107th Congress

FY 2002 House Appropriations on Chronic Diseases -- National Institutes of Health -- House Appropriations Subcommittee on Labor, HHS, and Education -- March 28, 2001

Members Present

Representatives Ralph Regula (D-OH), Chairman; Rosa DeLauro (D-CT); Kay Granger (R-TX); Steny Hoyer (D-MD); Ernest Istook (R-OK); Jesse L. Jackson, Jr. (D-IL); Patrick Kennedy (D-RI); Nita Lowey (D-NY); Dan Miller (R-FL); Anne Northup (R-KY); Nancy Pelosi (D-CA); John E. Peterson (R-PA); Don Sherwood (R-PA); Roger Wicker (R-MS).

Witnesses

Ruth Kirschstein, Acting Director, National Institutes of Health (NIH); Claude Lenfant, Director, National Heart, Lung, and Blood Institute (NHLBI); Allen Spiegel, Director, National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK); Jack McLaughlin, Acting Director, National Eye Institute (NEI); Lawrence Tabak, Director, National Institute of Dental and Craniofacial Research (NIDCR); Stephen Katz, Director, National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS); and Stephen Straus, Director, National Center for Complementary and Alternative Medicine (NCCAM).

Summary

Chairman Regula welcomed Dr. Kirschstein and her team. He commented that in the last two weeks they had listened to approximately 175 public witnesses-many asking for funding for NIH to find a cure for their diseases. He said NIH has had many successes and he looks forward to many more to come.

Dr. Kirschstein expressed delight in recently hosting a visit to NIH by Mr. Regula and other members. She stated that six institutes were chosen to present at this hearing though many more work on chronic diseases. She noted that all diseases are increasing in prevalence as life expectancy increases. Mr. Regula asked for a definition of "chronic". Dr Kirschstein replied that as opposed to acute disease, like the cold, chronic diseases persist and exacerbate by recurrence. Opening statements were then made by each of the six institute directors, followed by questions from the subcommittee.

Opening Statements

NHLBI: Dr. Lenfant gave his opening statement first. He noted that NHLBI has responsibility for a wide range of diseases-nearly all of them are chronic. He highlighted coronary heart disease, that now many people are treated and live longer lives, but recurring heart attacks eventually lead to heart failure. Many people with diabetes are dying from heart disease as well. He mentioned that NHLBI is pursuing new therapies such as gene therapy. Finally, he added that it is important to focus on prevention, treatment of disease and capitalizing on new scientific opportunities.

NIDDK: In his opening remarks, Dr. Spiegel stated they support a wide range of chronic diseases. Specifically mentioned was Type 1 and 2 diabetes and identification of a gene "resistin" which causes insulin resistance. They are funding several clinical trials to look at lifestyle and drug treatments. Heart disease is the leading cause of death in diabetics. There is an ongoing effort with NHLBI to understand and treat heart disease. The increased funding puts them in a good position to find the causes of diseases and then the treatments to cure them.

NEI:Dr. McLaughlin focused on chronic eye diseases. He made the following points: 1) diabetic retinopathy is the leading cause of new cases of blindness, 2) the aging process poses problems with the onset of macular degeneration, and 3) glaucoma is the number one cause of blindness in African-Americans. NEI is supporting clinical trials to look at this disease. Each of these diseases can cause blindness. Health education and communication are important for increasing awareness.

NIDCR: Dr. Tabak stated that at one time or another, we have all experienced some form of oral disease. He described NIDCR promoting research that will improve the general health of the public and said that research is ongoing in many areas. He said the NIDCR is supporting development of an artificial salivary gland. Initiatives are planned to research better ways to prevent and manage oral manifestations resulting from cancer treatment. In addition, plans are underway to host a consensus development conference on the diagnosis and management of tooth decay and periodontal disease. He noted that NIDCR also focuses on chronic pain and temporomandibular disorders. He said that clinical trials are underway to look at conservative versus surgical interventions, with preliminary findings indicating that costly surgical interventions offer no increased benefits. Dr. Tabak concluded by saying that without oral health, one couldn't be completely healthy.

