Innovative Technologies for the Molecular

Analysis of Cancer award provides assistance for

high-resolution cellular or molecular imaging

research, access to tissue samples, development of

preclinical models, and the conduct of clinical

investigations as an important extension of molecular analysis methods. The Applications of

Innovative Technologies for the Molecular

Analysis of Cancer awards will provide a means of

piloting and evaluating newly developed high-resolution imaging technologies.

70 The Nation’s Investment in Cancer Research
To promote the development, testing, and adoption of new imaging modalities and applications,

NCI has organized a unique forum that brings

together technology developers from academia

and industry with the funding agencies, regulators, and reimbursers of technology. NCI’s partners include the Food and Drug Administration

(FDA), the Health Care Financing Administration

(HCFA), other third-party payers and providers,

and major device manufacturers through the

National Electrical Manufacturers’ Association.

These partnerships promote communication and

progress in this critical area. The first forum was

held in September 1999 and the second is planned

for September 2000.

NCI, FDA, and HCFA also have formed an

Interagency Council on Biomedical Imaging in

Oncology to offer a multi-agency perspective on

communicating with government for investigators

and manufacturers attempting to bring emerging

medical imaging technology to the marketplace.

Council members have experience and knowledge

about their agency’s decision making processes concerning medical imaging products and will provide

advice on projects or proposals voluntarily submitted by investigators and technology developers in

industry and academia.

Because partnerships are critical in developing

new and emerging imaging technologies, NCI has

formed a partnership with the National Science

Foundation (NSF) to stimulate research on biophotonics – the study of light and its clinical applications. In addition, plans have been laid for NSF

to fund image processing research using a spiral CT

lung database developed through NCI-sponsored

research. 

NCI also plays a key role in the trans-NIH

Bioengineering Consortium (BECON – see

grants.nih.gov/grants/becon/becon2.htm), which

fosters new basic understandings, collaborations,

and transdisciplinary initiatives among the biological, medical, physical, engineering, and computational sciences related to bioengineering applications and advances. Research and collaborations

supported through BECON will help NCI develop

imaging technologies for detecting and treating

cancer. 

For example, NIH has created the

Bioengineering Research Partnership Programs to

support multidisciplinary research teams with bioengineering expertise that are applying an integrative, systems approach to developing knowledge or

methods to prevent, detect, diagnose, and treat disease and understand health and behavior. NIH also

has established the Bioengineering Research

Grants initiative with a similar scientific scope, but

designed for a limited number of investigators or

institutions.

For more information on NCI’s Cancer 

Imaging initiatives, go to cancer.gov/bip or 

cancer.gov/initiatives. 

Budget Increase Request for 2002 

Cancer Imaging

1. Strengthen the foundations of imaging 

technologies and methodologies. $11.0 M

2. Expand the development of novel 

imaging agents. 8.1 M

3. Integrate molecular and functional 

imaging methods into molecular target 

discovery, drug development, and early 

clinical trials. 5.0 M

4. Develop and use imaging as an endpoint 

in clinical trials. 48.5 M

5. Accelerate development and clinical 

testing of image-guided interventions. 5.0 M

6. Develop and conduct early clinical trials 

of innovative imaging technologies. 5.0 M

7. Establish information archives and 

repositories for the research community. 4.5 M

Management and Support 3.5 M

Total $90.6 M

Extraordinary Opportunities 71

Opportunities

Extraordinary
72 The Nation’s Investment in Cancer Research

THE PLAN — CANCER IMAGING

Goal 

Accelerate discovery and development of imaging methods to identify the biological and 

molecular properties of precancerous or cancerous cells that will predict clinical course and

response to interventions.

Objectives and Milestones for Fiscal Year 2002

1. Strengthen the foundations of imaging technologies and methodologies to assist in

fundamental investigations of cancer.

■ Fund 2 additional ICMICs. $4.00 M

■ Fund 8 to 10 supplements for collaborations between SAIRPs and other NCI 

programs such as the MMHCC. $3.00 M

■ Fund 8 to 10 seed grants and 6 Phased Innovation Awards for research on 

clinical applications of optical technologies, or integration of optical technologies

with other imaging. $4.00 M

2. Expand the development of novel imaging agents. 

■ Increase the number of imaging agents supported by the DCIDE program from 

6 to 12 per year. $6.00 M

■ Increase contract support for early clinical trials of imaging agents (safety and 

efficacy studies) from 8 to 12 trials per year. $2.00 M

■ Enhance the publicly available database of imaging agents by adding information 

on their properties. $0.10 M

3. Integrate molecular and functional imaging methods into molecular target 

discovery, drug development, and early clinical trials. 

■ Fund 8 supplements for imaging collaborations with the grantees in the Molecular

Target Drug Discovery initiative. $2.00 M

■ Fund 6 to 8 supplements for developing imaging cores within the Interdisciplinary

Research Teams for Molecular Target Assessment. $2.00 M

■ Fund imaging cores within the Molecular Target Laboratories. $1.00 M 

(For more information about molecular target initiatives, see pp. 82-83.)

4. Develop and use imaging as an endpoint in clinical trials. 

■ Fund supplements for 10 to 15 imaging cores within NCI-funded Cancer 

Centers to provide expertise in clinical trials that use imaging results as an 

endpoint. $5.00 M

■ Support expert panels to develop consensus criteria for using imaging results as

endpoints in clinical trials. $0.20 M

$11.0 M

$8.1 M

$5.0 M

$48.5 M
Opportunities

Extraordinary

Extraordinary Opportunities 73

■ Continue to fund the clinical trial to evaluate the potential clinical benefit of 

digital mammography compared to conventional mammography. $9.00 M

■ Continue to fund a randomized pilot study to assess feasibility of conducting 

a definitive clinical trial of spiral CT as a screening method for lung cancer 

detection. $2.00 M

■ Continue to fund a large definitive randomized clinical study of spiral CT as a

screening method for lung cancer detection if pilot study shows randomized 

design to be feasible. $30.00 M

■ Fund clinical studies to: compare CT colonography with endoscopic colonography 

for early detection of colon cancer and polyps; evaluate magnetic resonance spectroscopy for early detection and assessment of prostate cancer; and evaluate the

role of (F-18) – fluorodeoxyglucose PET studies for staging lung cancer. $2.25 M

5. Accelerate the development and clinical testing of image-guided interventions. 

■ Fund 6 to 10 Phased Innovation Awards for image-guided therapy research emphasizing a problem-solving, organ-specific approach and promoting interactions

between clinicians and bioengineers. $3.00 M

■ Fund 4 to 6 supplements for collaborations between the Clinical Trials Cooperative

Groups and the Diagnostic Imaging Network for testing promising, minimally invasive, image-guided interventions. $2.00 M

6. Develop innovative imaging technologies and conduct early clinical trials of 

new devices. 

■ Create a competitive program in which innovative equipment prototypes developed in industry and academia are provided to selected academic institutions for

feasibility testing, in collaboration with the developer. $5.00 M

7. Establish information archives and repositories for the research community. 

■ Establish data banks of standardized digital image data associated with known 

clinical outcomes to provide a research resource for investigators. $2.50 M

■ Fund 6 to 8 grants to develop and test image processing and analysis algorithms

using these standardized data sets. $2.00 M

Management and Support 

Total

$5.0 M

$5.0 M

$4.5 M

$3.5 M

$90.6 M
THE OPPORTUNITY

I

n the 19th century, the light microscope opened

a new frontier in the study of disease by opening

a window on the inner workings of the cell. With

this instrument, pathology – the branch of medicine

that deals with the essential nature of disease –

expanded to include the study of structural changes

in cells. For the first time, disease could be linked to

visible, recognizable changes in cells of the body. 

At the cusp of the 21st century, new molecularbased technologies are bringing us to a similar revelation. These new technologies are

enabling us to identify features of

individual cells in ways unimagined

by our 19th century predecessors. For

example, all cell types, depending on

their functions, have unique, identifiable “signatures” or special characteristics, such as which genes are

active and what proteins or other cellular products are manufactured by

the cell. Our new technologies are

enabling us to read and understand

those signatures. 

We have learned that during the

transformation of a normal cell to a

cancer cell, the signature changes,

and that change becomes a signal of the presence of

cancer. Further, we have learned that cells surrounding an incipient tumor may also undergo

changes, indicating that cancer is present. For

example, tobacco-induced molecular changes in

the mouth may predict the risk of developing lung

cancer, and cancers of the urinary tract may be signaled by cancer cells that are “shed” in the urine.

Reading the signatures of these easily accessed cells

may enable us to develop simple, non-invasive tests

to find cancers located deep within the body. 

The implications of these findings are profound.

By reading cellular signatures accurately, we may be

able to detect and diagnose cancers before they

have had a chance to invade nearby tissues. In fact,

with the tools we are developing, a single drop of

blood from a patient’s finger may be all that is needed to find a cancer, assess the threat it poses by

comparing its traits to profiles in an online library

of tumor characteristics, choose the best possible

treatment, and monitor the patient’s recovery. By

assessing the meaning of individual changes in the

cell’s signature, we may be able to determine which

cancers are most likely to progress and which are

less likely to do so. This will be especially valuable

to doctors and patients, such as those with prostate

cancer, who are sometimes confronted

with hard decisions and a desire to

avoid the consequences of unnecessary treatment. By studying the

sequence of changes a cell undergoes

as it transforms from normal to cancerous, we will gain important

insights into the etiology of the disease. By applying what we have

learned, we will be able to identify

new targets, at the molecular level, for

effective prevention and treatment. 

“Defining the Signatures of Cancer

Cells” was first identified as an area of

extraordinary scientific opportunity

in 1996, and it continues to be an area

rich in scientific promise today. For example, we

now are in a position to learn new ways to characterize tumors more efficiently – that is, to determine which genes are active and inactive, and the

levels of specific proteins that are present in a particular tumor. Such “molecular fingerprinting” will

markedly improve the specificity of cancer diagnosis by allowing us to differentiate among tumors at

the molecular level, and should enable us to devise

treatments targeted at cellular subtypes of different

cancers. See Molecular Targets Extraordinary

Opportunity, p. 80. We now can use changes in

molecular signatures to help us identify infectious

and environmental agents that may be responsible

74 The Nation’s Investment in Cancer Research

3

Extraordinary Opportunities for Investment

Defining the Signatures of Cancer Cells: 

Detection, Diagnosis, and Therapy

GOAL

Generate a catalog of 

distinguishing 

molecular signatures 

of cells to 

develop new diagnostic 

and therapeutic 

approaches and 

predict response. 
for the development or progression of a tumor. In

addition, while many of our previous efforts have

centered on identifying genes involved in cancer,

we are very interested in learning more about the

cellular functions of the proteins produced by these

genes. Finally, there has been a gap among the

identification of preclinical tumor changes, early

evaluation of new identification techniques, and

their clinical application. We now have the opportunity to synthesize these disparate findings into a

body of knowledge that will translate into real

health benefits. Our ultimate goal is to push back

the detection and diagnosis of cancer to the earliest

stages, thereby offering the potential to focus intervention efforts at preventing overt disease.

Clearly, we have made substantial progress, but

much remains to be done if we are to take full

advantage of the opportunities in this area.

Identifying the molecular signatures of cancer is

drawing a valuable, though static, picture of the

molecular composition of a cancer cell, providing an

opportunity to begin to develop targeted diagnostics

and therapeutics. However, a full understanding of

cancer initiation and progression will require a

dynamic understanding of the process. Thus it is

important to understand how alterations in a few

molecules might affect a variety of cellular functions. To achieve this understanding, we need to

assemble the molecular signatures information into

a complete picture of the living cancer cell. In addition, the diagnostic value of molecular-based methods must be confirmed and their practical benefits

established against the background of conventional

medicine. New technologies must be developed,

and new preclinical models must be created and validated to establish our findings. Finally, to validate

the predictive value of the new approaches, we must

develop the sophisticated computer systems, databases, and statistical methods needed to integrate

the complex information being generated by these

new technologies with the relevant clinical data.

