Foreword:	Director’s	Introduction	....................................... Page	3 

I.	 Empowering	Cancer	Research........................................ Page	7 

II.	 Reaching	All	Communities	Touched	by	Cancer	............. Page	19 

III.	 The	Promise	of	Prevention	and	Early	Diagnosis............ Page	7 

IV.	 NCI	at	a	Glance	.............................................................. Page	31 

V.	 The	009	Budget	(Professional	Judgment	Budget)	......... Page	35 

	 

	 

	 

	 

	 



Foreword: Director’s 

introDuction 

h tlaeHf os etutitsnIl anoitaN

John E. Niederhuber, M.D.


Director of the National Cancer Institute


J

eanne McCoy’s grandmother 

and mother both suffered from 

von Hippel-Lindau syndrome 

60 (VHL), a genetic disease that 

can manifest itself as a combination 

of nearly 600 types of cancer. For 

Jeanne’s grandmother, the tumors ap

peared first on her retina (she went 

blind at the age of 30) and also on 

her brain stem. Eventually the cancer 

invaded her kidneys. Jeanne’s mother, 

who was told at a young age that she 

did not have VHL, was shocked to learn 

later in her life that this diagnosis was 

wrong. That family history led Jeanne 

to search the Internet, where she learned 

of the recently-identified VHL gene. 

Jeanne sought out the National Cancer 

Institute, where Dr. Marston Linehan 

was leading a team of researchers who 

were working at the cutting edge of 

VHL—both in the laboratory and in 

the clinic. 

Because of the late stage of her cancer and 

her family history, Jeanne’s mother was 

quickly accepted into the clinical program 

at the NCI. There, she was confirmed to 

carry the VHL mutation. Several months 

later, Jeanne sent her own blood sample 

and records to NCI. The results 

confirmed that Jeanne also carried the 

VHL mutation. 

Jeanne had no symptoms of kidney 

cancer. In fact, at age 34, she had given 

birth to her third child a year earlier. 

Except for faint back pain, something 

many new mothers experience, Jeanne 

felt fine. But she also knew that the VHL 

mutation suggested a frightening fate. 

So Jeanne consulted her local 

oncologist, who advised her to 

undergo an ultrasound and CT-scan 

in a hospital near her Greenville, South 

Carolina, home. Within hours, she 

learned that both her kidneys were 

riddled with cysts and tumors that, if 

untreated, would eventually kill her. 

Within two weeks, Jeanne was headed 

for Bethesda, for more testing and 

consultation, and treatment at the 

National Cancer Institute. While 

hospitalized for the second of her kidney 

surgeries, just before Christmas 2003, 

Jeanne met Dr. Linehan, who came to see 

her in the surgical intensive care unit at 

the NIH Clinical Center, the world’s 

largest hospital dedicated to clinical 

research. “He sat down and talked to 

my husband and me,” she recalls. “And 

suddenly he put a real, personal face on 

the research; we realized that this wasn’t 

about scientists lost in a lab, it was about 

people, and about early diagnosis and 

detection, and about finding a cure.” 

Jeanne McCoy is doing well, but she is 

not cured. All of us owe a huge debt of 

gratitude to her and many other cancer 

patients for their willingness to join us 

in defeating this devastating disease, 

by participating in clinical research 

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NCI Strategic Objectives 

To Preempt Cancer at Every 

Opportunity 

To Ensure the Best Outcomes 

for All 

• Understand the causes and mechanisms 

of cancer. 

• Understand the factors that influence 

cancer outcomes. 

• Accelerate progress in cancer 


prevention. 


• Improve early detection and diagnosis. 

• Improve the quality of cancer care. 

• Improve the quality of life for cancer 

patients, survivors, and their families. 

• Develop effective and efficient 


treatments.


• Overcome cancer health disparities. 

3 
Why It’s Important: 

the Human and Economic 

Burden of Cancer* 

• 1,444,920 Americans were diagnosed with cancer in 2007 

• 559,650 Americans died of cancer in 2007 

• $206.3 billion was spent on healthcare costs for cancer in 2006 

Estimated Number of New Cancer Cases 


in the United States from 1998 to 2007


1,500,000


1,444,920 

1,399,790 1,400,000
 1,368,030 1,372,910 

1,334,100 

1,300,000
 1,284,900 1,268,000 

1,228,600 1,221,800 1,220,100 

1,200,000


1,100,000


1,000,000


1998 1999 2000 2001 2002 2003 2004 2005 2006 2007 

*Data source: American Cancer Society, Cancer Facts and Figures, 1998 to 2007 based on NCI SEER and NAACCR data. 

and trials. For the sake of Jeanne and her 

family, and the countless others like 

them, we need to make more progress 

more quickly. 

Everything we do at NCI begins and ends 

with people—those with cancer, those at 

risk for the disease, and those who care for 

them. That singular focus lies at the heart 

of all our work in basic, translational, 

and clinical science to better treat cancer; 

our outreach into communities across 

the United States; and our efforts to 

build cancer prevention strategies and 

programs. People with cancer are not just 

“patients” to us. They are human beings 

with family, friends, dreams, and lives 

that are all impacted by their diagnoses. 

Cancer patients are the cornerstone 

of cancer research, and we are forever 

indebted to those men and women who 

give so generously of themselves to make 

life better for others. 

Cancer, as a disease, is incredibly com

plex, both in its biology and in its social 

impact. We now know that cancer is 

not a single disease or even a 

small group of related diseases. 

Rather, we are finding that 

when viewed at the level of 

genes and proteins, cancer is 

a collection of many different diseases. It is this greater 

understanding of cancer at 

the molecular level that NCI believes 

will enable us to change the meaning 

of a cancer diagnosis in the future. The 

National Cancer Act of 1971 established 

the National Cancer Program and au

thorized NCI to coordinate its efforts. 

Encompassing academia, industry, the 

private sector, international partners, and 

other government agencies, NCI directs a 

massive multifaceted effort against 

cancer that continually strives to match 

the enormity of the disease itself. 

NCI is dedicated to the understanding, 

diagnosis, treatment, and prevention of 

cancer for all people. To these ends, NCI 

creates connections: basic science to clin

ical therapies; researchers to patients and 

doctors; NCI internal (“intramural”) 

work and external (“extramural”) efforts; 

public and private cancer-related institutions and research; and the U.S. effort to 

the wider global fight against cancer. 

We have made progress, but much work 

remains. The rate of cancer mortality 

continues to drop; however, cancer still 

remains a leading cause of death, second 

only to heart disease. The number of 

new cancer diagnoses continues to rise, 

with more than 1.4 million people hear

ing the dreaded words “you have cancer” 

this year in the United States alone. 

And we know this burden is dispropor

tionately shouldered by the poor, the 

elderly, and minority populations. 

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Our progress is thanks to the work of 

thousands of cancer researchers, both 

at the lab bench and in the clinic, and 

numerous patients who seek not only 

treatment for themselves but also to 

provide hope for others. As we now 

understand that cancer is a disease that 

results from changes in our genes, we 

are striving to comprehend the precise 

changes that occur before we are born 

or as we interact with our environment, 

and how to prevent or overcome them. 

We have pried open some of the secrets 

of how cancer begins, how it takes advantage of our normal biological makeup, 

and how it initiates its lethal spread, and 

we are pursuing potential new avenues 

to learn to stop it in its tracks. 

Each step forward we take opens up 

promising new opportunities to make 

further progress, which must be seized. 

NCI cannot do this alone. Thus, it is 

essential that NCI increase its capacity 

to enable and support the many critical connections that will bring about 

real changes in cancer prevention, 

diagnosis, and treatment, all the while 

ensuring that the latest scientific advances 

become available to all patients, not 

just those who have access to the NIH 

Clinical Center or other leading national 

cancer centers. 

Making these connections is not an 

easy task, and it is made even more 

difficult in light of the budgetary 

realities we face. Nearly 80 percent of 

our annual funding goes to support 

external scientists and researchers at 

academic institutions and centers across 

the country, including many who conduct 

the clinical trials necessary to translate 

basic scientific findings into cancer treatments. Much of the remainder supports 

our internal research operation, which 

facilitates collaborative opportunities between world class scientists, 

both at NCI and elsewhere, provides freedom to pursue new insights 

and opportunities (particularly for 

underserved populations), and trains 

and develops the next generation of 

cancer researchers. 

The below-inflation budgets of the 

last two years have impacted both our 

internal and external capabilities. Indeed, 

implications of our budgetary constraints 

have carried beyond the basic research 

laboratories, impacting the clinical 

research options available to patients. 

For example, the NCI’s Clinical Trial 

Cooperative Groups, which promote 

and support clinical trials of new cancer 

treatments, explore methods of cancer 

prevention and early detection, and 

study quality-of-life issues and rehabilitation during and after treatment, 

saw their funding cut by 10.9 percent 

between fiscal year 2002 and 2006. 

Although some of their lost funding was 

restored in FY 2007, fewer trials have 

been started, and fewer patients have 

been enrolled in available trials. This slows 

our ability to improve the standard of 

care for cancer patients through new 

drugs, devices, or interventions. 

Between the fiscal years 2004 and 

2007, NCI’s budget remained relatively 

flat. However, factoring in the rate of 

Dollars in billions

nci’s challenge 

5

Funds available 4.9

4.8 $4.8B $4.8B 

-12% Loss in 

Purchasing

Power Since 

2004 

4.7 

4.6 Adjusted by BRDPI* 

4.5 

4.4 

4.3 $4.3B 

4.2 

FY 2004 FY 2005 FY 2006 FY 2007 

* BRDPI: Biomedical Research and Development Price Index 

[ http://officeofbudget.od.nih.gov ] 

5 
The NCI is a unique 

national resource, one 

that can bring together 

the various aspects of 

the National Cancer 

Program by acting as a 

convener and facilitator 

of the public and private 

sectors to deliver on the 

promise that was made 

by the National Cancer 

Act of 1971. 

Getty Images 

biomedical inflation reveals a 12 percent 

loss of purchasing power over those same 

years. Investigators tell us that belowinflation funding often means the loss of 

a laboratory technician or two, a graduate student not brought into the lab, a 

postdoctoral fellow not hired, or research 

that must be scaled back to the point 

that scientific progress is significantly 

slowed and some opportunities are lost. 

The problem is compounded because 

these investigators—some of whom are 

turning away from biomedical science— 

are also the professors who nurture the 

careers of young scientists who will drive 

the future of cancer medicine. 

Aware of the fiscal situation we face, this 

document is a report on progress and opportunities, as well as a plan for critical 

scientific growth. It details the state of 

our science, the extraordinary promise of 

today’s research, and the resources we believe would allow NCI to hasten the day 

that the promise of personalized cancer 

medicine arrives. That day will also usher 

in a new era of cancer prevention, with 

new diagnostic tests that can predict the 

impending development of cancer long 

before the formation of a tumor. 

The NCI is a unique national resource, 

one that can bring together the various 

aspects of the National Cancer Program 

by acting as a convener and facilitator of 

the public and private sectors to deliver 

on the promise that was made by the National Cancer Act of 1971. The nurturing 

and careful stewardship of this national 

resource is our shared responsibility. 

Cancer touches virtually every American 

family, and NCI is committed to research that helps us provide access to the 

best standard of care for all in the communities where they live and work. And 

it is on behalf of every cancer patient 

that NCI will continue its vital research, 

until stories about successful battles 

against cancer are the only ones to tell. 



I. empowering cancer research. 

Every life is touched by cancer. As the leader of the U.S. efforts to understand, 

diagnose, treat, and prevent cancer in all of its forms, NCI must play the central 

role in bringing together basic and clinical researchers, patients, physicians, 

care givers, policy makers, and many others in a common cause. 

