Testimony delivered before the U.S. House Subcommittee on Labor-HHS-Education Appropriations, to discuss the NCI budget request for Fiscal Year 2008
John E. Niederhuber, M.D.
Director, National Cancer Institute
March 6, 2007
Mr. Chairman and Members of the Committee:
I am pleased to present the President's budget request for the National Cancer
Institute (NCI) of the National Institutes of Health (NIH). The Fiscal Year (FY) 2008
budget includes $4,782,114,000, a decrease of $9,094,000 below the FY 2007
annualized continuing resolution of $4,791,208,000, comparable for transfers proposed
in the President's request.
INTRODUCTION
I am most pleased to be before you today to report on the Nation's progress on
cancer research. While there has been a steady decline in the cancer mortality rate since
1991, we now have the excellent news that for the second year in a row, there was a
decline in the absolute number of cancer deaths. This notable decline in actual deaths
due to cancer is more significant considering the aging of our population and the
increasing number of citizens. The remarkable findings were highlighted by President
Bush during his visit to the National Institutes of Health (NIH) campus in January which
for the first time focused on the impact of cancer research. But we are not done - many
citizens continue to feel the pain of the devastating news of a diagnosis of cancer or the
loss of a loved one.
While the declining rate of deaths due to cancer is tremendous news, this change in
cancer mortality also has a significant economic impact. It has been projected that even
just a 1% decrease in cancer mortality will result in a $500 billion impact on the U.S.
economy (Murphy, K. and Topel, R., Journal of Political Economy, 2006; 114(5),
871-904). What is not as evident to the citizens of our country is the impact of cancer
research on our understanding of many other diseases. Cancer continues to be a model
system for other diseases. For example, the study of angiogenesis (blood vessel
development) associated with tumor growth has been applied to a greater understanding
and treatment of macular degeneration, ischemic heart disease, diabetic wound healing,
endometriosis and neurodegenerative illnesses. Advances in cancer accelerate progress
in other diseases. The identification of the AIDS virus, the development of assays to
screen banked blood for the AIDS virus and the current therapy was developed at the
National Cancer Institute (NCI).
The NCI is leading the way on a number of fronts to identify the genetic, molecular,
and cellular mechanisms associated with cancer. Building upon the sequencing of the
human genome, working in our newly developed "Center for Human Cancer Genomics"
NCI is systematically identifying all the important genetic alterations that are inherited
or acquired that contribute to cancer susceptibility. We are not just cataloguing the
genetic changes and other factors involved in a normal cell becoming malignant, but
applying this knowledge to identify people at increased risk for developing cancer, to be
able to prevent cancer and/or to detect cancer at its earliest stage of development when
there is the best hope for effective therapy, and to identify new targets for highly
selective and specific therapeutic agents.
We are already seeing the result of gene profiling applied to patient care. An
example is in non-Hodgkins lymphoma, where tumors that look the same under the
microscope actually have very different characteristics when their DNA is analyzed.
These genetic differences have been shown to correspond to good versus poor prognosis
for the patients. Using these genetic signatures, treatments are now tailored to a
particular patient and his/her tumor.
NCI must continue to make progress for the cancer patient. We must bring the best
science to the patients, the vast majority (85%) of whom is treated in community-based,
private practice oncology settings. With that in mind, NCI is launching the Community
Cancer Centers Program (NCCCP) pilot. This pilot project will study how best to
provide easily accessible, state-of-the-art, multispecialty cancer care and earliest phase
clinical trials research to patients in the communities where they live. Through this
program we will learn best how to educate patients concerning risk, healthier living,
screening practices, clinical trial participation, and survivorship issues.
A RECORD OF REAL SUCCESS
The past year in cancer research and development shows a record of substantial and
heartening achievement. We are expanding our knowledge and the technology tools to
understand the mechanisms of cancer. Importantly, we are seeing these advances
rapidly applied to patient care.
- An important public health milestone was reached last June when FDA approved a
vaccine that prevents infection by the two types of the human papillomavirus (HPV)
responsible for up to 70 percent of cervical cancer cases worldwide. Our Nation's
strong commitment and investment in cancer research at NCI led to this approval,
something in which we all can take great pride.
- NCI opened the first-in-human "Phase 0" clinical trials program in July 2006. The
Phase 0 trial, a step before the classic Phase I level of drug study, measures the
activity of a new drug in a small number of patients using a single, small dose of
the study agent prior to the traditional dose escalation, safety and tolerance studies.
The purpose is to get data upon which to design protocols based upon observed
activity in a patient; for example, determining the concentration of drug that gets
into the blood or actually seeing if an agent is getting to the specific target in the
tumor using imaging technology. This new step in speeding up the process of drug
development and approval is linked to our transition from highly toxic
chemotherapies to the new era of highly characterized tumors and specific, less
toxic molecularly targeted agents. This new paradigm in early phase clinical
testing will substantially compress the drug development time.
- NCI has long been at the forefront of research and development of biomarkers for
use in diagnosis and treatment for cancer. A Biomarkers Consortium was
launched this year which includes participants from the Foundation for the NIH,
NIH, FDA, CMS, and private industry with the goal of validating biological
markers for a variety of diseases, including cancer. The first project approved by
the Consortium is the evaluation of an imaging agent that detects an increase in
cell metabolism characteristic of tumor growth. NCI is conducting trials in lung
cancer and non-Hodgkin's lymphoma that use this ability to view cellular
metabolism to monitor tumor masses for increased activity (cell growth) or
decreased activity (cell death).