PROGRESS IN PURSUIT 

OF OUR GOAL

As a normal cell is transformed into a cancer cell,

its signature changes. To identify the changes

in signatures linked to major steps of tumor development, NCI is building the complete molecular

catalog of cancer through the Cancer 

Genome Anatomy Project (CGAP – see

ncbi.nlm.nih.gov/CGAP). Over 44,000 new genes

have been discovered through CGAP’s main component, the human Tumor Gene Index (TGI). To

help us better understand the genetic and molecular foundations of cancer and other human diseases,

NIH is creating a Mammalian Gene Collection

(MGC) that will enable the scientific community

to obtain individual full-length, human or mouse

complementary DNAs (cDNAs) for more rigorous

study of individual genes, their protein products,

and the role they play in human disease. 

NCI has established a number of programs to

develop and offer novel technologies to make

molecular and analytical resources available for

researchers to identify, characterize, and validate

signatures. Through the Unconventional

Innovations Program (UIP), NCI is supporting

peer-reviewed, novel, high-impact ideas with the

potential to revolutionize cancer research and cancer care. Five UIP contracts for “Novel

Technologies for Noninvasive Detection,

Diagnosis, and Treatment of Cancer” have been

awarded. The UIP is complemented by the

Innovative Molecular Analysis Technologies

Program, which has awarded over 80 grants focused

on development and pilot applications of new technologies for the molecular analysis of cancers and

their host environment. NCI also is working with

the National Aeronautics and Space

Administration (NASA) to develop minimally

invasive molecular biosensors and is sharing ideas

on the development of other high impact technologies. The agencies host the NASA/NCI

Biotechnology Forum, a Web-based forum that

brings together NCI and NASA scientists, technologists, and engineers. Finally, to identify leads

for cancer therapy and develop novel strategies for

marker discovery, NCI is funding combinatorial

libraries of small molecules, peptides, and antibodies. The libraries can be screened to identify molecules (ligands) that bind differently to tumor and

normal cells, or bind differently between tumors of

varying malignant potential. These ligands can be

used for new approaches to cancer diagnosis and

prognosis. 

Clinical specimens are a critical resource for the

discovery and research application of molecular 

signatures to problems in cancers. NCI initiatives

Extraordinary Opportunities for Investment 75

Opportunities

Extraordinary
in this area include Cooperative Tissue Resources

for Breast and Prostate Cancers and the

Cooperative Human Tissue Network. The

Institute is increasing access through a Tissue

Expediter, who helps researchers locate the specimens and related clinical data they need. NCI also

has created the Shared Pathology Informatics

Network, a consortium of institutions connected

by a model Web-based system that both provides

direct access to data related to archived specimens

and protects patient confidentiality. Tissue

resources will likewise be key as we conduct tissue

microarray technology research and development

through the Tissue Array Research Program

(TARP – see cancer.gov/tarp), supported by NCI

and the National Human Genome Research

Institute (NHGRI). Tissue microarrays can hold

hundreds of tissue samples on a single slide, and are

an important tool in the search for and validation

of molecular signatures. TARP will produce multitumor screening tissue microarrays for the research

community starting in Fall 2000, serve as an arraying facility for groups with unique tissue materials,

and disseminate tissue microarray technology by

providing training. TARP efforts will be supported

by NCI’s Advanced Technology Center (ATC)

where scientists use new technologies to address

biological, clinical, and genetic questions pertinent

to human cancers. The ATC, which also houses

CGAP, is a premier facility for developing tools for

molecular expression profiling studies.

Using experimental animal models that parallel

the behavior of human cancer and its response to

preventive and therapeutic interventions will greatly improve our understanding of molecular changes

that contribute to cancer and could enhance our

ability to evaluate biomarkers prior to clinical application. To meet this need, NCI launched the Mouse

Models of Human Cancer Consortium, which is

developing and making available to the research

community characterized and validated mouse models. In addition, NCI’s Mouse Cancer Genome

Anatomy Project (mCGAP) is investigating

molecular determinants of cancer in the mouse for

comparative studies in human tumors. The materials developed are being made publicly available to

support efforts to identify mouse modifier genes

involved in cancer etiology.

To identify and evaluate molecular biomarkers

and technologies for earlier detection and risk

assessment of all major cancers, NCI established

the Early Detection Research Network (EDRN –

see edrn.nci.nih.gov). The EDRN is a national network of academic and industry investigators with

expertise in laboratory and clinical sciences, biostatistics, informatics, and public health. Research

When cancer is detected early, lives are saved. But

finding a tumor in its earliest stages of development, when it is composed of only a few cells, is like

searching for a needle in a haystack. Scientists funded

by NCI’s Early Detection Research Network (see above)

have developed a novel approach to early detection

based on identifying genetic mutations found in the

DNA housed in a cell’s mitochondria – the specialized

parts of a cell responsible for generating energy.

These scientists found that mitochondrial DNA mutations

observed in the body fluids of cancer patients were identical to those found in their primary tumors. They first analyzed mitochondrial DNA from bladder, head and neck, and

lung tumors and identified specific mutations linked to

these cancers. Then, searching for similar mutations, they

studied urine, saliva, and cells washed from the lungs of

20 patients with these cancers. The researchers detected

mitochondrial mutations in all of the patients with bladder and lung tumors and most of those with head and

neck tumors. In the past, researchers have found similar

SPOTLIGHT ON RESEARCH

A New Detection Tool – Looking for DNA Mutations

76 The Nation’s Investment in Cancer Research
Opportunities

Extraordinary

funded through EDRN’s 18 Biomarker

Developmental Laboratories, three Biomarker

Validation Laboratories, eight Clinical/

Epidemiology Centers, and Data Management and

Coordinating Center already is yielding results.

EDRN-funded researchers have discovered a novel

approach for detecting cancer based on mutations

in mitochondrial DNA found outside a cell’s nucleus. (See Spotlight on Research below.)

The traditional classification of human tumors is

based on tumor structure, but structure alone does

not always accurately predict a tumor’s biological

behavior, treatment response, or prognosis. NCI is

seeking a more clinically predictive and useful classification system through the Director’s Challenge:

Toward a Molecular Classification of Tumors.

Investigators funded by this initiative are creating

comprehensive molecular profiles of tumors using

DNA, RNA, or protein-based technologies. These

profiles will identify clinically important tumor subsets and will provide more informative molecular

classification schemes for human cancers. To validate new approaches to diagnosis, prognosis, and

prediction of response to therapy, NCI has

launched the Program for the Assessment of

Clinical Cancer Tests. The program is a major

expansion of NCI efforts in this area and removes

the most frequently recognized barriers to progress

in translating new discoveries into clinical practice.

It helps NCI and investigators focus on linking

technological availability with clinical need.

For more information on NCI’s Signatures 

initiatives, go to cancer.gov/initiatives.

Budget Increase Request for 2002

Defining the Signatures of Cancer Cells

1. Expand the development and availability 

of molecular and analytic resources $35.5 M

2. Establish and make tissue resources 

available to researchers. 17.5 M

3. Develop molecular signatures to study 

and validate animal models. 8.5 M

4. Develop novel technologies for early detection 

and determination of biomarkers. 14.0 M

5. Validate molecular classification schemes 

and develop new diagnostic tests. 13.3 M

6. Characterize aberrant molecular 

interactions in cancer. 18.0 M

Management and Support 4.0 M

Total $110.8 M

Extraordinary Opportunities 77

matching mutations in primary tumors and in body fluids

when looking at DNA in a cell’s nucleus. However, a cell

has just one nucleus but many mitochondria, making

mitochondrial mutations much easier to find and potentially more ideal avenues for detection. 

Researchers envision using mitochondrial DNA for cancer

screening. For example, a person at higher risk of developing lung cancer because of tobacco use might provide his

or her doctor with a sputum sample. The initial sample

would be analyzed to obtain a baseline description of the

individual’s mitochondrial DNA. In subsequent visits, new

sputum samples would be collected and compared to the

baseline for changes. If changes indicating cancer were

observed in the mitochondrial DNA, doctors would be in a

better position to intervene while the cancer is in an

early stage. The identification of mitochondrial DNA mutations in a variety of tumors represents an opportunity to

develop new, noninvasive screening approaches for cancer

based on analysis of easily collected body fluids.
78 The Nation’s Investment in Cancer Research

THE PLAN – DEFINING THE SIGNATURES OF CANCER CELLS

Goal 

Generate a complete catalog of the distinguishing molecular signatures of normal, precancerous,

and cancer cells at all stages in all tissues, and use the catalog to develop diagnostic techniques for

the earliest detection of precancerous lesions and cancers; develop signature-based therapies; and

identify subsets of patients with different prognoses to predict therapeutic response. 

Objectives and Milestones for Fiscal Year 2002

1. Expand the development and availability of molecular and analytic resources. 

■ Complete the TGI for genes expressed in cells at all stages of tumor 

development. $2.50 M

■ Continue development of the MGC for full-length human and 

mouse cDNAs. $5.00 M

■ Double the number of Phased Innovation Awards to develop technologies 

relevant to discovering and measuring molecular signatures of cancer and 

precancer. $5.00 M

■ Expand development of biosensors for detecting human cancer and cancer 

development through the UIP. $20.00 M

■ Continue development of databases and analytic tools for comprehensive 

molecular analysis. $3.00 M

2. Establish and make available to researchers tissue resources to maximize the 

practical application of molecular signatures to problems in treating cancer.

■ Establish a national tissue resource system for all major cancers, including cancers

of the lung, breast, prostate, colon, head and neck, brain, soft tissue, blood, bone,

the gynecologic and genitourinary systems, and childhood malignancies. $4.00 M

■ Develop and expand tissue repositories of precancerous lesions in all 

major cancers. $4.00 M

■ Use Phased Innovation Awards to develop tissue preservation and sample preparation methods to increase their utility and compatibility with new technologies for

cancer and precancer. $4.00 M

■ Support tissue microarray technology development and expand the TARP for 

distributing tissue microarrays. $2.00 M

■ Develop a Web site with query and search capabilities to help investigators locate

appropriate national tissue repositories. $0.30 M

■ Enhance the Web-based system to query pathology information systems including 

pathology standardization and agreement on common data elements. $2.50 M

■ Provide automatic encryption features for personal identifiers associated with 

tissue resources. $0.20 M

■ Develop public education materials about tissue donation for research. $0.50 M

3. Develop molecular signatures to study and validate animal models for human cancer.

■ Complete the mCGAP to define the molecular anatomy of mouse cancer models.

Link the mCGAP to the mouse phenotype and tumor databases to provide a 

continuum of linked descriptors of cancer. $3.00 M

$35.5 M

$17.5 M

$8.5 M
Opportunities

Extraordinary

Extraordinary Opportunities 79

■ Enhance informatics tools to link the human and mCGAP data for use in 

validating mouse models as predictors of human cancer development and 

progression. $0.50 M

■ Continue development of preclinical mouse models and fund systematic analysis

and phenotyping to validate them. Use these models to validate new molecularbased approaches for early detection, diagnosis, treatment, and prognosis of 

human cancer. $5.00 M

4. Support novel technology development for early detection and for determining 

biomarkers of precancerous lesions and cancer.