The convergence of new biomedical technologies with 

information technologies has 

revealed to us just how complex cancer 

truly is. Indeed, the biology of cancer is 

intimately intertwined with the unique 

genetics of each person, making it an 

“individualized” disease. The ability to 

deliver individualized interventions to 

patients requires the integration and 

collaborations of disciplines not traditionally thought of as part of cancer 

research. This broader view of the 

cancer research community extends 

to mathematicians, physicists, and 

chemists as well as others in the physical sciences and relies on their skills 

and talents to enhance our ability to 

manage large amounts of data as well 

as developing novel applications in 

clinical research. 

THE PROMISE OF PERSONALIzED 

CANCER MEDICINE 

The progress we have made has opened 

up a vision of a future personalized 

cancer medicine, when doctors will 

determine prognosis and treatment 

options by understanding each 

patient’s unique genetic makeup and the 

genetic aberrations that have led to 

his or her cancer. That future includes 

developing combinations of therapeutic 

solutions that target the multiple pathways of cancer, earlier interventions 

that eradicate cancer long before the 

development of a tumor or the onset 

of symptoms, and effective prevention methods based on the individual. 

Personalized Cancer Medicine


Detection and diagnosis 

• Identify patterns of genes associated 

with the development of specific cancers. 

• Study how genetic variations that change 

the function of proteins cause cells to 

function abnormally. Investigate how those 

changes are further affected by lifestyle 

behaviors and environmental factors. 

• Develop biomarkers—blood tests and other 

tests using human specimens; imaging tech

niques; and other new methods—to detect 

and measure changes in protein and cellular

function associated with specific cancers. 

­

• Monitor the changes in an individual’s 

cellular function, in order to detect 

pre-cancerous changes and intervene to 

prevent those changes from progressing 

to disease. 

• Use genetic profiles to identify subsets 

of cancer types that define prognosis. 

Prognosis and prevention Treatment 

• Choose targeted therapies that minimize 

side effects and are based on both the 

type of cancer and the individual’s 

biological profile. 

• Use biospecimen tests and imaging 

techniques to measure the impact of 

interventions and refine treatment to 

improve outcomes. 

7 
In a development that 

is literally lifesaving, 

Dr. Staudt, who has been 

on the staff of NCI for 

nearly 20 years, 

has successfully used 

genomic technology 

to reliably distinguish 

Burkitt’s lymphoma 

from Diffuse Large B-Cell 

Lymphomas (DLBCL). 

National Institutes of HealthPhotographer: Bill Branson 

Louis M. Staudt, M.D., Ph.D., and members of his NCI laboratory 

This is a vision shared across the 

oncology research community. 

The NCI-designated Cancer Centers 

Directors’ report “Accelerating Successes 

Against Cancer”* also recognizes that 

advances in treatment will come from 

understanding molecular causes of dis

ease and using combination treatment 

approaches employing multiple modali

ties. The National Cancer Institute has 

not only embraced but is leading the 

way to that future, and is dedicated in 

all it does to ushering it in as rapidly 

as possible. 

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THE ESSENTIAL UNITY OF 

BASIC AND TRANSLATIONAL 

SCIENCE 

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In addressing the burden of cancer, there 

is an essential unity between fundamen

tal scientific studies on the molecular 

causes of cancer, research focused on 

translating those studies into the clinic, 

and actual clinical practice. The tradi

tional linear relationship from the bench 

to the bedside is no longer an effective 

and efficient model for medical progress. 

Insights from fundamental scientific 

* [NIH Publication No. 06-6080, September 2006]  

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research need to be tested in clinical 

settings, which in turn give rise to new 

research directions that can be pursued 

in the laboratory. 

Indeed, at the NIH Clinical Center 

and at many other cancer research and 

treatment centers across the country, 

laboratories and patient rooms are in 

close proximity. Cancer researchers 

within NCI and based at other in

stitutions are committed to working 

together to mine new insights from 

wherever they arise for truly effective 

cancer treatments. 

ON THE FRONT LINES: 


CONNECTING SCIENCE AND


CLINICAL PRACTICE


“CASE STUDIES”


Louis M. Staudt, M.d., Ph.d., studies 

lymphoma, a cancer of the lymphatic 

system that affects 23,000 patients 

yearly and causes 10,000 deaths 

Treatment can be difficult, because 

this cancer has many subtypes, which 

require different treatment options. 

For example, Burkitt’s lymphoma 

and diffuse large-B-cell lymphoma 

(DLBCL) are difficult to diagnose 

because the two cancers appear very 

similar under a microscope. However, 

the two are genetically distinct, and 

thus require very different treatments. 

And the stakes couldn’t be higher. 

“If Burkitt’s patients are treated with 

intensive therapy, there is roughly an 80 

percent survival rate,” says Dr. Staudt, 

deputy chief of the Metabolism Branch 

and head of the Molecular Biology of 

Lymphoid Malignancies Section in 

NCI’s Center for Cancer Research. 

“However, if they are misdiagnosed and 

treated with the lower intensity chem

otherapy recommended for DLBCL, 

the survival rate is 20 percent or even less.” 

In a development that is literally life

saving, Dr. Staudt, who has been 

on the staff of NCI for nearly 20 

years, has successfully used genomic 

technology to reliably distinguish 

Burkitt’s lymphoma from DLBCL. 

Dr. Staudt demonstrated that DNA 

microarrays, a technology rooted in the 

­

­
Human Genome Project, can accurately distinguish Burkitt’s 

lymphoma from DLBCL, thereby optimizing the treatment 

choice for each patient. “The current goal with the improved 

diagnostics technology is to make it uniform, reproducible, 

and available as a cost-effective test,” notes Dr. Staudt. 

In collaboration with a private sector company, researchers 

are now enrolling 2,000 to 4,000 patients for a large study 

of a new test designed to distinguish between all types of 

lymphomas and other, benign conditions. 

Even as work continues on better diagnostics, Dr. Staudt 

points out that improved lymphoma treatments are also being 

discovered. Dr. Wyndham Wilson, a clinician at the NIH 

Clinical Center, has developed a novel, less toxic form 

of chemotherapy to treat Burkitt’s lymphoma, called 

dose-adjusted EPOCH-R. In this treatment, the dosage of 

chemotherapeutic drugs is increased with every cycle until 

a maximum tolerated dosage is reached. “It’s an empirical 

way of adjusting the dosage to fit each patient, which 

avoids both under treatment and over treatment,” says 

Dr. Staudt. “This approach adjusts for differences in 

metabolism, age and genetics that can influence a patient’s 

response to drugs.” 

I CN

With DNA microarrays (shown to scale above), researchers are 

, td

beginning to unravel the complexities and highlight the unique 

ua

genetic features of the 30-plus forms of lymphoma. 

tSs iuoL

Lymphoma Genomic Profiling 

The Lymphoma/Leukemia Molecular Profiling Project (LLMPP) is a network of 11 clinical groups that maintain biospecimens and clinical 

data from hundreds of patients with this cancer of the lymphatic system. An international consortium of investigators, including NCI-funded 

investigators and researchers around the world, the LLMPP is classifying lymphoma by distinct molecular characteristics and is then using 

that information to more accurately guide diagnosis and to target treatment. 

9 
I EmpowEring CanCEr 

rEsEarCh 

0 

h tlaeHf os etutitsnIl anoitaN

Howard A. Fine, M.D., Chief of the Neuro-Oncology Branch at 

NCI’s Center for Cancer Research 

dr. Howard Fine “sees as many patients as want to 

see me.” As the Chief of NCI’s Neuro-Oncology Branch, 

he evaluates between 2,000 and 3,000 brain tumor patients a 

year. Brain cancer, he says, “is a disease that impacts patients 

physically, while eroding their cognition and personality, 

exacting a devastating toll on both the patient and caretaking 

families.” Unfortunately, as Dr. Fine points out, “the current 

standard of care is not optimal.” The disease is not amenable 

to prevention or early diagnosis, and care for brain cancer 

patients falls between the expertise of neurology and medi

cal and surgical oncology. Dr. Fine is committed to changing 

that situation. 

Gliomas are the most common tumors that originate in the 

brain, beginning in the cells that surround and sup

port nerve cells. Patients with low-grade glioma, which 

tends to grow more slowly, have a survival rate that aver-

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ages 5 to 10 years, while patients with high-grade, or 

aggressive, glioma (glioblastoma being most common) have 

a 14-month survival rate. The standard treatment for glio

blastoma is surgery, radiation, and chemotherapy. Dr. Fine 

explains that glioblastomas are “hugely different” from patient 

to patient, which makes the case for a better understanding of the 

underlying genetic abnormalities that distinguish one type 

from another. Although several key genes have been shown to 

play a role in glioblastoma, and drugs that inhibit these targets 

have been developed, many other genes remain to be identified. 

Dr. Fine envisioned an integrated program to study large 

numbers of glioma patients—comprised of 20 NCI-designated 

Cancer Centers, the pharmaceutical industry, brain tumor 

consortia, clinical trial cooperative groups, and other NIH 

Institutes. His concept, the Glioma Molecular Diagnostic 

Initiative, or GMDI, collects and correlates standardized data 

from tumor specimens that can be analyzed to identify patient 

genetic profiles and novel molecular targets—and to help 

develop patient-tailored therapy. For Dr. Fine, efforts such 

as this “represent a flagship initiative that brings together 

divergent programs in a war that couldn’t be won otherwise.” 

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HARNESSING THE POWER OF NEW 

TECHNOLOGIES TO UNDERSTAND, DIAGNOSE, 

TREAT, AND PREVENT 

Building upon the successful completion of the Human 

Genome Project and applying its insights to cancer diag

nosis, treatment, and prevention will require new tools 

that empower and connect the entire cancer enterprise. In 

addition to traditional single investigator/laboratory projects, 

NCI supports large projects that involve multi-disciplinary 

and multi-institutional teams of scientists. Both types of 

projects play critical and interconnected roles towards a 

single goal: rapidly translating new findings into effective new 

clinical practices. 

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Diagnosing Brain Cancer 

NCI’s Glioma Molecular Diagnostic Initiative (GMDI) aims to develop a comprehensive molecular classification system for this type of brain cancer, 

thus allowing consistent prediction of response to therapies for individual patients. The largest study ever conducted to merge genetic and clinical 

information, GMDI is a nationwide partnership of several NIH Institutes and NCI-funded consortia and scientists. GMDI investigators are conducting 

retrospective studies, which look backward to examine exposures to suspected risk, and prospective studies, which watch for outcomes over a long 

period of time, to build and validate molecular and genetic models of glioma. The molecular, genetic, and clinical data from GMDI are being compiled 

in REMBRANDT (REpository for Molecular BRAin Neoplasia DaTa), a publicly accessible online database and universal classification system. 

10
GENETICS OF CANCER 

Deciphering the human genome se

quence was a watershed event early this 

decade, providing a fundamental blue

print for beginning to understand the 

genetic contributions to human health 

and disease, including cancer. Howev

er, what truly matters is to understand 

the effects of various differences on an 

individual’s risk of developing cancer, 

on drug effectiveness for their tumor, 

and on their susceptibility to side ef

fects. These genetic changes can also 

be used as markers for monitoring the 

progression of cancer. Although the 

technology for sequencing each person’s 

genome remains prohibitively expensive, 

the work of many scientists—including 

those at NCI—has resulted in a catalog 

of common genetic variations across 

the human population (The Inter

national HapMap) that can be used 

as “landmarks” to quickly identify 

genetic elements relevant to cancer 

and therapy. 

This new approach, known collectively 

as Genome-Wide Association Studies 

(GWAS), is resulting in a flood of data 

about the underlying molecular contri

butions to different forms of cancer and 

other diseases, and it is opening up excit

ing new opportunities for discovering 

and developing therapeutic interventions 

that go to the root causes rather than just 

the symptoms of cancer. 

