The NIAMS Intramural Research Program (IRP), and the entire intramural program at NIH, offer unique resources in the biomedical research enterprise. The long-term, high-risk, high-reward focus of the IRP allows researchers to stretch the boundaries of innovation. They are able to build bridges across the traditional silos that tend to separate organizations, patients and scientists, and research results and clinical practice. These collaborations make an enormous difference for all Americans.
One of the clear successes in the NIAMS IRP, which is supported with 10 percent of the Institute’s budget, is the expansion and sharing of the expertise of its faculty around the nation through partnerships with extramural clinical research networks. A number of recently appointed faculty members have accelerated discovery through these partnerships. Peter Grayson, M.D., M.Sc., Head of the Vasculitis Translational Research Program, participates in the Rare Diseases Clinical Research Network as part of the Vasculitis Clinical Research Consortium External. This collaboration led to the recent discovery of a potential biomarker for predicting which patients with a disease known as ANCA-associated vasculitis are more likely to respond to treatment. In addition, Michael Ombrello, M.D., Head of the Translational Genetics and Genomics Unit, is an active member of the Childhood Arthritis & Rheumatology Research Alliance External Web Site Policy (CARRA) and a leader of the International Childhood Arthritis Genetics Consortium (INCHARGE). On behalf of INCHARGE and CARRA, Dr. Ombrello analyzed DNA from nearly 1000 children with systemic juvenile idiopathic arthritis. The researchers showed that changes in the HLA class II locus, a section of the genome related to immune cell function, are strongly associated with an increased risk of developing the condition. NIAMS IRP researcher Andrew Mammen, M.D., Ph.D., Head of the Muscle Diseases Unit, is also part of an important partnership — continuing to practice at the Johns Hopkins Myositis Center he helped create, while pursuing novel research at NIH to uncover how muscle diseases develop and could be treated. These activities demonstrate the power of our IRP scientific collaborations, and how they are helping to connect scientific institutions and speed discovery, for the benefit of patients.
NIAMS IRP researchers are also building bridges to reach additional patients through clinical trials. Over the past year, several new NIAMS-led clinical trials have begun enrolling patients in protocols at the NIH Clinical Center in Bethesda, Md. Among these are two novel interventional studies for lupus patients. One study, initiated by Mariana Kaplan, M.D., Chief of the Systemic Autoimmunity Branch is examining pioglitazone, a diabetes medication, and a second is studying tofacitinib, a rheumatoid arthritis medication. These studies will determine the safety of these medications in lupus patients and explore their potential to reduce symptoms and vascular complications of lupus. Other clinical research led by Raphaela Goldbach-Mansky, M.D., M.H.S., Acting Chief of the Translational Autoinflammatory Disease Section, has led to the identification of the genetic cause for several rare pediatric autoinflammatory diseases, including SAVI. Eric Hansen, M.D., Head of the Immunodeficiency and Inflammation Unit, and Richard Siegel, M.D., Ph.D., NIAMS IRP Clinical Director and Chief of the Autoimmunity Branch, have recently revealed how changes to a specific immune system regulator, called A20, and the molecules that associate with it, may lead to inflammatory disease. This research has paved the way for novel treatments for these conditions. These discoveries are only possible when patients and investigators become partners in research.
Finally, the IRP is closing the gap between scientific discovery and clinical practice. NIAMS IRP researcher Michael Ward, M.D., Chief of the Clinical Trials and Outcomes Branch, led the development of new treatment guidelines from the American College of Rheumatology for ankylosing spondylitis. NIAMS’ orthopaedics researcher Timothy Bhattacharyya, M.D., Head of the Clinical and Investigative Orthopedics Surgery Unit, has also used large surgical outcomes data sets to identify an association between the use of blood thinners and risk of complications after total joint replacement surgery. These types of findings and treatment recommendations provide clinicians with an up-to-date picture of the state of the science, and may enable more efficient and effective use of health care resources.
The NIAMS IRP is a model for facilitating scientific collaborations and patient partnerships, and improving clinical practice. We are eager to strengthen our existing bridges, and to build new ones. I encourage you to stay informed on the latest news from the IRP through the NIAMS website and our IRP Facebook page.
Stephen I. Katz, M.D., Ph.D.
National Institute of Arthritis and Musculoskeletal and Skin Diseases
National Institutes of Health