107th Congress

Report: FY 2003 Appropriations Theme Hearing: "Bench to Bedside"—Hearing Before the House Appropriations Subcommittee on Labor, HHS, and Education—March 14, 2002

Members Present

Ralph Regula (R-OH), Chairman; Dan Miller (R-FL); John Peterson (R-PA); Nancy Pelosi (D-CA); Jesse Jackson, Jr. (D-IL)

Witnesses

Ruth Kirschstein, Acting Director, National Institutes of Health (NIH); Andrew von Eschenbach, Director, National Cancer Institute (NCI); Anthony S. Fauci, Director, National Institute of Allergy and Infectious Diseases (NIAID); Allan Spiegel, Director, National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK); Richard Hodes, Director, National Institute on Aging (NIA); Audrey Penn, Acting Director, National Institute of Neurological Diseases and Stroke (NINDS). Also in attendance was Kerry Weems, Acting Deputy Assistant Secretary, Office of the Assistant Secretary for Budget, Technology, and Finance, DHHS.

Purpose of Hearing: This hearing was the second NIH fiscal year (FY) 2003 appropriations hearing and the first of five theme hearings. The hearing was held to highlight progress and plans in research addressed by the five institutes and to describe activities aimed at translating research knowledge into clinical practice. All witnesses appeared on a single panel.

Summary

Institute directors from NCI, NIAID, NIDDK, NIA, and NINDS provided updates primarily on research advances on cancer, diabetes, vaccines for HIV and other infectious diseases, Alzheimer's disease, and Parkinson's disease, respectively, and discussed the translation of new research results to patients. Both basic and clinical research were said to be important to our understanding of the cellular and disease processes, in development of therapies and vaccines and in discovery of new questions rising from clinical studies and trials.

Although Drs. von Eschenbach, Fauci, Spiegel, Hodes, and Penn received questions specifically targeted to activities of their respective institutes, several Members asked general questions of the entire panel. Both Representatives Regula and Peterson were interested in NIH activities related to dissemination of research information to health care providers and the public. Each of the directors cited multifacted approaches, including web sites, information clearinghouses, town meetings, radio and print media, community meetings, partnerships with schools, and other venues, as means used by their respective institutes to disseminate information. They also noted programs targeted to specific populations in which certain diseases are a particular problem. Representative Regula also was interested in the role stress plays in health. It was acknowledged that chronic stress may have some role in disease development and progression and may affect immune response; however, it was said we should be cautious in evoking stress as a cause of disease as other agents might be at play. It was also said that some stress responses actually may be healthy.

Representative Miller referenced a recent newspaper article discussing two papers on the usefulness of adult stem cells. He asked for comments on where we stand on stem cell and therapeutic cloning. Dr. Kirschstein responded that NIH has mechanisms in place to fund research on the approved stem cell lines, those which were available prior to the President's August 9, 2001, announcement. Regarding the two papers, Dr. Spiegel said research on adult stem cells is a controversial and rapidly evolving area of science, particularly regarding the potential of these cells to turn into many different types of cells. He said the papers, which have contrasting viewpoints, reflect the scientific uncertainty regarding the potential plasticity of adult stem cells versus the unequivocal potential of embryonic stem cells. With respect to therapeutic cloning, Dr. Kirschstein said the Administration has made its position very clear and that NIH supports the Administration's position. She noted a recent National Academy of Sciences report that said there is sufficient scientific potential to go forward on that work, although it equally mentions the need for further broad discussion of moral and ethical issues.

Statements of Witnesses

Dr. von Eschenbach began his remarks by applauding the commitment and leadership of both Drs. Ruth Kirschstein and Alan Rabson, Deputy Director, NCI, over their respective 46 year careers. Regarding NCI activities, he noted that budget increases have allowed the institute to pursue an aggressive path of research. Although there has been much progress, much remains to be done. He spoke of the decline in cancer death rates, the knowledge gained from both laboratory and clinical research, and the application of research results to the people. He noted, in particular, studies on cancer metastasis and cell surface receptors that were taken from the clinic to the laboratory. He said that future research dollars will support taking this knowledge from the laboratory back to the clinic and may enable development of interventions to halt the spread of certain cancers.

Dr. Fauci briefly described NIH's role in vaccine development and the important cost benefits that vaccines for infectious diseases provide. He highlighted steps in vaccine development from basic research to clinical evaluations, and said vaccine development would be at the top of everyone's short list of contributions NIH research makes in cost-benefit to the health of the nation's people. He cited four recent vaccines, hepatitis B, Haemophilus influenza, acellular pertussis, and pneumococcus conjugate, which were developed at NIH in conjunction with industrial partners. He also described their potential for reducing disability and saving lives. Dr. von Eschenbach also noted NIH vaccine research priorities—AIDS, malaria, tuberculosis, and vaccines for anthrax, smallpox, and ebola, all potential agents of bioterrorism.