■ Expand discovery, development, and validation of new early detection tests for all

major human cancers through the EDRN. $8.00 M

■ Develop high throughput technologies for isolating and enriching cells shed in 

body fluids. $2.00 M

■ Provide supplemental funding for biomarker evaluation to groups conducting 

prevention and therapy studies. $4.00 M

5. Validate molecular classification schemes of cancer and develop new diagnostic tests.

■ Fund expanded validation programs for each major cancer site as results emerge

from the Director’s Challenge and other programs. $10.00 M

■ Validate new diagnostic approaches through a Program for Assessment of Clinical

Cancer Tests to provide the research community with a means to evaluate and 

validate signatures with possible diagnostic value. $3.25 M

6. Support basic research aimed at characterizing aberrant molecular interactions 

in cancer. 

■ Generate a comprehensive map of all cellular signal transduction pathways 

and their links to one another through a Signal Transduction Annotation

Consortium. $3.00 M

■ Support basic research efforts for analysis of: higher order cellular architecture that

may be perturbed in cancer; organization of chromosomes into chromatin and their

localization within the nucleus; the structure and function of molecular machines;

and structure and function of membranes. $5.00 M

■ Fund 10 Phased Innovation Awards to develop technologies for analyzing cell-cell

interactions and communication that might be perturbed in cancer. $5.00 M

■ Develop an informatics system that will enable the modeling of dynamic and integrated cellular functions by establishing a “Virtual Cell” Consortium. $5.00 M

Management and Support 

Total

$14.0 M

$13.3 M

$18.0 M

$4.0 M

$110.8 M
80 The Nation’s Investment in Cancer Research

4

Extraordinary Opportunities for Investment

THE OPPORTUNITY

Our systematic search for drugs to combat cancer

began about 60 years ago. Throughout most of

this search, our understanding of cancer has been

limited by available technologies. The microscope,

for example, enabled us to see the structure of the

cancer cell, but revealed nothing about what actually makes a cell cancerous or about how the inner

workings of the cancer cell differ from a normal

cell. As a result, techniques for identifying drugs to

prevent or treat cancer were limited to tests that

measured inhibition of cancer’s development or its

growth in animals or test tubes.

Despite these limitations, scientists

have identified several synthetic and

natural substances that appear to

thwart cancer development, and they

have developed drugs that, alone or

with surgery or radiation, can cure

some cancers in people and ease

symptoms in others. 

Yet, most of the common tumors of

adults do not respond well to the

treatments available today. And even

when treatments successfully shrink

tumors or eliminate them from the

body, they can cause a variety of

short- or long-term side effects that

can have a devastating impact on a patient’s quality

of life. Many of chemotherapy’s most severe toxic

effects stem directly from their non-selective nature;

most available compounds that inhibit tumor cell

growth also inhibit the growth of healthy cells.

However, drugs that target the molecular differences

between tumor and normal cells – the altered genes

or proteins or corrupted pathways – promise to be

less toxic and more effective than our current drugs. 

The situation for prevention is similar.

Knowledge of the precise molecular steps that characterize premalignant change will provide the foundation for our search to find agents that reverse

these changes or block the steps critical to the full

development of cancer. 

Scientists working to discover effective prevention and treatment agents have always faced a formidable barrier: not knowing precisely what cancer is.

Now, with the evolution of molecular biology and

the emergence of new technologies, we are gathering

remarkable knowledge about the nature of a cancer

cell and the molecular changes that occur before and

during a tumor’s development. The extraordinary

opportunity before us to discover and exploit molecular targets for cancer prevention and treatment arises from the convergence of scientific advances in the

areas of cancer biology, synthetic and biosynthetic

chemistry, high throughput screening,

and medical imaging.

Cancer Biology

Evolving knowledge of how molecules

and pathways in premalignant or fully

malignant cells differ from their normal counterparts is enabling us to

refine human tumor classification in

terms of molecular changes and also is

giving us a new strategy for cancer

drug discovery. Every point of difference between premalignant or malignant cells and their normal counterparts is a potential target of opportunity for drug discovery – a molecular target of prevention or treatment. Targets also may be

revealed by understanding the consequences of fundamental molecular changes in cancer, such as those

that spur blood vessel growth to nourish tumors.

Still others are normal molecular machines that

take on particular significance in the context of cancer, such as hormone or growth factor systems.

Synthetic Chemistry

Traditionally, the chemicals used in anti-cancer

drugs have come from nature – from tropical rain

forests or organisms in the sea or the soil. Using

recently developed techniques, chemists now are

able to create enormously diverse collections of

Molecular Targets of Prevention and Treatment

GOAL

Accelerate discovery, 

development, and 

testing of prevention 

and treatment 

agents that 

target the molecular 

changes of cancer.
compounds, and we can screen both natural and

synthetically derived chemicals for possible anticancer effects. 

Biosynthetic Chemistry

Synthetic chemists have long been able to manipulate small molecules to produce useful medicines.

Now, the biotechnology revolution has cleared the

way for biochemists to mix and match genes to

design synthetic proteins. Changing proteins in

cells is a crucial step, since proteins form the “messages” that make up communication pathways that

determine a cell’s healthy or aberrant behavior.

Now scientists can change the messages sent by

protein molecules, creating a new class of drugs to

be tested for anti-cancer activity.

High Throughput Screening

Biotechnology advances also have made it possible

to devise highly sensitive, efficient tests for virtually any biological process. These tests, or “smart”

assays, can be used many different ways. For example, they can be used to screen cell lines and tissues

for the presence of particular genes, proteins, or

entire pathways, an essential step in identifying the

chain of events involved in every stage of cancer

development. They also can be used to screen

potential drugs for anti-cancer effects. Smart assays,

which can screen thousands of compounds each

week, can be performed on a micro scale with tiny

quantities of material, using computer-driven

robots to maximize efficiency.

Medical Imaging

Until now, imaging has been used in cancer research

and care to gain information about the occurrence,

size, and location of tumors. Refinements in imaging

technology are now allowing us to watch molecular

processes within the cell unfold, with unprecedented vividness and accuracy. Imaging techniques are

being developed to tell us not only the location of

a tumor but the kind of molecules it contains and

how its biochemical pathways work. (See p. 69 for

the Imaging Extraordinary Opportunity.)

The convergence of advances in these fields

presents us with the opportunity to revolutionize

cancer drug discovery and develop a whole new

generation of cancer preventives and treatments –

drugs that target the molecular features of cancer.

To ensure our success, however, we need to create

unprecedented conceptual and functional links

among drug discovery, development, and clinical

testing. To understand why, one must consider the

main questions that researchers need to pursue

about a new drug’s effect on malignant or precancerous cells: Does the drug kill the cancer or effectively block its growth and spread? What part of the

cell’s complex machinery does it disrupt, and how is

this disruption related to its anti-cancer effect?

Tools to address this second question have been

crude and inadequate, so our clinical testing has

focused only on the first.

We must gather the knowledge and develop the

tools to answer both of these questions. When we

can, we will be able to better address some of the

most important questions in cancer therapeutics: If

a drug is working well, why is it working, and if not,

why not? Are we giving a person the right amount,

or too much, or too little? Do we have to give people the maximum amount of a drug that they can

tolerate, or can we judge the right amount by

whether the drug is getting to the tumor and affecting its target? Will the drug harm the patient now or

in the future? Only when we can answer these questions will we be able to predict who is likely to

respond to a particular treatment and who will not.

Moreover, information from the clinic and from the

laboratory will reinforce each other, providing the

basis for designing even better drugs in the future. 

Extraordinary Opportunities 81

Opportunities

Extraordinary

Budget Increase Request for 2002

Molecular Targets of Prevention and

Treatment

1. Identify, characterize, validate, and produce 

targets for drug discovery. $16.5 M

2. Synthesize or acquire molecules for use in 

drug screening efforts. 30.5 M

3. Facilitate the steps to turn a promising 

compound into a drug. 28.0 M

4. Facilitate partnering. 41.5 M

5. Support special interdisciplinary initiatives. 25.0 M

Management and Support 5.0 M

Total $146.5 M
PROGRESS IN PURSUIT 

OF OUR GOAL 

I

dentifying molecular targets research as an

extraordinary investment opportunity reflects

NCI’s commitment to exploit the molecular underpinnings of cancer to achieve more effective prevention and treatment interventions. We have

launched a number of new initiatives and continue

to support ongoing programs to stimulate research

in this critical area. 

To encourage creative investigations for identifying, characterizing, and validating promising new

molecular targets, NCI has established Molecular

Target Drug Discovery (MTDD) grants in various

scientific disciplines to identify novel molecular

targets for prevention and treatment, validate the

targets as a basis for cancer drug discovery, and

develop tests to detect the effects of these agents on

their targets. 

Newly developed chemical and biological combinatorial techniques are enabling scientists to create, over the course of weeks or months rather than

years, millions of chemically diverse structures with

potential anti-cancer effects. And biotechnology

advances are enabling researchers to mix and

match genes to design synthetic proteins, creating a

new class of potential anti-cancer agents. To fully

exploit the new opportunity to synthesize or acquire

large numbers of possible drug molecules, NCI is continuing its support of six Biology-Chemistry

Centers, two of which were funded in Fiscal Year

2000. This novel interdisciplinary program facilitates collaborations among top researchers in

chemistry, biology, genetics, and computer science

for developing and refining robotic drug production

and screening technology. The Biology-Chemistry

Centers already have screened hundreds of thousands of compounds.

Another ambitious effort to develop new anti-cancer agents is the Rapid Access to Intervention

Development (RAID) program, designed to efficiently move novel treatment interventions developed in academic settings into the clinic. Because

academic institutions commonly lack the capacity

to develop drugs, promising ideas and candidate

molecules cannot always move forward in the drug

discovery process. The RAID program makes NCI’s

drug development resources available to investigators with molecules that hold promise for cancer

treatment. By providing the resources needed for

preclinical development of drugs and biological

agents, this program removes the most common

barriers between laboratory discovery and clinical

testing. Products developed through RAID are

returned directly to the originating laboratory for

clinical trial testing. Fourteen projects were funded

in Fiscal Years 1999 and 2000, and funding for additional projects is planned for Fiscal Year 2001.

82 The Nation’s Investment in Cancer Research

Each year, 25,000 people in this country are diagnosed with diffuse large B-cell lymphoma (DLBCL),

the most common form of non-Hodgkin’s lymphoma

(NHL). DLBCL is a form of cancer in which B cells in the

immune system proliferate uncontrollably. Physicians

have long questioned why standard chemotherapy cures

only 40 percent of their DLBCL patients while the majority relapse and die. A team of scientists recently used

the powerful new DNA microarray technology to make an

intriguing discovery that is a critical key for solving

this puzzle. 

The scientists created a novel microarray tool, which they

called the “lymphochip,” by mining the Cancer Genome

Anatomy Project (CGAP) database for information on over

18,000 genes important to both lymphoid malignancies

and the immune system and placing them on a device similar to a computer chip. The lymphochip enabled them to

compare gene activity of normal and cancerous B cells

and generate gene expression profiles, or signatures, of

the different cell types. After examining several different

forms of non-Hodgkin’s lymphoma, the scientists discovered that DLBCL cells showed two distinct patterns of

gene expression, suggesting that this diagnosis actually

SPOTLIGHT ON RESEARCH

Diffuse Large B-Cell Lymphoma: A Disease 

Within A Disease 
Opportunities

Extraordinary

Extraordinary Opportunities 83

While RAID focuses on developing promising

candidate molecules to treat cancer, the Rapid

Access to Preventive Intervention Development

(RAPID) program is providing funding and

resources to scientists working to develop agents to

prevent, reverse, or delay cancer development. Like

RAID, RAPID is designed to quickly move novel

preventive molecules and concepts from the laboratory to the clinic for efficacy testing in clinical trials. RAPID will accelerate the development process

for preventive agents by providing academic investigators with the contract resources needed for preclinical and early clinical drug development, ensuring the efficient translation of promising discoveries

even when investigators and their institutions lack

the requisite development capacity or clinical

expertise. Seven projects were funded through this

program in Fiscal Year 2000, and we expect to fund

additional projects in Fiscal Year 2001.