NIAMS: Dr. Katz opened by stating that NIAMS supports research in a broad array of diseases. He specifically mentioned that for osteoporosis there have been considerable advances in research and that this past year new drugs have come out that help manage the disease. He noted that other new drugs-biologicals-have provided considerable relief for those suffering from rheumatoid arthritis. Dr. Katz mentioned that NIAMS is working with NHLBI and other institutes to support research in a number of other chronic and disabling autoimmune diseases. He also mentioned a collaboration with other ICs to support further research on muscular dystrophy.

NCCAM: Dr. Straus was the last to provide opening remarks. He said that NCCAM is working in partnership with other ICs to explore complementary and alternative (CAM) approaches such as acupuncture and EDTA for those with debilitating and chronic diseases. He noted that for arthritis pain there is a very large study comparing acupuncture with a placebo to evaluate the long-term outcomes and cost-effectiveness of the acupuncture intervention. He highlighted other NCCAM studies focused on a Chinese herbal cocktail used for prostate cancer, soybean extracts for post-menopausal women, and alternative therapies used for dealing with the side effects and pain caused by cancer treatment.

Questions

Mr. Regula

Mr. Regula led the questioning. He stated that a list of Q's had been prepared. He would like to go around the committee, then if time permitted, go around again. He stated that questions for the record needed to be submitted to his office by noon on the following day. It was agreed that questions could be submitted on the subject of chronic disease for IC Directors not present.

NEI:. Mr. Regula asked if there has been research on the long-term effects of laser surgery. Dr. McLaughlin replied affirmatively that studies have been industry supported, and in long-term follow-up, the results are persisting. Mr. Regula asked if it is true that the eye gives one clues to their overall health. Dr. McLaughlin replied that eyes are the windows to the brain and many body conditions. The first signs of diabetes and arthritis can be seen through the eye.

NCCAM: Mr. Regula inquired how NCCAM is evaluating and informing the public about alternative medicines. Dr. Straus responded that we have the scientific tools to evaluate and that we ask the same questions as you would for a new device or procedure. He said the NCCAM's goal is to get answers that are clear and compelling and then to distribute results of the data via the Internet, an 800-phone number and town meetings.

Mr. Wicker

NCCAM: Mr. Wicker asked about initial impressions of chelation therapy. Dr. Straus replied that NCCAM is about to call for applications for a large controlled trial. In the past there have been small studies that have not shown benefit.

NIH OD: Mr. Wicker asked Dr. Kirschstein about stem cell research. He said that Congress has been very supportive in the effort to double the budget at NIH, but with controversial ethical problems such as embryonic stem cells, it is harder to gain widespread support. He referred to reports stating that non-embryonic cells can be just as beneficial. He said there is great promise in getting stem cells from other sources such as bone marrow, blood, spleen, and umbilical cORD and wondered why embryonic stem cells are emphasized so heavily. Dr. Kirschstein replied it is important to do the research to get scientific evidence, and there is great promise from all sources of stem cells. She said that NIH has not to date supported research using stem cells derived from embryonic cells. She said that no research has been done in parallel using both adult and embryonic stem cells to make a determination and that a comparison study must be done. She noted that promising research continues with adult stem cells. Mr. Wicker asked if embryonic stem cell research is being done in the private sector and suggested that the government leave the more controversial research to the private sector. Dr. Kirschstein replied that human embryonic stem cell research is occurring in the private sector, but that NIH doesn't receive information unless it is published in scientific journals, and it is not clear if the work done in the private sector is done with the same scientific rigor required with public funds.