NCI is leading the application of this 

exciting new technology, known as 

whole genome association studies, to 

cancer in several different initiatives, 

including: 

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The Cancer Genetic Markers of 


Susceptibility (CGeMS) Initiative: 


Initiated in 2005, CGEMS is a 

collaborative team of several research 

groups performing GWAS on the DNA 

of patients with prostate or breast cancer 

and comparing the results to those from 

patients without cancer. The results from 

this work are freely available to research

ers around the world to facilitate further 

study of the biological basis of cancer 

and also as the basis for new methods of 

diagnosis, intervention, and prevention 

of cancer. 

Patients 

Patient DNA 

Non-Patients 

e tutitsnIh craeseRe 

Non-Patient DNA 

moneGn amuHl anoitaN

Compare differences 

to discover 

SNPs associated 

with disease 

Disease-specific SNPS Non-disease-SNPS 

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GWAS Explained: How Are Genome-Wide 

Association Studies Conducted? 

To carry out a genome-wide association study, researchers analyze the DNA of two 

groups of participants: people with the disease being studied and similar people without 

the disease. Each person’s complete set of DNA, or genome, is placed on tiny chips and 

scanned on automated laboratory machines, which quickly survey each participant’s 

genome for strategically selected markers of genetic variation, which are called single 

nucleotide polymorphisms, or SNPs. 

If certain genetic variations are found more frequently in people with the disease 

compared to people without disease, the variations are said to be “associated” with the 

disease. The associated genetic variations can serve as powerful pointers to the region 

of the human genome where the disease-causing problem resides. 

However, the associated variants themselves may not directly cause the disease. They may 

just be “tagging along” with the actual causal variants. For this reason, researchers need 

to take additional steps, such as sequencing DNA base pairs in that particular region of the 

genome, to identify the exact genetic change involved in the disease. 

Genome-Wide Association Studies: Progress and Opportunities 

Genome-wide association studies involve rapidly scanning a collection of known genetic markers across the complete genomes of many 

people to find genetic variations that are associated with a particular disease. By using regularly spaced genetic markers across the 

genome, researchers can rapidly hone in on areas that are altered in people with a particular form of cancer, identify the specific genetic 

changes, and develop better strategies to detect, treat, and prevent the disease. 

11 
I EmpowEring CanCEr 

rEsEarCh 

The Cancer Genome Atlas (TCGA): TCGA, a joint 

effort by NCI and the National Human Genome Research 

Institute (NHGRI), is a coordinated research initiative to 

develop a comprehensive catalog, or atlas, of the many ge

netic changes that occur in cancers, from chromosome re

arrangements, to DNA mutations, to epigenetic changes 

(the chemical modifications of DNA that can turn genes 

on or off without altering the DNA sequence). The po

tential of a cancer genome “atlas” is tremendous, enabling 

detection of disease early when it is curable, distinguishing 

which patients will respond to therapy, providing new targets 

for drug development, and ultimately providing prevention 

strategies for people at risk for developing cancer. The TCGA 

Pilot Project is currently focused on three types of cancers: brain 

(glioblastoma multiforme), lung (squamous carcinoma), and 

ovarian (serous cystadenocarcinoma), which together account 

for more than 210,000 cancer cases each year in the United 

States alone. Data derived from these studies are rapidly made 

freely available for the use of cancer researchers everywhere. 

Childhood Cancer Therapeutically Applicable research 

to Generate effective Treatments (TArGeT) Initiative: The TARGET Initiative, a joint venture of NCI and 

the Foundation for NIH, is a public-private partnership 

to identify and validate therapeutic targets so that 

new, more effective treatments can be developed for 

children with cancer. Its immediate goal is to make 

major advances in identifying and validating therapeu

tic targets beginning with acute lymphoblastic leukemia 

and neuroblastoma. The TARGET initiative builds upon 

the experience and expertise NCI has gained in working 

with the NHGRI to build The Cancer Genome Atlas. 

TARGET will comprehensively characterize genomic 

profiles of selected childhood cancers and utilize DNA 

sequencing to identify those specific genes that are con

sistently altered in those cancers. Finally, high throughput 

screening methods that can rapidly identify active com

pounds, antibodies, or genes that modulate a particular 

biomolecular pathway will be applied to identify and 

validate therapeutic targets. 

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h tlaeHf os etutitsnIl anoitaN

Daniela S. Gerhard, Ph.D. 

Director, NCI’s Office of

Cancer Genomics 

Dr. Daniela Gerhard was appointed Director of the Office of 

Cancer Genomics at the National Cancer Institute in 2005. 

Among her responsibilities in overseeing many of NCI’s cancer 

genetics programs are her role as the project director of the 

Cancer Genetic Markers of Susceptibility (CGEMS) project, 

and serving as a member of The Cancer Genome Atlas (TCGA) 

management staff. She says, “For the first time, we have the 

tools to truly understand and address the fundamental causes 

of cancer, and make a profound difference in the lives of cancer 

patients everywhere.” 

Dr. Gerhard is a well-known human geneticist and molecular 

biologist. Her previous work at Washington University School 

of Medicine in St. Louis included identifying the key issues in 

the genetically complex bipolar affective disorder, the physical 

and genetic mapping of human chromosome 11, the cloning of 

the gene for multiple endocrine neoplasia, type 1 (located on 

chromosome 11), evaluation of genetic risk factors in prostate and 

cervical cancers, and the identification of a candidate region that 

harbors a gene relevant to cervical cancer development. 

1

UNDERSTANDING THE 

BIOLOGY OF CANCER 

Although understanding cancer genetics 

is critically important to our ability to 

diagnose, treat, and prevent cancer in 

all of its forms, the biological complexity 

of cancer extends far beyond its genetic 

changes, into changes in protein func

tion, structure, and metabolism that 

lead to cancer progression and 

metastasis. In order to develop a deeper 

understanding of these processes, 

NCI has undertaken several large 

multi-disciplinary team efforts to 

overcome obstacles to rapid prog

ress, including developing best 

practices for biospecimen handling, 

standardizing proteomic technologies 

for valid biomarker discovery, defin

ing the tumor microenvironment, and 

understanding cancer as a complex 

biological system. 

­

­

­

OVERCOMING OBSTACLES 

TO UNDERSTANDING BASIC 

CANCER BIOLOGY 

office of Biorepositories and 

Biospecimen research (oBBr): 

Almost all research into the molecular 

causes of cancer begins with tissue and 

DNA samples. In recent years, it has 

become clear to scientists that 

biorepositories with high quality bio

specimens and data are needed to 

enable this work. NCI’s OBBR is 

dedicated to providing leadership for 

biobanking activities that support all 

types of cancer research funded by the 

NCI. This is being done through a 

­

comprehensive approach to standard setting, biobanking science, and education 

with the aim of improving the quality of 

human biospecimens and biobanking 

operations nationally and internationally. 

The NCI Best Practices for Biospecimen 

Resources, issued in 2007, are a first 

attempt to standardize critical practices 

in biospecimen handling and banking 

in order to increase the meaningfulness 

and reproducibility of molecular data 

derived from experiments based on 

clinical samples from differing sources. 

Clinical Proteomic Technologies for 

Cancer Initiative: The study of the 

structure and function of proteins 

and their interactions are major cornerstones of cancer research. Evidence 

suggests that measurements of even small 

amounts of cancer-specific proteins and 

peptides could be reliable indicators of 

cancer initiation and progression. However, studies that have applied protein 

measurement technology—including 

mass spectrometry and affinity-based 

detection methods—to detect these 

potential “biomarkers” have not been 

as successful as anticipated, largely because of significant challenges in the 

technologies themselves. In order to 

address this need, NCI launched the 

Clinical Proteomic Technologies Initiative for Cancer, a cross-institutional and 

multi-disciplinary team approach that 

networks multiple research laboratories 

in 2006 to permit large-scale, real-time 

exchange and application of existing and 

newly developed protein measurement 

technologies, biological resources, and 

data dissemination; refine, standardize, 

and optimize technologies, reagents, 

methods, and analysis platforms that 

will ensure reliable and reproducible 

separation, capture, identification, 

quantification, and validation of 

protein measurements from complex biological mixtures; evaluate new 

technological approaches to separate 

and recognize proteins of significance 

related to the molecular and cellular 

events that occur during the process of 

cancer development. 

The Tumor Microenvironment 

Network (TMeN): Current cancer 

research reveals that tumors are not masses of cells developing independently, but 

function like organs composed of many 

interdependent cell types that contribute 

to tumor development and metastasis. 

Tumors and their surrounding cellular 

environment, collectively known as the 

stroma, evolve during tumor initiation 

and progression, and this interaction 

strongly affects the establishment and 

treatment of cancer. Evidence is emerging that critical stromal elements of the 

tumor are attractive targets for cancer 

prevention, because they primarily influencetumorcellsintheearlystagesofcancer 

development. The TMEN program was 

established to expand the understanding 

of the role of the microenvironment in 

which a tumor originates and the critical role it plays in tumor initiation and 

progression. TMEN is a multi-disciplinary network that includes pathologists, 

13 
EmpowEring CanCEr I rEsEarCh 

cancer biologists, cell biologists, oncol

ogists, and experts in bioengineering 

and bioinformatics. 

The Integrative Cancer Biology 

Program (ICBP): This effort focuses 

on the analysis of cancer as a complex 

biological system. The program brings 

clinical and basic cancer researchers 

together with researchers from 

mathematics, physics, information 

technology, imaging sciences, and 

computer science to work on key 

questions in cancer biology. The in

tegration of experimental biology with 

mathematical modeling will provide 

new insights in the biology and new 

approaches to the management of cancer. 

­

DEVELOPING THE 

ENABLING TECHNOLOGIES 

OF THE FUTURE 

NCI is exploring, developing, and 

sharing significant new cutting-edge 

technologies that will continue the 

transformation of our understand

ing of cancer and how to address it, 

and help usher in the cancer clini

cal practice of tomorrow. These 

efforts include constructing the first 

of its kind informational infrastruc

ture to link the diverse elements of the 

cancer community together in one united 

effort, exploring nanotechnology-based 

diagnosis and treatment possibilities, 

and finding new ways to image cancer at 

the subcellular level. 

­

­

­

­

­

NEW TECHNOLOGIES FOR 

CANCER RESEARCH 

Cancer Biomedical Informatics Grid 

(caBIGTM): As we begin to better under

stand cancer at the molecular level and 

personalized medicine becomes a reality 

in cancer patient care, researchers and 

clinicians will require more rapid access 

to—and easier methods to analyze and 

utilize—the multiple types of informa

tion involved. Earlier systems to help 

translate the necessary data into better 

patient outcomes were disconnected or 

underperforming. caBIGTM serves as the 

cornerstone of NCI’s biomedical in

formatics efforts to transform cancer 

­

­

­

1


research into a more collaborative, 

efficient, and effective endeavor. 

NCI launched caBIGTM to accelerate 

research discoveries and improve 

patient outcomes by linking research

ers, physicians, and patients throughout 

the cancer community. The caBIGTM 

community has already developed and 

released a variety of bioinformatics 

tools and capabilities that span the 

entire continuum of clinical research, 

pathology and genomics, and more are 

under development. These tools, though 

developed for the cancer research effort, 

are already widely applicable beyond the 

cancer community. 
The NCI Alliance for Nanotechnology 

in Cancer: NCI is engaged in efforts to 

harness the power of nanotechnology— 

the field of research that deals with the 

engineering and creation of things from 

materials that are less than 100 nanome

ters (one-billionth of a meter) in size— 

to radically change the way we diagnose, 

treat, and prevent cancer. The NCI Alli

ance for Nanotechnology in Cancer is a 

comprehensive, systematized initiative 

encompassing the public and private 

sectors, designed to accelerate the 

application of the best capabilities 

of nanotechnology to cancer. New 

nanotechnology approaches carry the 

potential to enable a new generation 

of targeted diagnostics, therapeutics, 

and preventives. Through the networked 

efforts of researchers at eight designated 

Centers of Cancer Nanotechnology 

Excellence, and the NCI’s Nanotechnolo

gy Characterization Laboratory (NCL) at 

its NCI-Frederick facility, NCI is helping 

build the cancer medicine of the future 

using technology developed today. 

