TABLE OF CONTENTS

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A Transformational Moment in Cancer Research

Budget Proposal & Priorities

Progress & Trends: Decreased Mortality, Increased Survival Rates

U.S. Trends

Racial/Ethnic Disparities

Global Trends

Obesity

The National Cancer Program: Driving Discovery

New Approaches to Funding Researchers

NCI-Designated Cancer Centers

NCI’s National Clinical Trials Enterprise

Overcoming Cancer Health Disparities

Addressing the Unique Challenges of Childhood Cancer Research

NCI’s Intramural Research Program

Bioinformatics to Accelerate Research

Frederick National Laboratory for Cancer Research 

Opportunities in Precision Oncology

Advancing Precision Medicine in Oncology

Expanding Precision Medicine Trials

Enhancing Drug Discovery and Development

Developing New Human Cancer Models

Harnessing the Promise of Immunotherapy

Strategies for the Prevention and Early Detection of Cancer

Preventing Cancer Caused by Viral Infections

Preventing Cancer with Drugs

Identifying New Biomarkers for Risk and Early Detection of Cancer

The Biology of Cancer Health Disparities
3

NCI’S ANNUAL PLAN & BUDGET PROPOSAL FOR FISCAL YEAR 2017

When I speak with leading cancer researchers in the United States and 

around the world, I hear unprecedented optimism that we are on 

the verge of pivotal advances in oncology. This sentiment is based on 

progress in many important areas, including immune-based therapies, genomics, 

advanced imaging technologies, new laboratory models of human cancer, precision 

medicine, and more.

Key aspects of our understanding of and approach to cancer have been transformed based 

on years of investment in biomedical research. We are increasingly able to treat cancer with 

greater precision by identifying the molecular abnormalities that drive each person’s cancer 

and targeting therapies to each patient, ultimately improving outcomes and providing hope.

This promise of precision medicine has already been realized for treating some cancers, and we 

foresee greater progress in preventing, screening, and treating other cancers and even other 

diseases. Cancer research, therefore, offers a model for other fields of biomedical research that 

seek to leverage genetic and other molecular information to administer precise and effective 

interventions to treat disease.

At NCI, we are advancing precision oncology while managing our resources to take full 

advantage of the most promising scientific opportunities. It is essential that NCI support the 

full continuum of scientific research—from basic biological research, to population-based 

studies, to cutting-edge clinical trials—as virtually all advances in cancer depend on many fields 

of science.

Although dramatic progress is being 

made, important scientific opportunities 

lie before us. With steady and sustained 

budget increases and a cadre of 

talented researchers, a new era of 

cancer medicine is well within reach.

Despite careful management of the 

NCI budget, many meritorious research 

proposals—including some bold concepts—must go unfunded each year due to the fiscal 

constraints we have been operating under for more than a decade. There is little doubt that 

budget constraints have resulted in missed scientific opportunities.

With the exception of the one-time increase allocated in the American Recovery and 

Reinvestment Act, federal investment in cancer research has been stagnant since 2003. During 

this same period, the costs of conducting research have escalated as inflation has substantially 

eroded NCI’s purchasing power. As a result, competition for NCI grants has been fierce, and 

some young researchers, frustrated by a lack of funding, have abandoned careers in medical 

research.

A Transformational Moment in Cancer Research

With steady and sustained budget increases 

and a cadre of talented researchers, a new 

era of cancer medicine is well within reach.
4

Read the full version of this document at plan.cancer.gov

The current budget situation has hindered NCI’s ability to optimally fulfill its mission and 

promise to the American public: to foster rapid progress and reduce the burden of cancer.

Working closely with its advisory boards, NCI senior leadership has made difficult funding 

choices, reducing funding for some worthwhile programs and initiatives and curtailing funding 

altogether in some cases.

In the budget table that follows, NCI recommends a funding increase of 7 percent over 

the fiscal year 2016 level to pursue promising research opportunities that improve our 

understanding of cancer and reduce the burden of the disease. These research opportunities 

are among those for which additional funding would greatly speed the progress against 

cancer.

But a 7 percent increase can 

only be viewed as an initial 

down payment. Steady funding 

increases, sustained over time, 

are necessary to restore NCI’s 

purchasing power and accelerate 

scientific discovery in ways that 

significantly reduce the burden 

for people with all types of cancer. 

An annual increase of 7 percent 

for the next 10 years is necessary to achieve these goals. These steady increases will result in a 

fiscal year 2026 budget for NCI that is twice what it is today.

NCI recommends a funding increase of 7 percent over the fiscal year 2016 level to pursue 

promising research opportunities that improve our understanding of cancer and reduce the 

burden of the disease.

As NCI’s leader, I am continually inspired by the incredible dedication and passion of the 

institute’s staff and researchers, as well as the dedication and passion of cancer researchers 

across the country and around the world. We understand that patients and their loved 

ones expect and deserve continued progress and that we have an obligation to meet their 

expectations.

