February 2007 (historical)

Authorizing Legislation: Section 301 of the Public Health Service Act, as amended.

Budget Authority:

  FY 2006 Actual FY 2007 Continuing Resolution FY 2008 Estimate Increase or Decrease
BA $507,416,000 $507,752,000 $508,082,000 +$330,000
FTE 211 214 217 +3

This document provides justification for the Fiscal Year (FY) 2008 activities of the National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS), including HIV/AIDS activities. Details of the FY 2008 HIV/AIDS activities are in the "Office of AIDS Research (OAR)" section of the Overview. Details on the Roadmap/Common Fund are located in the Overview, Volume One.

Director's Overview

Institute Mission

The NIAMS supports a broad range of research, training, and health information activities related to arthritis, musculoskeletal, and skin diseases. The diseases studied are both common and rare, affect individuals across the life span, and have a major impact on quality of life and disability. People of all racial and ethnic backgrounds, as well as economic strata, are affected, and in many cases, women and minorities are disproportionately afflicted. Since the Institute's inception twenty years ago, significant progress has been made to better understand the causes of many disorders of the bones, muscles, joints, and skin. NIAMS has also supported the development of new treatments for conditions ranging from osteoporosis, a common disorder that weakens the bones, making them susceptible to fractures, to neonatal-onset multi-system inflammatory disease, a rare and debilitating disorder that affects numerous organs and body systems. An important component of the Institute's stewardship involves strategic planning to identify research and training opportunities and gaps that NIAMS is uniquely positioned to address. The Institute is committed to pursuing high-priority projects across NIAMS mission areas, in collaboration with other NIH Institutes and Centers, and non-Federal partners.

Recent Progress

In the summer of 2006, NIAMS released its Long-Range Research Plan for FYs 2006 to 2009. The plan serves as a broad scientific outline for NIAMS, and the scientific and lay communities, as it identifies compelling research opportunities that will inform the Institute's priority-setting process. Over time, the plan - which includes both cross-cutting and program-specific sections - will help propel research progress related to the understanding, diagnosis, treatment, and prevention of arthritis, musculoskeletal, and skin diseases. Areas of recent emphasis covered in the plan include enhancing research translation across our core disciplines, and strengthening collaborative efforts with both Federal and non-Federal partners in fields of shared scientific interest.

With respect to research training, the long-range plan articulates a broad need to develop a cadre of clinician scientists who are positioned to pursue epidemiology, clinical trials, and health outcomes research across NIAMS mission areas. To facilitate this, the Institute recently partnered with the Orthopaedic Research and Education Foundation and the American Skin Association to encourage applications for individual National Research Service Award post-doctoral fellowships. NIAMS anticipates the first set of these fellowships will be funded in FY 2007. The Institute is also strengthening research training offered to rheumatology fellows in the intramural program, to build a more multi-disciplinary approach and take advantage of diverse faculty and shared resources.

Future Directions

In FY 2008, NIAMS will pursue a number of scientific opportunities identified through the Institute's planning process. These include an emphasis on genome-wide association studies related to diseases of interest to the Institute; support for a new extramural program in musculoskeletal development, tissue engineering, and regenerative medicine; and a continued commitment to the Senator Paul D. Wellstone Muscular Dystrophy Cooperative Research Centers, that promote side-by-side basic, translational, and clinical research. The Institute will also build on its efforts to enhance innovative public-private partnerships, such as the Osteoarthritis Initiative and the Collaborative Initiative on Bone Strength. In health information, NIAMS is committed to develop and disseminate science-based materials for patients, health providers, and the public. The Institute will enhance its efforts to reach underserved populations who may be particularly affected by the diseases NIAMS studies. Finally, NIAMS will integrate insights from on-going program evaluations research training and career development, as well as bone genetics, to ensure that these programs are operating as effectively as possible.

FY 2008 Justification by Activity Detail

Overall Budget Policy

Investigator-initiated research projects and research conducted by new scientists continue to be the Institute's highest priorities. Consistent with NIH policies for FY 2007, the NIAMS will not provide inflationary increases for its grants in FY 2008. The NIAMS will continue its policy of not accepting unsolicited applications for new program project grants, and competing continuation applications for program project grants will only be considered for a second competing award for a total project period of up to 10 years. As in previous years, the NIAMS will reserve a portion of its budget to support high priority research or meritorious applications beyond the established payline. Particular priority will be given to new investigators and first-time renewals.