The many steps involved in turning a potential

anti-cancer agent into a drug appropriate for human use

can take years and an investment of several million

dollars. The process includes discovery, efficacy

testing, development of lead agents, pharmacology

and toxicology studies of the potential drug,

Investigational New Drug (IND) application filing

with the Food and Drug Administration, and clinical evaluation. To support small business participation in this process, NCI established the Flexible

System to Advance Innovative Research

(FLAIR) to provide small businesses the budgets

and time required to identify promising agents and

develop them into drugs that can be evaluated 

clinically. 

To discover, develop, and validate innovative tools

to help scientists determine whether a potential

agent actually affects an intended molecular target,

NCI has set aside funding for Interdisciplinary

Research Teams for Molecular Target Assessment

(IRTMTA). Each multidisciplinary team will focus

on a critical biological process thought to contain

high priority targets for cancer prevention or treatment drug discovery. They will work to develop

tools, probes, assays, and imaging approaches to

assess the effects of drugs on their targets.

NCI recently announced the Molecular Targets

Laboratory (MTL) initiative that will focus intensively on developing a resource of biological assays

and chemical probes for biological studies of cancer.

The MTLs will emphasize collaboration between

chemists and biologists to produce libraries of

potential anti-cancer compounds for public distribution, develop screening assays suitable for high

throughput screening of chemical libraries of

potential agents, and confirm a drug’s ability to

alter the drug target in cancer cells. 

For more information on NCI’s Molecular

Targets initiatives, go to cancer.gov/initiatives.

has lumped together two subtypes of NHL. While unable

to distinguish one from the other under a microscope, the

tool conventionally used for diagnosis and cell typing,

scientists now are able to use advanced technology to

sort out these two biologically distinct subtypes of lymphoma and to subsequently identify two distinct clinical

courses that DLBCL can take. Although more research is

needed, scientists are optimistic that expression signatures will lead them to more precise diagnoses and ultimately to more effective treatments for specific subtypes

of this disease. 

Over the coming years, we expect to be able to use gene

expression signatures like these to vastly improve our

ability to diagnose, classify, and treat not only lymphoma

but all types of cancer. By analyzing differences at the

molecular level, we can identify the genes and cell pathways that are important to cancer development and progression. These molecular differences will provide the

clues to early detection and diagnosis and serve as the

targets of present and future cancer drugs.
84 The Nation’s Investment in Cancer Research

THE PLAN – MOLECULAR TARGETS OF PREVENTION AND TREATMENT

Goal 

Accelerate the discovery, development, and testing of prevention and treatment agents that target

the molecular changes underlying the various stages of cancer initiation and progression.

Objectives and Milestones for Fiscal Year 2002

1. Identify, characterize, validate, and produce targets in precancerous and cancerous

cells for drug discovery. We will identify novel targets that represent critically 

vulnerable sites – the true Achilles’ heels – within cells, determine the role they play,

validate their biological importance in cancer growth, and mass produce promising

targets for use in testing the potential effects of various agents. 

■ Expand the MTDD program to include 10 grants in each of 3 focus areas involved

in cell signaling events: cell cycle regulation, cell death and immortality, and cell

invasion and metastasis. $9.00 M

■ In conjunction with the Mouse Models of Human Cancer Consortium, develop 

transgenic animal models (mice genetically altered to express human genes relevant to cancer) to verify the potential drug responsive characteristics of selected

targets and define their associated pathways. $3.00 M

■ Co-fund with the National Institute for General Medical Sciences the expansion

of technology in the National Beam Laboratories to enable researchers to rapidly

determine the structure of important molecular targets, thereby permitting 

computer modeling of potential agents. $4.50 M

2. Synthesize or acquire large numbers of diverse candidate molecules for use in drug

screening efforts. We will encourage chemists and biologists to work collaboratively

to synthesize novel structures aimed at defined molecular targets, support expanded

efforts to collect candidate agents from nature, and promote research efforts to 

synthesize biological agents (e.g., vaccines, recombinant proteins) directed at target

molecules.

■ Procure and distribute libraries of chemically diverse compounds to cancer 

drug discovery researchers, including MTDD grantees, to rapidly determine the 

effectiveness of these compounds against novel targets in drug screens guided by 

target structure. $5.00 M

■ Expand the collection of natural product extracts from threatened ecologic niches

and previously unexplored biorealms. Increase operations to augment the library of

available natural product extracts to assure a constantly renewable supply of this

valuable resource. Create a centralized distribution center for formatted natural

product extracts. $8.00 M

■ Expand support for RAID and RAPID to assist investigators in producing sufficient 

quantities of potential biological agents for testing. Partner investigators with

appropriate commercial, government, or academic production facilities. $6.00 M

■ Create a repository of public domain libraries of biological agents (e.g., peptides,

carbohydrates) relevant to target cells or organs. $4.00 M

■ Create 2 or 3 Centers of Excellence in Biologics to support approaches for 

designing, producing, validating, and clinically testing vaccines, viral vector constructs for agent delivery, and/or recombinant proteins directed at cancer-related 

target molecules. $7.50 M

$16.50 M

$30.50 M
Opportunities

Extraordinary

Extraordinary Opportunities 85

3. Facilitate the steps necessary to turn a promising target-directed compound into a

drug appropriate for human use.

■ Fund studies of promising target-directed compounds to determine toxicity and

modify them as needed to be more effective and well tolerated by patients.

Information gleaned from target structure and animal model studies will assist in

this process. $8.00 M

■ Assist small businesses in cancer drug discovery by expanding the FLAIR program

to develop novel approaches to molecular toxicology, formulation advances, and

clinical evaluation and by using a RAID-like process to provide contract research

resources to advance concepts. $6.00 M

■ Expand the IRTMTA from 2 or 3 to at least 10 centers to develop a “toolbox” of

valid assays for assessing a drug’s affect on its intended target, thereby speeding

movement of candidate drug molecules to the clinic. Develop a formal process for

clinical investigators to access expertise for developing assays in support of early

clinical trials focused on assessing whether drugs affect their molecular target of 

reference. $14.00 M

4. Facilitate partnering of academic, commercial, and government resources to 

promote cancer drug discovery and development.

■ Expand from 12 to 16 the number of National Cooperative Drug Discovery Groups

to facilitate partnerships with industry and academia to foster multidisciplinary

approaches to the discovery of novel prevention or treatment strategies. $16.00 M

■ Support the creation of publicly available databases and data mining tools for compound and target information. These databases and tools will provide an informatics infrastructure for MTDD and MTLs and serve as an archive for MTDD and

MTL information. Sponsor yearly interdisciplinary chemistry/biology/genomics/

proteomics workshops for academic and small business researchers to enhance

interactions. Design and build a Web-based workshop to continue such interactions. $3.50 M

■ Double the number of academic Phase I trials supported by NCI and promote partnering between NCI-funded centers and commercial firms with suitable test agents.

Provide infrastructure to centers doing Phase I studies to allow verification of the

molecularly targeted nature of drug action. $7.00 M

■ Establish 5 new high priority Phase II trials through Specialized Programs of

Research Excellence (SPOREs) and cancer centers focusing on breast, prostate,

colorectal, other gastrointestinal, and gynecologic cancers, and leukemia/

lymphoma. $15.00 M

5. Support for special interdisciplinary initiatives

■ Increase resources allocated to the MTLs to develop a resource of biological assays

and chemical probes for biological studies of cancer. 

Management and Support

Total

$28.0 M

$41.5 M

$25.0 M

$5.0 M

$146.5 M
86 The Nation’s Investment in Cancer Research

5

Extraordinary Opportunities for Investment

THE OPPORTUNITY

T

he numbers are alarming. An estimated 450,000

people in the U.S. will die this year alone from

tobacco-related diseases – the most preventable and

costly cause of death in our Nation. Every day more

than 3,000 youths start smoking, placing themselves at increased risk for a host of cancers, particularly lung cancer, which causes approximately

160,000 deaths a year. Mouth, pharynx, larynx,

esophagus, pancreas, cervix, kidney, and bladder

cancer, as well as heart disease and many other

conditions are all associated with

tobacco use. Of those who continue

to smoke, approximately half will die

prematurely, losing an average of 20

to 25 years of their life expectancy.

The global picture is even more

sobering: More than one billion people smoke worldwide and an estimated three million die annually from

tobacco-related illness. By 2025, the

number of deaths is expected to reach

ten million per year.

These statistics illustrate dramatically that the use of cigarettes, cigars,

pipes, and smokeless tobacco products

is a major worldwide threat to health that must be

addressed. Although their risk for lung cancer

remains higher than if they never had smoked,

strong evidence shows that people who stop smoking – regardless of age – live longer than those who

continue to smoke. 

Developing Optimal Prevention and

Cessation Strategies 

Studies indicate that while the majority of smokers

want to stop smoking completely, they struggle to

quit. Most adults who smoke regret ever starting. It

is critical that strategies be developed to prevent

people from ever starting to smoke and help those

who currently smoke to stop. 

The past three decades have brought many

achievements in this area – we have developed and

implemented physician training and office protocols

for smoking cessation, confirmed the effectiveness of

primary care medical and pharmacologic interventions, and developed both effective self-help interventions and tailored interventions designed to meet

the needs of individual smokers. Mass media interventions to reach large numbers of people with prevention and cessation messages have been developed, and strategies to reach minority, ethnic, and

high-risk populations have been tested. Recent

large-scale programs, like the Community

Intervention Trial for Smoking

Cessation (COMMIT) and the

American Stop Smoking Intervention

Study (ASSIST) have shown both the

potential and the limitations of community and state tobacco control

interventions for changing attitudes

about tobacco use, changing tobacco

use behaviors, and reducing the tobacco-related cancer burden. Still, we do

not fully understand the most critical

elements of tobacco prevention and

treatment strategies, their timing, how

best to target high-risk subgroups and

settings, and how to tailor messages

and materials appropriately for different populations. 

Identifying and Targeting Populations at

High Risk for Tobacco-Related Cancers

and Nicotine Addiction

We have made enormous progress in understanding

at the molecular level the transformation of a normal cell to a cancer cell following exposure to

tobacco carcinogens. Scientists have identified

many cancer-causing agents contained in tobacco

smoke and shown that different tobacco products

and methods of nicotine delivery influence the type

and quantity of exposure to these agents. 

Researchers also have determined that these

multiple agents seem to induce similar changes,

Research on Tobacco and Tobacco-Related Cancers 

GOAL

Understand the causes 

of tobacco use, 

addiction, and 

related cancers and 

apply this knowledge 

to their prevention 

and treatment. 
Opportunities

Extraordinary

regardless of the cell’s location in the body. The

challenge now is to learn more about how and why

elements in tobacco smoke target particular genes

and how tobacco-induced cellular damage initiates

and promotes cancer development. Such knowledge will help us identify precancerous lesions and

markers that may predict tobacco-induced cancer.

Identifying markers that detect DNA damage and

other antecedents of cancer will enable us to test

different prevention and treatment strategies and

develop new early detection methods. 

This research also could provide important

insights into why some people may be particularly

vulnerable to harm from tobacco. For example,

women develop more lung cancers than men per

cigarette smoked, and certain ethnic groups also

appear to be at increased risk for lung cancer. While

the reasons for these differences are not clear, smokers with certain gene variants are more likely to

develop lung cancer. New preclinical models are

needed to enable us to identify these harmful variants that lead to increased susceptibility to tobaccorelated cancers. Using this information and knowledge of how inherited susceptibilities and tobacco

exposure in combination contribute to cancer, we

will be able to develop specific prevention and

detection strategies for vulnerable individuals. 