Dr. Spiegel noted that NIDDK's mission includes research on a wide array of chronic diseases. He spoke of diabetes research, in particular, noting progress in understanding the disease process that, if left unchecked, can lead to blindness, amputations, kidney failure and death. He noted fundamental research that has lead to the discovery of approaches to block damage to insulin-producing beta cells of the pancreas, an important step in preventing Type 1 diabetes. Dr. Spiegel also briefly discussed the results of a diabetes prevention trial showing that modest changes in diet and exercise can help prevent Type 2 (adult onset) diabetes. Translation of this discovery could potentially prevent onset in millions of people who might otherwise be at risk.

Dr. Hodes discussed progress in Alzheimer's disease (AD), including basic research that has identified a number of risk factors for AD, including mutated genes, and led to development of mouse models for the disease. Gene insertion studies in this model may lead to the development of therapies for AD. He said budget increases have enabled the institute to fund a number of clinical trials, including seven large-scale trials. Dr. Hodes highlighted research on beta amyloid plaques that develop in the brains of AD patients and noted the identification of two enzymes that work in concert to create these plaques. This improved understanding may lead to improved treatment options for AD patients.

Parkinson's disease (PD) was cited by Dr. Penn as one of the important debilitating diseases studied by NINDS. She noted the development years ago of L-dopa to replace dopamine loss in PD patients as a significant discovery, but she said it is not a perfect therapy. She described the recent identification of specific key targets deep in the brain that can be stimulated by microelectrodes, and noted the significant relief this provides to PD patients. Dr. Penn also noted investigations into mechanisms of degeneration and cellular roles of certain proteins as leading to important new information. NINDS is funding 11 Udall centers focused on PD. Dr. Penn said the rate of progress is impressive and that current therapeutic strategies arose from fundamental bench research cross-fertilized from applied research on other neurodegenerative diseases. Dr. Penn said that, although basic research is an important step on the path to clinical trials, clinical studies also must continue to help identify new questions for basic research to address.

Questions

Representative Regula:

When was NIH started? What was the genesis?

If I put a cancer cell on a slide and another cell on a slide, what would be the difference? Do you know what makes the difference happen? Does a cancer cell start as a good cell? Do we all have some cancer cells in our bodies? Your mission is to monitor the cancer cells to keep them from changing? The new therapy is to control it rather than excise it? The killing mechanism is that it crowds out other cells?

I saw a recent television program where they took an AD gene from a cell? Can you tell us about that?

How do 250 million Americans gain access to your information?

How are your information booklets distributed?

If you had a publication on what information you have and you worked with the Administration on Aging, these could be distributed by Meals on Wheels to older Americans. You could include information on stroke, for example, and they could check the box and send it in. Would that work?

You spoke of the intramural program, do you move information back and forth through NIH? Is there a process?

What role does stress play in health?

You have a tough job on translating research results internationally. What are the barriers on a global level to translation of research results? Do all of you have contacts with institutions beyond our borders? Do you get good responses? Do you gain from interactions with these countries? You have a regular free flow of information? Do you develop drugs or is this done by industry? Is it the role of the NIH? Do you have contractual arrangements with these drug companies?

I would like each of you to tell us how you disseminate information.

Representative Miller:

(Noting a newspaper article about closure of a bookstore that specializes in books on Russian studies) do you see your successors talking about these same issues in 2012? Where are we headed? Will you be out of business?

What's in the pipeline for HIV therapies?

Whose responsibility is it to get the wORD out (about research advances)?

The last big advance (in HIV/AIDS) was development of protease inhibitors several years ago. What's new?

Regarding stem cells and therapeutic cloning, where do you stand on these issues?

Representative Peterson:

As the Administration and Congress push for more research funding, do we have a way to measure the impact on people? There seems to be a disconnect in what we know and the health status of the nation. All this information is not being used by the public.

Most Americans do not benefit from what you're doing; they don't understand that what you do can affect your health. Isn't that correct?

I suggest you engage the health care community. How are patients to make lifestyle changes if their doctors won't talk to them about obesity?

Representative Pelosi:

What are you doing on brain tumors?

In your budget, are you getting additional funding for bioterrorism or will you have to get money from other areas?

Although the number of new HIV infections has decreased overall, the number in young gay men and minority populations is increasing. Can you address what we need to do? The new HIV vaccine is promising?

Regarding cancer and the environment, what is being done to look at environmental causes?

Representative Jackson:

Stroke disproportionately affects minorities, particularly African Americans. Can you tell us how NINDS is addressing minority health disparities and stroke?

The Diabetes Research Working Group has provided an important roadmap for diabetes research. What are you doing to update this?

Are there any cancer centers at minority serving institutions? What are NCI's plans to address prostate cancer in minorities? What about training at minority serving institutions?

Prepared by Roz Gray/OD/OLPA, March 21, 2002