TESTIMONY OF MARVIN GERSHENGORN, MD CHAIR, RESEARCH AFFAIRS COMMITTEE

TESTIFYING ON BEHALF OF THE ENDOCRINE SOCIETY REGARDING FISCAL YEAR 2001 APPROPRIATIONS FOR SUPPORT OF MEDICAL RESEARCH FUNDING

BEFORE THE SUBCOMMITTEE ON LABOR, HHS, AND EDUCATION COMMITTEE ON APPROPRIATIONS U.S. HOUSE OF REPRESENTATIVES
April 5, 2000

Mr. Chairman and Members of the Subcommittee:

My name is Marvin Gershengorn and I am a Professor of Medicine at the Weill Medical College of Cornell University in New York City. I am a laboratory-based researcher and physician, with a clinical specialty in endocrinology. I teach medical and graduate students, and train these students and post-doctoral fellows in laboratory science. My research involves molecular and cellular studies of receptors. In particular, I study how receptors and their signal transduction pathways regulate cell function. Two areas of my research are in the field of endocrinology, specifically thyroid physiology and disease, and bone metabolism and osteoporosis. I also study two aspects related to cancer. I study a tumor called Kaposi's sarcoma and angiogenesis, the development of new blood vessels.

I am pleased to testify before you today on behalf of The Endocrine Society, where I serve as Chair of the Research Affairs Committee. We commend you on the outstanding leadership and support you have shown for biomedical research in the past.

The Endocrine Society, founded in 1916, consists of over 9000 scientists and physicians who are dedicated to the advancement, promulgation, and clinical application of knowledge related to endocrinology. Our members include academic researchers and educators as well as clinicians involved in the daily treatment of patients with diabetes, hyperthyroidism, hypothyroidism, osteoporosis, infertility, menopause, pituitary tumors, and other endocrine disorders. We publish four peer-reviewed journals: Endocrinology, Endocrine Reviews, The Journal of Clinical Endocrinology and Metabolism, and Molecular Endocrinology.

As an organization dedicated to the advancement of research and knowledge as well as the treatment of patients, The Endocrine Society urges the Congress continue research support with a 15% increase for the National Institutes of Health (NIH) and continued support to the Centers for Disease Control and Prevention (CDC).

Support for Training and Development of America's Researchers

This Subcommittee clearly recognizes the importance of medical research, as shown by your efforts to double the NIH budget over five years. Further increases for NIH will help science by providing more grants to support not only research, but also the development of researchers themselves. Members of The Endocrine Society rely on NIH grants to fund training programs as well as to support their research. One of our biggest challenges is attracting and supporting the work of young students training in fields of medical research. The choices made by this Subcommittee may well determine whether or not young people will choose to become medical researchers.

We are particularly concerned about the future of physician-scientists. Physician-scientists, biomedical researchers who hold M.D. or M.D./Ph.D. degrees, play a vital role in American health care and medical research. By bridging the fields of research and practice, physician-scientists translate achievements in the laboratory into treatment for patients. They combine research and clinical experience to advance the science of medicine, while educating the next generation of doctors. Physician-scientists have made enormous contributions to medical education, practice, and research.

Unfortunately, financial pressures on scientists at all stages of their careers have reduced the number of physician-scientists. Young physicians, who graduate with tremendous loan burdens, are being pulled away from research and teaching to more lucrative specialties. At the same time, experienced physician-scientists are finding less support for their research as academic medical centers come under increasing financial stress. We have fewer students aiming for careers in research and declining numbers of mentors to guide and teach those who do choose research careers. To ensure the continued supply of talented physician-scientists, we ask for your support for programs that enable scientists to pursue investigative careers. The Endocrine Society supports the proposal put forward by the Federation of American Societies for Experimental Biology (FASEB) to encourage more young physicians to pursue research and to provide continued support to current physician-scientists. This proposal contains four elements that will allow the supply of physician-scientists to meet America's research needs. We recognize that the third and fourth prongs of FASEB's proposal require enactment of substantive legislation and hope the committee report will endorse these changes.

1. Double current funding for Medical Scientist Training Programs (MSTPs). Graduates of these joint M.D./Ph.D. programs have had great success in establishing productive research careers. About one third of all NIH grants to physician-scientists are held by MSTP graduates. We thus request an appropriation of $60 million for MSTPs.
2. Provide support for the training and mentoring of early career physician-scientists by expanding post-doctoral research training programs. We seek an appropriation of $40 million for post-doctoral research programs administered by NIH.
3. Authorize NIH to institute a national program of debt forgiveness to alleviate the financial burden that prevents medical students and young physicians from entering research careers.
4. Eliminate the statutory salary caps on NIH awards to extramural investigators to make career opportunities in medical centers more attractive to physicians.

We applaud continued efforts to increase the diversity of backgrounds represented in medical research. Minority institutions and medical programs enrich the our profession by expanding opportunities to communities that historically have been underrepresented. Your support for minority medical programs and institutions nationwide will encourage the best and the brightest young people, who come from a wide variety of intellectual and cultural backgrounds, to become the independent investigators of tomorrow.

National Institutes of Health

The Endocrine Society recommends an appropriation of $20.485 billion in fiscal year 2001 for the National Institutes of Health (NIH). This 15% increase would keep NIH on track to double its budget by FY 2003, allowing a significant expansion in the number of investigator-initiated research grants that can be awarded.

America's remarkable successes in biomedical research can be attributed not only to how much funding we provide for research, but also to how we allocate our scarce resources. NIH relies on a merit-based peer review system to determine how most of its grants will be distributed. This system fosters healthy competition among scientists by requiring them to demonstrate the importance and quality of their research in order to receive funding. As funding is increased for NIH, the peer review system must remain in place to ensure that America's research dollars are allocated efficiently.

