About Us

Budget Request
FY 2000

FY 2000 Statement to the House and Senate Appropriations Subcommittees

February 1999 (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 budget request for the National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS) for Fiscal Year 2000, a sum of $310 million. Including the estimated allocation for AIDS research, total support proposed for the NIAMS is $314.75 million, an increase of $7.368 million or 2.4 percent over the comparable FY 1999 appropriation. Funds for NIAMS efforts in AIDS research are included within the Office of AIDS Research budget request.

I am honored to appear before this Subcommittee, to express my appreciation for the FY 1999 appropriation, to share with you how we have invested these funds, and to talk about some of the scientific opportunities that we plan to pursue in FY 2000. The FY 1999 budget increase provided an opportunity for us to invest in key areas of public health needs, with a particular emphasis on clinical studies. Specifically we are launching a new clinical study of low back pain, expanding our clinical and basic studies of the many autoimmune diseases that we are concerned with, and investing in the next generation of clinical researchers. Let me tell you briefly about each of these.

First, I want to expand on low back pain -- a major problem for our society that affects people at home, at work, and in their recreational activities. We have initiated a multicenter clinical trial on low back pain that will assess the effectiveness of back surgery versus non-surgical treatment for the three most common diagnoses for which surgery is performed. The study has the potential to have a major impact on clinical practice and on costs of medical services. Second, with regard to autoimmune diseases, we are encouraging additional research on the molecular pathways and the genetic basis of the target organ that is involved in autoimmune diseases - what is it about the kidney, the brain, or the heart that makes them the target in lupus in some people and not in others, and what is it about the hair that makes it the target in alopecia areata, for example. Third, we are encouraging pilot clinical trials in rheumatic and skin diseases as well as clinical trials in osteoporosis. Fourth, we are responding to concern about building the pipeline of researchers who can conduct clinical studies by making a commitment to increase our support of training and sustaining clinical investigators who can work with basic scientists and use their knowledge to improve public health. These exciting new studies and support mechanisms are important additions to our research portfolio of fundamental and clinical studies of bone, muscles, skin, and joints. Now I want to share with you some highlights of progress and other opportunities in the NIAMS.

Autoimmunity

While our understanding of autoimmune diseases has improved significantly, researchers do not yet fully understand why some patients are affected with diseases in which their bodies' immune cells attack various vital organs. Diseases in this category include 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. This year, we have witnessed significant, exciting research advances in several of these diseases.

Rheumatoid arthritis

is a chronic autoimmune disease that causes progressive destruction of the joints in affected people causing pain, suffering, and decreased mobility, and it has compromised quality of life and productivity for many Americans. There are now new medications that have been developed for patients with rheumatoid arthritis. This development is an excellent example of how fundamental knowledge can have an impact on health. Basic studies in recent years identified a particular molecule (called tumor necrosis factor alpha) that is important in causing the joints to become inflamed, and pharmaceutical companies were then able to target this molecule and try to eliminate it before it destroys the joint. The new treatments are either artificial decoys that bind the culprit molecule or are antibodies to the culprit molecule. Other new drugs for rheumatoid arthritis block enzymes that enhance joint inflammation. These drugs, known as COX-2 inhibitors, are thought to target joint inflammation more specifically than do the currently available nonsteroidal anti-inflammatory drugs. There is also a newly available immunosuppressive drug that targets fast-growing blood cells that are involved in joint inflammation in patients with rheumatoid arthritis and other forms of inflammatory arthritis. As more disease-causing or amplifying molecules or cells are identified, they will be targeted for elimination in a similar manner.

On a more fundamental level, NIAMS intramural scientists continue their forefront research on the genetics of rheumatoid arthritis and have provided critical information on the role of genes in influencing disease susceptibility in animal models of rheumatoid arthritis and other autoimmune disease. During the next few years, we are going to invest in developing these animal models further because of their relevance to our ongoing genetic studies of families affected with rheumatoid arthritis.

Scleroderma

is an autoimmune disease that occurs much more frequently in women than in men, and it is characterized by widespread hardening of the skin and other tissues. NIAMS-supported researchers have made progress in three areas of research related to scleroderma: (1) a new study in Oklahoma Choctaw Native Americans suggests that the gene for the protein fibrillin-1 is a possible susceptibility gene for scleroderma; this finding is particularly significant because we know that this gene plays an important role in an animal model of scleroderma; (2) an intriguing discovery that has identified the persistence of fetal cells in the skin and blood of women with scleroderma suggests that these persisting immune cells may start attacking the patients' own vital organs; and (3) a potentially very important study that has improved our understanding of the molecular pathways of fibrosis -- the determination that cells from scleroderma patients have twice as many receptors for a particular molecule, transforming growth factor (TGF ), as cells from persons without scleroderma. We know that the binding of TGF to its receptors sends a signal to the cell to produce more collagen. This cycle then results in increased collagen formation and hardening of tissues. These three advances provide exciting research avenues to be pursued to improve our understanding of scleroderma.