Mr. Sherwood

NHLBI: Mr. Sherwood noted that part of the Healthy 2010 goals includes working on translation of research to the bedside and asked how NHLBI is applying its research to treatment of patients. Dr. Lenfant replied that many findings have been translated to patients, but not as many as he would like. He noted that guidelines on risk factors continue to be developed, and that the National Heart Attack Program has been successful in reducing the time to access care. Dr. Lenfant mentioned a new sleep disorder program in which NHLBI uses Garfield the comic-strip cat to get the message to elementary school children.

Ms. Pelosi

NIAMS: Ms. Pelosi asked if there was progress to report on two diseases affecting women- Scleroderma and Ehlers-Danlos Syndrome. Dr. Katz replied that NIAMS views itself as an institute of women's health, that many autoimmune diseases affect women 9 to 1. He said NIAMS is working closely with Dr. Pinn in ORWH. For scleroderma, NIAMS has had national and international meetings to identify research opportunities/needs in the community; they have received 28 applications to address the many aspects of scleroderma. For Ehlers-Danlos Syndrome, NIAMS is meeting with several organizations and scientists to identify scientific opportunities to include therapeutic intervention, and encouraging scientists to apply to do research in this area.

Ms. DeLauro

NHLBI: Ms. DeLauro expressed concern about the enormous toll of heart attack and cardiovascular disease on women. She asked Dr. Lenfant to address the controversy over differences between men and women on how they are diagnosed. Dr. Lenfant stated that we do know quite a bit, however, there have been barriers in translating treatment to women. He said that we need to be sure women and their physicians know what needs to be done and NHLBI is working on this at a conference this week.

Ms DeLauro applauded the "Wise Woman Program" and mentioned that Connecticut rates among the worst in asthma cases. She wondered what is being done in the area of exposure of children and what is being done with CDC. Dr. Lenfant replied that the gold standard treatment for children-inhaled corticosteroids-is safe. He also responded that they are working on asthma with many agencies including the CDC and EPA.

Mr. Hoyer

NIH OD: Mr. Hoyer asked Dr. Kirschstein to respond to the impact of funding increases that have occurred over the last 3 years on pay lines for each IC, on science, and on young new scientists going into biomedical research. Dr. Kirschstein responded that there has been a sense of continuity and stability in the biomedical research community and that, in general, about one-third of applications are funded. She stated that there has been an increase in applications, both by new applicants and existing grantees. Dr. Katz added that there had been a concern about young people being able to compete for funds. The funding increase has helped support K grants so there is more opportunity for young researchers. In addition, the K-24, for example, has helped mid-level scientists. Mr. Hoyer asked that the pay line for the ICs, particularly the low and the high, be provided for the record.

NIA: Mr. Hoyer asked about the effect of cholesterol lowering drugs on Alzheimer's disease. Dr. Kirschstein suggested he save that question for the next hearing on Life Span, when Dr. Hodes would testify.

Mr. Jackson

Mr. Jackson stated he would submit all of his Q's for the record and wanted to make a statement on stem cells. He said he understands the dilemma of stem cells and that he debated these issues while at seminary school. But he said he believes in using government money to fund good research. He stated that the prohibition is for government research, not private research. It serves to protect advances for the private sector and increase costs to the general public. If it is to be done at all (public or private), it should be done with careful oversight of the government.

NHLBI: Mr. Jackson asked how the increase in the budget has affected NHLBI, specifically for research in sickle cell disease. Dr. Lenfant responded that a core component of the research is ten centers that study sickle cell disease, as well as a number of clinical trials. Mr. Jackson expressed concern that the ten center grants are about to expire and will be renewed for 5 years but they presently don't include a minority institution. He asked if NHLBI provides any assistance to help minority serving institutions to compete. Dr. Lenfant replied that they are working with minority institutions, encouraging them to apply. He also stated that the awards are made based on scientific merit and that they are ensuring the review boards have a large minority representation. Mr. Jackson emphasized he assumes good science will be funded but is concerned by the lack of minority representation.

