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Statement for the Record
House Subcommittee on Labor-HHS-Education Appropriations
March 20, 2012 (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 budget request for the National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS) of the National Institutes of Health (NIH). The Fiscal Year (FY) 2013 NIAMS budget of $535,610,000 includes an increase of $462,000 over the comparable FY 2012 level of $535,148,000.
As the primary Federal agency for supporting medical research on diseases of the bones, joints, muscles, and skin, NIAMS touches the lives of nearly every American. Training the basic and clinical scientists who carry out this research, and disseminating information on research progress in these diseases, are two other important components of the NIAMS mission.
Using Science to Inform Health Care Decisions
Over the past two decades, the NIH Study of Osteoporotic Fractures (SOF) has provided information that health care providers are using to assess people’s bone health. SOF’s finding that bone mineral density (BMD) relates closely to fracture risk, for example, contributed to Medicare’s decision to pay for numerous people to get their BMD measured every two years. Many started taking bone-preserving drugs because of their results, and the rate of hip fractures dropped nearly 25 percent among female beneficiaries1. New, longer-term data from SOF could refine the screening guidance: women at the highest risk of osteoporosis might benefit from annual exams, while frequent measurements may be unnecessary for others. In fact, women with the lowest risk could be tested much less frequently unless other aspects of their health change.
1 Brauer CA, et al. JAMA. 2009. PMID: 19826027
As multiple treatments become available for various conditions, research is needed to help clinicians decide which options are best for their patients. Studies of adults who have rheumatoid arthritis (RA) suggest that aggressive treatment is more beneficial than waiting until the disease progresses. A group of rheumatologists tested whether a similar approach would reduce the disability and health care costs of juvenile idiopathic arthritis (JIA). They compared two therapies and determined that early treatment with either strategy increased the likelihood that the joint-destroying processes would stop.
Many diseases within the NIAMS mission involve pain, fatigue, and other difficult-to-measure symptoms. The ability to quantify changes in these parameters could enhance clinical outcomes research and, ultimately, clinical practice. NIAMS is one of several NIH components engaged in the Patient-Reported Outcomes Measurement Information System (PROMIS) initiative to develop such a tool. In addition to managing PROMIS on behalf of NIH, NIAMS is encouraging researchers to use the resource in ongoing clinical studies of rheumatic, musculoskeletal, and skin diseases.
For the past decade, researchers have been monitoring the health of people who have low back pain due to intervertebral disk herniation, lumbar spinal stenosis, or degenerative spondylolisthesis. Early findings showed that, in general, most surgical patients fared better than patients who received non-operative care, although many patients got better without surgery. Recent data show that the cost-effectiveness of surgery for low back pain due to these disorders—four years after an operation—is comparable to that of other common treatments for non-musculoskeletal conditions.
Community engagement is a key component for translating interventions into health care and integrating lifestyle changes into daily living. To address the well-documented disparities in medical knowledge and research participation, NIAMS will continue its Multicultural Outreach Initiative to improve access to health information for underserved minority populations. FY 2013 plans include field testing program materials and creating an electronic toolkit to facilitate their dissemination.
Investing in Basic Research
Itch is an often difficult and sometimes debilitating symptom of many skin diseases and other disorders within the NIAMS mission. Poor knowledge of the mechanisms underlying chronic itch has hampered the development of pharmacologic treatments. In FY 2013, NIAMS will encourage basic and translational studies in this area.
NIAMS maintains a considerable investment into the genetic and cellular basis of osteoarthritis (OA), with the goal of identifying potential targets for therapies that halt tissue degeneration. Even after researchers develop treatments to stop or reverse OA progression, however, some patients will require total joint replacement. With support from the American Recovery and Reinvestment Act of 2009, researchers made a surprising discovery about the lubricating layer that forms around metal-on-metal hip implants. Instead of cell-based fluid made by the patient, the lubricant is a synthetic material produced through friction. This finding could lead to longer-lasting materials which, in turn, could improve the surgeries’ success and reduce their long-term costs.
With the advent of new laboratory and data mining tools, investigators are making connections among biologic processes and organ systems that previously were viewed independently. For example, researchers are learning that inflammation, which plays an important role in RA and other autoimmune joint diseases, is involved in OA onset and osteoarthritic joint degeneration. Others are exploring how normally harmless microorganisms can lead to RA by causing the immune system to attack healthy tissue.
The technologic advances related to genome-wide analyses have enabled investigators to identify a genetic mutation that causes a rare childhood disease characterized predominantly by inflammation and fat loss. The disorder, named chronic atypical neutrophilic dermatosis with lipodystrophy and elevated temperature (CANDLE), may actually represent a spectrum of diseases that have been described in the literature under a variety of names. More importantly, since no treatment for this disease exists, the findings may have uncovered a possible target for future therapies.
Advancing Translational Science
NIAMS supports several large programs to encourage teams of translational researchers. In FY 2013, it again will partner with other NIH institutes to fund applications for the Wellstone Muscular Dystrophy Cooperative Research Centers program. The Centers have facilitated numerous basic discoveries and animal tests since their establishment in 2003. A group of investigators that includes Wellstone researchers recently published preclinical data about small molecules that target the defective RNA that causes myotonic dystrophy type 1. The cell-culture and mouse-model findings have the potential to benefit people who have myotonic dystrophy type 1; their promise also extends to other conditions that might be amenable to RNA-targeted therapies.
NIAMS strengthened its Small Business Innovation Research (SBIR) program in recent years by inviting eligible companies to propose studies on specific topics that complement the Institute’s other grants. Results from the targeted efforts include a cell-derived human skin substitute for use in consumer product testing, drug discovery, and toxicity screening. NIAMS will continue to look for opportunities that could benefit from an SBIR focus and will solicit applications as areas are identified.
The advances described above are just a few of the contributions that NIAMS-funded investigators have made to save and improve millions of American lives. Collectively, the Institute’s research, training, and health information programs have significantly advanced our understanding of how to treat or prevent many common, chronic, costly diseases. Looking forward, this progress will serve as a strong foundation for the future, as the burden that these conditions place on individuals and society is reduced and, over time, eliminated.