Growing evidence indicates that genes interact

with environmental factors to influence whether an

individual will start smoking, how early he or she

will start, and how difficult it will be to quit. Just as

we now know more about the biological basis of

tobacco-related cancers, we also have learned a

great deal about the psychosocial, biobehavioral,

and biological determinants of tobacco use and

addiction. We know, for example, that adolescent

tobacco use is tied to peer and family influences and

low self-esteem. We know that continued smoking

by adults is associated with nicotine addiction,

depression, and stress. Recent research breakthroughs have provided important insights into the

biological basis of tobacco use and nicotine addiction, including the role of genetic factors in nicotine metabolism. For example, certain genes modify nicotine metabolism and regulate brain chemicals that affect the pleasurable feelings triggered by

nicotine. By determining how these preexisting vulnerabilities interact with sociocultural and psychological influences on tobacco use, and by improving

our ability to assess risks quantitatively, we will be

able to develop more effective prevention and cessation interventions and tailor these interventions

to the people most likely to benefit from them. We

also will be able to identify effective new drugs and

combinations of proven drugs for treating nicotine

addiction. Combining pharmacologic and behavioral tailoring may prove particularly effective in

accelerating smoking cessation rates. 

Capitalizing on Social, Legal, and Public

Policy Developments 

Important social, legal, and public policy developments are converging with scientific advances to

create a unique opportunity to tackle tobacco use.

Public attitudes reflect decreasing acceptance of

tobacco use as a social norm. Government agencies,

academic institutions, and professional and voluntary organizations are making major commitments

to reduce tobacco use and exposure to tobacco carcinogens. Through lawsuits, states are recovering

billions of dollars lost to the treatment of diseases

caused by smoking. However, while state political

leaders have been urged to use these funds to

expand tobacco control programs, they almost certainly will use most of the funds to address other

priorities. This dilemma underscores the need for

NCI to expand its investment in tobacco control

research to ensure that the best scientific evidence

informs state and community programs. 

In summary, we have an unprecedented opportunity to reduce the enormous burden of tobacco use

on our Nation’s public health. The investment proposed here will enable us to gather knowledge that

Extraordinary Opportunities 87

Budget Increase Request for 2002

Research on Tobacco and 

Tobacco-Related Cancers

1. Define the biological, behavioral, and social 

bases of tobacco use and addiction. $12.0 M

2. Understand the interplay among tobacco, 

other exposures, and host susceptibility 

on cancer risk. 23.0 M

3. Develop, test, and disseminate more effective 

interventions to prevent and treat tobacco 

use and tobacco-related cancers. 29.0 M

Management and Support 3.0 M

Total $67.0 M
88 The Nation’s Investment in Cancer Research

THE PLAN – RESEARCH ON TOBACCO AND TOBACCO-RELATED CANCERS 

Goal 

Understand the causes of tobacco use, addiction, and tobacco-related cancers and apply this 

knowledge to their prevention and treatment. 

Objectives and Milestones for Fiscal Year 2002

1. Expand efforts to define the biological, behavioral, and social bases of tobacco use

and addiction.

■ Develop and implement specialized surveys and incorporate biospecimen collection

in cohort studies to better understand youth tobacco use and addiction. $4.00 M

■ Establish regional repository resources to house large numbers of biospecimens and

encourage collaborative studies to identify genetic predisposition for tobacco use 

and addiction. $4.00 M

■ Conduct national tobacco control surveys on activities that impact tobacco control

through supplements to the Current Population Survey. The data, covering diverse

populations, are used increasingly by Federal and non-Federal researchers to assess

progress in cancer control. $2.00 M

■ Create a public use research resource of regional, state, and local tobacco-related

policy and legislation to expand capacity to assess the effect of these factors on

regional and local tobacco control progress. $2.00 M

2. Accelerate progress in understanding the interplay among tobacco, other exposures

such as alcohol and asbestos, and host susceptibility on cancer risk. 

■ Expand existing prospective studies by incorporating biospecimens, developing

standard measures and shared data instruments, and supporting cooperative efforts

in order to identify and characterize genetic and biological factors affecting vulnerability to tobacco-related cancers. $10.00 M

■ Fund innovative studies to determine how tobacco use contributes to cancers 

other than lung and oral cancers, such as pancreatic, cervical, kidney, and bladder

cancers. $5.00 M

$12.0 M

$23.0 M

will inform policy makers and public health practitioners about the best strategies for preventing and

treating tobacco use and tobacco-related cancers. 

PROGRESS IN PURSUIT 

OF OUR GOAL

NCI has a number of projects underway in its

intramural cancer epidemiology and genetics

program to better understand the causes and mechanisms of tobacco-related cancer. NCI is conducting large case-control studies of lung, bladder, renal,

non-Hodgkin’s lymphoma, biliary tract, and gastric

cancer. These studies include genetic and biomarker components to complement traditional epidemiological approaches for assessing risk. Populationbased cohort studies on lymphomas, leukemias and

cervical cancer are assessing the role of tobacco use.

The Prostate, Lung, Colorectal, and Ovarian

(PLCO) cancer study is evaluating screening strategies for multiple smoking-related cancers and for

colonic polyps, a pre-cancerous condition related to

tobacco. A study of family members with Von

Hippel-Lindau syndrome, an inherited cancer syndrome, is establishing whether tobacco influences
Opportunities

Extraordinary

Extraordinary Opportunities 89

■ Support the development of animal models for tobacco-related 

carcinogenesis. $1.00 M

■ Support research that combines population and family approaches to better understand the interplay of genes and environment in tobacco-related cancers. $5.00 M

■ Collaborate with other NIH Institutes to support studies to further define the

adverse health effects of environmental tobacco smoke exposure. $2.00 M

3. Develop, test, and disseminate more effective interventions to prevent and treat

tobacco use and tobacco-related cancers, especially in high-risk individuals and groups.

■ Support pilot projects that incorporate biomarkers in developing and evaluating

chemopreventive agents in populations of former smokers at high risk for tobaccorelated cancers. $3.00 M

■ Support the development of biomarkers of tobacco exposure and risk. Such correlative laboratory projects will complement activities ongoing in the Early Detection 

Research Network. $3.00 M

■ Fund studies to develop and test new behavioral, pharmacological, and combination therapies to treat nicotine dependence, with special emphasis on populations 

at high risk. $6.00 M

■ Support rapid, standardized evaluations of current tobacco cessation programs for

adolescents and young adults. $2.00 M

■ Support studies in collaboration with public and private tobacco research funding

organizations to identify successful tobacco use prevention interventions. $5.00 M

■ Expand support for research to evaluate the impact of new state and community 

tobacco control programs. $10.00 M

Management and Support

Total

$29.0 M

$3.0 M

$67.0 M

cancer in cancer-prone families. To advance our

understanding of genetic and environmental interactions that influence smoking behavior and lung

cancer in particular, NCI will support a genetic epidemiology of lung cancer and smoking study. This

interdisciplinary case-control study will explore how

tobacco and genes influence both lung cancer and

smoking by incorporating the study of siblings and

an extensive biospecimen collection. 

Seven Transdisciplinary Tobacco Use Research

Centers (TTURCs) – launched in 1999 under the

joint sponsorship of NCI, the National Institute on

Drug Abuse, and the Robert Wood Johnson

Foundation – are helping to provide the needed

infrastructure for tobacco research across many disciplines. TTURC researchers are tackling a wide

range of topics, including genetic susceptibility, animal models of behavior, sociocultural factors, innovative treatments, and research on health care policy and bioethical implications of tobacco control.

The Centers will accelerate tobacco control intervention development, speed the transfer of these

approaches to communities nationwide, and train a

new generation of tobacco control researchers.

To further understand youth tobacco use and

addiction, NCI, in collaboration with other NIH

Institutes, is supporting youth prevention and cessation research. This research includes projects on
adolescent tobacco use prevention, experimentation related to the onset of regular tobacco use,

dependence and withdrawal, and cessation and

treatment of tobacco use. 

Tobacco-related deaths among U.S. women continue to rise. NCI is supporting several studies on

tobacco use and addiction in women. Studies on

reducing tobacco use by pregnant women are focused

on helping low-income women quit, testing the ability of women’s partners to assist them in quitting,

and preventing relapse after delivery. Another study

examines the relationship between smoking and

major depressive disorder, a problem that disproportionately affects women. NCI also is funding a major

study of African American women’s health that

includes an examination of smoking behavior. 

We still need to know how best to design and

conduct state and community tobacco control programs. NCI is supporting 12 new research projects

on innovative tobacco prevention and control

interventions at the community, state, or multistate level. Questions being addressed concern the

impact of media campaigns on tobacco use behaviors, readiness to quit, and attitudes and how to tailor media and policy interventions to influence

high-risk groups.

NCI is working with public and private partners

to build a comprehensive and integrated surveillance system to monitor tobacco control progress at

the local, regional, and national levels. Through

the Tobacco Use Supplement to the Current

Population Surveys (CPS), final data from the

1990s will be made available by the Census Bureau

in Fall 2000 as a public use data resource (wwwdccps.ims.nci.nih.gov/ARP/RiskFactor/

tobacco.html). NCI will provide supplements to

existing tobacco control grants to encourage

researchers to analyze these data. NCI also is beginning pilot work to determine the best approach to

creating a public use research resource of regional,

state, and local tobacco-related policy and legislation

to expand capacity to assess the effect of various factors on regional and local tobacco control progress. 

Because effective communication and information dissemination about emerging public health

issues in smoking and tobacco control are critical to

reducing adult and youth tobacco use, NCI established the Smoking and Tobacco Control

Monograph series in 1991. Recent data from the

Tobacco Use Supplement to the CPS have been

used extensively in this series, for example, in

Changing Adolescent Smoking Behavior and Cigars:

Health Effects and Trends, State and Local Legislative

Action to Reduce Tobacco Use. Data from the cigar

monograph were used to support the June 2000

Federal Trade Commission’s requirement that the

seven U.S. cigar companies include warnings about

significant adverse health risks of cigar use in their

advertising and packaging. These monographs can

be viewed on NCI’s Web site at rex.nci.

nih.gov/NCI_MONOGRAPHS/LIST.HTM.

In August 2000, NCI cosponsored and participated in the 11th World Conference on Tobacco

OR Health, a meeting that brought together

researchers, clinicians, public policy officials, tobacco control advocates, public health workers, and

educators from around the world. The international conference showcased the state of the art in

tobacco control and provided a forum for sharing

ideas and developing consensus on global

approaches. 

90 The Nation’s Investment in Cancer Research

About Tobacco 

■ Tobacco is responsible for more than 30 percent

of all cancers and nearly one in five deaths in

the U.S. every year.

■ In the last decade, more than four million 

people in this country lost their lives to 

tobacco-related diseases, including, according 

to Environmental Protection Agency estimates,

more than 30,000 nonsmokers who died of 

lung cancer caused by breathing smoke from

others people’s cigarettes.

■ Lung cancer deaths are still increasing among

women.

■ Over the course of their lives, current and former

smokers generate an estimated $501 billion in

excess health care costs. Tobacco costs Medicare

a staggering $10 billion each year and Medicaid

more than $12.9 billion annually.

■ The latest figures confirm a disturbing increase

in youth smoking over the past decade. More

than one-third (34.8%) of high school students

reported recently using some form of tobacco.

■ Adult smoking rates did not decrease from 1995-

1997, but smoking increased among those 18 to

24 years of age.
Opportunities

Extraordinary

Extraordinary Opportunities 91

6

Extraordinary Opportunities for Investment

Cancer Communications

THE OPPORTUNITY

We are in the midst of a communications revolution unparalleled since Gutenberg introduced movable type to the western world in the

15th century. At no other time in history has it

been so easy for so many people to access such a

vast wealth of information. Radio and television

continue to be powerful communication tools, and

the Internet has multiplied exponentially our ability to make large amounts of information available

to a wide audience quickly and easily.