Ms. Lowey

NCCAM: Ms. Lowey stated she looks forward to hearing the results of efforts to look at the efficacy of acupuncture. She is interested in knowing if it is effective in providing pain relief, and also if it can target the cause of pain. Dr. Straus responded that a large share of the NCCAM portfolio is looking at pain. They are looking at the responses in the brain. Acupuncture seems to quiet pain and is registered in the mind. He said that many small studies have been done, but NCCAM is looking at larger studies to get compelling evidence in order to make recommendations to the public.

Ms. Lowey stated that research is beginning on alternate therapies for cancer. She asked if alternate remedies could be used in primary treatment. Dr. Straus replied that cancer affects patients so seriously. They become so desperate they resort to any kind of treatment. Currently NCCAM is funding a study of a dietary approach to treating pancreatic cancer. They are partnering with NCI to look at alternative approaches to primary treatment.

NIAMS: Ms. Lowey asked about glucosamine in treatment of arthritis. Dr. Katz responded that glucosamine is being used for relief and there is new evidence that it helps. They are partnering with NCCAM on osteoarthritis; the study is longer term and more rigorous to look at pain as well as functional benefit and will take another 2 years to get results. Ms. Lowey responded that she thought the idea was beyond relieving pain but to rebuild cartilage. Dr. Straus commented that they are doing studies to see how it works.

Mr. Regula

NIAMS: Chairman Regula asked how many suffer from arthritis. Dr. Katz replied that there are over 100 forms of arthritis. One kind, rheumatoid arthritis, occurs in over 2 million people. Arthritis affects about half of the population over 65 years of age.

Mr. Peterson

Mr. Peterson said he was delighted to be on the Subcommittee. He said he was sorry he missed the tour but hoped there would be another opportunity for those that didn't get to make the last tour.

NIDCR: Mr. Peterson asked Dr. Tabak if he agreed with public witnesses' statements recommending increasing clinical research now. Dr. Tabak stated he supported having more clinics, that findings have matured and are now ready for more clinical research. He stated he would provide a percentage for the record.

NIAMS: Mr. Peterson stated his wife is taking pain medication for arthritis and has had several side effects. He stated they tried a new medicine and she felt even sicker. He wondered if he should report these side effects to the company. Dr. Katz replied that he should first report the effects to their physician and stop taking the medication. Dr. Katz noted that one should be aware that anything you take could have serious side effects.

OD: Mr. Peterson stated he represents rural Pennsylvania and many of his constituents use tertiary health centers. In terms of communication with rural providers, he asked how we develop systems of disseminating information quickly to all physicians. Dr. Kirschstein responded that this is an effort we are all taking on-getting the wORD out. As an example, the NLM has a big program on telemedicine. Dr. Katz added that NIH has another system-consensus development conferences-to look at new findings and determine what should be taken to the physicians. Then the information is disseminated through journals and to the physicians themselves.

Mr. Regula asked if CDC is used as an agency to distribute information. Dr. Kirschstein replied that they are used but NIH also distributes its own information. The consensus development conference gets the information into the medical journals. The new Center on Minority Health and Health Disparities will also focus on getting the information out. In addition, all clinical trials funded by NIH are available on its website, and soon private trials will be in the database as well.

Mr. Kennedy

NIMH: Mr. Kennedy commented that he found it interesting and distressing that NIMH was not at the hearing since there is such a behavioral interrelation to chronic disease. Dr. Kirschstein responded that with the schedule it was unfortunate that not all of the ICs could be present but that they would all be at the budget hearing next month. Chairman Regula also commented that Q's relevant to today could be submitted to Directors not at the hearing.

Mr. Kennedy was concerned with the effects of these diseases on mental health-depression is a major issue. Dr. Spiegel replied that a workshop on depression and chronic illness was sponsored recently; as an outcome the conjunction of mental health will be studied. Dr. Straus added that mental health is a concern with all diseases. He noted that NCCAM is currently looking at the effects of St. John's Wort in the treatment of depression.

