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Witness appearing before the Senate Subcommittee on Labor-HHS-Education Appropriations
May 21, 2009 (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 President’s Fiscal Year 2010 Budget for the National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS) of the National Institutes of Health (NIH). The FY 2010 budget includes $530,825,000, which is $5,953,000 more than the FY 2009 appropriation of $524,872,000.
As the primary Federal agency for supporting medical research on diseases of the bones, joints, muscles, and skin, the NIAMS touches the lives of nearly every American. For example, the U.S. Bone and Joint Decade notes that one in two people will experience back pain each year, and one in five will have pain that affects their ability to work. The National Arthritis Data Working Group estimates that 21 percent of adults have arthritis in at least one joint, a figure that is likely to grow as the population ages. Likewise, one of every two women and one in four men aged 50 years and older suffer fractures each year because of osteoporosis; researchers project that the number of osteoporotic fractures in the United States will grow from 2 million to more than 3 million in the next two decades. The NIAMS is committed to preventing disabilities and reducing costs associated with these and other conditions through balanced basic, translational, and clinical research investments.
As the Institute sets priorities, it is considering how recent advances have positioned its research community for discoveries to prevent disease and improve each American’s life. It is soliciting input from researchers, health care providers, patients, and the public on promising areas of inquiry; pressing scientific needs; programs to ensure a continuing supply of well-trained researchers; and strategies to eliminate health disparities. An important consideration is how investigators can engage in multidisciplinary opportunities. Chronic pain, for example, is an aspect of many diseases that are part of the NIAMS portfolio; staff are exploring partnerships through the Trans-NIH Pain Consortium. Prospects for stem cell research are growing rapidly as researchers isolate stem cells from skin and other organs, and as more lines become available under the Nation’s policy for Federal support of embryonic stem cell research.
Consistent with the Federal commitment to double NIH-wide cancer research spending, the NIAMS will continue to pursue collaborations with the National Cancer Institute in support of high quality projects that relate directly to diseases and organ systems within the NIAMS mission, particularly the bones and the skin. Already, the NIAMS supports research on mechanisms underlying skin cancers, and investigators have uncovered a strategy that kills tumor cells with less damage to healthy skin.
Research to identify susceptibilities to and initial symptoms of disease, and to develop strategies to slow disease progression, is a NIAMS priority. Building on findings that early, aggressive therapy alters the course of rheumatoid arthritis (RA), NIAMS is comparing treatments against a related disease—juvenile idiopathic arthritis.
The NIAMS and the National Institute on Aging lead the Osteoarthritis Initiative (OAI), a public-private partnership to identify and evaluate biomarkers of osteoarthritis (OA). NIH and its partners, with input from the Food and Drug Administration, launched the OAI in 2001. Over 1100 researchers worldwide have accessed OAI data to explore issues such as differences in OA progression, or why only some people with x-ray evidence of OA develop pain. In 2010, the NIH will extend the OAI for six years. It expects the OAI to suggest approaches for slowing joint damage, facilitate clinical testing of interventions and allow clinicians to identify risk factors for OA development, predict severity, and personalize treatments for their patients.
COMPLEX GENETIC DISEASES
The NIAMS community is benefiting from another public-private partnership, the Genetic Association Information Network (GAIN). Since GAIN’s inception, NIAMS investigators have been involved in its Collaborative Association Study of Psoriasis, an ambitious effort to combine genetic and clinical information from people affected by psoriatic skin disease and psoriatic arthritis. The project has yielded a wealth of data that researchers are using to develop diagnosis, treatment, and prevention strategies.
NIAMS-funded investigators have uncovered genetic susceptibility markers of alopecia areata and other autoimmune or auto-inflammatory skin and joint diseases, including lupus. Collaboration among U.S. and European researchers recently linked a component of the immune system and RA. At the NIH Clinical Center, sample collection has begun for a genomic analysis of Behçet's disease, a complex disorder of inflammation affecting skin, eyes, gastrointestinal tract, lungs, vasculature, and joints.
COLLABORATIONS AND TEAM SCIENCE
Behçet’s disease is one of many conditions researchers are studying through the new NIH-wide Center for Human Immunology, Autoimmunity, and Inflammation. NIAMS’ intramural program is taking a leadership role in the Center. Collaborations among scientists from several NIH institutes who are studying related disease systems will facilitate studies about conditions associated with defective immune or inflammatory responses, and will allow them to apply their results to the development of interventions and, ultimately, disease prevention strategies.
In collaboration with orthopaedic surgeons at the Walter Reed Army Medical Center, NIAMS researchers recently discovered that tissue commonly discarded as waste contains special cells that feature many of the same properties as adult stem cells. The cells can be used for regenerative medicine, such as treating war-traumatized muscle, without subjecting patients to additional surgeries and related complications.
The NIAMS participates in the multi-Institute Senator Paul D. Wellstone Muscular Dystrophy Cooperative Research Centers program. In addition to conducting research, scientists at the Centers maintain core resources that all who are studying muscular dystrophy can use. A group of NIAMS-funded muscle researchers showed that defects in blood vessel constriction are associated with the severe fatigue that people with muscular dystrophy experience; mouse experiments suggest that compounds with FDA-approval for other conditions may improve symptoms. Other scientists uncovered molecules that confer many of the benefits of exercise, at least in mice; the findings might lead to treatments for conditions that leave patients unable to exercise.
The scale and complexity of today's research problems and their solutions demand that the NIH explore new models for team science. In FY 2008, the NIAMS started a program, Building Interdisciplinary Research Teams (BIRT), to promote partnerships among fields that share interests, but historically do not interact. Because collaborations proposed in the first round of applications suggested that modest investments in the program will provide great dividends, the NIAMS opened BIRT up to additional communities and expects to make another set of awards at the end of FY 2009.