­

­

­

TRANSLATING RESEARCH 

INTO CLINICAL PRACTICE: 

REDEFINING CLINICAL TRIALS 

The enormous potential for more 

“personalized” cancer treatment, coupled 

with the complexity of evaluating new, 

highly specific therapeutic agents and 

diagnostic tests, demands a national 

clinical trials enterprise that integrates 

the knowledge, insights, and skills of 

multiplefieldsintoanewkindofcross-disci

plinary, scientifically-driven, cooperative 

­

research endeavor. Creating such an 

endeavor, endorsed by cancer centers 

across the U.S., will require greater inte

gration of the successful, but functionally 

diverse, elements of the current clinical 

trials system supported by NCI. 

To address this goal, the Clinical Trials 

Working Group (CTWG) of the Na

tional Cancer Advisory Board (NCAB) 

developed a detailed blueprint for “Re

structuring the National Cancer Clinical 

Trials Enterprise.” The strategy developed 

by the CTWG focuses on leveraging the 

unique strengths of the entire current 

NCI-supported clinical trials enterprise 

to bring about a system that is more 

effective, efficient, and facile. 

This strategy specifically recognizes 

the role of NCI-designated Cancer 

Centers as a key institutional home 

for a large number of cancer clinical 

investigators, the strength of Special

ized Programs of Research Excellence 

(SPOREs) in disease-oriented trans

lational studies, the stable clinical 

trials infrastructure provided by 

the Cooperative Groups, and the 

ability of Community Clinical 

Oncology Programs (CCOPs) and 

other community oncologists to 

provide clinical trials in a local 

environment. The proposed restruc

turing preserves and strengthens all of 

the existing components of the NCI 

clinical trials system and enhances their 

ability to work together in fundamentally 

different ways. 

­

­

­

­

­

­

The strategy developed by the CTwG 

addresses four important goals: 

• Enhance coordination and cooperation 

by ensuring that comprehensive infor

mation on cancer clinical trials is readily 

available for all, that collaborative team 

science as well as individual achieve

ments are rewarded, and that NCI clini

cal trials are effectively coordinated with 

federal regulatory systems. 

• Enhance scientific quality and pri

oritization so that NCI supports the 

best-designed trials that address the most 

important questions, thereby leveraging 

significant scientific advances. 

• Enhance standardization of tools 

and procedures for trial design, data 

capture, data sharing, and administrative 

functions to decrease effort and mini

mize duplication. 

• Enhance operational efficiency by 

increasing the rate of patient accrual 

and reducing operational barriers so 

that trials can be conducted in a timely, 

cost-effective manner. In order to oversee 

implementation of the recommendations 

of the CTWG, an extramural advisory 

committee, the Clinical Trials Advisory 

Committee (CTAC), has been chartered. 

In addition, NCI has developed a coordi

nated, internal organizational structure to 

manage the entire clinical trials enterprise 

supported by the Institute. The Clinical 

Trials Operations Committee (CTOC) 

provides strategic oversight of the NCI’s 

clinical trials enterprise while the Coordi

nating Center for ClinicalTrials (CCCT) 

manages the implementation of the 

CTWG initiatives. 

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15 
I EmpowEring CanCEr 

rEsEarCh 

Locations of NCI-Designated Cancer 

Centers in the United States. 

Comprehensive Cancer Centers Cancer Centers Planning Centers 

NCI-Designated Cancer Centers 

The NCI’s Cancer Centers Program is a critical component of the 

Institute’s effort to provide infrastructure and manpower support for 

NCI-sponsored extramural science. The Cancer Centers Program 

supports 63 NCI-designated Cancer Centers nationwide that are 

actively engaged in transdisciplinary research to reduce cancer 

incidence, morbidity, and mortality. The NCI-designated Cancer Cen

ters are a major source of discovery of the fundamental nature 

of cancer and serve as a key mechanism for translating those 

discoveries to patient care. These Centers, as well as other cancer 

centers throughout the country, deliver medical advances to patients

and their families, educate healthcare professionals and the public, 

and reach out to underserved populations. 

­

The NCI-designated Cancer Centers are directed by some of the 

finest leaders in the cancer community. The Cancer Center Directors 

have spent much of the last year examining the opportunities that 

exist to further the fields of cancer prevention, early detection, 

cancer treatment, and survivorship. They have identified areas 

for collaboration and areas of particular need. Their recommenda

tions are outlined in the report, “Accelerating Successes Against 

Cancer” and serve as a valuable resource to the cancer research 

community and the NCI. Their recommendations have been 

incorporated into our overall strategic plan and are reflected in 

our need to expand certain areas of research. 

­

Implementing the recommendations of 

the CTWG will require considerable 

effort by all stakeholders as well as 

financial investment on the part of NCI. 

This renewed commitment and the associated resources required are crucial 

for ensuring that the large, ongoing 

national investment in cancer clinical 

trials is effective and efficient in bringing effective new therapies to patients. 

By embracing this restructuring, NCI, 

cancer centers, and the oncology research 

community will be positioned to ensure 

that the advances in understanding the 

biological basis of cancer, generated by 

the past 40 years of research, are harnessed 

effectively to bring measurable, meaningful benefits to patients today and in 

the future. 

Building on the plans outlined by the 

CTWG, NCI recently completed a 

two-year examination of early translational research and the opportunities 

and barriers that exist. The Translational 

Research Working Group (TRWG), 

a working group of the NCAB, was 

established to conduct a discussion with 

the broader cancer research community 

to develop recommendations about how 

NCI can best organize its investment to 

further translational research. The recommendations of this group were received 

by NCI in June, 2007, and the Institute 

is working on an implementation plan 

that integrates these activities with the 

infrastructure and support being created 

to support the CTWG. 

1

A genomic health scientist reviews a slide to insure the tissue 

sample is suitable. 

Genomic Healthwww.genomichealth.com/pressroom/downloadablefiles.aspx 

DISCOVERING AND DEVELOPING THE NExT 

GENERATION OF EFFECTIVE AND EFFICIENT 

CANCER THERAPIES 

NCI is not content to only understand the biology of cancer. 

NCI researchers are working tirelessly to find novel cancer 

treatments that do not harm healthy tissue, particularly for 

those types of cancer that are not being addressed by the 

private sector. We seek to find and evaluate such therapies, 

and then to develop collaborations with the private sector that 

will ensure that the therapies progress quickly to regulatory 

approval and clinical practice. To facilitate application, the 

NCI-designated Cancer Center Directors’ report recommends 

enhanced collaborations among national organizations such 

as Centers for Disease Control and Prevention, Centers for 

Medicare and Medicaid Services, Health Resources and 

Services Administration, and Agency for Healthcare 

Research and Quality as key to successful dissemination of 

best practices for cancer care to reduce treatment heterogeneity 

or suboptimal care. 

At the heart of NCI’s internal efforts to find and develop 

new cancer drugs is the NCI’s Developmental Therapeutics 

Program (DTP). In keeping with its goal to turn 

molecules into medicine for the public health, DTP, 

created by Congress in 1955 as the Cancer Chemotherapy 

National Service Center, serves as a vital resource in 

acquiring preclinical information and providing 

research materials, including vialed and plated compounds, 

tumor cells, animals, and bulk drugs for investigational new 

drug (IND)-directed studies. DTP has been involved in the 

discovery or development of more than 70 percent of the 

anticancer therapeutics on the market today. Successes include 

paclitaxel (Taxol®), one of the most widely prescribed anticancer 

drugs on the market, and bortezomib (Velcade®). 

Paclitaxel, a natural product, was first harvested by researchers 

working under a joint U.S. Department of Agriculture-NCI 

grant. It was a DTP contractor who formulated the drug for 

use in clinical trials. 

In cooperation with the commercial sponsor, bortezomib 

was screened and formulated by DTP. Approved by the 

FDA in 2003, it remains the first treatment in more than a 

decade to be approved for patients with multiple myeloma. 

Although many academic and private-industry laboratories also are focused on drug discovery, NCI sees an 

opportunity to mediate novel academic-industry partnerships 

to overcome financial and technical barriers that may keep 

promising therapeutics from reaching patients. For example, 

a new direction for therapeutic drug development, made 

possible by the FDA Exploratory IND Guidance, is the 

Phase 0 clinical trial. These early studies are designed 

to determine if a drug shows favorable biodistribution, 

mechanisms of action and/or binding characteristics. 

These determinations are made in a limited number of 

patients using microdoses of the drug under investigation 

enabling “go, no-go” decisions earlier in the drug development 

process. NCI is developing these protocols, in many cases 

with industry partners, expediting the development and testing 

of promising therapeutics for cancer. 

Clinical Trials: Optimizing Breast Cancer Treatment 

The Trial Assigning IndividuaLized Options for Treatment (Rx), or TAILORx, was launched in May, 2006. Currently, the majority of women 

with early-stage breast cancer are advised to receive chemotherapy in addition to radiation and hormonal therapy, yet research has not 

demonstrated that chemotherapy benefits all of them equally. TAILORx seeks to incorporate a molecular profiling test into clinical decision 

making, and thus spare women unnecessary treatment if chemotherapy is not likely to be of substantial benefit. TAILORx is one of the first 

trials to examine a methodology for personalizing cancer treatment, and will ultimately enroll over 10,000 women at 900 sites in the United 

States and Canada. TAILORx is sponsored by the NCI, and is coordinated by the Eastern Cooperative Oncology Group (ECOG). 

17 
I EmpowEring CanCEr 

rEsEarCh 

Estimated Number Cancer Survivors in the United States from 1971 to 2007 

12 11,941,043 

11 

10 

Numbers in Millions 

9 

8 

7 

6 

5 

4 

3 

2 

1 

0 

1971197219731974197519761977197819791980198119821983198419851986198719881989199019911992199319941995199619971998199920002001200220032004200520062007 

Year 

Data sources: NCI SEER November 2006 Submission, U.S. Estimated Prevalence counts were estimated by applying U.S. populations to SEER-9 and historical 

Connecticut Limited Duration Prevalence proportions. Populations from January 2004 were based on the average of the July 2003 and July 2004 population 

estimates from the U.S. Bureau of Census. 

1 

2004 Erbitux® (NSC 632307) 

2003 Velcade® (NSC 681239) 

1998 Ontak® (NSC 697979) 

1996 Gliadel® (NSC 714372) 

Topotecan (NSC 609699) 

1995 All-t-retinoic acid (NSC 122758) 

1992 Chorodeoxyadenosine (NSC 105014) 

Taxol® (NSC 125973) 

Teniposide (NSC 122819) 

1991 Fludarabine Phosphate (NSC 312887) 

Pentostatin (NSC 218321) 

1990 Hexamethylmelamine (NSC 13875) 

Levamisole (NSC 177023) 

1989 Carboplatin (NSC 241240) 

1988 Ifosfamide (NSC 109724) 

1987 Mitoxantrone (NSC 301739) 

1983 Etoposide (NSC 141540) 

1982 Streptozotocin (NSC 85998) 

1979 Daunorubicin (NSC 82151) 

1978 cis-Platinum (NSC 119875) 

1977 BCNU (NSC 409962) 

1976 CCNU (NSC 9037) 

1975 Dacarbazine (NSC 45388) 

1974 Doxorubicin (NSC 123127) 

Mitomycin C (NSC 26980) 

1973 Bleomycin (NSC 125066) 

1970 FUDR (NSC 27640) 

Mithramycin (NSC 24559) 

o-p’-DDD (NSC 38721) 

1969 Ara-C (NSC 63878) 

Procarbazine (NSC 77213) 

1967 Hydroxyurea (NSC 32065) 

1966 Pipobroman (NSC 25154) 

Thioguanine (NSC 752) 

1964 Melphalan (NSC 8806) 

Actinomycin D (NSC 3053) 

1963 Vincristine (NSC 67574) 

1962 Fluorouracil (NSC 19893) 

1961 Vinblastine (NSC 49842) 

1959 Cyclophosphamide (NSC 26271) 

Thiotepa (NSC 6396) 

1957 Chlorambucil (NSC 3088) 

19 

Approved Cancer Treatment Drugs Developed with DTP Involvement 
II. reaching all communities toucheD 

by cancer. Although the sheer complexity of cancer itself remains 

our most significant challenge, it is almost matched by the heterogeneity 

of the actual practice of cancer medicine across our nation. Part of NCI’s 

mandate is to disseminate new knowledge and best practices, as well as 

clinical trial opportunities, to physicians and patients, regardless of location 

or social standing. It is a charge we take seriously, and we are making 

significant progress. In addition, we know that progress will continue to be 

made only if we make a concerted effort to identify, nurture, and train the 

next generation of talented researchers and clinicians. 