Douglas Lowy, M.D.

Acting Director, National Cancer Institute

NCI recommends a funding increase of 7 percent over 

the fiscal year 2016 level to pursue promising research 

opportunities that improve our understanding of 

cancer and reduce the burden of the disease.

Progress & Trends: 

Decreased Mortality, 

Increased Survival 

Rates
7

NCI’S ANNUAL PLAN & BUDGET PROPOSAL FOR FISCAL YEAR 2017

Decreasing cancer death rates and increasing numbers of cancer 

survivors are important indicators of the progress we are making 

in diagnosing and treating cancer, but much work remains. Too 

many people still face a diagnosis of cancer, and far too many still die 

from the disease.

U.S.Trends

Before 1990, overall age-adjusted cancer mortality rates increased for 

several decades. Since the mid-1990s, however, they have dropped 

steadily. Mortality rates are decreasing for most cancers, including those 

that arise at the four most common sites: the lung, breast, colon/rectum, 

and prostate.

These improvements in mortality rates have been accompanied by a 

substantial increase in the number of cancer survivors. Whereas cancer 

survivors accounted for about 2.5 percent of the U.S. population in 

1992, it is estimated that they will account for about 5.4 percent of the 

population in 2024.

For many of these survivors, long-term effects, both physical and 

psychological, may remain. Recognizing this, survivorship research 

remains a key component in NCI’s research portfolio.

Despite these advances, it is estimated that approximately 590,000 adults 

and nearly 2,000 children and adolescents will die from cancer in the 

United States in 2015 and that 1.66 million new adult cancer cases and 

15,000 new childhood and adolescent cancer cases will be diagnosed.

In addition, our progress in preventing, diagnosing, and treating cancers 

is not universal for all forms of the disease. The mortality rates for some 

cancers have actually increased. 

Because older age is a major risk factor for developing cancer, improved 

life expectancy by itself will lead to a rise in the total number of cancer 

cases in the United States. Therefore, although the age-adjusted rates at 

which cancer develops are expected to continue to decline, the aging of 

the U.S. population means that the total number of cancers will increase.

These projected increases imply that intensive and sustained research 

efforts against cancer will continue to be critical. NCI continues to employ 

every aspect of its portfolio to better prevent, diagnose, and treat cancer, 

but these efforts must be enhanced to keep up with the changing 

demographics of our society.

THE OVERALL CANCER DEATH RATE

IN THE UNITED STATES

23%

FROM

FELL BY

Source: SEER Cancer Statistics Review 1975-2012

www.cancer.gov

1990 2012

MORE PEOPLE SURVIVE CANCER

Where We Were

How Far We’ve Come

Where We Will Be

Source: American Cancer Society, Cancer Treatment & 

Survivorship Facts & Figures 2014-2015

www.cancer.gov
8

Read the full version of this document at plan.cancer.gov

Racial/Ethnic Disparities

Although declines in overall cancer incidence and mortality have been 

observed for most racial and ethnic groups in the United States, certain 

groups continue to have higher incidence and mortality rates for some 

cancers than the general population.

For example, in 2012, the rates of colorectal cancer incidence and 

mortality among African Americans were 22 percent and 36 percent 

higher, respectively, than the rates observed for the general population. 

For multiple myeloma, the incidence and mortality rates in 2012 were 116 

percent and 85 percent higher, respectively, for African Americans than 

for the general population.

Among gender-specific cancers, the rates of prostate cancer incidence 

and mortality among African American men from 2008 through 2012 

were 56 percent and 116 percent higher, respectively, than the rates for 

men in the general population. During the same period, the rates for 

cervical cancer incidence were 29 percent and 19 percent higher among 

Hispanic and African American women, respectively, than among women 

in the general population, and the corresponding rates of cervical cancer 

mortality were 17 percent and 74 percent higher for Hispanic and African 

American women, respectively.

ALL RACES

NEW CASES DEATHS

WHITE

BLACK

HISPANIC

ASIAN/PACIFIC

ISLANDER

AMERICAN INDIAN/

ALASKA NATIVE

171.2

202.0

170.9

119.3

106.6

454.8

478.0

463.3

349.9

306.8

319.7 117.4

NUMBER OF NEW CASES & DEATHS

BY RACE/ETHNICITY: ALL CANCER SITES

PER 100,000 PERSONS

www.cancer.gov

Source: SEER 18, 2008-2012
9

NCI’S ANNUAL PLAN & BUDGET PROPOSAL FOR FISCAL YEAR 2017

Global Trends

Improvements in nutrition, health care, and other factors are increasing 

life expectancy for most of the world. However, this benefit is 

accompanied by an increase in many diseases associated with an aging 

population, including cancer, and this trend is being exacerbated by 

greater tobacco consumption in many low- and middle-income countries.

The projected increases in cancer rates in the U.S. and worldwide imply 

that intensive and sustained research efforts against cancer will continue 

to be critical. NCI continues to employ every aspect of its portfolio to 

better prevent, diagnose, and treat cancer, but these efforts must be 

enhanced to keep up with changing U.S. and global demographics.

