About Us

Budget Request
FY 2002

FY 2002 Statement to the House and Senate Appropriations Subcommittees

May 16, 2001 (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 Committee:

I am pleased to present the President's budget request for the National Institute of Arthritis and Musculoskeletal and Skin Diseases for FY 2002, a sum of $443,565,000, which reflects an increase of $46,962,000 over the comparable Fiscal Year 2001 appropriation.

It is an honor for me to have this opportunity to share stories of progress and opportunity in the research within our mission areas. Improving daily life is the driving force for the research that we support and conduct at the National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS). Virtually every home in America is touched by diseases affecting bones, joints, muscles, and skin. We are committed to better understanding, diagnosis, treatment, and prevention of these diseases and disorders that are often chronic and disabling, many of which disproportionately affect women and minority populations.

Health Disparities

Research has revealed that many of the diseases within our mandate affect groups such as African Americans, Hispanic Americans, American Indians, Alaskan Natives, and Asian Americans both in increased numbers and increased severity. For example, the prevalence of lupus is higher among African Americans and Hispanic Americans, and these groups also experience more complications of lupus; African Americans also have higher rates of hip and knee osteoarthritis; scleroderma occurs with greater frequency in Choctaw American Indians; and African American people are also disproportionately affected by overgrowth of scar tissue (keloids) and by loss of pigmentation (vitiligo), both of which may be severely disfiguring.

Health Partnership Program. The health of a nation depends on the health of its communities. Recognizing this, the NIAMS is launching the first phase of its Health Partnership Program - A NIAMS Diversity Outreach Initiative, a new program to address the health disparities in joint, muscle, bone and skin diseases that exist in minority communities. The initial phase of this Program has begun as a model community-based program in the African American community in the metropolitan Washington, D.C., area, with the focus on rheumatic diseases. As a component of this partnership, plans are also underway for a new rheumatology clinic to be located in a centrally accessible area of Washington, D.C.

Recruitment to Research Careers. Specific strategies are underway and planned to increase the number of underrepresented minority investigators in the biomedical research fields related to the diseases within our mandate. The Institute, along with many other NIH Institutes, has recently issued a Request for Applications for planning grants for clinical research training in minority institutions as a first phase of this initiative. The second phase will be a five-year grant to assist in the actual development of clinical research curricula. A successful program will produce well-trained clinical researchers who can lead clinical research projects.

Research in Children

The NIAMS has undertaken a number of programs and activities focused on children to enhance our understanding of childhood diseases and to develop improved treatments for our younger generation. For example, the NIAMS Intramural Research Program launched an exciting and promising initiative in the fall of 2000 at the NIH research hospital - the new NIH Pediatric Rheumatology Clinic. The clinic offers diagnosis, evaluation, and treatments for children with arthritis and other rheumatic diseases. The clinic will provide children with a place where they can be diagnosed and treated in a state-of-the-art facility, and researchers can learn much more about rheumatic diseases. In addition, treatment for juvenile rheumatoid arthritis has been significantly improved with the results of a recent clinical trial that showed Enbrel® (etanercept) is a safe and effective drug in the treatment of children and teenagers with juvenile rheumatoid arthritis (JRA). This clinical trial was conducted by researchers at one of the NIAMS Multipurpose Arthritis and Musculoskeletal Diseases Centers and investigators in the Pediatric Rheumatology Collaborative Study Group. The success of this clinical trial is also the culmination of many years of basic research supported by the NIAMS and other NIH components. These findings offer hope for children with juvenile rheumatoid arthritis, hope that they may live their lives as active children. In other research involving children, we now understand that osteoporosis may actually start in childhood. Research studies in young girls revealed that minor variations in a gene for the bone protein collagen can lead to lower bone density. These minor variations in this gene, while not causing apparent disease, may define a high susceptibility group for osteoporosis later in life. Identifying and understanding genetic susceptibility to osteoporosis early in life may facilitate the targeting of interventions to those who will most profit from them.