One of the major strengths of the NIAMS research program is that many of its activities cut across disciplines and involve the collaboration of a diverse array of specialists from both basic and clinical areas. For example, the NIAMS partners with other Institutes, Centers, and non-NIH entities to support research endeavors of mutual interest. Its intramural researchers exchange information with extramural grantees across the United States and other experts around the world. It also is committed to bringing basic and clinical researchers together in a targeted and organized way to facilitate the translation of research findings into practical applications that improve the public's health (see portrait).

Portrait of a Program: NIAMS Centers of Research Translation (CORT)

FY 2007 Level: $11,890,000
FY 2008 Level: $11,890,000
Change: $

A key ingredient in research success is translation of laboratory bench insights to the patient bedside and back again, to inspire new laboratory investigations that ultimately improve patient care and public health. In this vein, NIAMS launched its Centers of Research Translation (CORT) program to unite basic and clinical research in a way that translates basic discoveries into diagnostic approaches and treatments. The first set of centers, focusing on lupus, orthopaedic trauma care, scleroderma, and a genetic form of rickets (a childhood disorder characterized by a softening and weakening of bones), began in FY 2006 and are funded through FY 2011. Funding for a second set of centers is scheduled to begin in FY 2007 and continue through FY 2012.

CORT is a new funding mechanism for NIAMS, developed after an extensive review of the Specialized Centers of Research (SCOR) program. In summary, reviewers concluded that the SCOR program successfully broke down research silos by bringing together scientists who would not have otherwise interacted, and made positive and long-lasting changes in the research culture of the home institution. However, the program had not been designed to function under the modern-day "bench-to-bedside and back-again" paradigm, and it needed to be replaced by a program that would specifically emphasize and support translational research.

The new program includes many SCOR elements that are relevant to translation (e.g., participation of basic and clinical scientists, projects focused on a common disease theme, and core resources). Unlike the SCOR, the CORT program has an overarching goal of translating basic discoveries into clinical investigations, and includes resources and an administrative structure to facilitate this goal. Principal investigators are responsible for identifying diseases that their centers will address, and for outlining research topics on which translation efforts are based. In addition, each center's scientific advisory group helps the translational process, and while their lay members bring the patient perspective to CORT activities.

Arthritis and Rheumatic Diseases

The overall goals of this program are to advance high-quality basic, translational, and clinical biomedical and biopsychosocial research to treat, cure, and prevent arthritis and rheumatic diseases. This includes work that advances the understanding of the natural history of these disorders, as well as mechanisms of disease susceptibility and development. The program supports research in rheumatoid arthritis, adjuvant and chemically-induced inflammatory arthritis, systemic lupus erythematosus, systemic scleroderma, spondyloarthropathies, dermatomyositis and myositis, vasculitis, fibromyalgia, juvenile arthritis and general autoimmunity; basic biology of cartilage and related diseases, such as osteoarthritis, and heritable disorders of connective tissue, such as Marfan's syndrome; gout, Lyme disease, and infection-related arthritis. An important dimension involves taking advantage of new insights in the fields of genetics, genomics, proteomics, and imaging related to arthritis and rheumatic diseases. The NIAMS is committed to pursuing new opportunities that identify risk factors for these disorders, to enhance disease prediction and advance prevention strategies.

Budget Policy: The 2008 budget estimate for the Arthritis and Rheumatic Diseases program is $137,535,000, a decrease of $48,000 or -0.03 percent from the FY 2007 Continuing Resolution. NIAMS plans for FY 2008 include a continued emphasis on the development and validation of biomarkers related to osteoarthritis, and for autoimmune diseases such as rheumatoid arthritis, juvenile arthritis, and systemic lupus erythematosus. The Institute will also support studies to further illuminate the role of genetics in various autoimmune disorders. In addition, it anticipates increased focus on innovative therapies for rare rheumatic diseases, such as vasculitis, and on translational research that facilitates the development of new treatment and prevention approaches.

Musculoskeletal Biology and Diseases

The Musculoskeletal Biology and Diseases program focuses on understanding the fundamental biology of tissues that constitute the musculoskeletal system, and on translating and applying this knowledge to a variety of diseases and conditions. It studies the causes and treatment of acute and chronic injuries-including carpal tunnel syndrome, repetitive stress injury, and low back pain-and clinical and epidemiological studies of osteoarthritis. The program supports the development of new technologies, such as methods for imaging bone and cartilage to improve the diagnosis and treatment of skeletal disorders, or facilitate repair of damage caused by trauma to otherwise healthy musculoskeletal tissue. Therapeutic approaches of interest in the program include drugs, nutritional interventions, joint replacement (including biomaterials and implant science), bone and cartilage transplantation, and gene therapy. Tissue engineering, regenerative medicine, sports medicine, and musculoskeletal fitness are areas of special emphasis.