Obesity

Over the past few decades, the incidence of obesity has risen markedly 

in the United States and in many other countries around the world. 

Although the so-called obesity epidemic has been most commonly linked 

to the rising incidence of diabetes and related conditions, it also has 

substantial implications for cancer research and cancer control, since 

obesity is associated with increased risks of developing cancer at many 

sites.

WORLDWIDE CANCER CASES

WORLDWIDE CANCER DEATHS

WILL INCREASE BY

WILL INCREASE BY

52%

61%

FROM IN 2012

2030

14.1 million

TO 21.4 millionIN

8.2 million

13.2 million

FROM IN 2012

TO IN 2030

Source: American Cancer Society: Global Cancer Facts & Figures, Second Edition

www.cancer.gov
The National 

Cancer Program: 

Driving 

Discovery
11

NCI’S ANNUAL PLAN & BUDGET PROPOSAL FOR FISCAL YEAR 2017

NCI coordinates the National Cancer Program and supports it by 

providing funding and resources to individual researchers and 

institutions, providing leadership to national infrastructures that 

care for patients and develop new methods to treat and prevent cancer, 

and conducting research in especially challenging areas.

Progress against cancer depends on many types of research—including 

basic, translational, and clinical—across different research areas, from 

the biology of cancer cells to studies of large populations.

Regardless of the research type or area, supporting the best science and 

the best scientists is of paramount importance to NCI.

New Approaches to Funding Researchers

NCI continues to develop new funding opportunities that adapt to 

changes in the way that science is conducted.

Support for the best science underpins everything NCI does; therefore, 

recruiting outstanding young investigators with great potential is a 

priority for the institute. However, the uncertainty of a successful career 

due to a lack of funding opportunities has made attracting and retaining 

the best minds to the field of cancer research today a challenge.

NCI is also committed to supporting the training and development of a 

strong workforce of cancer researchers that spans the career continuum. 

Due to current limitations in funding, however, the early careers of 

graduate students, postdoctoral fellows, and young investigators may be 

hampered by low salaries, many years spent in training positions, and still 

more years in an independent research position before they obtain an 

NIH grant for their research.
12

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For most of the past 50 years, at least 30 percent of grant applications 

were funded, dipping to 25 percent during periods of lean budgets. 

Today, the percentage of successful applications hovers in the mid-teens, 

far lower than at any other time.

Although NCI is actively building a cancer research workforce for 

the future, these efforts will appear hollow without strong support 

for funding these young scientists. Even established researchers are 

now forced to devote too much time to securing funding rather than 

conducting research or training the next generation of scientists.

High-School 

Students

Pre- & PostBaccalaureates

Clinical 

Fellows

Early-Career

Scientists

Graduate & 

Medical Students

Postdoctoral & 

Research Fellows

Individuals supported 

by NCI training, career 

development grants, 

and intramural research 

experiences

* Numbers do not include students and postdoctoral fellows supported by NCI research project grants, cancer center grants, and other non-training mechanisms

† Does not distinguish between summer research experiences and part- or full-time appointments

†

428 205 841 1,526 123 485

3,608

InFY 2014, NCI supported 3,608 emerging cancer researchers through training and career development grants and intramural research experiences.

*

Training The Workforce

NATIONAL CANCER INSTITUTE

Source: http://www.cancer.gov/cct | http://crchd.cancer.gov

www.cancer.gov
13

NCI’S ANNUAL PLAN & BUDGET PROPOSAL FOR FISCAL YEAR 2017

Moreover, when funds are limited, there is a tendency to support less 

risky research. However, high-risk research, the fruits of which may take 

several years to realize, is often where the biggest breakthroughs are 

made. Therefore, NCI has taken steps to provide incentives to researchers 

who propose and conduct high-risk research.

In 2014, NCI established the Outstanding Investigator Award, and the 

first awardees will be announced in the fall of 2015. This new R35 funding 

mechanism is designed to provide longer-term support—7 years—and 

more than twice the dollar amount of an average R01 investigatorinitiated grant. These awards are given to experienced investigators 

who are likely to continue conducting seminal cancer research and 

mentoring the next generation of cancer researchers. With this support, 

investigators may pursue research that might be viewed as too high-risk 

to be funded through regular grant mechanisms.

In addition, through mechanisms such as R21 exploratory grants, NCI 

seeks to help investigators conduct other potentially new and exciting 

but high-risk studies.

The Provocative Questions initiative is another approach developed by 

NCI to stimulate the research community and provide a new form of 

funding. Through this initiative, NCI is supporting research, including 

basic and applied, in a number of important areas that have been 

understudied, neglected, paradoxical, or difficult to address in the past.