THE NEED FOR 

RESEARCH ON ACCESS 

TO CANCER CARE 

Although we are rapidly gaining new 

knowledge about cancer, we must 

get that new knowledge—the latest 

science—to patients. NCI maintains a 

network of 63 Cancer Centers, which 

are principally located at our country’s 

premier research universities. Thirtynine of those facilities, which conduct 

research and provide state-of-the-art 

cancer care, are designated as Compre

hensive Cancer Centers. Yet, we are also 

aware that only approximately 15 per

cent of cancer patients are treated in 

these centers, with the great majority 

of the nation’s cancer patients being 

treated in the communities where they live 

­

­

and work. 

To address this significant access 

problem, NCI launched the NCI 

Community Cancer Centers Program 

(NCCCP) earlier this year, which is 

designed to study ways to bring new 

discoveries closer to the home of each 

cancer patient. The NCCCP, now in a 

three-year pilot phase, will encourage 

the collaboration of private-practice 

medical, surgical, and radiation oncologists 

and provide close links to NCI research 

and to the network of NCI-designated 

Cancer Centers. The pilot program will 

also study new and enhanced ways to 

assist, educate, and treat the needs of 

underserved populations—including 

elderly, rural, inner-city, and low-income patients—as well as racial and 

ethnic groups with unusually high 

cancer rates. Through this program, we 

will also learn how best to educate 

patients concerning cancer risk, healthier 

living, screening practices, clinical trial 

participation, survivorship, and end-oflife issues. 

The NCCCP pilot will also study ways 

to bring early-phase clinical research 

to the community setting, along with 

emphasis on the standardized collection 

of biological specimens for research and 

the institution of electronic medical 

records. The NCCCP is designed to work 

in concert with other initiatives, in new 

settings, and with greater benefits for 

more patients. 

19 
rEaChing all CommunitiEs II touChEd by CanCEr 

National Institutes of Health 

“Mindful of our mission to 

conduct research in all areas 

of science—including the 

behavioral sciences, 

such as how best to provide 

patient education and access 

to optimal care—NCI is 

launching the pilot phase 

of the national Community 

Cancer Centers Program 

that, if fully implemented, 

will help bring state-of-theart cancer care to patients 

in community hospitals 

across the United States.” 

Dr. John Niederhuber 

Director, National Cancer Institute 

Hearing Senate Subcommittee on 

Labor-HHS-Education Appropriations 

May 21, 2007 

ONGOING OUTREACH 

INITIATIVES 

The Community Clinical Oncology 

Program (CCOP) network enables the 

participation by community physicians 

in national clinical trials studies. Participating in these trials makes it easier for 

community physicians to more quickly 

put successful regimens into practice. In 

fact, over its 23 years of operation, the 

CCOP, with more than 4,000 physicians 

participating at over 400 hospitals, has 

enrolled more than 200,000 people in 

treatment and prevention trials. 

COMMUNITY-BASED 

INTERVENTIONS TO REDUCE 

CANCER HEALTH DISPARITIES 

NCI’s commitment to research that 

improves the quality of cancer care is 

a theme that runs through all of the 

Institute’s research. Indeed, we still have 

much to learn about why some fare better with cancer than others, why some 

racial and ethnic groups have higher incidences of cancer than others, and how 

to assist those who may lack language 

capacity, mobility, or even the knowledge 

to navigate our healthcare system. 

Up Close with a CCOP Patient 

Sue Duyser was leading a busy life in Grand Rapids, Mich., running a pharmacy with her husband. Then she found a lump in her breast. 


Her doctors initially thought the lump was a cyst. “I was so busy, I let it go,” says Ms. Duyser. Six months later, a friend who is a breast cancer 


survivor urged her to follow up with her doctors, and Ms. Duyser was found to have a late-stage cancer.


While extended family knew oncologists at the University of Texas M . D . Anderson Cancer Center, travel was not an option. Ms. Duyser is 


the primary caregiver for her husband, who has multiple sclerosis and uses a wheelchair. Networking through her community, she found the 


Grand Rapids Clinical Oncology Program, a CCOP site. There she found Dr. Marianne Lange, a dedicated physician willing to go the “extra 


mile” to get patients the best possible care. Ms. Duyser joined an NCI-sponsored clinical trial and though the treatment took many long and 


difficult months, Ms. Duyser knew she “was in good hands with the CCOP doctors” and was thankful to be able to be home with her husband, 


family, and friends. She is, today, a disease-free, 10-year breast cancer survivor. 


0

Two community-based programs 

of note—the Community Networks 

Program and the Patient Navigator 

Program—are further proof of that 

commitment to all of our citizens. 

The Community Networks Program 

(CNP), which began in May, 2005 as 

a follow-up to our successful Special 

Population Networks program, works 

toward the reduction of disparities in 

cancer care, prevention, and survivorship 

through community-based participatory education, training, and research 

among racial and ethnic minorities 

and underserved populations. To date, 

NCI has awarded $95 million in 

5-year grants to 25 institutions located 

in regions of the United States where 

the cancer burden is disproportionately high. Most sites have formed 

clinical partnerships that provide 

prevention services, including tobacco 

cessation, diet and exercise programs, 

and screening services for breast, 

prostate, colorectal, and cervical 

cancer. More than 640 partnerships 

with major cancer collaborators, such 

as the American Cancer Society, Susan 

G. Komen For the Cure Foundation, 

the National Urban League, state and 

local health departments, clinics, and 

hospitals, as well as regional offices of 

NCI’s Cancer Information Service, 

help to build a strong and sustainable 

community-based infrastructure. 

NCI’s Patient Navigation Research 

Program (PNRP) focuses on developing 

and testing interventions to promote 

cancer awareness within communities 

and to help patients and their families 

manage cancer diagnoses and overcome 

common barriers to obtaining timely 

and appropriate cancer care and treatment. The primary participants for this 

research program are those experiencing 

cancer health disparities, encompassing racial/ethnic minorities, individuals 

with lower socioeconomic status, and 

residents of rural areas. PNRP captures valuable data for comprehensive 

evaluations of the effectiveness of its 

interventions in reducing cancer 

health disparities. For example, the 

program analyzes patient and navigator demographics and data on access to 

screening, diagnosis, and treament; 

quality of life; patient satisfaction; and 

cost effectiveness. 

Health Disparities in Estrogen Receptor (ER) 

Negative Breast Cancer 

1 

About two-thirds of all breast cancer 

cells are ER-positive, meaning the 

tumors contain significant levels of 

receptors for the female hormone estro

gen. The other third of breast cancers, 

ER-negative cancer, tend to grow more 

aggressively; they are more likely to 

metastasize, and are associated with a 

worse prognosis. Researchers suspect 

that the higher proportion of ER-negative 

tumors found in African American breast 

cancer patients, compared with white 

patients, may contribute to the higher 

­

breast cancer mortality rates seen in the 

African American population. Research 

to improve outcomes for patients with 

ER-negative breast cancer is an impor

tant strategy for addressing this health 

disparity. NCI is supporting identification 

and exploration of the basic biologic 

attributes of ER-negative breast cancer, 

including molecular mechanisms that 

can be targeted with prevention, early 

detection, and treatment interventions. 

­

Two-thirds of breast cancer cells express the estrogen receptor (right), 

and one third—which tend to grow more aggressively—do not (left). National Institutes of Health 

National Institutes of Health 

Cancer.gov en Español 

More and more Americans are 

benefiting from advances in early 

detection and treatment of cancer. 

However, many Hispanics and Latinos 

in the United States have not heard 

this message and are unable to take 

full advantage of these options. 

By 2050, it is estimated that Latinos 

will make up a quarter of the U.S. 

population. National efforts to control 

cancer must include interventions and 

information directed at this group. 

NCI’s Spanish-language Web site 

(http://www.cancer.gov/espanol) 

strongly communicates the message 

that cancer can be prevented and 

treated, in addition to offering informa

tion on all aspects of the disease. 

The site is tailored to meet the needs 

of Latinos who seek cancer information 

online. Rather than simply translating 

the English version of cancer.gov, 

the site was designed specifically for 

this audience. Cancer.gov en Español 

currently contains pages on different 

types of cancer, more than 100 peerreviewed cancer treatment summaries 

for health professionals and patients, 

and a dictionary that includes 5,000 

terms and definitions in both Spanish 

and English. NCI will continue to test 

and update the site to ensure that it 

meets the information needs of 

Spanish speakers. 

­
II rEaChing all CommunitiEs 

touChEd by CanCEr 

National Institutes of Health 

Members of the NCI Minority Institute/Cancer Center Partnership Program 

Life is different 

after cancer— 

emotionally and 

physically. Remembering 

that cancer is a process,

 and not an event, 

NCI leads the nation’s 

research in cancer 

survivorship. 

CANCER SURVIVORS 

Life is different after cancer—emotion

ally and physically. Remembering that 

cancer is a process, and not an event, 

NCI leads the nation’s research in 

cancer survivorship. The NCI-designated 

Cancer Center Directors’ report identi

fied survivorship as a major theme and 

recommended increased support to 

understanding all factors that affect a 

cancer patient’s response to their disease 

and treatment. NCI researchers report 

that nearly one in four people diag

nosed with cancer has participated in 

a support group. Furthermore, use of 

support groups varies considerably by 

cancer type, and few survivors receive 

physician referrals to such programs. 

Studies to show the value of such as

sistance may help clinicians recognize 

the importance of support groups for 

cancer patients. 

­

­

­

­

BUILDING PARTNERSHIPS 

The NCI Minority Institution/Cancer 

Center Partnership (MI/CCP) brings 

together NCI-designated Cancer Cen

ters and Minority-Serving Institutions 

(MSI)—colleges and universities that are 

committed to the special encouragement 

of students from ethnic minority groups, 

including African Americans, Hispanic/ 

Latino Americans, American Indians, 

Alaska Natives, and American Pacific 

Islanders. In addition to training, this 

collaboration focuses on reducing the 

disproportionate cancer incidence and 

mortality in minority populations. MSI 

and Cancer Center investigators are 

collaborating on more than 120 research 

projects. One example is the partnership 

between Nashville’s Meharry Medical 

College—the nation’s largest private, 

independent historically black institution 

dedicated solely to educating health pro

fessionals—and the Vanderbilt-Ingram 

­

­



Cancer Center. Through their alliance, 

the two institutions are learning from 

each other and advancing student educa

tion, patient care, and research. 

­

RESEARCH TRAINING AND 

CAREER DEVELOPMENT 

As one way to continue the advance

ment of the entire cancer community, 

NCI is committed to training a future 

generation of American and interna

tional researchers dedicated to reducing 

cancer incidence, mortality, and suffering. 

Cancer researchers in the decades 

ahead will face a new landscape of 

challenges and opportunities. For this 

reason, NCI continually adapts its train

ing programs to accommodate rapid 

developments in the frontiers of science 

and technology. 