Alopecia areata

is another example of an autoimmune disease and it is the most common form of acquired hair disease (excluding male pattern baldness). There has been a real expansion in our understanding of normal hair growth, and much of this enhanced knowledge comes from critical animal models that have been developed for studying this disease. In November 1998, the NIAMS joined the National Alopecia Areata Foundation in cosponsoring the Third International Research Workshop on Alopecia Areata at which research advances and many promising opportunities in understanding hair development, in developing better approaches to animal models, in searching for the antigenic targets in hair, and in attempting to define a better classification of disease were identified. We plan to develop a program announcement in this area in FY 2000.

Osteoporosis

Studies of basic bone biology have given us important insights into how bone is built up and broken down normally in the body, and how this balanced process can go awry in conditions like osteoporosis, where the bone thins and fracture susceptibility increases. Research has increased our understanding of why estrogens are beneficial for people with osteoporosis, and why steroid drugs called glucocorticoids are deleterious and cause thinning of bones. Glucocorticoids are important in the prevention of rejection of transplanted organs and in the treatment of many common inflammatory diseases like rheumatoid arthritis and asthma, but their use can cause bone loss that leads to fractures and disability. New observations suggest that the bone loss may be explained in part by a reduction in the rate at which bone-building cells form, along with higher rates of cell death in bone. Investigators are currently attempting to identify the pathways by which these changes occur.

The NIAMS is also expanding its studies on osteoporosis from those primarily focused on women to those seeking to understand the causes and improve the treatments for men with osteoporosis. Osteoporosis is a significant public health issue that affects many Americans and threatens to affect many more as our population ages. The good news is that we have substantial research progress in this area. We have improved diagnostic approaches to osteoporosis, we have effective treatments available that were not on the market a decade ago, and we know much more about lifestyle practices that enhance bone health. Another initiative that the NIAMS is undertaking is the study of combinations of drugs for osteoporosis. This is an area in which the federal government can provide real leadership, because companies generally do not support studies that combine their drug with a drug from another company. The use of various drugs in combination has the potential to make an important contribution to the treatment of osteoporosis and thus to improve public health. Finally, information dissemination about osteoporosis, and indeed every other disease under the purview of the NIAMS, to all segments of the population remains a key priority of the Institute. The NIAMS joined with six other components of the Department of Health and Human Services in awarding a cooperative agreement for the NIH Osteoporosis and Related Bone Diseases -- National Resource Center. Also, in FY 2000 the NIAMS and other NIH institutes and other federal agencies will sponsor a Consensus Development Conference on Osteoporosis that will serve to educate physicians as well as other health care providers and the public with vital substantiated information about the diagnosis, treatment, and prevention of osteoporosis.

Health Disparities

The NIAMS is concerned that there are disparities in the health status of Americans. One example is the finding from studies in osteoarthritis that African American people have much lower rates of total knee replacements than whites, even when adjusted for age, sex, and insurance coverage. Understanding the reasons for this disparity will help us to target particular populations to develop prevention strategies. In addition, studies in behavioral research have demonstrated that Hispanic and African-American lupus patients have more severe disease at the time of presentation than Caucasian patients. Genetic and ethnic factors appear to be more important than socioeconomic factors in influencing disease activity at the time of disease onset. Furthermore, differences in the disease course and outcome in lupus patients also appear to be caused by many factors -- including the ways in which patients themselves respond to their illness. We already know a lot about the importance of "self-efficacy" and how patients manage their disease. Many chronic diseases like osteoarthritis and lupus affect women and minorities disproportionately, and we are actively seeking to understand the causes of these gender and ethnic differences.

Exercise Physiology and Sport Injuries

Every day more and more Americans are undertaking some sort of fitness program or exercise activity. While this is good news -- as we are all encouraged to be more active -- it is also accompanied by a significant increase in sports injuries, particularly in women. We are not yet sure why, but women are particularly vulnerable to some types of injuries when they participate in sports, especially injuries of their knee joints. We are joining with the American Academy of Orthopaedic Surgeons to sponsor a meeting on women and sports injuries this June, just prior to the 1999 Women's World Cup Soccer Tournament in Washington, DC. We intend to use this opportunity to put a spotlight on women in sports, and to try to understand the particular injuries that women suffer. We are working to identify the causes of sports and exercise injuries, and to develop effective strategies to avoid and treat them.

Medical Research Makes a Difference in People's Lives

As the illustrations just cited reveal, considerable progress has been made in identifying and alleviating many of the physical and social consequences of chronic diseases, and the investigations underway and planned promise to continue to improve life. We are proud of the achievements of the scientific programs we have supported, of the individual scientists who devote their lives to research, and of the value of research to every day life. We remain very clear in our goal: to support high quality science that will continue to improve the health of the American people.

The activities of the National Institute of Arthritis and Musculoskeletal and Skin Diseases are covered within the NIH-wide Annual Performance Plan required under the Government Performance and Results Act (GPRA). The FY 2000 performance goals and measures for NIH are detailed in this performance plan and are linked to both the budget and the HHS GPRA Strategic Plan which was transmitted to Congress on September 30, 1997. NIH's performance targets in the Plan are partially a function of resource levels requested in the President's Budget and could change based upon final Congressional Appropriations action. NIH looks forward to Congress' feedback on the usefulness of its Performance Plan, as well as to working with Congress on achieving the NIH goals laid out in this Plan.

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