THE PROVOCATIVE

HAS FUNDED

THE INITIATIVE

HAS IDENTIFIED QUESTIONS IN CANCER 

RESEARCH THAT HAVE BEEN

UNDERSTUDIED, NEGLECTED, PARADOXICAL,

OR DIFFICULT TO ADDRESS IN THE PAST.

GRANTS

188

WITH MORE THAN

MILLION

$70

Source: NCI Center for Strategic Scientific Initiatives

QUESTIONS INITIATIVE

www.cancer.gov

University of Kentucky Markey Cancer Center
14

Read the full version of this document at plan.cancer.gov

NCI-Designated Cancer Centers

The NCI-Designated Cancer Centers program is a major anchor of the 

nation’s cancer research effort.

At any given time, hundreds of cutting-edge research studies are under 

way at NCI-Designated Cancer Centers, ranging from basic laboratory 

research to clinical assessments of new treatments.

Each NCI-Designated Cancer Center receives a core support grant 

from NCI that funds the critical research infrastructure of the center, in 

addition to the funding received from individual competitive research 

grants and contracts with NCI.

The funding provided by the core grants is essential for the efficient 

conduct of research at the centers and for driving the nation’s progress 

against cancer.

NCI-DESIGNATED CANCER CENTERS

www.cancer.gov

The 69 NCI-Designated Cancer Centers are at the forefront of NCI-supported efforts at universities and cancer research centers across the United 

States. The centers are developing and translating scientific knowledge from promising laboratory discoveries into new treatments for cancer 

patients. There are 17 cancer centers, 45 comprehensive cancer centers, and 7 research centers.

NCI-DESIGNATED CANCER CENTERS

The 69 NCI-Designated Cancer Centers are at the forefront of NCI-supported efforts at universities and cancer research centers across the United 

States. The centers are developing and translating scientific knowledge from promising laboratory discoveries into new treatments for cancer 

patients. There are 17 cancer centers, 45 comprehensive cancer centers, and 7 research centers.
15

NCI’S ANNUAL PLAN & BUDGET PROPOSAL FOR FISCAL YEAR 2017

NCI’s National Clinical Trials Enterprise 

NCI has a long history of supporting both small early-phase trials as 

well as large-scale trials, many of which have led to changes in standard 

medical practice. People with cancer now live longer lives in part because 

of strategies that have come from NCI’s clinical trials program.

To respond rapidly to new scientific opportunities, especially in 

conducting the new generation of precision medicine clinical trials, NCI 

created the National Clinical Trials Network (NCTN).

Precision medicine trials require sophisticated and expensive 

technologies and clinical processes that have not typically been a part of 

most clinical trials.

In addition to the higher cost of conducting precision medicine clinical 

trials, NCI is now providing a higher percentage of cost reimbursement to 

the physicians who are treating participants enrolled in trials.

Although the NCTN is designed to carry out all aspects of precision 

medicine trials, NCI is unable to fully support it and must rely on the 

institutions and others involved in the network to cover some costs.

ALLIANCE

CANADIAN

NETWORK

GROUP

SWOG

NATIONAL CANCER INSTITUTE

COG

(PEDIATRIC)

Centralized Functions:

• Centralized Institutional 

Review Board

• Cancer Trials Support Unit

• Imaging and Radiation 

Oncology Core (IROC) Group

• Common Data Management 

System Central Hosting

ECOGACRIN

NRG

ONCOLOGY

NCTNCENTRALIZED

FUNCTIONS

NCORP

SITE

PARTICIPATION

National Clinical Trials Network

www.cancer.gov/clinicaltrials/nctn

30 Lead Academic 

Participating Sites 

(LAPS)

The National Clinical Trials Network (NCTN) has four U.S. adult

cooperative groups (Alliance, ECOG-ACRIN, NRG Oncology, and SWOG)

and one pediatric cooperative group (COG). The NCTN also includes

a Canadian Network Group because the NCI has had long-standing

collaborations with Canadian investigators in clinical trials. Sites that

are part of the NCI Community Oncology Research Program (NCORP)

can also participate in NCTN clinical trials.

www.cancer.gov
16

Read the full version of this document at plan.cancer.gov

Although these partnerships are highly effective and appropriate 

for some trials, there are some clinical questions for which there are 

no obvious partners. It is NCI’s responsibility to pursue these trials, 

especially in the case of rare cancers, but, as in other areas of NCIsupported research, current funding levels constrain maximizing their 

pursuit.

In addition, in 2014, the institute launched the NCI Community Oncology 

Research Program (NCORP), a community-based initiative that brings 

clinical trials, as well as research aimed at improving the efficiency and 

quality of delivered health care, to people in their own communities. 

NCORP provides an important connection to community-based cancer 

care, ensuring that people have access to the benefits of the latest 

research regardless of where they live.

Overcoming Cancer Health Disparities

NCI’s Center to Reduce Cancer Health Disparities (CRCHD) is a major 

component of the institute’s efforts to address the unequal burdens of 

cancer experienced by certain racial and ethnic groups in our society. 