Budget Policy: The 2008 budget estimate for the Musculoskeletal Biology and Diseases program is $73,436,000, a decrease of $26,000 or -0.03 percent from the FY 2007 Continuing Resolution. NIAMS plans for FY 2008 include partnering with other NIH components, the Food and Drug Administration, industry, and academic centers to launch a new program called the Collaborative Initiative on Bone Strength. It will enable researchers to identify markers of bone strength to be used as surrogate endpoints for fractures in clinical trials, and to find measurements that are more accurate than bone density to predict risk of fracture. This need is more pressing given the aging of the U.S. population and the risk of fractures in this demographic group. Information collected through this effort will facilitate development of new treatments to prevent fractures because it enables the design of clinical trials that are smaller, shorter, and less expensive than current studies. The Musculoskeletal Biology and Diseases program remains committed to another major effort to expedite clinical research-the Osteoarthritis Initiative (OAI). The OAI is a long-term effort, developed with support from numerous NIH components and private-sector sponsors, to create a research resource to identify and evaluate biomarkers of osteoarthritis for use in clinical research. The study has 5,000 participants who are at high risk for knee osteoarthritis and, as of early FY 2007, clinical data from approximately 2,000 of them were available for research projects. All data and resources developed in the OAI will become publicly available for scientific investigation.

Bone Biology and Diseases

The Bone Biology and Diseases program covers a broad spectrum of research to better understand genetic and cellular mechanisms involved in the build-up and break down of bone. It studies regulation of bone remodeling; mechanisms of bone formation, bone resorption, and mineralization; and effects of hormones, growth factors, and cytokines on bone cells. The program emphasizes application of fundamental knowledge of bone cell biology to the development of drug and gene therapies for bone diseases, especially osteoporosis. It supports several large epidemiologic cohorts for characterization of the natural history of osteoporosis, and for identification of genetic and environmental risk factors that contribute to bone disease. Like other cohort studies supported by NIAMS, the ultimate goals are to contribute to the development of better diagnostic tools, treatments, and prevention strategies.

Budget Policy: The 2008 budget estimate for the Bone Biology and Diseases program is $82,370,000, a decrease of $28,000 or -0.03 percent from the FY 2007 Continuing Resolution. Program plans for FY 2008 include continued support of a multi-center study of osteoporosis and fractures in nearly 6,000 older men. Thus far, researchers identified lifestyle, medical, and demographic characteristics that are associated with low bone mass and potential fracture risk in men over age 65-information that health care providers can use to identify men who are at high risk of fracture so they can begin treatment. NIAMS will continue to fund meritorious investigator-initiated grants on bone biology and diseases and encourage research collaborations to mine genetic resources that NIAMS-funded researchers have previously developed.

Muscle Biology and Diseases

The NIAMS' Muscle Biology and Diseases program supports a wide range of basic, translational, and clinical research projects in skeletal muscle biology and diseases. It focuses on fundamental biology of muscle development, physiology, and muscle imaging. Particular interests include the basic biology of satellite and muscle stem cells, excitation-contraction coupling, muscle metabolism, and adaptation of muscle to exercise. It addresses a need for translational research to develop these discoveries into advances in treatment, and improvements in the management of muscle, musculoskeletal diseases, and disorders. Its overarching objective is to advance the understanding of, and, ultimately, prevent and treat the muscular dystrophies, inflammatory myopathies, muscle ion channel diseases, and muscle disorders such as disuse atrophy and age-related loss of muscle mass.

Budget Policy: The 2008 budget estimate for the Muscle Biology and Diseases program is $70,467,000, a decrease of $25,000 or -0.03 percent from the FY 2007 Continuing Resolution. Program plans for FY 2008 include continued support of research efforts related to muscular dystrophy. The original Senator Paul D. Wellstone Muscular Dystrophy Cooperative Research Centers are scheduled to end during FY 2008, and as there is considerable interest in maintaining the program from the scientific community, Congress, and the public (see portrait), the NIAMS will participate in a multi-institute effort to solicit applications from institutions that are interested in participating in the program for FY 2008 and beyond. The Institute will continue to fund meritorious investigator-initiated applications submitted in response to announcements that encourage basic and translational muscular dystrophy research.