From primary prevention to survivorship and

end of life issues, communication

empowers people to make informed

cancer-related decisions and to

engage in behaviors that will

improve their health. The Science

Panel on Interactive Communication and Health convened by the

Department of Health and Human

Services concluded that few other

health-related interventions have

the potential of interactive health

communications to simultaneously

improve health outcomes, decrease

health care costs, and enhance consumer satisfaction. Scientists and

communications experts studying the

process of effective communication and its impact

on health for more than 25 years have produced

increasingly refined theories of health communications, including those that focus on how people

process health information and how they respond

to cancer-related risks. These theories have been

applied to interventions that have contributed to

declining smoking rates among many groups in the

United States, to the increasing proportions of

Americans who are eating more fruits and vegetables each day, and to the larger numbers of people

who are getting screened for breast, cervical, and

colorectal cancers. 

Despite our progress in refining health communications theories, however, major gaps remain in

our understanding of how consumers use health

information. We must learn how to help people distinguish important from insignificant health risks

and deal with contradictory or inaccurate health

messages so they can make informed choices. We

must provide accurate and balanced information

about all areas of cancer treatment and care, including complementary and alternative therapies. 

In addition, we need to narrow the enormous gap

between what is known about cancer communications and what is practiced. We must find the best

ways to inform physicians, nurses, and other health

care providers of emerging best practices in patient care. We must help

them to become more effective communicators and to integrate cancer

communications into all aspects of

cancer care. The power of health communications must be harnessed to

reduce cancer-related health disparities. To achieve these aims, more

research is needed, and the cadre of

health communications scientists and

practitioners who can conduct communications research and apply the

results must be expanded.

NCI, its grantees, and contractors

have long been leaders in health communications. Now it is time to seize the opportunity presented by new knowledge of health behavior,

new technologies, and our arsenal of proven strategies to further empower people to increase their

knowledge, use the health care system more effectively, and understand and modify their health risk

behaviors. By increasing patient access to and participation in clinical trials, we also can speed the

pace of discovery. Through these efforts, we will

have a far richer understanding of how people use

communications technologies of all kinds, and we

will use that understanding to improve outcomes in

cancer prevention, early detection, treatment, and

survivorship.

GOAL

Understand and apply 

the most effective 

communications 

approaches to 

maximize access to 

and use of cancer 

information by all 

who need it.
92 The Nation’s Investment in Cancer Research

THE PLAN – CANCER COMMUNICATIONS

Goal

Increase knowledge about, tools for, access to, and use of cancer communications by the public, 

consumers, patients, survivors, and health professionals – with a special focus on diverse 

populations – to accelerate reductions in the U.S. cancer burden. 

Objectives and Milestones for Fiscal Year 2002

1. Support cancer communications planning, research, evaluation, dissemination, and 

marketing by establishing new data collection and analysis strategies. 

■ Sponsor the Health Information National Trends Survey. $1.00 M

■ Access relevant commercial data on use of the new media for health communications to inform NCI’s planning and evaluation efforts about which audiences use

which new media, and how they use them. $1.00 M

■ Fund Pilot Projects to Overcome the Digital Divide to test strategies to increase

access to and use of online and other interactive cancer communications. These

competitive grant supplements will be made to existing Cancer Information

Service (CIS) contractors to conduct pilot research in their regions. $1.50 M

2. Accelerate research, development, and interventions in cancer communications.

■ Support Centers of Excellence in Cancer Communications Research.

■ Provide support for investigators to diffuse and disseminate NCI-funded evidencebased interventions. 

3. Develop a menu of communication choices to meet the needs of all users, and 

especially to increase knowledge about, tools for, access to, and use of these 

choices by diverse populations. 

■ Conduct a series of pilot tests to explore the feasibility of new directions, using 

different CIS offices and cancer centers as test beds. $3.00 M

$3.5 M

$12.0 M

$6.0 M

PROGRESS IN PURSUIT 

OF OUR GOAL

NCI is already making progress in pursuit of our

cancer communications goal. For example, we

are taking the lead on developing the Health

Information National Trends Survey (HINTS),

the first nationally representative survey of the

American public – with emphasis on underserved

populations – about their access to and use of cancer-related health information. NCI is assisted in

this biennial, longitudinal data collection effort by

national experts in health communications and

representatives of other parts of the Department of

Health and Human Services, including the Centers

for Disease Control and Prevention, the National

Institute for Occupational Safety and Health, and

the National Library of Medicine at NIH. 

NCI is conducting ten research projects focused

on maximizing effective cancer communications

through message tailoring and increasing the

impact of behavioral counseling on cancer control
Opportunities

Extraordinary

Extraordinary Opportunities 93

$3.0 M

$2.0 M

$1.0 M

$27.5 M

■ Develop new communication products to facilitate cancer communications for the

public, patients and their caregivers, underserved populations, advocacy groups,

health professionals, and cancer communicators. Continue work with the AHRQ

to fund research on decision aids, and link with Pilot Projects to Overcome the

Digital Divide to promote dissemination and use of interactive communication

tools and collect information on current levels of and barriers to use. $3.00 M

4. Increase the Nation’s capability and capacity for cancer communications by training the health communications scientists, researchers, and practitioners needed to

achieve our scientific and health communications objectives. 

■ Encourage the development of interdisciplinary training programs that, at a minimum, include people in the fields of health behavior, marketing, engineering, 

communications, public health, and medicine. $1.00 M

■ Fund existing health communications research laboratories to conduct intensive

training programs and provide opportunities for research professionals in growing

areas, including risk communications and interactive health communications.

These will include intensive short-term training programs, grants for interdisciplinary training, and distance learning programs. $2.00 M

5. Enhance and refocus NCI’s communications activities to provide a comprehensive,

technology-supported capability for imparting information about cancer that is 

easily accessible, timely, and appropriate. 

■ Enhance the accessibility and user-friendliness of NCI’s databases and Web sites. 

■ Centralize the coordination of external and internal communications activities to

maximize proactive responses to communications priorities and challenges, ensure

optimal public access to NCI’s information resources and products, and promote

NCI’s identity as an accurate source of information and as the national leader in

cancer research and discovery. 

Management and Support

Total

interventions. In collaboration with the Agency for

Healthcare Research and Quality (AHRQ), we are

funding a review of the research evidence on 

cancer-related “decision aids” – interventions

designed to help people make specific and deliberate choices by providing information on options

and outcomes specific to a person’s health status.

The review will provide guidance about what

research is needed in this important area.

NCI and AHRQ are co-funding a demonstration

project to enhance consumer and patient use of

information about health care quality. The investigators are developing new tools for communicating about cancer-related risks. The demonstration

project will develop and test methods for providing

information on quality for consumers and patients

to use when making health care decisions. The

projects also will evaluate the impact of strategies

to provide information about quality to these 

audiences.

We are creating Centers of Excellence in

Cancer Communications Research to speed

advances in cancer communications knowledge.

Interdisciplinary teams of researchers will develop,
implement, and evaluate strategies to improve both

access to cancer information and its efficacy , effectiveness, and dissemination. The Centers will collaborate with other NCI-funded centers, such as

the Comprehensive Cancer Centers,

Transdisciplinary T obacco Use Research Centers,

the Special Populations Networks for Cancer

Awareness Research and T raining, and the Cancer

Information Service (CIS).

NCI is using new technologies to implement an

integrated cancer knowledge management system.

A new Publications Locator lets users view and

order NCI publications online from the NCI W eb

site. Users can access publications on various types

of cancer, treatment options, clinical trials, genetics, risk factors, cancer causes, prevention, and

managing side effects and pain. In addition, pilot

projects are underway to develop an online Cancer

Information Service (eCIS) that would provide

another alternative for people to get answers to

their questions about cancer and research findings.

Unequal access to cancer information is a critical problem for cancer communications. In 2000,

NCI will provide supplemental funding, in conjunction with our CIS regional offices, for Pilot

Projects to Overcome the Digital Divide. These

projects will provide the foundation for larger scale

projects to respond to the access needs of underserved populations. 

NCI’s Operation J-O-L-T (Joining

Organizations with Leading Technologies) is

working with numerous other groups to bridge the

gap between emerging technologies and their application to cancer communications. NCI coordinates

activities with the W orld Wide Web Consortium

on Cancer to collaborate, share information, and

support one another in the delivery of authoritative

cancer knowledge via survivor -run and other W eb

sites. We have arrangements with commercial

online vendors to provide their customers information on NCI resources, publications, and the CIS

hotline (1-800-4CANCER). NCI is working with

an Internet access hardware and software provider

to enable low income families to add cancer information to their Web portal. In addition, we are collaborating with a maker of novel devices for inexpensive Internet access to include links to NCI and

to customize for use by cancer patients. 

In 2000, NCI established the Eleanor Nealon

Extraordinary Communicators Lecture Series,

which pays tribute to outstanding communicators

who have advanced the science of communication

or the communication of science. NCI also has supported several creative meetings and workshops on

cancer and health communications-related topics,

including a Media and Health Education workshop

(co-sponsored with Rutgers University), the Future

of Health Technology Summit 2000 (co-sponsored

with the Massachusetts Institute of T echnology),

and the Strategic Education and T raining in Health

Communication workshop.

NCI reorganized its Office of Communications

in May 2000 to provide a comprehensive, integrated, and technology-based communication structure

that will increase the effectiveness of our interactions with the public. New organizational units will

enhance our capabilities in W eb design and evaluation, maximize our use of emerging technologies,

optimize our ability to help people with cancer

inquiries navigate through the NCI communications structure, and increase our readiness to form

partnerships with outside organizations. A new

Office of Education also has been established as an

essential NCI communication arm.

For more information on NCI’s Cancer Communications initiatives, go to dccps.nci.nih.gov/eocc or

cancer.gov/initiatives.

Budget Increase Request for 2002 

Cancer Communications

1. Support cancer communications planning, 

research, evaluation, dissemination, 

and marketing. $3.5 M

2. Create Centers of Excellence in Cancer 

Communications Research. 12.0 M

3. Develop a menu of communication choices. 6.0 M

4. Train health communications scientists, 

researchers, and practitioners. 3.0 M

5. Continue to integrate and restructure NCI’s 

communications activities. 2.0 M

Management and Support 1.0 M

Total $27.5 M

94 The Nation’s Investment in Cancer Research
Opportunities

Extraordinary

Extraordinary Opportunities 95

school education, and to be ethnic minorities. We

must work to eliminate this divide. How can we promote knowledge about, tools for, access to, and use of

cancer information, given the high national rates of

medical illiteracy? How can we ensure that cancer

communications and interventions are relevant, meaningful, and culturally sensitive to diverse audiences?

How can we know what does not work and why? We

are challenged to help physicians and other health

care providers optimize their communications about

cancer, and to redesign information systems so they

give people the information they want how, when, and

where they want it. 

New information technologies must complement,

not replace, older but effective strategies, such as

the mass media, one-to-one counseling, and targeted

print communications. For example, most people still

want to talk directly with a knowledgeable and supportive person. Such interactions can enhance outcomes of and satisfaction with care. As we develop new

technologies, we must not lose sight of the importance

of personal interactions and the need for continuing

research and practice to strengthen one-to-one communications. We also must take into account that consumers, patients, and health professionals alike are

coming to expect seamless, integrated, accessible, 

tailored communication choices. They want to move

easily among these new and older information technologies – for example, to go from a Cancer

Information Service (CIS) information specialist to

CancerNet or to talk to the specialist while viewing a

Web site.