Finding ways to reduce these cancer health disparities is also a major 

focus of NCORP.

NCI supports a wide range of research, including basic biological 

research, to increase our understanding of the causes of cancer health 

disparities and to develop ways to mitigate their effects and, ultimately, 

eliminate them. 

NCI also supports studies of health care access and utilization, their 

effects on health-related outcomes, and epidemiologic studies examining 

the role of obesity and other risk factors in cancer health disparities.

NCI is also committed to training underrepresented minorities in 

cancer research in general and cancer health disparities in particular. 

These training programs include the Continuing Umbrella of Research 

Experiences (CURE) and Partnerships to Advance Cancer Health Equity 

(PACHE).

The NCI Community Oncology Research Program 

provides care in community-based health care 

practices across the United States.
17

NCI’S ANNUAL PLAN & BUDGET PROPOSAL FOR FISCAL YEAR 2017

Addressing the Unique Challenges of Childhood Cancer Research 

NCI recognizes the importance of research that focuses on cancers in 

children and adolescents and has an extensive biomedical research 

portfolio that relates, directly or indirectly, to childhood cancer. 

This portfolio includes basic scientific research, which enhances our 

understanding of the fundamental mechanisms of cancer, and clinical 

research, which evaluates new therapies and other interventions for 

safety and effectiveness. 

NCI’s Intramural Research Program 

Some of NCI’s budget supports the intramural research program and the 

research of scientists who work at the NIH Clinical Center and in offices 

and laboratories located in Bethesda, Rockville, and Frederick, Maryland. 

These intramural investigators conduct basic, clinical, and populationbased research, including the study of rare cancers, and are encouraged 

to explore the translation of relevant findings from the laboratory to the 

clinic.

Intramural researchers at this center are able to quickly test new 

approaches to cancer prevention and treatment. The ability of the NIH 

Clinical Center to treat patients from all over the world facilitates clinical 

research on rare cancers, which may help patients with these diseases 

and produce insights relevant to more common cancers.

The NIH Clinical Center does not come without expense. NCI and the 

other NIH institutes and centers support, financially and professionally, 

the operations of the clinical center, with NCI covering more of the costs 

than any other institute.

With both academic and private sector partners, intramural researchers 

can undertake longer term projects that may be difficult, if not 

impossible, through traditional funding mechanisms.

Bioinformatics to Accelerate Research

Bioinformatics, which enables the management and use of very large sets 

of complex molecular and clinical data, has become a core component of 

NCI’s research enterprise.

The data center at NCI’s Frederick National Laboratory for Cancer 

Research in Frederick, Maryland, provides high-performance, highcapacity computing support for these efforts.

The Cancer Genome Atlas, a joint effort by NCI and the National 

17

Annette Abrams, a patient of Daniel H. Fowler, 

M.D., of NCI’s Experimental Transplantation and 

Immunology Branch, stands by a mosaic she created 

while undergoing treatment. The art is on display at 

the NIH Clinical Center.

Credit: National Cancer Institute (Rhoda Baer)
18

Read the full version of this document at plan.cancer.gov

Human Genome Research Institute to survey the landscape of genomic 

alterations in more than 30 different types of cancer, has generated 2.5 

petabytes (2.5 million gigabytes) of next-generation sequencing data 

from several different technology platforms.

NCI is establishing the Genomic Data Commons (GDC) to store, analyze, 

and distribute cancer genomics data generated by NCI and other 

research organizations. Currently, these data and associated information 

are being stored in many different repositories, which is inefficient, 

confusing, and a barrier to research. The GDC, a centralized repository, 

will address these problems by putting the data in one place that will be 

widely accessible.

An additional impetus for the GDC is to reduce the total cost of ownership 

of genomic data for NCI. Having many smaller repositories makes it 

harder for researchers to find and retrieve the data they are looking for. 

It also creates many systems, all at least slightly different, that need to be 

maintained and upgraded as the state of science changes. This is costly 

and hard to manage, coordinate, and sustain.

Strong, continuous funding for bioinformatics is needed to:

• increase the capacity of the GDC and enable it to handle additional 

data types generated by precision medicine research

• explore the development of clinical bioinformatics, in which 

biological information is integrated with medical information to 

enhance precision health care

• support the use of new experimental models of human cancer in 

cancer drug discovery and development and in studies of cancer cell 

biology

Frederick National Laboratory for Cancer Research

What is today the Frederick National Laboratory for Cancer Research 

(FNLCR) had its beginning in a 1971 designation by President Nixon. In 

1975, it became the only Federally Funded Research and Development 

Center (FFRDC) dedicated to biomedical research.

Overseen by NCI, this FFRDC provides rapid response capabilities and 

one-of-a-kind resources for the entire biomedical research community 

using a unique contract mechanism that brings public and private 

partners together to solve difficult medical research challenges.

The FNLCR provides scientific tools, services, and information to support 

Dan Oleyar collects data center metrics at the 

Advanced Technology Research Facility at the 

Frederick National Laboratory for Cancer Research 

in Frederick, Maryland.