In the past year, the NIAMS has made considerable progress in leading a trans-NIH partnership with the National Aeronautics and Space Administration. By designating the U.S. portion of the International Space Station (ISS) as a National Laboratory, Congress underscored the significance that Americans place on the ISS’ research potential. The NIH shares this belief and, for the next three years, will accept applications for studies that use the ISS for experiments directly related to the NIH goals of understanding human physiology and promoting the public’s health.
One element of improving the Nation’s health is to support clinical studies on which physicians can rely when discussing treatment options with patients. Before the Spine Patient Outcomes Research Trial (SPORT), many who had low back pain were conflicted about surgery. Now, patients can be assured that surgery relieves pain from herniated disks, but—if the pain is tolerable and not worsening—it will likely subside without surgery. Similarly, people who have pain due to spinal stenosis (a narrowing of the spinal column that occurs with age) are likely to benefit more from surgery than from non-invasive treatments such as physical therapy; but, they are not causing more damage if they adopt a “wait-and-see” approach before committing to an operation. Recently, SPORT offered guidance to help people who suffer from herniated disks personalize their treatment decisions by reporting that study participants who had surgery on an upper lumbar disk improved more than those with damage further down.
For decades, the NIAMS has invested heavily in efforts to understand fracture risk and to uncover strategies to prevent and treat bone loss. Although physicians now have an array of medications for people who are at risk of osteoporosis, many patients fail to benefit fully because they do not follow the treatment regimens. Because a method to improve compliance could immediately slow the growing health and economic burden that osteoporosis places on society, the NIAMS is funding research in this area.
The discoveries and activities highlighted above are just a few examples of research that will continue to benefit Americans from all walks of life. In partnership with government and private entities, the NIAMS also develops and distributes science-based health information directly to patients, health care providers, and the public. The Institute will continue outreach to diverse populations through research, training, and information dissemination. Collectively, NIAMS programs have spurred understanding of many common, chronic, and costly diseases. Looking forward to the next decades, this progress provides a foundation for an era in which the burden of these debilitating conditions is reduced and—with time, continued support from the American public, and the dedication of our Nation’s researchers—eliminated for millions of affected adults, children, and families.
Stephen I. Katz, M.D., Ph.D. has been Director of the National Institute of Arthritis and Musculoskeletal and Skin Diseases since August 1995 and is also a Senior Investigator in the Dermatology Branch of the National Cancer Institute. He was born in New York in 1941 and his early years were spent in the Washington, D.C., and Bethesda, Maryland areas. After attending the University of Maryland, where he graduated with honors, he graduated from the Tulane University Medical School with honors in 1966. He completed a medical internship at Los Angeles County Hospital and did his dermatology residency at the University of Miami Medical Center from 1967 to 1970. He served in the U.S. military at Walter Reed Army Medical Center from 1970 to 1972. From 1972 to1974, Dr. Katz did a postdoctoral fellowship at the Royal College of Surgeons of England and obtained a Ph.D. degree in immunology from the University of London in 1974. He then became Senior Investigator in the Dermatology Branch of the National Cancer Institute and assumed the position of Acting Chief in 1977. In 1980, he became Chief of the Branch, a position he held until 2001. In 1989, Dr. Katz also assumed the position of Marion B. Sulzberger Professor of Dermatology at the Uniformed Services University of the Health Sciences in Bethesda, Maryland, a position from which he resigned in 1995.
Dr. Katz has focused his studies on immunology and the skin. His research has demonstrated that skin is an important component of the immune system both in its normal function and as a target in immunologically-mediated disease. In addition to studying Langerhans cells and epidermally-derived cytokines, Dr. Katz and his colleagues have added considerable new knowledge about inherited and acquired blistering skin diseases.
Dr. Katz has trained a large number of outstanding immunodermatologists in the U.S., Japan, and Europe. Many of these individuals are now leading their own high-quality, independent research programs. He has served many professional societies in leadership positions including as a member of the Board of Directors and President of the Society for Investigative Dermatology, on the Board of the Association of Professors of Dermatology, as Secretary-General of the 18th World Congress of Dermatology in New York in 1992, as Secretary-Treasurer of the Clinical Immunology Society, and as President of both the International League of Dermatological Societies and the International Committee of Dermatology. Dr. Katz has also served on the editorial boards of a number of clinical and investigative dermatology journals, as well as several immunology journals. He has received many honors and awards, including the Master Dermatologist Award and the Sulzberger Lecture Award of the American Academy of Dermatology, the National Cancer Institute=s Outstanding Mentor Award, honorary membership in numerous international dermatological societies, election into the Institute of Medicine of the National Academy of Sciences (USA), the Alfred Marchionini Gold Medal, an Honorary Doctor of Medicine Degree from the University of Munich, the Lifetime Achievement Award of the American Skin Association, and a Doctor Honoris Causa Degree from the Semmelweis University in Budapest, Hungary, and the University of Athens in Greece. Dr. Katz has twice received the Meritorious Rank Award and has also received the Distinguished Executive Presidential Rank Award, the highest honor that can be bestowed upon a civil servant. In July 2006, he received the Harvey J. Bullock, Jr., EEO Award in recognition of his extraordinary leadership in scientific, programmatic, and administrative arenas, and in Sept. 2006, he received the Excellence in Leadership Award from the Intl. Pemphigus Foundation. Dr. Katz received the AChange It@ Champion 2007 Award from Parent Project Muscular Dystrophy. He received the Marriott Lifetime Achievement Award from the Arthritis Foundation, Nov. 2007. Dr. Katz received a Doctor Honoris Causa Degree from the University of Athens in Greece on Oct. 2008. He will receive the 2009 Albert M. Kligman/Philip Frost Leadership Lecture and Award from the SID in Montreal on May 2009.