About Us

Budget Request
FY 2001

FY 2001 Statement to the House and Senate Appropriations Subcommittees

February 2000 (historical)

Statement by
Stephen I. Katz, M.D., Ph.D., Director
National Institute of Arthritis and Musculoskeletal and Skin Diseases

Mr. Chairman and Members of the Subcommittee:

I am pleased to present the President's non-AIDS budget request for the NIAMS for FY 2001, a sum of $363,479,000, which reflects an increase of $19,021,000 over the comparable Fiscal Year 2000 appropriation. Including the estimated allocation for AIDS, total support requested for the NIAMS is $368,712,000, an increase of $19,232,000 over the Fiscal Year 2000 appropriation. Funds for the NIAMS efforts in AIDS are included within the Office of AIDS Research budget request.

I am honored to appear before this Subcommittee. I want to begin by providing a context for the research mission of our Institute. As the Director of the NIAMS, I am acutely aware that the every day life of the American people is enormously improved by the research that our Institute supports. Children on the playground, women and ethnic minorities who are disproportionately afflicted by the many diseases that affect their daily activities, adults in the work place, and elderly Americans striving to live independently -- all have their lives enhanced or compromised by the health and functioning of their bones, joints, muscle, and skin. Ensuring improved quality of life and increased longevity of life for all Americans is our primary mission.

I want to express my appreciation for the FY 2000 appropriation -- the second year of an extraordinary budget that enabled us to invest in medical research with much more breadth and depth than ever before. I will share specific examples of ways in which we invested our budget over the last year and some of the research directions we are pursuing.

Clinical Research

The focus of most of my remarks today will be on clinical research. While the backbone of medical science has been and will continue to be fundamental research on how the body functions, the ultimate goal of research supported by the NIH is improving public health. In order to achieve this, we must also serve as translators - translating the basic research findings from the laboratory to improving patient care at the bedside as well as utilizing the information learned at the patient bedside to inform more focused and sophisticated basic studies. This translation is the cornerstone of the research supported by the NIAMS.

Highlights of particularly exciting and promising translational research on bones, joints, muscles and skin include the findings that lower doses of estrogen than we normally prescribe can be effective in preventing osteoporosis, that some of the new treatments for rheumatoid arthritis can be targeted directly at the disease-causing factors, that normal hair growth is controlled by genes that encode for proteins that may be excellent targets for new therapies, and that proteins that are essential for normal muscle formation can, in mouse models, be produced by injecting particular genes into the muscles that contain the defective proteins. These examples provide only a broad sample of some of the most recent findings from research studies that we have supported.

The reality is that clinical research is vitally important, but is also very costly. The significant increase in our appropriation last year enabled us to launch a number of clinical initiatives in particularly challenging areas of health, including: (1) Pilot studies in rheumatic diseases and skin diseases - These studies include expanded research on some of the most difficult public health challenges such as leg ulcers, rheumatoid arthritis, scleroderma, lupus, spondylitis and others where new treatment approaches are needed. (2) Osteoporosis in men - A major clinical trial will determine the extent to which the risk of fracture in men is related to bone mass and structure, biochemistry, lifestyle, tendency to fall, and other factors. The study will also seek to determine if bone mass can be correlated with an increased risk of prostate cancer. (3) Combination therapies for osteoporosis -- It is important to determine whether combinations of drugs, often produced by different pharmaceutical companies, will be more effective and have fewer side effects than any of the drugs used alone for osteoporosis. (4) Treatment of back pain - Back pain is very common and has a serious impact on people's personal and professional lives, and results in significant costs to businesses and the American economy. We have launched a major clinical trial studying surgical versus nonsurgical treatment of three different back disorders. We anticipate that this study will have a significant effect on clinical practice and the cost of medical services for people with any of these three back disorders. (5) The Spondylitis Consortium - The NIAMS has partnered with the American Spondylitis Association in establishing the North American Spondylitis Consortium to search for genes that determine susceptibility to ankylosing spondylitis, a painful inflammatory disease of the spine, also known as arthritis of the spine. and (6) Clinical research training and career development - Because we need to develop and maintain a pipeline of researchers who are training to plan, conduct, analyze, and disseminate the findings of clinical research, the NIAMS has enthusiastically embraced and participated in the NIH initiatives to develop and maintain careers in clinical research.

Autoimmunity

The NIAMS supports a broad and diverse portfolio of research on autoimmunity, including studies of rheumatoid arthritis, systemic lupus erythematosus, Sjogren's syndrome, scleroderma, alopecia areata, and many blistering skin diseases -- all potentially devastating chronic diseases which exact a huge toll in human suffering and economic costs. The additional funds provided by Congress for the Autoimmunity Initiative last year provided the opportunity for the NIAMS to expand its research in the following key areas: (1) pilot trials on innovative therapies for rheumatoid arthritis and scleroderma; (2) target organ damage in autoimmune diseases, also focusing on scleroderma and rheumatoid arthritis; (3) autoimmune rat repository and transgenic resource - a national resource and development center for rat models of autoimmune disorders; (4) NIAMS patient data registries - in which information will be collected on neonatal lupus and on juvenile rheumatoid arthritis, with an expansion to include genetic studies; and (5) new imaging technologies for autoimmune diseases, through a project involving in vivo imaging of tiny blood vessels in animal models of rheumatoid arthritis.

Fibromyalgia

The NIAMS has a firm commitment to identifying the causes of fibromyalgia and improving the daily life of people affected by this debilitating disease, and we have a broad portfolio of research in this area. Last year the NIAMS as well as the NIDCR, NINDS, and the ORWH funded fifteen new clinical and basic research studies on fibromyalgia, and we are confident that these new studies will provide much-needed information on the causes of fibromyalgia as well as new strategies for treatments.