In addition to using the peer review system, we believe that funding for NIH should not be earmarked for particular diseases. Scientific breakthroughs rarely benefit only one illness, and often apply to many more conditions than expected. For example, research into the genetic causes of breast cancer resulted in the identification of a gene related to the development of brain tissue. Advances like this can be difficult to predict and could be blocked unintentionally by earmarks for certain diseases.

My field, endocrinology, provides many examples of the ways in which seemingly distinct research topics are related. Endocrinology deals with the role of hormones in regulating bodily functions. Hormone imbalances can cause diabetes, heart disease, tumor growth, infertility and birth defects. With such a wide variety in the systems affected by hormones, it is not surprising that research into endocrine disorders is conducted at many, if not all, of the 21 Institutes and Centers at NIH. My own research is supported by funding from the National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) and the National Cancer Institute (NCI). The National Institute of Child Health and Human Development (NICHD), National Heart, Lung, and Blood Institute (NHLBI), and National Human Genome Research Institute (NHGRI) are just a few of the other institutes that support research into endocrine disorders. Although these institutes appear to focus on unique fields of medicine, the work of each institute contributes to the others by enhancing our understanding of the causes and treatment of endocrine disorders. Allow me to give you a few examples of their work:

NICHD:

When NICHD was founded 35 years ago, one of its primary goals was to help people have healthy children at the time they want them. Recent projects at NICHD provide examples of the progress we can make toward this goal. One study has identified a drug that may combat the most common cause of infertility, polycystic ovarian syndrome. Another NICHD-funded project is developing a protocol for prenatal hypothyroidism screening. Testing women for hypothyroidism early in their pregnancies may help prevent mental retardation in their children. Additional funding will enable NICHD to continue its exciting progress in helping all hopeful parents have healthy babies.

NCI:

Studies funded by NCI have enhanced our knowledge about the interaction between hormones and genes. One recently reported study built off research tools developed by earlier researchers to track the movement of receptors, molecular configurations that bind to hormones, to and from genes. The research revealed that, rather than binding to one spot, receptors take a "hit and run" approach to genes, further advancing our comprehension of how hormones trigger reactions in genes.

Other NCI projects have examined the relationship between hormone replacement therapy (HRT) and breast cancer. One study, whose results were released in January, revealed that use of a combined estrogen-progestin replacement therapy is associated with a greater risk of breast cancer than use of estrogen alone or no HRT. The study's results also suggest that short-term use of HRT is not associated with increased risk of breast cancer. The study's results provide women and their physicians with more information to help weigh the risks of hormone replacement therapy against its benefits.

NHLBI:

One of the suspected advantages of hormone replacement therapy is its ability to prevent coronary heart disease (CHD). The National Heart, Lung, and Blood Institute supports several randomized trials examining the effects of HRT on heart disease. When completed these trials should provide the first comprehensive evaluation of the benefits and risks of long-term hormone replacement therapy.

NIDDK:

The National Institute of Diabetes and Digestive and Kidney Diseases has supported landmark studies into the causes and treatment of diabetes. Researchers at NIDDK have made significant progress toward identifying the genetic causes of diabetes. By identifying at-risk people early, researchers hope to apply preventative measures to stop or limit the destruction of insulin-secreting beta cells. For people who currently are living with type I diabetes, the Diabetes Control and Complications Trail showed that careful control of blood sugar levels can have long-lasting benefits. By comparing the long-term outcomes of patients who used intensive treatment to those who received conventional diabetes treatment, the trial found that intensive treatment delayed or prevented diabetic eye, nerve, and kidney complications. These findings demonstrate the need for intensive therapy to begin earlier and continue longer, and lend support for efforts to improve glucose-monitoring and insulin-delivery systems.

NHGRI:

Knowledge about the human genome has become key to understanding many diseases. As you can see from the examples I have already given of projects at other Institutes, the National Human Genome Research Institute has contributed to research throughout NIH by developing research tools that allow scientists to evaluate the human genome. Additionally, the map of the human genome being produced by researchers funded by NHGRI has identified genes that cause endocrine disorders. For example, scientists at NHGRI discovered a gene, AIB1, that is linked to the estrogen response pathway. Further study of this gene may reveal the basic biology of tumor cells that are responsive to steroid hormones. This will expand our knowledge of ovarian and prostate cancer as well as breast cancer.

By continuing on the path to doubling the budget for NIH, you will allow NIH to support more important research projects, such as these examples, that give so much back to our country in terms of scientific progress and improved health care.

Centers for Disease Control and Prevention

The Centers for Disease Control and Prevention (CDC) has the expertise to translate NIH research into better outcomes in public health. One program that combines the strengths of NIH and CDC is the National Diabetes Education Program (NDEP). This joint program of NIDDK and CDC involves both public and private partners to improve the treatment and outcomes for people with diabetes, to promote early diagnosis, and ultimately to prevent the onset of diabetes. NDEP's goal is to reduce the morbidity and mortality of diabetes and its complications by increasing public awareness regarding the risk factors and potential prevention strategies for diabetes, promoting diabetes self-management behaviors, educating health care providers about diabetes and its control through an integrated approach to care, and promoting health care policies that improve the quality of and access to diabetes care. The Endocrine Society enthusiastically supports NDEP and its program goals and objectives. We believe that NDEP should serve as a model for other cooperative, innovative programs that translate research breakthroughs to improved public health outcomes. Unless CDC's budget is increased together with NIH's budget, we will not reap the true benefits of NIH's exciting research breakthroughs.

Conclusion

The Endocrine Society appreciates this opportunity to testify on these very important appropriations issues. We are at an exciting threshold in biomedical research. With the continued strong support and leadership of this Subcommittee, we can seize the opportunity to make an investment today that will continue to realize great dividends for our nation in the many decades to come.