To be effective, cancer communications must be

integrated into the cancer continuum from prevention

through treatment to survivorship and to end-of-life

issues, including palliative care and pain management.

Communication should be an integral component of

quality cancer care for all, regardless of race, ethnicity,

health status, education, income, age, gender, or geographic region.

C hanges in the role and accessibility of information are altering health care practices, patientphysician relationships, and the way consumers and

patients acquire and use information. Where once

physicians were the main source of health information,

now many consumers are actively using a variety of

information sources to meet their needs. Both consumers and health professionals now have an array of

new opportunities for creating, distributing, and

acquiring health information from sources such as the

World Wide Web, electronic mail, individually tailored

print and multimedia materials, interactive computer

games, interactive kiosks, and wireless pagers. 

These “new media” already have had an enormous

impact: 

■ According to a poll released by Harris Interactive in

February 2000, more than half of U.S. households

are now online and of these, 90% use the Internet. 

■ ACNielsen reported in May 2000 that nearly twothirds of Americans over age 12 have access to the

Internet, and half go online every day.

■ A 1999 Harris Poll found that approximately 70 

million (74 percent) of adults who use the Internet

searched online for health information. Cancer was

the third most sought-after health topic.

■ In 1999, cancer-related listservs (e-mail groups

devoted to a specific topic) hosted by the

Association of Cancer Online Resources processed

1.9 million pieces of e-mail each week.

Substantial barriers still prevent major segments of

the population from seeking and/or using cancer

information. Some people continue to lack access to

the array of cancer communications media. Others are

faced with content that is unintelligible to them (in

the wrong language or in language that is too complex), culturally inappropriate, or simply ineffective.

There clearly is a “digital divide” in our Nation and

around the world. Those who lack access to information

and new online tools – the information “have nots” –

are more likely to be poor, to have less than a high

The Changing Scene for Cancer Communications

“Science and communications are always inextricably entwined. 

From research to the patient, the imperative to communicate deepens.

Communication empowers, enlightens, soothes, clarifies life and death...”

— Dr. Richard Klausner
Planning National Agendas in 

Disease-Specific Research

R

eports on the toll that cancer places on the

American people are overwhelming. 1.2 million new cases diagnosed in 2000. A half million

people dead this year. Nine million with a history of

cancer right now. But these daunting statistics fail to

communicate the tremendous burden borne by

patients, families, and friends who want to better

understand and deal with a specific form of cancer. 

Five years ago, NCI instituted a comprehensive,

dynamic planning process that incorporated three

separate – but equally vital – elements. The first of

these focuses on our continuing efforts to support

scientific discovery and application of discovery to

human health – a range of programs that currently

form the basis for the “Challenge” section of this

document. The second involves investment in

areas of research that are broadly applicable to all

cancers, as discussed in this document’s

“Extraordinary Opportunities” section. The third

element of our process is disease-based planning.

When NCI began its new approach to planning,

we first identified the broadly applicable scientific

opportunities through which vital progress could be

made most quickly. We then began a comprehensive review of the large programs that support discovery. Once these assessments were well underway, we stepped back and asked ourselves, “Can

these broad investments be tailored to specific cancers? Have we developed the tools and infrastructure to conduct disease-specific research, and to do

it well? Have we defined those broad-based research

areas that can best support our disease-specific

efforts?” Our efforts to answer these questions 

culminated in the creation of a new vehicle for

planning disease-specific research: the Progress

Review Group.

PRGs: THE PEOPLE, THE PROCESS,

THE PLAN

Progress Review Groups (PRGs) are panels of 

20-30 experts that are convened to develop a

national research framework for individual types of

cancer. The PRGs assess the state of the science,

identify gaps in knowledge and barriers to progress,

and identify research priorities. Beginning with an

assessment of the NCI’s current research portfolio,

the members of each PRG identify and prioritize

future research areas for the particular cancer under

study, and summarize their deliberations in a written report. 

Each PRG is given a compelling charge: To imagine and build the future. In doing so, the groups

engage the scientific, medical, and advocacy communities in a dialogue on potential directions for

Schedule of Completed and Planned

Progress Review Groups 

Completed August 1998 Breast Cancer

Completed August 1998 Prostate Cancer

June 1999 - November 2000 Colorectal Cancer

November 1999 - February 2001 Brain Tumors

February 2000 - May 2001 Pancreatic Cancer

April 2000 - August 2001 Leukemia-LymphomaMyeloma

August 2000 - November 2001 Lung Cancer

December 2000 - February 2002 Gynecologic Cancers

March 2001 - May 2002 Kidney/Bladder Cancer

June 2001 - August 2002 Stomach/

Esophageal Cancer

September 2001 - November 2002 Liver/Bile Duct Cancer

December 2001 - February 2003 Skin Cancers

96 The Nation’s Investment in Cancer Research
Planning Agendas

research. In spotlighting the gaps in our knowledge,

and recommending how those gaps can be filled,

the PRGs give NCI the tools it needs to build the

future and kindle the interest of the best scientific

minds in research on specific cancers. 

The PRG process is intensive and extends over a

number of months. To date, PRGs have been completed for breast, prostate, and colorectal cancers.

A Brain Tumor PRG, conducted in collaboration

with the National Institute of Neurological

Disorders and Stroke, is nearing completion. PRGs

for all of the other most common types of cancer

are planned through 2002; ongoing and planned

PRGs are outlined in the chart on page 96.

OUR COMMITMENT: 

WE WILL LISTEN AND WE WILL ACT

NCI is committed to responding rapidly and

enthusiastically to recommendations from the

Progress Review Groups. For each PRG, we:

■ Determine to what extent the PRG’s recommendations are being addressed or could be addressed

by existing programs or efforts.

■ Develop an implementation plan for the recommendations and discuss that plan with the members of the PRG.

■ Communicate our plans to the scientific community, and enlist their active participation in

the implementation process.

■ Track progress toward goals and report on the

results.

Based on PRG recommendations, NCI may

modify existing programs or activities, or create

new ones. For example, the Breast Cancer PRG

noted that our lack of understanding of the biology

and developmental genetics of the normal breast

was a significant barrier to progress against the disease. NCI responded by releasing a Program

Announcement, in conjunction with several other

NIH Institutes, seeking applications for research on

normal breast development, as well as on changes

in the breast throughout the progression of early

and advanced cancer. 

NCI also responds to the findings and recommendations of Progress Review Groups in other

ways. Proposed research projects that address priorThe Common Scientific

Outline 

“

What research are you doing on prostate 

cancer?” “How much of your funding for

basic research is focused on prevention?” These are

common questions, but they can be surprisingly

difficult to answer.

In order to arrive at the answers to these questions, planners and policy makers must have the

necessary information readily at hand. To respond

to this need, the NCI developed a new coding 

system to categorize its research simply and 

systematically, by type of cancer and area of 

science. This new coding system, known as the

Common Scientific Outline (CSO), grew out of the

NCI’s experience with PRGs and disease-specific

task forces over the last several years and has

been developed in conjunction with the

Department of Defense (DOD) U.S. Army Medical

Research and Materiel Command because of its

work in cancer research.

Both NCI and DOD have now used the CSO to 

categorize their research projects for Fiscal Years 

1997-1999. In addition, several national and international organizations – both public and private –

have been invited to participate in a pilot project

to use the CSO to classify and analyze their

research portfolios.

Because of the simplicity and accessibility of 

its design, the CSO and the reporting and analysis

tools it makes possible are likely to have an

impact on shaping disease-related research and

scientific planning. The CSO will also foster 

coordination between NCI, DOD, and other 

organizations that support cancer research by

identifying gaps in funding.

Planning Agendas 97
ities identified by PRGs but rank just below the

cut-off for funding receive special consideration in

the funding exceptions process.

WHAT WE ARE LEARNING

With reports from three Progress Review Groups

completed, and several more underway, NCI

has begun to take stock of its efforts to support disease-specific research. The results to date have

been encouraging. About 80 percent of the recommendations resulting from PRGs to date can be

addressed by programs that are already in place or

with minor modifications to existing programs. In

fact, there are core scientific needs – for example,

the need for research training, or for the development of disease biomarkers – that appear to be universal, regardless of the disease. Many of the

remaining recommendations represent genuine

gaps in research or infrastructure that NCI must

and will address. A few of these, however, fall outside the Institute’s purview (e.g., Medicare reform),

and NCI’s response to these recommendations will

necessarily involve advising other organizations or

agencies, rather than acting directly.

These results indicate to us that we are on the

right track. But the larger question that remains is

whether this approach to disease-based research

planning makes a difference in research and discovery over the long term. To that end, we are

beginning to develop a plan for evaluating the PRG

process and outcomes, and we are exploring strategies to reassess diseases that remain areas of high

concern. Starting with the Breast and Prostate

PRGs, we will analyze changes in funding levels,

types of research funded, and grant application

response that occur after the completion of a PRG.

We will issue a status report two to three years after

each PRG, and the members will reconvene to discuss the status of the recommendations and NCI’s

response and to recommend further action. This

process will be in place by 2001, and will provide

valuable information and insight about our directions in disease-based planning.

For NCI Progress Review reports and schedules,

go to planning.cancer.gov. 

98 The Nation’s Investment in Cancer Research

Why do we need Progress Review Groups?

PRGs allow us to convene a group with diverse backgrounds to step back and take a look at where we are,

and to speculate on where we should be going in 

cancer research for maximum benefit. PRGs also serve

to show Congress and the public that NCI really does

have a reasonable planning process, and they indicate

directions for future research.

This attracted me to the process when Dr. Klausner

asked me to serve as Co-Chair. It looked like chairing

the PRG would be fun, and it seemed that through the

process, we might come out with broad recommendations for breast cancer research funding. 

What is realistic to expect from the PRG

process? What are its limitations?

It’s realistic to expect PRGs to give a picture of where

the research is now, and a broad outline of where it

should be going. PRGs are also good for identifying

areas of critical need. 

In terms of limitations the PRGs can only give a

“snapshot in time” of the state of the science, or of

research needs, or of exciting opportunities. Things

change. The danger would be if the NCI tried to adhere

too rigorously to a plan made in the past. A PRG report

is like any strategic plan – it’s a work in progress that

needs to be modified continuously to address changing

opportunities and needs.

“There’s So Much More We Can Do” 

A Talk with a PRG Co-Chair

Harold Moses, M.D., is Director of the Vanderbilt Cancer Center in Nashville. In 1998, he served as Co-Chair of 

NCI’s Breast Cancer Progress Review Group. Recently, we talked to Dr. Moses about the PRG and the future 

of breast cancer research. 
Planning Agendas

Planning Agendas 99

Looking specifically at the Breast Cancer

PRG, what do you consider to be the most

valuable things to come out of the process?

Well, it’s difficult to place a value judgment on this.

But the realization of how little we know about the

basic biology of the mammary gland, and about the

progression of cancer from normal to precancer through

metastasis, was certainly very important. The group

recognized how important an understanding of normal

breast biology would be to designing clinical trials, as

well as identifying surrogate endpoints and markers for

studies of all kinds, including epidemiologic and prevention studies.

While the Breast Cancer PRG was going on, NCI had

many other ongoing working groups, and they were

making similar recommendations. It’s amazing how 

similar the recommendations coming out of the breast

group were to those coming out of the Prostate PRG,

for example. So the recommendations we were coming

up with were not necessarily unique to breast cancer,

but they were critical to the advancement of science in

a number of areas. 

Are you satisfied with NCI’s response to 

the PRG’s recommendations?