NIDDK: Mr. Kennedy asked what was being done with stem cell research, especially in finding islet cells that can address juvenile diabetes. Dr. Spiegel responded that they have great hopes for a potential cure for juvenile diabetes. They are looking at islet cell transplantation, not stem cells. However, for the 800,000 to one million affected, there will never be enough islet cells available to treat everyone. So, NIDDK is vigorously supporting research on adult human and mouse embryonic stem cells to address the supply issue.

NHLBI: Mr. Kennedy asked about the clinical trial network and treatment of asthma. Dr. Lenfant replied that treatment in adults is advancing. The research community would state that no one should die from asthma. We must use what we know and address behavioral factors. Three things play an important role: genetic factors, environmental factors and behavioral factors. We have to look at them individually and in combination.

Mr. Istook

OD ORD: Mr. Istook said he was interested in hearing results in the future on alternative treatments. He also asked if there is a focus on rare diseases, as there is no Center for this. Dr. Kirschstein said the Office of Rare Diseases does a terrific job of coordinating efforts on rare diseases. Each disease has a home within an IC where the expertise is. ORD brings it all together as a single source to go to for information.

Mr. Istook stated one of the challenges in assimilating new resources is to ensure sufficient quality in applications. Another challenge being the ability of practitioners to absorb advances and apply them in the treatment of people. He mentioned a brochure where Blue Cross Blue Shield explained that the recent escalation in cost of resources for treatment is causing an escalation in health care costs and expressed his concern. He asked how NIH is ensuring cost effective treatments-advances being done in a way to bring down costs. Dr. Kirschstein responded that this issue would not be resolved quickly. Much of what has been learned has resulted in expensive drugs and technology. There are, however, opportunities to compare one drug or technology with another. She gave the flu shot as an example: it turns out a smaller dose is just as effective. Dr. Speigel added another example, that during the committee's recent visit to NIDDK's new transplant wing at the Clinical Center, they saw a patient who received a transplant and biological drugs, showing that a transplant could be done more cost effectively. The biological agents decreased the need for expensive immunosuppressive drugs. Finally, Dr. Kirschstein added that NIH is looking more and more into prevention, which reduces the need for therapy.

Ms. Granger

NIDA/NIAAA: Ms. Granger referenced recent reports indicating that current treatments for drug and alcohol abuse are wrong and asked where NIH is on this. Dr. Kirschstein replied that she would be happy to take that question and give it to the proper IC Director to respond. Dr. Straus added that NCCAM is looking at alternative therapies to address cocaine addiction.

Ms. Granger stated she had a friend who received a fetal tissue transplant in Denver for Parkinson's disease. She asked about the results of that trial. Dr. Kirschstein responded that patients can go to the Denver center, which is supported through private funds. She also stated that NINDS has conducted controlled clinical trials of the transplants. The results have recently been published in the New England Journal of Medicine, and that given the results, the whole approach is being reevaluated.

Mr. Miller

NHLBI: Mr. Miller asked for a status report on gene therapy and hemophilia. Dr. Lenfant responded that they are guarded but optimistic about using gene therapy for hemophilia. There are five phase I studies underway. He believes they have been very successful and are ready to move into human clinical studies. He noted that if gene therapy works this could eliminate the need for what is currently very expensive treatment.

NIDDK: Mr. Miller commented that the Hepatitis C incidence has grown. He asked the status on the future research on this-is research only carried out in NIDDK? Dr. Spiegel responded that through efforts of NHLBI we have protected the blood supply, and that NIAID and NIDDK are working on both basic and clinical aspects. The Institutes are working on prevention through development of a vaccine. Dr. Spiegel noted that major clinical trials are underway to look at more effective therapy.

Mr. Regula

Mr. Regula concluded stating the subcommittee was very appreciative of NIH's efforts. He stated that the proof that what NIH is doing gets results is the increased life span for people today. He then adjourned the meeting.

Prepared by OB