Credit: National Cancer Institute
19

NCI’S ANNUAL PLAN & BUDGET PROPOSAL FOR FISCAL YEAR 2017

researchers and to enable and expedite their investigations. Examples 

include support for drug discovery and development and for the 

characterization and quality-control assessments of nanoparticles being 

developed for cancer diagnosis and treatment.

As a national resource, the FNLCR provides cancer researchers a bridge 

between basic research and clinical practice with support that is not 

readily available elsewhere. 

19

The Frederick National Laboratory for Cancer 

Research provides its researchers with scientific tools, 

services, and information to enable and expedite 

their investigations.

Credit: National Cancer Institute
Opportunities 

in Precision 

Oncology
21

NCI’S ANNUAL PLAN & BUDGET PROPOSAL FOR FISCAL YEAR 2017

I

n the very near future, a patient’s cancer will be extensively 

characterized for mutations and other molecular abnormalities, and 

his or her treatment will be based on the identified molecular changes 

instead of where the cancer originated in the body. This approach is part 

of what is called precision (or personalized) medicine.

The greater molecular and mechanistic understanding of cancer that we 

are gaining—and applying toward the development of new therapies—is 

also directly applicable to cancer prevention and screening.

To speed progress in this area, President Obama announced the 

Precision Medicine Initiative (PMI) on January 30, 2015. The PMI is a $215 

million proposed investment to accelerate biomedical research and 

provide clinicians with new therapies and tools to select the treatments 

and interventions that will be most effective for individual patients.

Advancing Precision Medicine in Oncology

The PMI includes $70 million in increased funding for NCI to advance the 

field of precision medicine in oncology. Oncology is an appropriate choice 

to begin this ambitious initiative, since precision medicine focuses mainly 

on genes, and cancer is a disease of the genome.

At the time of the PMI’s announcement, NCI was well along the path 

toward precision medicine, but steady and sustained funding is needed 

to capitalize on the exciting opportunities now at hand.

Expanding Precision Medicine Trials

NCI’s efforts in precision medicine include the development of clinical 

trials for patients whose cancers will be profiled and whose treatments 

will be based on the molecular abnormalities in their tumors. These trials 

include Lung-MAP, ALCHEMIST, and NCI-MATCH.

More robust funding would allow NCI to:

• expand NCI-MATCH to include additional targeted drugs and 

increase the number of genetic alterations being investigated

• accelerate the launch of Pediatric MATCH

• collect and analyze genomic data sets to increase our understanding 

of the genetic alterations associated with cancer

Shridar Ganesan, M.D., Ph.D. (right), of Rutgers 

Cancer Institute of New Jersey, studies genomic 

changes in cancers that can be therapeutically 

targeted.

Credit: National Cancer Institute

23

NCI’S ANNUAL PLAN & BUDGET PROPOSAL FOR FISCAL YEAR 2017

• establish a national knowledge network to house and integrate 

genomic information from tumors—annotated with clinical 

information, including treatment response and patient outcomes 

information—as a resource for scientists, health care workers, and 

patients

Enhancing Drug Discovery and Development

Another goal of precision medicine is the development of new treatments 

that are more effective and less toxic than traditional treatments to 

improve the care of patients with cancer.

Historically, NCI has played a vital role in cancer drug discovery and 

development, and, today, that role continues. Frequently, NCI’s drug 

development efforts focus on unmet needs that are not being adequately 

addressed by the private sector. The success of NCI’s efforts is reflected 

in the fact that the institute has been involved in the discovery or 

development of a large percentage of the anticancer therapeutics on the 

market today.

To speed the delivery of these therapies to patients with cancer, NCI’s 

Experimental Therapeutics Program (NExT) supports the most promising 

new drug and biologic agent discovery and development projects 

through collaborations with partners in academia, government, and 

industry.

The PMI would provide clinicians with new therapies and information to 

select treatments that will work best for each patient.

Under the initiative, NCI would:

• greatly expand the number of human cancer models to facilitate the 

study of human cancer biology, drug discovery and development, 

and therapy selection

• test new therapies against some childhood cancers and common 

cancers in adults

• investigate and develop approaches for overcoming the difficult 

problem of acquired resistance to cancer treatments

While in NCI’s Center for Cancer Research, Susan 

Bates, M.D., studied a compound that would become 

the cancer drug romidepsin (Istodax®).

Credit: National Cancer Institute (Bill Branson)
24

Read the full version of this document at plan.cancer.gov

Cancer cell spheres, or spheroids, formed during 

three-dimensional culture of cells from a breast 

cancer cell line. Spheroids can be used to screen 

drugs or study cancer cell biology.

Credit: Joan Brugge, Ph.D., Harvard Medical School

Developing New Human Cancer Models

To support NCI’s anticancer drug discovery program, the institute 

established the NCI-60 Human Tumor Cell Line Screen in 1990. Tens of 

thousands of compounds have been tested using this screen, generating 

the largest cancer pharmacology database in the world.

