About Us

Budget Request
FY 2003

Witness appearing before the House Subcommittee on Labor-HHS-Education Appropriations

March 13, 2002 (historical)

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

Kerry Weems, Acting Deputy Assistant Secretary, Budget

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 2003, a sum of $488,228,000, which reflects an increase of $37,988,000 over the comparable Fiscal Year 2002 appropriation.

It is an honor for me to have this opportunity to share stories of research advances as well as highlights of the many opportunities we have in research on bones, muscles, joints, and skin. The mission areas of our Institute touch the daily life of millions of Americans, and we are committed to improving quality of life as well as longevity. Diseases within our mandate know no barriers in terms of age, gender, ethnicity, or socioeconomic status. In fact, many of the diseases in our mission areas disproportionately affect women and minority individuals, and we are committed to determining why this is the case.

Research in Children

While we typically associate chronic diseases with the elderly, the fact is that they can affect people of all ages, and can rob a child of the joys and activities of the young. The other reality is that children are not small adults - diseases affect them in different ways and treatments may have different effects in children than adults. In light of these and other realities, 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, it has been said that osteoporosis is actually a disease of childhood that is manifested in later years. We know how vitally important it is that children develop a strong skeleton in childhood so that they can withstand the age-related changes that occur in their bones later in life. Research supported by the NIAMS has resulted in the design of a 7-month, high intensity jumping regimen that will increase peak bone mass at two clinically critical sites, the hip and the spine. Investigators discovered that children who participated in the jumping program had a significantly greater change in bone mineral content in both the hip and spine compared with a control group, as well as showing positive differences in bone mineral density and bone area. This regimen, which can easily be incorporated into the regular elementary school curriculum, has potentially important public health implications with respect to optimizing peak bone mass attainment in young people.

The NIAMS has also placed an enhanced emphasis on research on osteogenesis imperfecta (OI), one of the most common genetic diseases of bone. OI is characterized by brittle bones that fracture easily, and is caused by mutations in the gene for a protein called type I collagen. NIAMS-supported researchers have recently reported very exciting progress in both the controlled introduction of genes into bone cells, as well as the ability to inactivate mutant genes that can cause disease. Further progress in OI research is expected as a result of several new grant awards from the NIAMS for projects ranging from cutting-edge gene and cell therapies to testing drug treatments in mouse models of the disease.

In other research related to children, the NIAMS continues to lead the NIH's Pediatric Rheumatology Clinic. In addition to providing diagnosis, evaluation, and treatment of juvenile arthritis and other rheumatic diseases, the clinic facilitates the translation of research advances to improve patient care. A new study underway at the clinic is designed to determine the best medication combinations for treating children with juvenile rheumatoid arthritis. We recognize that we have much to learn about diseases in children and we are currently developing a new, broad initiative that will focus on multidisciplinary translational research projects in rheumatic and immuno-inflammatory skin and muscle diseases of children so that we can target those areas that present special challenges in children.

Arthritis and Other Rheumatic Diseases

Research on osteoarthritis, a degenerative joint disease, took a big step forward with the launching of the new public-private partnership that teams several NIH entities, the FDA, and four pharmaceutical companies in the Osteoarthritis Initiative. Clinical research on osteoarthritis has been severely hampered by the lack of biological markers needed to assess the progression of this most common form of arthritis. The significant commitment required to undertake such a study has been beyond the scope of either government or industry alone, but is feasible and indeed underway through this new partnership. The NIAMS teamed with our colleagues in the National Institute on Aging in leading this effort to fund from four to six clinical research centers to establish and maintain a natural history database for osteoarthritis. The database will include clinical evaluation data and radiological images, as well as a biospecimen repository. All data and images collected will be available to qualified researchers worldwide to help hasten the pace of scientific studies and biomarker identification. In a separate effort, the NIAMS is supporting work to develop biomarkers for two chronic inflammatory diseases which affect many Americans, rheumatoid arthritis and lupus.

Lupus is a serious and potentially fatal autoimmune disease that occurs with greater frequency and intensity in African American women, and it affects many organ systems of the body. One of the challenging manifestations of lupus is the involvement of the nervous system, and researchers supported by the NIAMS have recently reported significant advances in our understanding of the molecular mechanisms involved in the changes that can occur in the brains of people with lupus. The identification of the particular antibodies involved not only helps us to understand the nervous system complications in lupus, but also provides some new therapeutic possibilities for this aspect of lupus that can be difficult and challenging for affected patients, their families, and their health care providers. To further enhance research in this area, the Institute has recently released a solicitation for applications on neuropsychiatric lupus, in an effort to stimulate additional study of the neurological and psychiatric syndromes associated with this chronic disease.