I was absolutely amazed at NCI’s response; it was 

fantastic, in my view. Some of the most exciting

aspects of NCI’s response, to me, include the increased

funding for investigator-initiated grants, especially

those in basic biology and early progression; the

renewed emphasis on mouse models; and the CGAP

[Cancer Genome Anatomy Project] movement to incorporate some of our recommendations, such as the

development of gene expression profiles for normal

breast tissue in addition to breast cancer. 

What did the Breast Cancer PRG reveal

about the current state of breast 

cancer research?

It showed us that we’ve come a long way relative to

five or ten years ago. We’ve made enormous advances,

and that includes advances in basic science that are

applicable to all types of cancer. It’s now becoming

clear in the survival rates that breast cancer can be

more of a chronic disease, not a disease that is 

invariably fatal. 

Another thing that came out of this group was the 

critical importance of basic research to progress in

breast cancer, and the recognition of its importance to

all the groups at the table – basic scientists, clinicians,

epidemiologists, prevention researchers, and so on. 

PRG members in all of those disciplines recognized 

the urgent need for advances in basic research.

What would you like to see happen in the

future in breast cancer research?

I would like to see us figure out better preventive

measures against breast cancer, as well as treatments

that will cure the disease with minimal side effects. 

A major need is to figure out how to do Phase II 

clinical trials more quickly and efficiently. There are 

so many amazing treatment agents in the pipeline B

targeted agents with very low toxicity. Figuring out

how to use those in the most appropriate way is a

major challenge.

We also need better diagnostic criteria. We need to

know which pathways are altered in precancer, early

cancer, metastatic cancer. In addition, we need to keep

plugging along in normal biology. 

What I would foresee is that someday, we’ll be able to

get the gene expression profile for each individual cancer, then administer a treatment that’s targeted to that

cancer at the molecular level, likely in combination

with a standard treatment. It’s unlikely that any single

treatment will be effective against all breast cancers.

There are a lot of opportunities right now in breast

cancer research, but they involve a lot of work. 

It’s exciting; there have been many important accomplishments – there’s so much more we can do.
100 The Nation’s Investment in Cancer Research

How It All Comes Together

T

he programs and initiatives described in this document

promise to one day increase our understanding of 

cancer and improve the options available to patients, their

families, and the physicians who care for them. Examining

how these challenges and opportunities apply to lung 

cancer – the leading cause of cancer death in the United

States – illustrates the potential they hold for improving

our ability to prevent, detect and diagnose, and treat 

cancer in all its forms. 

Prevention

In one of many efforts to respond to the challenge of

studying emerging trends in cancer (page 38), detailed

maps are developed to displayed data on cancer deaths in

the Atlas of Cancer Mortality. Researchers use these data

to identify previously unrecognized geographic patterns in

cancer mortality and formulate hypotheses for further

research. A few years ago, investigators using the atlas

observed that men living along the Southeast coast of the

United States were dying of lung cancer at unusually high

rates. Follow-up investigation led to the discovery that

many of these men had been exposed to asbestos while

working in the shipyards during World War II, a key piece

of evidence linking asbestos to lung cancer and the need

to prevent future exposures to asbestos. 

As a result of the extraordinary opportunities identified for

research on genes and the environment (page 64) and

tobacco and tobacco-related cancers (page 86), scientists

are turning their attention to the perplexing question of

why some individuals exposed to cancer-causing agents

develop lung cancer while others do not. Researchers are

beginning to find the answers by combining genetics and

epidemiology to examine the relative roles of genes, smoking, and other environmental and lifestyle factors in lung

cancer development.

Through the extraordinary opportunity for cancer communications (page 91), we hope to find the best ways to tailor and target prevention messages about smoking and

other risk factors for lung cancer to teens, adults, or

health care providers. These communications must give

special attention to the distinct smoking patterns and

preferences of different racial and ethnic groups. For

example, African American smokers are more likely than

others to choose menthol cigarettes, a preference that

may inadvertently result in the absorption of higher levels

of toxic substances from tobacco smoke.

Detection and Diagnosis

Investigators pursuing the extraordinary opportunity for

research on the signatures of cancer cells (page 74) are

focused on learning as much as possible about the unique

characteristics of normal cells and the changes that occur

when a normal cell becomes cancerous. As scientists identify and catalog the molecular changes that accompany

different types and stages of cancer, they may one day be

able – through a simple test of body fluid or cells – to use

these biomarkers as tools to detect and diagnose lung

cancer at its earliest stages. For example, now that we

know that lung cancer development is accompanied by

alterations in the cells that line the bronchial passages,

investigators are closer to finding an accurate and reliable

biomarker for early detection of lung cancer. 

Because only 15 percent of lung cancers are diagnosed in

the early stages when they are most curable, a reliable,

cost-effective tool for screening could considerably

increase the chances of successful treatment. Through 

NCI’s national clinical trials program (page 33), researchers

pursuing the extraordinary opportunity for cancer imaging

(page 69) are evaluating the use of spiral computed

tomography (CT) for early detection of lung cancer. They

are trying to determine whether this relatively new imaging

technology detects more cases of lung cancer than chest 

x-rays and whether its use will ultimately save lives.

Treatment

Eventually, we hope that what we learn about molecular

signatures can be applied, through the extraordinary

opportunity for research on molecular targets of prevention and treatment (page 80), to develop therapies targeted for maximum benefit in treating lung cancer. 

Currently, three Specialized Programs of Research

Excellence (page 31) are dedicated to developing and evaluating new treatments for lung cancer. They bring teams of

scientists together in interdisciplinary research centers to

focus on specific cancers and hasten the movement of

research findings from the laboratory to the clinic.

Next Steps

NCI will convene a Progress Review Group in the fall of

2000 to assess the state of the science in lung cancer,

identify gaps in knowledge and barriers to progress, and

rank research priorities for lung cancer-related research.

(See page 96.) The report from this group is expected to

guide our lung cancer research for years to come. 
NIH Publication No. 00-4373 T813

October 2000 

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cancerTrials at Information about clinical trials for patients, physicians,

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Extraordinary Opportunity Initiatives at Details on the NCI initiatives related to the extraordinary 

cancer.gov/initiatives opportunities for investment described in this document.

NCI Funding Opportunities at Information on grants, contracts, and other forms of 

deainfo.nci.nih.gov research funding available through NCI.

NCI Research Resources at cancer.gov/resources A directory of over 100 research services and tools 

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Atlas of Cancer Mortality at Maps, text, tables and figures on cancer death rates in the 

cancer.gov/atlas U. S. 1950-1994 for about 40 cancers. Includes 

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Cell Biology and Cancer at An introduction to the biology of cancer for high school 

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Acknowledgments

The National Cancer Institute’s The

Nation’s Investment in Cancer Research

is a collaborative effort involving countless people both within NCI and in

research, professional, and advocacy organizations throughout the country who

contribute their insights, perspectives,

and expertise.

The NCI Office of Science Planning and

Assessment (OSPA) and Financial

Management Branch (FMB) play key roles

in developing and assembling The Nation’s

Investment in Cancer Research. Several

members of these groups deserve special

recognition for their contributions. Cherie

Nichols served as mentor and advisor to

the OSPA Planning Team. Kathie Reed, as

team leader, artfully guided each step of

the process from conceptualization to

production. With exceptional dedication

and skill, she helped to shape this document into a valuable resource for the cancer community. Catherine Law, Jane

Lockmuller, Kate Nagy, Suzanne Reuben,

Jennifer Sutton, and Anne Tatem worked

tirelessly alongside the Champions to

conceptualize, write, and ensure the accuracy of the material. D. J. Joya kept up

the internal development Web site.

Several other OSPA staff played behindthe-scenes supporting roles including

Marilyn Duncan, Kevin Callahan, Bernard

Glassman, and Anna Levy. All members of

the FMB worked to ensure the accuracy

and comprehensiveness of the budget figures. Special recognition goes to John

Hartinger and Ngan Nguyen. In the Office

of Communications, Maggie Bartlett and

Paul LaMasters helped put final touches

on the document.

Extraordinary Opportunity and NCI

Challenge Champions

Dr. Richard Klausner served as Editor-inChief. Several NCI staff members served

as “champions” for the Extraordinary

Opportunity and NCI Challenge areas.

They developed the goals, plans, and

resource requirements for their areas 

and worked with OSPA staff to develop

the background narrative. 

Extraordinary Opportunity Champions

Genes and the Environment –

Dr. Robert Hiatt, Dr. Robert Hoover, 

Dr. Kenneth Buetow

Cancer Imaging – Dr. Ellen Feigal*, 

Dr. Daniel Sullivan

Defining the Signatures of Cancer Cells –

Dr. Robert Strausberg, Dr. Dinah Singer

Molecular Targets of Prevention and 

Treatment – Dr. Robert Wittes, 

Dr. Edward Sausville

Research on Tobacco and Tobacco-Related

Cancers – Dr. Robert Croyle, 

Dr. Neil Caporaso

Cancer Communications –

Dr. Barbara Rimer, Dr. Susan Sieber

NCI Challenge Champions

Investigator-Initiated Research –

Dr. Marvin Kalt

Centers, Networks, and Consortia –

Dr. Ellen Feigal, Dr. Brian Kimes

National Clinical Trials Program –

Dr. Robert Wittes, Dr. Michaele Christian

Studying Emerging Trends in Cancer –

Dr. Rachel Ballard-Barbash, 

Dr. Brenda Edwards

Quality of Cancer Care –

Dr. Joseph Lipscomb, Dr. Martin Brown

Reducing Cancer-Related Health 

Disparities – Dr. Robert Hiatt, 

Dr. Jon Kerner

Informatics and Information Flow –

Dr. John Silva, Ms. MaryAnn Guerra

Training, Education, and Career 

Development – Dr. Brian Kimes

In addition to these Champions, 

Dr. Joseph Fraumeni, Ms. Jill Johnson, 

Dr. Barry Kramer*, Ms. Mary McCabe, 

and Ms. Susan Waldrop made significant

contributions to the content and accuracy

of this document.

Fiscal Year 2002 Planning Committee

Members of this committee helped to

ensure that this document clearly and

accurately describes the excitement and

promise of NCI’s efforts, our needs and

plans, and the vision for the future of our

research programs.

Dr. Richard Klausner

Director, NCI

Dr. Alan Rabson

Deputy Director, NCI

Dr. Robert Wittes

Deputy Director, Extramural Science and

Director, Division of Cancer Treatment and

Diagnosis 

Dr. Carl Barrett*

Director, Division of Basic Sciences

Dr. Joseph Fraumeni*

Director, Division of Cancer Epidemiology

and Genetics

Dr. Peter Greenwald

Director, Division of Cancer Prevention

Ms. MaryAnn Guerra

Director, Office of Management

Dr. Joseph Harford

Associate Director, Special Projects

Mr. John Hartinger*

Associate Director, Financial Management

Dr. Marvin Kalt

Director, Division of Extramural Activities

Dr. Edison Liu*

Director, Division of Clinical Sciences

Dr. Suresh Mohla, Chair

Extramural Advisory Board

Ms. Cherie Nichols

Director, Office of Science Planning and

Assessment

Dr. Barbara Rimer*

Director, Division of Cancer Control and

Population Sciences

Dr. Susan Sieber* 

Director, Office of Communications

Dr. Dinah Singer*

Director, Division of Cancer Biology

Ms. Susan Waldrop*

Director, Office of Science Opportunities

Dr. Allan Weissman, Chair

Intramural Advisory Board

Dr. Frederick Appelbaum, Chair

Board of Scientific Advisors

Dr. Martin Abeloff, Co-Chair 

Dr. Bruce Stillman, Co-Chair

Board of Scientific Counselors

Ms. Lillouise Rogers, Member

Director’s Consumer Liaison Group

Dr. Phillip Sharp, Chair

Ms. Ellen Stovall, Member

National Cancer Advisory Board

*Editorial Board member