Muscle Diseases

People affected by muscle diseases face profound changes and challenges in their every day lives. We now know that many of these diseases are caused by genetic mutations, and we are supporting research to further define these mutations and to overcome the current barriers to effective gene therapy of muscle diseases. We are optimistic that genetic manipulation of skeletal muscle will improve therapy for muscle diseases such as Duchenne muscular dystrophy and Facioscapulohumeral dystrophy. The Institute, in collaboration with the National Institute of Neurological Disorders and Stroke, is sponsoring workshops in both of these areas in May 2000 and we are looking forward to hearing recommendations from experts in these fields on research directions we could pursue in FY 2001.

Osteoarthritis

As many Americans are well aware, osteoarthritis is the most common disease of joints. The NIAMS continues to support a substantial amount of research across the full spectrum of scientific approaches and strategies to understand this disease and improve life for affected people. In July 1999 we sponsored a major conference on osteoarthritis with the participation of leading experts in this field and many related fields. We used this opportunity to have a tandem educational meeting for patients whose daily lives are affected by osteoarthritis. We learned a great deal about the disease from both kinds of experts - those doing research on osteoarthritis and those living with it. We are carefully considering the recommendations made from both sets of experts to strengthen our portfolio in osteoarthritis using the FY 2001 appropriation, particularly in the area of prevention. The NIAMS is also partnering with our colleagues in the National Institute on Aging, the Food and Drug Administration, many pharmaceutical companies, and several professional and voluntary lay organizations to try to create a public-private partnership to identify biomarkers for osteoarthritis. Our goal is to support clinical and laboratory evaluations of biomarkers and imaging techniques as potential surrogate endpoints for clinical trials that would assess the efficacy of osteoarthritis disease-modifying interventions.

Osteoporosis

We have known for some time that osteoporosis has genetic components, but the genes that are actually associated with fractures themselves had not previously been identified. Scientists have found that older women who have the gene for apolipoprotein E, also known as APOE*4, are at increased risk for hip and wrist fractures. We know about this gene from other studies of its association with common, late-onset forms of Alzheimer's disease and with osteoporosis in patients on dialysis. This represents a promising area for further study in patients with osteoporosis as well as those with Alzheimer's disease, who are known to have a higher risk of hip fracture.

In March 2000 the NIAMS and other NIH components will hold a Consensus Development Conference on osteoporosis to address many key questions and to bring a focus to scientific opportunities that could be pursued in this major public health problem that compromises daily activities for millions of Americans of all ages. The information that comes from this Conference will be broadly disseminated to physicians and other caregivers, as well as to the public.

Systemic Lupus Erythematosus

In genetic studies of lupus, researchers have found an association between the disease and a region on chromosome 1. Fine mapping of this region has identified another candidate gene involved in immune function. Studies of the genetics of lupus may identify potential therapeutic targets and may facilitate the development of markers of disease activity. While medical research has certainly made a significant difference in the daily lives of people (primarily women) with lupus, this remains a major public health problem that compromises the quality and longevity of life for many Americans. In addition to the many systems of the body that are affected, patients with lupus also have a higher risk of neuropsychiatric manifestations and accelerated atherosclerosis. In 1999 the NIAMS sponsored workshops in both of these areas, and we intend to follow-up on the many excellent recommendations from experts who participated in these two meetings.

Skin Diseases

Gene therapy has potential for treating many skin diseases, diseases that significantly compromise daily life for millions of Americans both physically and psychologically. In addition, skin can also serve as a factory for the production of molecules including hormones such as insulin and human growth hormone that are used in the treatment of many systemic diseases. Skin provides a number of advantages for gene therapy approaches, including the ability to remove genetically altered skin by simple excision if problems develop. The NIAMS is supporting a scientific conference in March 2000 to increase the level of interest in gene therapy using skin and to identify scientific opportunities and needs in this area.

Health Disparities

While we know that disease can strike people at every age, of either gender, and in every ethic group, we also know that many diseases affect women and members of minority groups disproportionately -- both in increased numbers and increased severity of the diseases. Even if variables such as socioeconomic status, access to health care, and insurance coverage are eliminated, the fact remains that diseases like lupus, scleroderma, osteoarthritis, and vitiligo all account for disparities in the health of women and minorities. We are actively seeking to understand the causes of these gender and ethnic differences, and we are expanding our commitment to better understanding of health disparities. We are also increasing our efforts to disseminate health information to minority populations and have established a toll-free line in both Spanish and English as well as produced a number of our publications in Spanish. Plans are underway for a workshop to address promising opportunities for research in this area. In addition, members of our Intramural Research Program are designing an outreach program targeted toward the minority community. While it is still in preliminary stages, it will ultimately include both local and national outreach efforts.

Conclusion

Bones, joints, muscles, and skin are central components of the human body. When the functions of any of these areas are affected, the daily lives of people are compromised. We are committed to continuing to support basic research as well as the many clinical studies underway, including those exploring the roles of genetics, the environment, diet, and behavior in disease. I cite our scientific achievements with pride, and I pledge to continue my commitment to support high quality science that will continue to improve the health of the American people.

The NIH budget request includes the performance information required by the Government Performance Results Act (GPRA) of 1993. Prominent in the performance data is NIH's first performance report which compares our FY 1999 results to the goals in our FY 1999 performance plan. As our performance measures mature and performance trends emerge, the GPRA data will serve as indicators to support the identification of strategies and objectives to continuously improve programs across the NIH and the Department.

I will be happy to answer any questions you may have.