Portrait of a Program: Senator Paul D. Wellstone Muscular Dystrophy Cooperative Research Centers

FY 2007 Level: $3,258,000
FY 2008 Level: $3,258,000
Change: $

A major component of the NIAMS muscular dystrophy research portfolio includes funding for two of the six NIH-supported Senator Paul D. Wellstone Muscular Dystrophy Cooperative Research Centers. Each center brings together expertise, infrastructure, and resources focused on major questions about muscular dystrophy. The Wellstone Centers promote side-by-side basic, translational, and clinical research, and provide resources that can be used by the national muscle biology and neuromuscular research communities. The first NIAMS-supported center is located at the University of Pittsburgh where researchers are examining the use of gene and stem cell therapies to treat Duchenne muscular dystrophy (DMD), the most common childhood form of muscular dystrophy. The second center is located at the University of Pennsylvania where researchers are investigating strategies to inhibit muscle degeneration and to promote muscle growth. These approaches could be applicable to a wide range of muscular dystrophies and other muscle diseases. Although funding for the original Wellstone Centers is scheduled to end during FY 2008, NIAMS will participate in a multi-institute effort to solicit applications from institutions that are interested in participating in the program for FY 2008 and beyond.

To enhance the ongoing activities of the Wellstone Centers, NIAMS recently joined several other NIH components in announcing the availability of administrative supplements to the Centers. The first supplement promotes collaborations by the Centers, and maximizes opportunities for career development among junior investigators affiliated with the Centers. The second supplement encourages the Directors of the Wellstone Centers, in collaboration with other muscular dystrophy researchers and representatives from voluntary health organizations, to apply for administrative supplements to support workshops or conferences focused on specific topics in muscular dystrophy research. These workshops fill a specific need in the muscular dystrophy field to bring investigators together to achieve a range of objectives, including developing collaborations, focusing efforts and resources, and reaching consensus on research and patient care strategies. Funding for the supplements and workshops will be available through FY 2009.

Skin Biology and Diseases

This program supports a broad portfolio of basic and translational research in skin. These efforts include work on the developmental and molecular biology of skin, the study of skin as an immune organ, and the genetics of skin diseases. Areas of particular emphasis include investigations of stem cells found in skin; metabolic studies of skin, such as the effects of hormones and interactions with enzymes; and immunologically-mediated cutaneous disorders, such as atopic dermatitis, contact dermatitis, and vasculitis. Research is underway to better understand keratinizing disorders such as psoriasis and ichthyosis; disorders of pigmentation such as vitiligo; and bullous diseases such as pemphigus, pemphigoid, and epidermolysis bullosa. It studies acne and the physiologic activity of the sebaceous glands, as well as disorders of the hair, such as alopecia areata. Tremendous opportunities exist in the field of skin diseases research, from work toward a deeper understanding of the basic biology of skin, to new approaches for developing artificial skin, to advances in imaging technologies for diagnosis and tracking of skin disease progression. The NIAMS is committed to pursuing these and other avenues of research to improve health outcomes for patients with skin diseases.

Budget Policy: The 2008 budget estimate for the Skin Biology and Diseases program is $63,376,000, a decrease of $22,000 or -0.03 percent from the FY 2007 Continuing Resolution. NIAMS plans for FY 2008 include continued support for studies focused on wound healing, to better understand the molecular mechanisms underlying this process with the goal of finding ways to accelerate wound healing and to improve patient outcomes. In addition, the budget estimate anticipates an enhanced emphasis on the genetic factors that contribute to skin diseases such as psoriasis, building on current projects supported by NIAMS and through the Genetic Association Information Network (GAIN).

Intramural Research Program

The mission of the intramural research program of NIAMS is to conduct innovative basic and translational research relevant to the health concerns of the Institute, and to provide training for investigators interested in careers in these areas. The ultimate goals are to provide new insights into the normal function of joints, bone, skin, and muscle-and diseases that affect them-and to generate a cadre of well-trained investigators equipped to continue the progress in understanding these structures and related disorders. NIAMS intramural investigators pursue diverse projects in biomedical research ranging from fundamental analyses of protein structure and function involving crystallography, cryoelectron microscopy, and atomic force microscopy, through protein chemistry, cell biology, signal transduction, gene regulation, tissue development and differentiation, genetics, and immunology, to animal models of disease and direct clinical studies on the genetics, etiology, pathogenesis, and treatment of a variety of rheumatic, autoimmune, inflammatory, joint, skin, and muscle diseases.