DELVING DEEPLY INTO THE CANCER CELL ENVIRONMENT
At the NCI, we have pioneered efforts to identify all the genetic alterations that are
inherited and that predispose a person to be at increased risk for developing cancer. The
Cancer Genetic Markers of Susceptibility (CGEMS) project is identifying the common
genetic changes that contribute to prostate and breast cancer risk using single nucleotide
polymorphism (SNP) analysis or small genetic regions showing changes associated with
malignancy. A similar analysis for pancreatic cancer is just beginning. The ability to
identify at risk patients or to detect the disease at its earliest stage of development has
been proven to provide the best hope for effective therapy and high quality of life.
Similarly, NCI and the National Human Genome Research Institute have launched
The Cancer Genome Atlas (TCGA) to determine the feasibility of using large-scale
genome analysis technology to identify important genetic changes involved in cancer.
A TCGA Pilot Project is studying lung, brain (glioblastoma), and ovarian cancers
which, collectively, account for more than 210,000 cancer cases each year in the United
States.
Other initiatives are expanding our study of not only the cancer cell but the
networks and the cellular microenvironment that also appear to be significantly involved
in tumor development and metastasis. These studies of molecular carcinogenesis are
being conducted at the single cell or subcellular level using high-resolution, three-dimensional
electron microscopy. These technologies allow us to look within the
nucleus to study differences in chromosome movement and location during stages of
abnormal cell growth.
There is increasing evidence that cancer "stem cells" or "cancer initiator" cells are
the driving force behind many cancers and are the basis for long term risk. The presence
of such cells, first demonstrated in acute myeloid leukemia patients, provides a different
and exciting model with which to further explore cancer biology. NCI is establishing a
trans-NIH group of scientists interested in embryogenesis and cancer stem cell biology
to advance the study of the underlying mechanisms in these processes.
TRAINING THE NEXT GENERATION OF CANCER RESEARCHERS
Cancer is one of the most exciting and innovative areas of medical research. It takes
a superbly trained, highly effective workforce to make these discoveries, to translate
them into new interventions, and to put the improved knowledge base and these cutting-edge
tools to work for patients. NCI will continue to play an important role in
developing the cancer research workforce in the United States and in other countries.
We stand firmly by the Institute's commitment to provide unparalleled training
opportunities for talented researchers from a wide variety of disciplines to advance their
careers. In fact, many of the current programs at NIH had their origin in the NCI.
Of special significance, are minority training programs which encompass the
continuum from talented high school minority students through programs, including the
Continuing Umbrella of Research Experiences (CURE), that direct long-term funding to
qualified minority students interested in scientific, cancer research-related careers.
ADVANCED TECHNOLOGIES ACCELERATE PROGRESS
It is clear that the area of advanced technologies development is absolutely essential
and critical in creating tools for speeding up and enabling the discovery process. In
addition to the genomic technology projects (CGEMS and TCGA) already mentioned,
NCI is investing in the development of critical technology platforms in a number of
other strategic areas - such as nanobiology, proteomics and computational biology.
The NCI, in recognition of the key role of biospecimens in all of biomedical
research as well as cancer research, has led a pioneering effort to provide the first
guidelines that standardize and enhance specimen collection and biorepositories. Using
these guidelines, NCI has developed a common biorepository infrastructure that
promotes resource sharing and enables data comparison among research laboratories
while ensuring patient protection and ethical integrity.
We also believe that advanced imaging technologies will play a significant role in
the prevention and preemption of cancer as well as in making "go or no-go" decisions
for early oncologic drug development. The NCI is working now in the subcellular space
to be able to view in real time drug-cellular target interaction and functional change
secondary to such interactions. The NCI is developing new targeted and non-targeted
molecular imaging agents for use as lymphatic markers, angiogenic markers, and
surrogate markers for drugs that enhance quantitative methods to measure early, real-time
tumor response.
INTERAGENCY COLLABORATIONS
Addressing the cancer problem requires that NCI work across institutional and
sector boundaries, share knowledge, and bring together the diverse members of the
Department of Health and Human Services (DHHS) family of agencies, as well as other
federal offices, and the private sector, in partnerships that can help develop systems-based
solutions to the cancer problem. Last year marked the launch of an unprecedented
public-private research partnership in the Biomarkers Consortium that was described
previously.
The joint NCI-FDA Interagency Oncology Task Force (IOTF), which was
established in 2003 to enhance and accelerate the overall process of developing new
cancer interventions, released two new guidance documents and a final rule intended to
streamline the early clinical development of new drugs and biologics for cancer and
other diseases. This has enabled the first Phase 0 trial described previously.
REACHING THE PATIENT AND COMMUNITY
The advances being made in these and many other areas offer real hope for
continuous progress. However, even the new progress report based on NCI-SEER data
from the American Cancer Society (ACS) confirms that minority and low-income
populations shoulder a disproportionate cancer burden and aren't benefiting equally
from these important advances. One way NCI is attempting to address this problem is by
bringing the results of genomic and proteomic science to patients where they live
through the NCI Community Cancer Centers Program (NCCCP) pilot, with the hope of
broadening access to clinical trials and to cutting-edge prevention, diagnosis, and
treatment interventions.
Through partnerships with NCI-designated Cancer Centers, the NCCCP's goals are
to expand access to cancer prevention, screening, treatment, survivorship follow-up, and
end-of-life care, as well as increasing participation in early phase clinical trials and
reducing healthcare disparities in community hospital-based settings where 85% of our
patients actually receive their treatment.
There is great cause for optimism, but an optimism that should be tempered by an
understanding of the very real hurdles to progress we still face. These are challenges that
we must address as a community. In doing so, such encouraging trends will become the
rule, not the exception.
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