As valuable a resource as the NCI-60 cell line screen has been, it does 

have limitations. Aside from including only a few cell lines for just a few 

cancer types, we don’t know how representative the cell lines are with 

respect to their corresponding cancer types.

New human cancer models and refinements in the methods used to 

generate them are making human cancer models resemble human 

disease more closely. Furthermore, technological advances are making 

it easier to study the biological effects of specific gene mutations and 

potential therapies in these models.

These advances have considerable potential to provide additional insight 

into:

• the development of cancer

• the role of the tumor microenvironment

• responses to new therapeutic interventions

• treatment selection

• understanding and overcoming drug resistance

These new human cancer models should enhance our drug discovery and 

development efforts and increase our understanding of human cancer 

cell biology.

With adequate funding, the institute would be able to molecularly 

characterize the tumors of every patient treated in an NCI-sponsored 

clinical trial and generate models from those tumors, greatly accelerating 

the evolution of precision medicine and its implementation into routine 

cancer care.

Harnessing the Promise of Immunotherapy

Research in cancer immunotherapy has led to the development of several 

new methods of treating cancer by restoring or enhancing the immune 

system’s ability to fight the disease.
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NCI’S ANNUAL PLAN & BUDGET PROPOSAL FOR FISCAL YEAR 2017

As researchers develop new approaches to overcoming tumor avoidance 

of immune destruction and new methods for identifying antigens on 

tumor cells that can be targeted most effectively, immunotherapy is 

becoming an integral part of precision medicine. NCI continues to 

support many grantees and intramural investigators working to treat 

cancers with immunotherapy, including:

• inhibitors of immune checkpoint molecules (proteins that normally 

limit the strength of immune responses to prevent damage to 

normal tissues)

• immune cell therapy (adoptive T-cell transfer and chimeric antigen 

receptor T-cell therapy)

• therapeutic antibodies (antibodies linked to toxic substances and 

antibodies that partner with immune cells to kill cancer cells through 

a process known as antibody-dependent cell-mediated cytotoxicity)

• immune modifying agents (cytokines and growth factors)

• therapeutic vaccines that elicit immune responses against normal 

(i.e., nonmutant) antigens that are highly expressed by cancer cells 

or “neoantigens” that are generated by mutation during tumor 

development

Although many patients are deriving benefit from immunotherapy, 

research is still needed to:

• increase our understanding of what enables this approach to work 

in some patients but not in others who have the same cancer

• find ways of expanding the use of immunotherapy to more types of 

cancer

• better understand how to use immunotherapies in combination with 

targeted therapies and standard treatments, such as chemotherapy 

and radiation therapy

Strategies for the Prevention and Early Detection of Cancer

The prevention of cancer and the development of more effective 

strategies to detect cancer precursors and early-stage cancers, when 

treatment may be most effective, remain critical goals.

Although the pharmaceutical industry regularly funds diagnostic and 

treatment research, the majority of prevention research is funded by the 

public sector. This is because preventive agents and interventions provide 

minimal or no potential for commercial profit.

Michelle Becker-Hapak, a research lab supervisor at 

Washington University School of Medicine in St. Louis, 

works on a personalized melanoma vaccine.

Credit: National Cancer Institute
26

Read the full version of this document at plan.cancer.gov

Nevertheless, prevention has the potential to save more lives from cancer 

than treatment and spares individuals the physical and psychological 

morbidity caused by cancer diagnosis and treatment. For these reasons, 

strong support for this area of research is important.

Preventing Cancer Caused by Viral Infections

Identifying infectious agents that cause cancer provides several paths to 

prevention, including reduction or elimination of exposure to the agent, 

developing a vaccine that prevents infection, or treating the infection 

before cancer develops.

Recent developments for three oncogenic viruses—human 

papillomavirus (HPV), Epstein-Barr virus (EBV), and hepatitis C virus 

(HCV)—illustrate how research advances have the potential to 

substantially reduce cancers attributable to these agents.

Further NCI-funded research aims to:

• confirm whether administering fewer than the recommended 

three doses of FDA-approved HPV vaccines will provide long-term 

protection against HPV-associated cancers

• determine whether treating high-grade anal dysplasia, which is 

associated with HPV infection, can reduce the likelihood of invasive 

anal cancer

• increase the accuracy and efficiency of cervical screening tests

• aid in the development of a prophylactic vaccine against EBV 

infection, which is thought to account for more than 200,000 new 

cases of cancer each year worldwide

• develop a prophylactic vaccine against infection with HCV, which is a 

major cause of liver cancer

Preventing Cancer with Drugs

NCI has investigated many natural and synthetic compounds to see if 

they have chemopreventive properties—properties that help reduce the 

risk of various types of cancer. The two most notable successes arising 

from this research are the approvals by the FDA of the drugs tamoxifen 

(Nolvadex®) and raloxifene (Evista®) to reduce the risk of breast cancer 

in women at increased risk of the disease.

Research has indicated that regular aspirin use may be associated 

with reduced risks of death for several types of cancer, with the 
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NCI’S ANNUAL PLAN & BUDGET PROPOSAL FOR FISCAL YEAR 2017

greatest benefit observed for gastrointestinal cancers. Currently, 

NCI is collaborating with the National Institute on Aging on a 5-year 

study of aspirin’s cancer preventive benefits and side effects, in hopes 

of providing information that will better guide the use of aspirin for 

chemoprevention.

Long-term chemoprevention of cancer with aspirin has been limited 

by concerns about side effects. Genomics and precision medicine may 

enable identification of individuals who are most likely to benefit from the 

use of aspirin or other chemopreventive agents, those who will not, and 

those who may actually be harmed by these interventions.

Consequently, NCI is supporting research in this area. One example is 

a recent study, funded largely by NCI, in which participants who carried 

specific genetic variants were found to have reduced risks of colorectal 

cancer with regular aspirin and/or nonsteroidal anti-inflammatory 

drug (NSAID) use, whereas participants with other genetic variants had 

increased risks of the disease.

Identifying New Biomarkers for Risk and Early Detection of 

Cancer

Discovering biological markers, or biomarkers, that can accurately 

identify people at increased risk of cancer or who have early cancers 

provides opportunities for medical intervention to reduce risk, for 

ongoing surveillance and deferral of treatment until necessary, or for 

early intervention when cancer therapy may be most effective.

Our ever-increasing understanding of the mechanisms of cancer, 

greatly facilitated by advanced genomic, proteomic, and other molecular 

technologies, is amplifying opportunities for cancer biomarker discovery 

and development. The potential rewards—in terms of cancer cases and 

deaths avoided—make this an extremely important area of investigation.

Although many candidate cancer biomarkers have been identified in 

recent years, few have been validated as being clinically useful. Therefore, 

guidelines and frameworks for the development and validation of 

biomarkers have recently been developed.

Current research aims to:

• discover and validate biomarkers that can help diagnose cancer at 

its earliest stages more easily, cheaply, and less invasively

• identify people at increased risk of cancer who may need to be 

closely monitored for cancer development

Breast cancer cells that overexpress the HER2 

biomarker, with the cell nuclei stained blue and the 

HER2 protein in bright red at the cell membranes. 

Trastuzumab (Herceptin®) can be used to treat 

cancers that overexpress HER2.

Credit: National Institute of Standards and Technology
28

Read the full version of this document at plan.cancer.gov

• identify biomarkers that can discriminate between life-threatening 

cancers and cancers that will not cause symptoms or threaten life 

and between precancerous growths that will progress to invasive 

cancer and those that will not

• correlate findings from studies testing various imaging modalities 

for the early detection of cancer with similar biomarker studies

Examples of biomarkers currently being developed include tumor DNA 

and cancer cell exosomes (membrane-enclosed cell fragments that 

contain proteins, DNA, and RNA) shed into the blood.

The Biology of Cancer Health Disparities

Cancer health disparities, differences in cancer incidence and outcomes 

among different racial and ethnic groups, are thought to reflect the 

interplay of socioeconomic factors, culture, diet, stress, the environment, 

and biology.

The least understood of these factors is the role of biology. But with 

advanced genomics and other molecular technologies and the growing 

sophistication of computational tools, understanding how biology 

contributes to health disparities may finally be within reach.

Until fairly recently, a major barrier to research on the role of biology 

in cancer health disparities was the lack of large samples of DNA from 

individuals across a wide range of racial and ethnic groups. Supporting 

ongoing efforts to build large repositories of samples and data for this 

research remains a priority.

NCI currently supports a broad range of research aimed at identifying 

and understanding biological differences in cancers that arise among 

different racial and ethnic populations, and how biological factors 

interplay with other factors, such as diet and the environment, in 

contributing to cancer health disparities.

Examples include research to identify:

• genetic mechanisms that explain the biological differences between 

triple-negative breast cancers that arise in African American women 

and those that arise in women of other racial and ethnic groups

• genetic mutations that appear to be preferentially associated with 

colorectal cancers that arise in African American men and women 

compared with white men and women
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• genetic variations that may explain the higher risk of prostate cancer 

among African American men compared with white men

• epigenetic differences and differences in gene expression patterns 

and other molecular characteristics that may explain differences in 

the aggressiveness of prostate cancers between African American 

men and white men

As our understanding of the role biology plays in cancer health 

disparities grows, new opportunities for prevention, early detection, and 

intervention will undoubtedly be revealed. However, as a society, we 

must also continue to explore innovative ways to mitigate the effects of 

nonbiological factors that contribute to cancer health disparities, such 

as limited access to and use of health care services, including cancer 

screening programs, and the ability to pay for care.

Melanie Nix is a survivor of triple-negative 

breast cancer.

Credit: National Cancer Institute