Budget Policy: The 2008 budget estimate for the Intramural Research Program is $50,236,000, a decrease of $354,000 or -0.6 percent from the FY 2007 Continuing Resolution. NIAMS plans for FY 2008 include an enhanced focus on translational research, in order to facilitate patient-oriented studies in the areas of arthritis, musculoskeletal, and skin diseases, including their genetic, inflammatory, and immune mechanisms. The Institute will strengthen its efforts in pediatric rheumatic diseases, as well as in musculoskeletal medicine and health outcomes research. Key recruitments in all of these areas are currently underway. In addition, it anticipates an increased emphasis on the development of state-of-the-art research resources, such as core facilities for genetic analyses using chip-based sequencing techniques, to enable cutting-edge investigations across its mission areas. Finally, NIAMS will continue its commitment to multidisciplinary training of rheumatology research fellows, to strengthen the pipeline of highly qualified physician-scientists in this field.

Portrait of a Program: NIAMS Intramural Research Program

FY 2007 Level: $50,590,000
FY 2008 Level: $50,236,000
Change: $ -354,000

Since the founding of NIAMS twenty years ago, its intramural program has pioneered a number of clinical and translational research successes. These include, but are not limited to, landmark clinical trials that established the efficacy of cyclophosphamide to treat patients with lupus nephritis; studies of the natural history, pathogenesis, and treatment of inflammatory disorders such as polymyositis and dermatomyositis; identification of a gene mutation which causes severe combined immunodeficiency, or SCID, and development of a new class of immunosuppressive drugs to treat the disorder; and discovery of the genetic causes of several systemic autoinflammatory diseases, as well as therapies to treat them effectively. Building on these successes to understand and treat inflammatory and immune system disorders, NIAMS will continue its program in clinical and translational research. The overall goal is to facilitate patient-oriented intramural research in the areas of arthritis, musculoskeletal, and skin diseases, including their genetic, inflammatory, and immune mechanisms.

Within funds allocated, NIAMS will pursue novel research studies in the following high-priority areas: investigations into the pathophysiology of human disease; studies of conditions for which it is difficult to assemble adequate patient cohorts in academic health centers; innovative interventional trials in serious disorders for which effective treatment options do not exist; studies capitalizing on the sophisticated imaging and laboratory technologies available in the NIH Clinical Research Center; and development of trans-Institute initiatives in translational research. These cross-cutting activities will establish the foundation for a multidisciplinary, collaborative program in inflammation and immunity. It will enable the development of multi-Institute natural history and treatment protocols, as well as sharing of innovative laboratory resources for clinical samples. In addition, a clinical scholars training program will be pursued to foster interactions among existing training programs with common scientific interests in rheumatology, dermatology, transplantation, gastroenterology, infectious diseases, and allergic disorders.

To ensure the intramural program is operating as efficiently as possible, scientific staff will be realigned to better reflect the high-priority program areas. In addition, emerging technologies will be used to improve the overall management of scientific programs and streamline research activities, as appropriate.

Research Management and Support (RMS)

NIAMS' RMS supports the scientific, administrative management, and information technology expenses associated with the Institute's day-to-day operations. It reflects costs associated with long-term investments in the research enterprise: the review of applications for grants and contracts by special panels of outside experts, and the management and evaluation of grants and contracts after they are awarded. Expenses incurred to disseminate research results and health information to patients, health care providers, and the American public are also included. The Institute currently oversees more than 1,240 research grants and centers, as well as 55 research and development contracts and 125 individual and institutional research training grants. NIAMS supports 421 clinical research projects, including 52 clinical trials.

Budget Policy: The 2008 budget estimate for RMS is $23,976,000, an increase of $237,000 or 1.0 percent from the FY 2007 Continuing Resolution. NIAMS plans for FY 2008 include continued support for the community-based Health Partnership Program, an outreach initiative to address health disparities that exist among local minority groups with rheumatic diseases. The NIAMS will build on a successful feasibility study to systematically evaluate its extramural training and career development awards programs to determine if they are producing the scientists to advance fields relevant to the NIAMS mission. RMS funds will be used to assess the need for a consortium to combine the clinical and genetic resources generated by NIAMS-funded investigations into factors influencing bone mass and fracture risk in humans. Finally, the Institute will continue to sponsor roundtable discussions and a scientific retreat with extramural investigators and lay representatives to inform the research priority-setting and strategic planning process.

Last Reviewed: