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FY 2006 Statement to the House and Senate Appropriations Subcommittees
April 6, 2005 (historical)
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 Fiscal Year (FY) 2006 President's budget request for the National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS). The FY 2006 budget includes $513,063,000, an increase of $1,906,000 over the comparable FY 2005 enacted level of $511,157,000 comparable for transfers proposed in the President's request.
Improving daily life is the driving force for the research that we support and conduct at the NIAMS. Virtually every home in America is touched by diseases affecting bones, joints, muscles, and skin. We are committed to improving our understanding, diagnosis, treatment, and prevention of these diseases and disorders that are typically costly, chronic, and disabling, many of which disproportionately affect women and minority populations. I am delighted to share highlights of our research progress as well as our plans.
The NIH Roadmap for Medical Research
The NIAMS is pleased to partner with other NIH components in the many dimensions of the NIH Roadmap, and the Institute has responsibility for the management of an initiative for a patient-reported outcomes measurement information system - or PROMIS - network. The goal of this initiative is to develop ways to measure patient-reported symptoms such as pain and fatigue and aspects of health-related quality of life across a wide variety of chronic diseases and conditions. The PROMIS initiative will develop a publicly available computerized adaptive test for the clinical research community. Many diseases that compromise daily life involve pain, fatigue, and other difficult-to-measure quality of life outcomes, and the development of a test to measure changes in these symptoms will be of benefit to patients and their health care providers.
Research in Children
When arthritis and other rheumatic diseases affect children, they can significantly compromise a child's ability to enjoy an active life. NIAMS-supported researchers have launched a state-of-the-art genomics project, and the goal of this project is to take full advantage of the tremendous progress that has been realized in genetics and genomics, and to uncover gene expression patterns (groups of genes that are "turned on" or "turned off") that contribute to the development of pediatric arthritis. The NIAMS and a chapter of the Arthritis Foundation and the Schmidlapp Trust are supporting this study of children newly diagnosed with a variety of pediatric diseases such as juvenile rheumatoid arthritis, juvenile ankylosing spondylitis (or spinal arthritis) and other related immune disorders. Identifying the gene expression patterns for different types of arthritis in children will help to improve diagnosis as well as to predict the severity of disease for affected children.
In other studies supported by the NIAMS, the promise of genetic studies was underscored by the identification of a gene variant that increases susceptibility to juvenile arthritis. The NIAMS and the Arthritis Research Campaign funded researchers from around the world who worked collaboratively in collecting DNA samples from children with juvenile rheumatoid arthritis and their parents. Research findings suggest that there may be distinct genetic profiles for the disease that result in differences in age of onset as well as disease severity.
Biomarkers of Disease
Progress in identifying the onset and progression of disease is a challenge in many chronic diseases, and the NIAMS has taken the lead in three initiatives to address this challenge: the first is the Osteoarthritis Initiative - a public-private partnership that the NIAMS, the National Institute on Aging, several other NIH components, and three pharmaceutical companies support that is working to develop clinical research resources for the discovery and evaluation of biomarkers and surrogate endpoints for clinical trials on osteoarthritis (the most common form of arthritis). Data and images collected will be available to researchers around the world to speed the pace of research in biomarker identification, and this consortium is expected to serve as a model for initiatives in the future that involve public and private partnerships. We have already enrolled 1900 individuals to participate in this Initiative. The second initiative is the creation of the Osteoarthritis Biomarkers Network involving institutions in the U.S. and Sweden. This Network facilitates the sharing of clinical, biological, and human resources to more rapidly and more effectively identify biomarkers for osteoarthritis. In the third biomarker initiative, the NIAMS supports the Autoimmune Biomarkers Collaborative Network which includes efforts to identify and validate biomarkers for lupus -- a serious and potentially fatal autoimmune disease that occurs with greater frequency and intensity in African American women, and that affects many organ systems of the body.
Arthritis and Other Rheumatic Diseases
Rheumatoid arthritis is an autoimmune disease, and affected individuals often must be treated with powerful drugs that may help to keep the disease better controlled, but also suppress the immune system - leaving patients particularly vulnerable to infection. NIAMS-supported researchers have identified a potential treatment that will suppress the abnormal, autoimmune response that causes the rheumatoid arthritis, but does not diminish the patient's ability to fight bacteria and viruses. The treatment is a synthetic peptide (a chain of amino acids) called dnaJP1- a particular section of a protein that has the same characteristic amino acid sequence as that found in patients with rheumatoid arthritis. In initial studies a synthetic version of the dnaJP1 peptide was given to patients with rheumatoid arthritis with the goal of blocking the immune response, and the immune system responses were normal in these treated patients. The NIAMS partnered with the National Institute of Allergy and Infectious Diseases, the Royal Netherlands Academy of Arts and Sciences, and the Dutch Organization for Scientific Research in funding this study. A new larger study will be undertaken to pursue studies of this promising synthetic peptide for people with rheumatoid arthritis.
Fibromyalgia is a disease that affects many systems of the body, affects women far more commonly than men, and is characterized by low pain thresholds at specific tender points in the body. NIAMS-supported researchers have furthered our understanding of fibromyalgia in recent studies that determined that fibromyalgia was strongly aggregated in families, and that the number of tender points as well as total muscle pain scores were strongly associated with fibromyalgia in families. In addition, there was an increase in the presence of mood disorders in relatives of fibromyalgia patients. This aggregation of fibromyalgia in families suggests that genetic factors may play an important role in this disease. The NIAMS supported a workshop in November 2004 that reviewed the state of the science and a view to future studies in fibromyalgia.
Bone and Musculoskeletal Diseases
Osteoporosis is characterized by bone thinning that results in increased susceptibility to fracture. A particular clinical challenge has been that often the first indication of osteoporosis is when a person (most often a woman) has a bone fracture, and by then the bone has already thinned. Better methods are needed to screen for osteoporosis and for those who are at high risk for fractures. Researchers have recently learned that bony regions of conventional dental x-rays may be useful in evaluating both the current micro-architecture of bone as well as following changes in bone over time. Bone quality plays a critical role in osteoporosis and other bone diseases, and the NIAMS has partnered with the American Society for Bone and Mineral Research in sponsoring a meeting in May 2005 to evaluate the current status of assessment methods to serve as surrogates for fracture and bone fragility, as well as to determine the next steps that must be taken to validate these methods and incorporate them into clinical trials. In other studies with relevance for osteoporosis, basic scientists have identified a particular gene (Alox15) that is strongly associated with changes in bone mineral density -- a measure of vulnerability for osteoporosis. Researchers had previously identified the involvement of Alox15 in fat metabolism, so the identification of its role in bone links metabolic pathways and bone changes, and also provides a new drug target for osteoporosis.
One of the most active and productive areas within the Institute's research portfolio is in the muscular dystrophies - a group of genetic diseases characterized by progressive weakness and degeneration of the skeletal or voluntary muscles which control movement. NIAMS research has made progress in defining the genetic mutations and in overcoming the current barriers to effective gene therapy of Duchenne muscular dystrophy, Facioscapulohumeral dystrophy, and other muscle diseases. For example, scientists supported by the NIAMS and the Muscular Dystrophy Association recently reported that a particular method of gene therapy was able to reach all damaged muscles in a muscular dystrophy (MD) mouse, with implications for delivering genetic therapy for MD and perhaps other diseases of the muscle or heart. Previous work showed that MD could be prevented from occurring in a mouse model of the disease by replacing the gene for dystrophin, which is defective in people with the Duchenne form of the disease with a corrected copy of the gene. However, until now, no one had found a way to deliver a new gene to all muscles of an adult animal, including muscles that had already developed MD.
The NIAMS has teamed with the National Institute of Neurological Disorders and Stroke (NINDS) and the National Institute of Child Health and Human Development (NICHD) to bring a strong focus to basic and clinical studies of MD. Activities include the efforts related to the new Muscular Dystrophy Coordinating Committee (MDCC), and the Muscular Dystrophy Research and Education Plan for the NIH that was developed by the MDCC and released in September 2004. In addition, in FY 2003, the NIAMS, along with NINDS and NICHD, each funded a Muscular Dystrophy Cooperative Research Center for which additional funding was provided by the Muscular Dystrophy Association. In FY 2004, the three institutes re-issued the solicitation for centers - now known as Senator Paul D. Wellstone Muscular Dystrophy Cooperative Research Centers, and expect to fund two to three additional meritorious centers in FY 2005.
The NIAMS, NINDS, NICHD and the Centers for Disease Control and Prevention sponsored a workshop on the burden of muscle diseases in January 2005. The participants in this workshop identified existing data on the costs and scope of muscle diseases, with a focus on the muscular dystrophies, and recommended strategies for developing new information sources.
Skin diseases significantly compromise daily life for millions of Americans, both physically and psychologically. Researchers supported by the NIAMS have made great progress in our understanding of basic skin biology as well as understanding the bases for skin diseases.
A particular area of focus in the NIAMS portfolio is on the roles of genes in skin diseases, and scientists have advanced our understanding in a number of areas, including identifying two genes on chromosome 17 which are associated with psoriasis. Other studies have identified susceptibility genes for keloids, which are an abnormal form of scarring that disproportionately affects people of color. Investigators studying the physiologic basis for keloid formation were able to determine that a blood vessel growth factor was likely to be associated with keloid formation. This suggests that it may be possible to suppress keloid formation by topical application of an inhibitor of this molecule. In a third area of genetics research, investigators have identified a new mouse model of alopecia areata that has allowed genetic susceptibility studies to be undertaken, and two new regions on chromosomes 8 and 15 were identified. The availability of this new animal model will allow better identification of the genetic basis of alopecia areata as well as provide a basis for testing potential interventions.
Significant progress has been made in our understanding of fundamental life processes and how they go awry in diseases of bone, joints, muscles, and skin. We are proud of the advances that scientists supported by the NIAMS have achieved, and we are excited about initiatives that we have launched. Our goal remains, as always, to improve the health of the American public -- to reduce the burden of disease and to enrich the quality of life for all Americans.
I will be happy to answer any questions that you may have.