Bone Biology and Bone Diseases

Basic researchers have reported new insights into the complex effects of estrogen on bone. We know that the most common cause of bone loss is the decline in the female sex hormone, estrogen, in women after menopause. Estrogen also appears to be important in maintaining bone mass in men, although men have more of the male sex hormone androgen than estrogen. Recent research reports from work supported by the NIAMS have provided important clues to the complex relationship between estrogen and bone, and revealed-as many research investigations do-that we still have much to learn about the action of estrogen as well as the function of estrogen receptors. The most recent research reports indicate that either estrogen or androgen can act to increase bone formation and prevent net bone loss. In other research, scientists have shown that particular cells of the immune system called T cells can contribute to the bone loss that occurs when estrogen levels are low. These and other basic studies funded by the NIAMS are adding to the foundation of knowledge of normal function in bone biology and the changes that occur in bone diseases. Recent initiatives to stimulate further work in the bone sciences include the release of solicitations to encourage applications on new research strategies for the evaluation and assessment of bone quality, and one on basic and applied stem cell research for arthritis and musculoskeletal diseases.

Muscle Biology and Muscle Diseases

This has been a very active year in the whole field of the muscular dystrophies as the NIAMS has joined our colleagues in the NINDS in targeting research in this area. Over the last two years, we have supported two successful scientific conferences, and issued research solicitations to the research community targeting those areas of particular opportunity that were identified by experts at the conferences. As a result of these activities, the NIAMS and NINDS recently awarded several new grants to support both basic and clinical research studies in facioscapulohumeral dystrophy (FSHD), the third most common genetic disease of skeletal muscle. We have also funded a number of projects in follow-up to a solicitation for proposals on therapeutic and pathogenic approaches for the muscular dystrophies. In addition, we continue to support a research registry in particular forms of muscular dystrophy that serves as an invaluable resource for scientists to collect and analyze new research data in their pursuit of better treatments for muscular dystrophies.

Skin Biology and Skin Diseases

Chronic wounds are a significant public health challenge, particularly in the elderly and people with diseases like diabetes that affect skin healing. A new living skin substitute showed a significant improvement in wound healing and a decrease in time to complete closure of the wound in people with diabetic foot ulcers. Newer technologies such as artificial skin equivalent systems can improve the rate of healing of existing wounds, as well as minimize or reduce the incidence of severe complications.

Pseudoxanthoma Elasticum (PXE) is a systemic inherited disorder that affects the elastic tissue in the skin, eyes, and cardiovascular system, and it can result in severe and even fatal problems in affected individuals. The fascinating new dimension to our understanding of PXE is that, contrary to earlier beliefs, PXE is actually a metabolic disorder. The recognition that this is a metabolic disease offers new hope for the development of treatments based on metabolic modifications potentially including such approaches as diet manipulation or drug therapy. There is also the potential for PXE to be identified in affected people early so that treatment can be instituted before signs and symptoms of the disease actually occur. To boost research on PXE and other heritable disorders of connective tissue, such as Marfan syndrome and Ehlers-Danlos syndrome, the Institute recently released a solicitation, along with our colleagues at the National Heart, Lung, and Blood Institute, to encourage more basic and clinical studies of these disorders.

Health Disparities

A number of diseases within the mission areas of the NIAMS affect women and members of minority groups disproportionately, including lupus, scleroderma, osteoarthritis, vitiligo, and keloids. In addition to the vigorous research portfolio that the NIAMS funds in these areas, I want to cite two programs that the Institute supports that address the critically important area of health disparities. We continue our active involvement in the Health Partnership Program, a model community-based research program to study rheumatic diseases in the African American and Hispanic/Latino communities in the metropolitan, Washington, D.C., area. In addition, we enthusiastically support a newly initiated program that the NIAMS was active in creating - a new strategy for enhancing clinical research training in minority-serving institutions. The goal of this program is to produce well-trained clinical researchers who will go on to lead clinical research projects. Finally, in follow-up to a major scientific conference organized by the Institute, the NIAMS is developing a new initiative on health disparities in rheumatic and skin diseases.

Intramural Research Program

The NIAMS Intramural Research Program (IRP) is a vital and growing program that has become a national and international resource, as well as a recognized site for scientific excellence on the NIH campus. A major new program that the IRP has undertaken is the initiation of a trans-NIH collaboration in musculoskeletal medicine. This effort will include the development of innovative fundamental science, clinical studies, and translational research. The collaboration is designed to build on strengths that are already present at the NIH, as well as foster the growth of new research and training programs in the critical and under-served area of musculoskeletal medicine.

Conclusion

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 typically chronic, costly, common, and disabling. The vitality of our bones, joints, muscles, and skin is key to the length and quality of our lives. 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.

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 compared our FY 2001 results to the goals in our FY 2001 performance plan.

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