NCI devotes approximately four per

cent of its annual budget to institutional 

and individual research training and 

career development-related grants and 

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programs. This investment provides 

support for scientists throughout their 

careers. There has been an emergence 

of new disciplines, changes in how 

cancer patients are treated, and an 

increase emphasis on communicating 

research-based information on cancer. 

NCI annually funds over 2,200 research 

training and career development awards. 

This number, however, represents only 

a portion of the training effort which 

is also supported in SPORE grants, 

the intramural research program and 

throughout the Research Project Grant 

(RPG) pool. 

NCI uses these diverse training and development resources to provide support 

for individuals, from graduate students 

to established investigators, who needprotected time away from administrative 

responsibilities to expand their research 

programs and mentor junior investigators. 

These investments will ensure a steady 

flow of well-trained investigators to focus 

on the challenges of fighting cancer and 

ultimately increase the diversity of the 

cancer research workforce. 

A GLOBAL OBLIGATION 

Cancer is not just a U.S. domestic 

issue. Each year more than 11 mil

lion people are diagnosed with cancer 

and more than 6 million die world

wide. That toll is greater than deaths 

from AIDS, tuberculosis, and ma

laria combined. NCI’s international 

efforts are designed to foster research 

and build research capacity globally. 

NCI funds foreign contracts and grants, 

develops partnerships with other nations 

and organizations, and provides training 

opportunities for American scientists 

abroad and foreign scientists in the 

United States. NCI remains commit

ted to supporting and expanding its 

international research efforts. 

Dr. Grace Butler 

Dr. Grace Butler does not just talk about 

cancer; she has lived it. Dr. Butler, a 

professor emeritus at the University of 

Houston, is a survivor of colorectal cancer 

who founded the non-profit organization 

called Hope Through Grace in 2002. The 

organization covers the cost of colon cancer 

screening for uninsured and underinsured 

populations and provides cancer awareness 

programs at shelters, churches, and 

higher education institutions, emphasizing 

underserved communities, including 

senior survivors of Hurricane Katrina who 

relocated to the Houston area. 

Dr. Butler also freely gives her time to 

the service of all cancer patients. She 

is a member of both the NCI’s Director’s 

Consumer Liaison Group and the Consumer 

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Advocates in Research and Related 

Activities (CARRA) program. Dr. Butler also 

attends important NCI meetings, such as 

the annual cancer health disparities summit, 

where she is an avid participant, sharing 

her passion for a healthcare system that 

reaches all cancer patients. In January 2007, 

President Bush visited the Clinical Center 

on the NIH campus in Bethesda, Md. He 

toured laboratories, met with patients, and 

conducted a roundtable discussion on the 

state of cancer research and treatment. 

Grace Butler was at that table too, 

representing those who have suffered with 

cancer and survived, determined to do 

something to help others. 

3 

h tlaeHf os etutitsnIl anoitaN

Grace Butler, Ph.D., and President George W. Bush at NCI 
II rEaChing all CommunitiEs 

touChEd by CanCEr 

Highlights of NCI’s international 

efforts include: 

Partnerships. The Middle East Cancer 

Consortium (MECC) includes Cyprus, 

Egypt, Israel, Jordan, the Palestinian 

Authority, and Turkey. Over the past 

decade, MECC has supported popu

lation-based cancer registries. NCI 

also acts as a sustaining partner of the 

Breast Health Global Initiative, which 

strives to develop, implement, and study 

guidelines to improve interventions 

for breast cancer in countries with 

limited resources. 

Research. A global approach to research 

enables explorations of interactions 

between genes and specific environ

mental exposures found in diverse 

geographical settings. With the Chi

nese Center for Disease Control, NCI 

researchers have evaluated cancer risk 

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associated with occupational exposure to benzene. The results of these 

studies were considered by the U.S. 

Environmental Protection Agency (EPA) 

in establishing a rule to limit the benzene 

content in gasoline, significantly reducing 

emissions of hazardous air pollutants in 

the United States. NCI-funded U.S. 

and Turkish researchers have studied 

malignant mesothelioma (MM), which 

accounts for 50 percent of all deaths in 

certain Turkish villages and has been 

linked with use of erionite, a carcinogenic 

mineral fiber. The researchers found that 

only some families suffer from a high 

incidence of the disease, because they 

possess a genetic predisposition that 

influences the development of MM. 

In addition to contributing to prevention 

and treatment for MM in Turkey, these 

studies also may improve prevention 

and treatment in the United States, 

where about 2,000 new cases of MM, 

primarily attributable to asbestos exposure, are diagnosed each year. 

Cancer researchers in the 

decades ahead will face 

a new landscape of 

challenges and opportunities. 

For this reason, NCI 

continually adapts its 

training programs to 

accommodate rapid 

developments in the frontiers 

of science and technology. 

Preparing The Next Generation 

Dr. Sapna Syngal is an overachiever. 

She trained in internal medicine and 

gastrointestinal cancer, she focuses on the 

study of genetics, screening, and primary 

prevention of gastrointestinal tumors, 

primarily colorectal cancer. An assistant 

professor at the Harvard Medical School 

and a researcher at Dana-Farber Cancer 

Institute, Dr. Syngal sees patients, teaches 

postdoctoral students, and conducts cancer 

prevention research. 

In 2000, Dr. Syngal received a Mentored 

Research Career Development Award. 

Her research to help reduce cancer incidence 

and mortality in individuals at high risk of 

colon cancer resulted in several other 

grants investigating genetic mutations 

in colorectal cancers. 

In 2005, Dr. Syngal won “protected time” 

through an NCI-supported Mid-Career 

Investigator Award, which freed her from 

administrative, teaching, and clinical 

duties so she could focus on mentoring 

young clinician investigators in patientoriented research In 2005, Dr. Syngal 

worked with 14 postdoctoral clinicians, 

while continuing to further her research. 

This outstanding clinical researcher is 

just one example of the people NCI and 

the NIH are honored to support through 

unique career development opportunities. 

Sapna Syngal, M.D., M.P.H., is currently the Director 

of the Brigham and Women’s Hospital/Dana-Farber 

Cancer Institute Familial Gastrointestinal Cancer 

Program. 

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na

n 

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a

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ra

y 

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-

o 

a 

t

n

o

a

h

D

 P
global cancer inciDence anD mortality Global Cancer Incidence and Mortality 

Iceland 

1,000 / 500 

Sweden 

Norway 43,000 / 22,000 

21,000 / 11,000 

Japan Canada 521,000 / 311,000 

138,000 / 66,000 

Estonia 

Ireland 5,000 / 3,000 

13,000 / 8,000 Germany

United States of 408,000 / 218,000 

America	

1.4M / 566,000 

United Kingdom

277,000 / 156,000 

China


2.2M / 1.6M
 Austria 

37,000 / 19,000 Korea

109,000 / 62,000 France 

269,000 / 149,000 Switzerland 

35,000 / 17,000 

7.6 million


people died of

cancer in 2005 

(out of 58 million deaths) 

Republic of


Singapore


10,000 / 6,000 Australia


86,000 / 37,000

> 70% in low and


middle income


countires Cancer Incidence / Mortality per year


Source: International Agency for Research on Cancer, GLOBOCAN database 

NCI’s international efforts are 

designed to foster research and build 

research capacity globally. NCI funds 

foreign contracts and grants, develops 

partnerships with other nations 

and organizations, and provides 

training opportunities for American 

scientists abroad and foreign scientists 

in the United States. 

5

II rEaChing all CommunitiEs 

touChEd by CanCEr 

Getty Images 

Air pollution, a major contribution to cancer risk, in Shanghai, China 

Cancer is not just a U.S. 


domestic issue. Each year 


more than 11 million people 


are diagnosed with cancer 


and more than 6 million 


die worldwide.


 
III. the promise of prevention anD 

early Diagnosis. Despite many decades of investigation and 

progress made in early diagnosis and treatment, the exact causes of most 

cancers remain unknown. For most of the cancers we treat, there exists a mix 

of genetic changes and numerous environmental influences that challenge 

the development of simple prevention strategies. NCI’s approach to cancer 

prevention is defined by the use of advanced tools and technologies—such as 

those employed in genomics, proteomics, and metabolomics—to dissect the 

molecular events associated with the molecular mechanisms and early signs 

of cancer development. 

Anew era of cancer prevention 

will require understanding 

genetic alterations, both those 

we are born with and those we accumulate throughout our lifetime, which 

can alter protein expression patterns 

and cellular function. These events will 

be further impacted by the need to 

understand environmental exposures 

and lifestyle factors. All of these layers 

point to the difficulty of finding effective, nontoxic preventive agents. Part of 

the solution involves identifying biomarkers of risk, as well as genome-wide 

association studies. For example, NCI’s 

Early Detection Research Network is 

supporting researchers who are making 

important inroads in this area, including, promising preliminary work on 

an early detection assay for pancreatic 

cancer. Despite the complexity, NCI’s 

extensive cancer prevention research 

program has generated some remarkable 

success stories, including the dramatic 

drop in smoking rates over the past 

two decades, as well as the approval of 

tamoxifen and raloxifene for the prevention of breast cancer. Thus, we continue 

to support research into lifestyle and 

environmental factors that influence 

cancer risk. These efforts include antiobesity programs aimed at minorities 

conducted under the auspices of the 

Transdisciplinary Research on Energetics in Cancer (TREC) program as well 

as the development of new chemopreventive agents. 

Prevention is, and will continue to be, 

an integral part of reducing cancer’s 

burden. Identifying and quantifying an 

individual’s risk of cancer and providing 

a tailored prevention strategy will 

be the cornerstones of a truly effective 

cancer prevention effort. NCI is actively 

working toward that goal. 

7

III thE promisE of prEvEntion 

and Early diagnosis 

UNDERSTANDING THE RISK 

OF DEVELOPING CANCER: 

THE NCI COHORT 

CONSORTIUM 

The study of genetic and environmen

tal risk factors for cancer has advanced 

considerably due to our investment in 

population studies. Researchers are us

ing new genomic technologies, pooling 

resources, and sharing findings from 

large-scale studies, which are identify

ing gene variants that affect cancer risk, 

diagnosis, and prognosis. Underpinning 

the success of these studies is a long-term 

investment in the NCI Cohort Consor

tium. Comprised of 32 separate study 

groups including 4 million people, this 

cooperative, international endeavor col

lects biological specimens and associated 

risk factor data from a diverse group of 

populations. It provides a coordinated, 

interdisciplinary approach to tackling 

important scientific questions, while 

affording opportunities to quicken the 

pace of research and work across a col

laborative network of investigators. 

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NEW PREVENTIVE THERAPIES: 

EARLY PREVENTION CLINICAL 

TRIALS CONSORTIA 

To assess the cancer prevention prop

erties of promising drugs and other 

agents, NCI is funding six chemopre

vention research consortia. These groups 

will design and conduct Phase I and 

Phase II clinical trials, which determine 

safety and efficacy. The ultimate goal is 

to identify agents and strategies that 

can be tested in larger Phase III trials to 

determine whether a drug meets criteria 

for FDA approval. Consortia researchers 

will study the effects of these agents on 

molecular targets of cancer prevention, 

as well as on other biological events 

associated with cancer development, 

such as cell proliferation, cell death, the 

expression of growth factors, and the 

development of genes that cause cancer. 

Researchers will also correlate effects 

of the agents with clinical outcomes 

of trials, including survival and disease 

progression. The first 18 early-phase 

chemoprevention trials are now accruing 

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patients and the consortia are developing 

17 more trials. 