Bone Biology and Bone Diseases

Bone is metabolically a very active tissue, constantly undergoing build up of new bone and resorption of old bone. Bone remodeling is a normal, but carefully balanced process. Bone diseases like osteoporosis can result from an imbalance in this process and osteogenesis imperfecta can result from the mutation of bone-producing genes, and both diseases result in low bone density, fragile bones, and increased susceptibility to fracture. Research has taught us that many factors affect bone density and strength, including genetic, nutritional, environmental, and others. Basic research has provided the foundation for our understanding of bone and has revealed some intriguing and potentially important scientific opportunities. For example, researchers found that statins, drugs that lower serum cholesterol, increase the production of a bone-enhancing molecule. This is leading to work on the development of similar drugs that can be directly delivered to the bone for maximum effect. Other studies showed us that a protein called leptin, which has an established role in controlling food intake and other aspects of behavior and physiology, seems to inhibit bone formation in animal models. Researchers will pursue this finding with the goal of designing drugs to specifically block leptin's action on bone and restore lost bone.

Muscle Biology and Muscle Diseases

There are many forms of muscular dystrophy, and the NIAMS has teamed with our colleagues in other components of the NIH, particularly the National Institute of Neurological Disorders and Stroke, to bring a strong focus to basic and clinical studies of muscular dystrophy. Last year we sponsored major scientific conferences in both Duchenne Muscular Dystrophy (DMD) and Facioscapulohumeral dystrophy (FSHD), issued research solicitations signaling our strong interest in the submission of high quality research applications in understanding and treating muscular dystrophy, and have funded a research registry for FSHD and myotonic dystrophy that will facilitate research by serving as a liaison between families affected by these diseases, and researchers who want to study these disorders.

Skin Biology and Skin Diseases

This has been a particular productive year in research on skin biology as well as skin diseases. Highlights of progress include: (1) ground-breaking research on impetigo, a common infection among children aged 2 to 6. The bacterium Staphylococcus aureus, cause of the common skin infection bullous impetigo, produces a toxin that attacks a protein highly specific for cell-to-cell binding in the outermost layer of the skin. Researchers have reported that breakup of this protein not only brings about the characteristic blistering, but also gives the bacterium a specific mechanism to circumvent the skin's protective barrier and spread further. (2) The gene causing Pseudoxanthoma Elasticum has been identified. Pseudoxanthoma elasticum is an inherited disorder characterized by progressive calcification of elastic fibers in the skin, eye and cardiovascular system. This disease is inherited and can have severe manifestations in these organ systems. Work is continuing to determine the function of the gene and how mutations in the gene result in the clinical disease. This discovery should allow for the eventual determination of the cause and, ultimately, allow the design of therapeutic interventions for the treatment of this disease. (3) Advances in understanding hair development and treating hair diseases have been reported. A number of skin diseases affect hair cycle resulting in various abnormal types of hair loss as well as the hair loss normally associated with aging. An understanding of the events in hair development, cycling, and the mechanism of hair loss in various diseases will allow for the development of treatments to correct these abnormalities. Knowledge of the molecular mechanisms involved in the continuously repeated cycle of resting, shedding, and regrowth means that hair biology is useful not only as a way to understand hair diseases such as alopecia areata, but also for understanding of other cycling and regenerating tissues. The research advances that have increased our understanding of the hair follicle system and the chemicals and signaling molecules involved in its cycling will allow the development of specific interventions to treat hair diseases, both naturally occurring, such as alopecia areata, and those induced by certain cancer chemotherapeutic treatments.

Conclusion

The vitality of our bones, joints, muscles, and skin is key to the length and quality of our lives. Basic research has taught us much about how these components of our bodies function normally and what goes awry and causes the enormous number of diseases and disorders affecting bones, joints, muscles, and skin. Clinical research helps us to understand the nature of disease, and improves our ability to diagnose, treat, and prevent disease. Medical research supported by the NIAMS has made significant strides in improving health and quality of life, and we are committed to pursuing promising research opportunities that will continue to improve the health of the American people. We are investing in the future health of our nation, and American people of all ages and population groups will benefit from these investments.

The NIH budget request includes the performance information required by the Government Performance and Results Act (GPRA) of 1993. Prominent in the performance data is the NIH's second annual performance report which compares our FY 2000 results to the goals in our FY 2000 performance plan. As performance trends on research outcomes emerge, the GPRA data will help NIH to identify strategies and objectives to continuously improve its programs.

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