TOBACCO AND TOBACCO

RELATED CANCERS 

Tobacco use and exposure to tobacco 

smoke are the leading preventable 

causes of illness and death in the United 

States. Each year, more than 440,000 

Americans die of tobacco-related disease, 

or one in every five deaths. Cigarette 

smoking is responsible for more than 

30 percent of all cancer deaths annually 

in the United States. According to 

the World Health Organization, cut

ting tobacco consumption in half 

by the year 2020 would prevent 

approximately 180 million tobaccorelated deaths around the globe. 

NCI works with national and inter

national partners, to understand 

the etiology of tobacco use, prevent 

initiation, develop and promote 

effective smoking cessation inter

ventions, and improve survival of 

patients with tobacco-related cancers by 

developing earlier, more accurate diag

nostic techniques and targeted therapies. 

For example, NCI-supported researchers 

investigate the biologic, genetic, and 

behavioral foundations of nicotine 

addiction and tobacco cessation; the 

causes of tobacco-related cancers; and 

the genetic and genomic factors affecting 

patient response to treatment and 

disease progression. NCI is commit

ted to translating findings from this 

research into evidence-based prevention, 

cessation, and treatment interventions. 

NCI plans to broaden initiatives to 

examine the complex social, behavioral, 

environmental, biological, and genetic 

factors that influence tobacco use and 

tobacco-related cancers, including in

ternational consortia to address tobacco 

consumption in lower income nations 

Obesity and Cancer 

The Transdisciplinary Research on 

Energetics and Cancer (TREC) integrates 

the study of diet, weight, and physical 

activity and their effects on cancer 

by focusing on energy balance and 

energetics (the study of the flow and 

transformation of energy through living 

systems). NCI currently funds four 

research centers and one coordinating 

center as part of the TREC initiative. The 

TREC centers are fostering collaboration 

among transdisciplinary teams of 

scientists with the goal of accelerating 

progress toward reducing cancer 

incidence, morbidity, and mortality 

associated with obesity, low levels of 

physical activity, and poor diet. They also 

provide training opportunities for new 

and established scientists who can carry 

out integrative research on energetics 

and energy balance. The TREC initiative 

complements NCI’s other energy 

balance research endeavors and efforts 

of the NIH Obesity Task Force. 

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and populations. The Transdisciplinary 

Tobacco Use Research Centers 

(TTURCs) seek to understand the 

determinants of tobacco use, nico

tine addiction, and relapse, along with 

methods for preventing tobacco use 

across cultures. 

Specialized Programs of Research 

Excellence (SPOREs) in lung, head 

and neck, pancreatic, and bladder 

cancers conduct translational research 

related to tobacco and tobacco-related 

cancers. For example, the University of 

Texas-M. D . Anderson Head and Neck 

Cancer SPORE is developing a tyrosine 

kinase inhibitor that shows antitumor 

activity in a tobacco carcinogen-based 

mouse model for both head and neck 

squamous cell carcinoma and non-small 

cell lung cancer. 

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TOBACCO CONTROL: 

PREVENTION AND CESSATION 

OF TOBACCO USE TO 

REDUCE DISEASE 

The NCI-supported Cancer Inter

vention and Surveillance Modeling 

­

Network (CISNET) uses information 

on national trends in cigarette smoking 

to model how tobacco control interven

tions, screening modalities, treatments, 

and public policies may affect lung 

cancer incidence and mortality. Other 

studies help to identify tobacco control 

policies with the greatest potential for 

preventing tobacco use and second-hand 

(environmental) smoke exposure in 

populations disproportionately affected 

by tobacco use or tobacco company 

marketing. NCI also supports research to 

develop and disseminate culturally 

tailored, gender-specific, and languageappropriate tobacco prevention and 

cessation methods. 

­

NCI supports research to improve peo

ple’s ability to stop smoking. For example, 

researchers are learning how tobacco 

cessation treatments may be affected 

by genetic factors. Others studies are 

identifying gene variants associated with 

nicotine metabolism and risk of addiction 

that may provide new targets for cessation 

treatments. Still other researchers seek to 

understand and overcome barriers to 

quitting smoking, such as weight gain. 

NCI research also assesses the toxic and 

addictive properties of cigarettes and 

other tobacco products marketed as 

causing “reduced harm,” and on smokeless 

tobacco. 

NCI initiatives such as the Tobacco 

Research Network on Disparities 

(TReND), help to increase smoking 

cessation in underserved, low income 

populations. TReND includes the Low 

Socioeconomic Status (SES) Women 

­

and Girls Project to stimulate new 

research that will inform policies and 

programs to reduce tobacco use among 

this population. 

TOBACCO-RELATED DISEASE: 

LUNG CANCER 

Examples of NCI research to lessen 

the burden of lung cancer and other 

tobacco-related diseases include the 

following: 

etiology: Population studies uncover 

the biologic, genetic, epidemiologic, 

environmental, social, and behavior

al factors that interact with cigarette 

smoking and other tobacco exposures 

to cause cancer. NCI-supported casecontrol studies (which compare people 

who have a condition with people who 

do not) explore the genetics of lung can

cer development, smoking persistence, 

and patient survival. Research on the 

link between the changes in the lung 

microenvironment caused by inflam

mation will help inform the process of 

cancer development and may inform our 

understanding of lung cancer in smokers, 

former smokers, and non-smokers. 

Prevention: The Lung Cancer Biomark

ers and Chemoprevention Consortium 

(LCBCC) conducts clinical trials and 

studies to identify blood-based biomark

ers that can be used to assess response to 

chemoprevention agents. 

early detection: The National Lung 

Screening Trial (NLST) and the Prostate, 

Lung, Colorectal, and Ovarian (PLCO) 

cancer screening trials are following large 

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9

III thE promisE of prEvEntion 

and Early diagnosis 

Getty Images 

A scanning electron microscopic image of a cancer cell and its processes on a cellular surface 

groups of participants to compare the 

effectiveness of spiral-computed tomography (CT) against standard chest x-rays 

in reducing lung cancer mortality and 

to test the effectiveness of regular chest 

x-rays for lung cancer detection. 

Treatment: NCI’s lung cancer clinical 

trials are studying new treatment 

approaches to inhibit factors that 

stimulate growth of lung tumors. NCI’s 

Imaging and Lung Cancer teams are 

planning research to advance the use 

of advanced imaging, such as FDGPET (positron emission tomography), 

an imaging technique that produces a 

three-dimensional image of functional 

processes in the body, to detect a 

patient’s response to a targeted therapy. 

Work to translate findings from this 

and other research into interventions 

to reduce the burden of tobacco-related 

cancers affords a potentially major 

opportunity to improve the health of 

people across the globe. For example, 

a drug called erlotinib (Tarceva®) 

is one of the “targeted drugs” that 

more specifically attack cancer cells, 

sparing normal cells. Erlotinib targets 

a protein called the epidermal growth 

factor receptor (EGFR). A protein that 

helps cells divide, EGFR is found at 

abnormally high levels on the surface 

of many types of cancer cells, including 

many cases of non-small cell lung cancer. 

By interfering with EGFR, erlotinib 

may keep tumors from growing. NCI 

is working to identify those patients for 

whom the drug, now approved by the 

FDA, offers the best outcome. 

30

IV. nci at a glance. As the leader of the National 

Cancer Program, NCI is committed to enabling the connection between 

academia, the private sector, and other agencies within the federal 

government to drive progress for cancer patients and their families. 

By bringing unique resources to the table, NCI is able to leverage those 

with the expertise and support of the rest of the cancer community. 

Across its intramural and extramural efforts NCI supports basic, 

translational, clinical, and population research, technology development 

initiatives, research training and career development programs, research and 

management support infrastructure, and assessments activities. 

I

NTRAMURAL RESEARCH 

PROGRAM 

NCI is home to many of the world’s 

leaders in cancer research. The NCI 

Intramural Research Program is 

allocated approximately 9 percent of 

NCI’s research dollars and provides 

an unmatched environment that 

supports high-risk projects in basic, 

translational, and clinical research, along 

with epidemiological research studies 

that probe the incidence and distribution 

of cancer. A special strength of the intra

mural program is its ability to rapidly 

launch initiatives in response to emerging 

scientific needs and new opportunities. 

­

NCI’s intramural scientists, who in 

essence form the largest academic 

medicine research university in the 

world, have been tremendously successful 

largely thanks to their close access to 

the NIH Clinical Center, the world’s 

largest hospital dedicated to clinical 

research. This state-of-the art facility provides an ideal environment for 

molecular epidemiology studies and 

genomic research to identify and validate genetic pathways and molecular 

targets that can lead to new approaches 

to cancer prevention, diagnosis, detection, and treatment. It is an ideal venue 

for testing drugs in their earliest phases 

of development. New approaches such 

as Phase 0 trials show great promise 

to shorten drug development time, 

improve drug safety, and drive down 

development cost. 

ExTRAMURAL 

RESEARCH PROGRAM 

Nearly 80 percent of NCI’s budget funds 

extramural research activities—research 

taking place at institutions across the 

country and around the world. The Ex

tramural Research Program supports 

cancer research in nearly 650 univer

sities, hospitals, Cancer Centers, and 

other sites throughout the United 

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31 
IV nCi at a glanCE 

States and in more than 20 countries. 

The majority of NCI’s extramural 

funding supports investigator-initiated Research Project Grants (RPGs). 

For Fiscal Year 2007, NCI invested 

more than $2.1 billion in support of 

over 5,200 RPGs. NCI’s extramural 

budget also supports cancer control, 

investigator training, health disparities 

research, collaborations with the private 

sector, and other types of research and 

development activities. 

Program experts within NCI guide and 

administer the investment in extramural 

biomedical research. They participate in 

the decision making process that identifies research goals and objectives, and 

recommend action for resource allocation. 

They also collaborate and maintain 

effective liaisons with scientists in their 

program area and monitor technologic, 

scientific, and policy developments to 

help identify future research opportunities 

and priorities. The extramural funding 

supports areas such as basic research into 

the workings and mechanism of cancer 

and its cells; research focused on cancer 

prevention, early intervention, symptom management, and supportive care; 

cancer diagnostic and therapeutic cancer interventions research; and research 

in surveillance, epidemiology, health 

services, behavioral sciences, and 

cancer survivorship. 

NCI is leading an initiative to facilitate 

collaborations between intramural and 

extramural investigators by leveraging 

the NIH Clinical Center. At this national “clinical research laboratory” an 

important early-phase clinical trial for an 

aggressive form of a rare cancer, hereditary medullary thyroid carcinoma 

(MTC) was launched in July, 2007. The 

trial will determine whether the investigational agent vandetanib may be the 

first effective nonsurgical treatment in 

young patients with this cancer. But this 

trial is significant for another reason: It 

is the first being conducted at the NIH 

Clinical Center under the joint leadership of an NIH intramural clinical 

investigator and an extramural scientist. 

Dr. Frank Balis, from NCI’s intramural 

research program, is the principal investigator (PI), while Dr. Samuel Wells, 

Collaborations across Intramural and Extramural/ Public and Private Sectors 

When she arrived at the Clinical Center at the National Institutes 

of Health, our patient couldn’t even make a fist. Her hands, 

wrists, elbows, hands, and knees could scarcely bend. Her skin 

was cracking; her face was swollen and disfigured. A once-vibrant 

woman in her late 20’s, she was now severely anemic, wheelchair 

bound, and wrapped in blankets, to preserve the body heat her 

skin could no longer retain. Over two years, as she suffered the 

disabling manifestations of cutaneous T-cell lymphoma, the nights 

spent in the hospital had come to greatly outnumber those she 

spent at home. She was in hospice care and lacked the strength 

to be with her two small children. She came to the Clinical Center 

virtually out of treatment options—and once there, an initial short 

list of experimental treatments had all failed. 

Having apparently run out of all hope, our patient came into the 

care of Dr. Martin E. Gutierrez, a staff clinician with the NCI’s 

Medical Oncology Branch. Dr. Gutierrez, who has spent his career 

working on new therapies for T-cell lymphoma patients, decided 

to try a new drug being developed through NCI’s Rapid Access to 

Intervention Development (RAID) program. RAID exists to speed 

the translation of novel anticancer therapies from laboratories 

to patients. And in this case, the new drug paid off dramatically. 

Within the first few doses, Dr. Gutierrez began to see improvement. 

Within seven months, the patient’s symptoms were gone. 

Today, a year after her arrival at the Clinical Center, the patient’s 

tests show no evidence of disease. 

The drug utilized by Dr. Gutierrez was one that was developed, but 

never marketed, by a pharmaceutical company as a drug to prevent 

cancer. It was brought to the RAID program by an extramural 

investigator from Los Angeles, who developed it as 

a chemotherapeutic drug. Through 

its RAID program, NCI is able to take 

another look at agents that may not 

have fared well in a commercial 

environment, where the potential for 

profitability and market share are 

factors that must be considered. 

RAID offers the opportunity to further 

develop promising approaches for 

prevention, detection, and treatment. 

3 

h tlaeHf os etutitsnIl anoitaN

Martin E. Gutierrez, M.D. 

NCI Staff Clinician 
c nI, soidutSn ossiS, ekpmeLe nauDy by hpargotohpl aireA

The Clinical Center at the National Institutes of Health in Bethesda, Md., is the nation’s largest hospital devoted to clinical research. 

from Washington University in St. Louis 

and one of the world’s foremost MTC 

experts, is the adjunct PI. Dr. Wells was 

involved in the discovery of a proto

oncogene, called RET, associated with 

the hereditary disorder multiple endo

crine neoplasia (MEN). Patients with 

subtypes of MEN are at high risk of 

developing MTC and other endocrine 

tumors. Dr. Wells also helped to establish 

the efficacy of prophylactic thyroidec

tomy in children with RET mutations 

associated with these MEN subtypes. 

After leading early-phase trials that 

demonstrated the activity of vande

tanib in adults with MTC, Dr. Wells 

approached NCI about conducting a 

trial in children. It offered an ideal opportu

nity to pursue an intramural/extramural 

scientist-led trial. NCI worked with 

NIH leaders to make the arrangements 

allowing Dr. Wells to serve as a PI of a 

Clinical Center trial. 

It is with rare cancers that the Clinical 

Center’s value truly shines. Because NIH 

can recruit patients from around the 

country, trials performed at the 

Clinical Center are more likely to 

enroll enough patients with rare 

diseases to provide the statistical 

power needed to produce meaningful 

results and inform clinical care. 

Conducting the trial at the Clinical 

Center also allows investigators from 

other NIH institutes with expertise in 

endocrinopathies and MEN syndromes 

to participate. In addition to 

helping to write the trial protocol, 

these investigators provide clinical care 

to trial participants. And many patients 

in this trial—as well as members of 

their families, since this is a hereditary 

syndrome—will likely participate in 

additional studies, ensuring that the 

most scientific value from and clinical 

benefit for each patient during their 

treatment can be achieved. 

The trial’s Phase I/II design allows it to 

both determine the safest drug dose that 

can be given to pediatric patients and to 

evaluate its potential efficacy. The trial 

also involves biomarker and pharma

cokinetic analyses, as well as analyses of 

tumor samples in the laboratory of NCI’s 

Dr. Paul Meltzer to study whether there 

are genetic mutations associated with 

resistance to the agent. 

This trial highlights a new vision of 

collaboration and partnership. It maxi

mizes the potential of a single trial to 

provide as much data as possible so that 

the results can be quickly translated into 

patient benefit. 

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33 
IV nCi at a glanCE 

It is with rare cancers that 

the Clinical Center’s value 

truly shines. Because NIH 

can recruit patients from 

around the country, 

trials performed at the 

Clinical Center are more 

likely to enroll enough 

patients with rare diseases 

to provide the statistical 

power needed to produce 

meaningful results and 

inform clinical care. 

htlaeHf os etutitsnIl anoitaN

Patient rooms are in close proximity to cutting-edge laboratories 

doing related research. 

h tlaeHf os etutitsnIl anoitaN

3 
V. the 2009 buDget. (Professional Judgment Budget) 

Successful outcomes from cancer research are the product of significant 

investments, not just of financial resources, but of time and professional 

dedication. Future success depends on sustained commitment to research 

so that we can truly deliver on the promise of personalized medicine. 

In order to fulfill this vital mission, the principles of the highest-quality 

scientific investigation, the development of enabling advanced 

technologies, and a pipeline of well-trained scientists and clinicians 

must be sustained. 

OPPORTUNITIES 

The National Cancer Institute 

is acting strategically to capture 

and leverage the opportunities before us, 

by building on previous research successes and wisely spending every dollar we 

receive. Emerging and expanding opportunities, reflected in this document, have 

been identified not in a vacuum, but by 

carefully listening to the entire oncology 

research community. NCI has the mandate to lead this effort, in addressing the 

fundamental issues that accompany the 

“preemption of cancer at every opportunity” and “assurance of the best outcomes 

for all”: the dual core of the National 

Cancer Program. 

The Impact of an Increased 

Budget: recruitment and Funding 

of Scientists 

NCI’s ability to attract new, talented 

people into cancer research hinges on 

many factors, including continued 

financial investment. These young 

scientists are crucial for generating 

new ideas and translating them into 

scientific discoveries that benefit 

patients everywhere. An increase in 

scientific grant-supported research is 

absolutely necessary to encourage 

new scientists to bring their talents to 

cancer research rather than pursuing 

other well-funded areas, or to leave 

science all together. 

A sign of this growing need can be seen 

in the fact that the average age of a firsttime NIH grant recipient is now over 

41—up from 34 in 1970. Grants receiving scores that in times of greater 

resources were competitive, now are not 

funded. Furthermore, the number of 

training awards for scientists has stayed 

level in recent years, even though the 

number of applications has continued 

to rise. This reality can be discouraging 

for seasoned scientists as well as trainees 

who, unable to obtain funding, are pursuing other careers at a time when we 

need them the most. 

NCI recognizes the need to train individuals in new technologies, to foster team 

science, and to increase our research 

capacity. We are hopeful that the resources can be found to support training 

and career development. 

Seizing the opportunities at Hand 

This plan—and the accompanying 

budget request—outline NCI’s highest 

priorities for Fiscal Year 2009. With 

sufficient national investment: 

35 
thE 2009 budgEt V (profEssional JudgmEnt budgEt) 

Getty Images 

1.	 Our Nation’s position as a leader in 

biomedical research and development 

will remain strong. 

2.	 We will enhance our ability to translate research findings to improve the 

quality of care and life for our increasing number of cancer survivors. 

3.	 We can fully and rapidly utilize promising advances in cancer research, and 

ultimately realize the new possibilities 

for cancer prevention, early detection, and treatment for all. 

The timing of these priorities coincides 

with a period when our country is experiencing an increase in new cancer cases, 

as the first wave of “baby boomers” 

begins to pass the age of 60 into the age 

bracket in which most cancers occur. 

Meanwhile, cancer will continue to affect people of all ages, and virtually every 

American will know someone affected 

by this disease. Our national investment 

in cancer research and treatment must be 

equal to the passion of our scientists and 

clinicians to ensure that the burden of 

cancer be lifted for all people. 

3


These young scientists 

are crucial for generating 

new ideas and translating 

them into scientific 

discoveries that benefit 

patients everywhere. 
This budget consists of two components: (1) the increase required to maintain our present level of operations or 

“current services”, and (2) the increase required to initiate new and expand existing initiatives. The current services 

increase is the amount that will be required to sustain NCI programs, restore some of the funding cuts that have been 

implemented over the past several fiscal years, and provide for some minimal growth. Noncompeting Research 

Project Grants (RPGs) would be funded at committed levels, the success rate for competing RPGs would be maintained 

at its current rate, there would be a small growth in the number of Cancer Centers, and most other mechanisms would 

receive sufficient increases to cover cost of living adjustments based on the Biomedical Research and Development 

Price Index (BRDPI). This budget level also includes funds to make critically needed capital repairs and improvements 

at the NCI-Frederick Federally Funded Research and Development Center. The funds needed to initiate new and expand 

existing initiatives are add-ons to the current services level. 

at a glance (dollars in thousands) 

37

 Fiscal Year 2008 Estimate
 $4,925,740

Current Services Increase
 334,638 

Subtotal 5,260,378 

Fiscal Year 2009 Additional Resources

 Increase Success Rate for Research Project Grants 65,000 

 Basic and Translational Science for Personalized Medicine 289,902 

 Linking Science and Technology 209,600 

 Reaching All Communities 72,100 

 Prevention and Early Detection 131,406 

Subtotal 768,008 

TOTAL, NCI 6,028,386 

* Does not include the NCI contribution to the NIH Roadmap. 
the 2009 nci professional JuDgment buDget 

(dollars in thousands) 

Research Project Grants 

FY 2007 

Obligations 

FY 2008 

Estimate 

Current 

Services 

Increase 

FY 2009 

Current 

Services 

FY 2009 

Additional 

Resources 

Total 

FY 2009 

Bypass 

% Inc Over 

FY 2008 

Estimate 

Noncompeting $1,597,609 $1,593,908 $101,303 $1,695,211 $0 $1,695,211 6.4% 

Administrative Supplements 39,864 50,000 7,500 57,500 - 57,500 15.0% 

Competing 440,193 495,950 

2,139,858 

89,700 

2,229,558 

98,050 

206,853 

3,319 

210,172 

594,000 

2,346,711 

93,019 

2,439,730 

306,000 

306,000 

12,000 

318,000 

900,000 

2,652,711 

105,019 

2,757,730 

81.5% 

24.0% 

17.1% 

23.7% 

Subtotal 2,077,666 

SBIR/STTR 93,677 

Subtotal, RPG 

Research Centers & SPOREs 

2,171,343 

Cancer Centers (P30/P20)
 274,504 283,304 15,482 298,786 15,000 313,786 10.8% 

SPORE (P50/P20)
 159,478 160,478 5,938 166,416 29,200 195,616 21.9% 

Other Specialized (U54/P50)
 85,838 97,092 

540,874 

3,592 

25,012 

100,684 

565,886 

46,500 

90,700 

147,184 

656,586 

51.6% 

Subtotal, Centers 21.4% 

Other Research 

519,820 

Research Careers 79,595 80,955 5,995 86,950 11,000 97,950 21.0% 

Cancer Education 31,337 32,837 1,215 34,052 5,550 39,602 20.6% 

Cooperative Clinical Research 238,403 245,603 9,087 254,690 28,250 282,940 15.2% 

Minority Biomed. Res. Support 2,435 2,435 90 2,525 - 2,525 3.7% 

Other 63,559 65,160 

426,990 

3,197,422 

2,411 

18,799 

253,983 

67,571 

445,789 

3,451,405 

7,000 

51,800 

460,500 

74,571 

497,589 

3,911,905 

14.4% 

16.5% 

22.3% 

Subtotal, Other Research 

Total Research Grants 

415,329 

3,106,492 

National Research Service Awards 68,223 69,933 3,588 73,521 10,000 83,521 19.4% 

R & D Contracts 550,590 578,114 

20,000 

715,129 

21,390 

740 

26,460 

599,504 

20,740 

741,589 

180,000 

15,000 

55,485 

779,504 

35,740 

797,074 

34.8% 

78.7% 

11.5% 

SBIR/STTR
 12,387 

Intramural Research 706,179 

Research Management & Support 353,211 357,222 13,217 370,439 20,023 390,462 9.3% 

Construction (Capital Improvements) - - 6,000 6,000 24,000 30,000 ~

Buildings & Facilities 7,920 7,920 10,000 17,920 18,000 35,920 353.5% 

TOTAL, NCI 4,792,615 4,925,740 334,638 5,260,378 768,008 6,028,386 22.4% 

3

NIH Publication No. 07-6238


Printed January 2008