Approximately 10 percent of the NIH, and in turn NIAMS’, annual budget is devoted to research conducted right here in Bethesda, Maryland, through our Intramural Research Program (IRP). This investment is yielding high returns, and I would like to take this opportunity to tell you more about our IRP and the talented group of scientists who work in it.
The intramural research program at NIH, comprised of scientists from 23 Institutes and Centers, provides a stimulating environment where long-term, high-impact projects can be pursued. The diversity of scientists and research interests across the NIH provides a unique incubator where truly innovative solutions can be explored. Here at the NIAMS, our IRP is home to 21 principal investigators, led by Scientific Director John O’Shea, M.D., and Clinical Director Richard Siegel, M.D., Ph.D. Our program spans eight primary focus areas, including bone biology, clinical and epidemiological research, genomics and epigenomics, immunology, muscle biology, skin biology, structural biology, and stem cell and induced pluripotent stem cell biology. Our intramural researchers’ work is frequently published in top-tier journals, including The New England Journal of Medicine, Science, Nature, and Cell, and they train numerous post-doctoral research fellows and students, helping to build the next generation of researchers.
I am proud to say that over the past year, two drugs have been approved by the U.S. Food and Drug Administration (FDA) based in part on research from the NIAMS IRP. The first, tofacitinib (brand name Xeljanz), was developed through a public-private partnership between Dr. O’Shea and the pharmaceutical company Pfizer. In the early 1990’s, Dr. O’Shea’s research group identified a group of proteins, called Janus kinases, that are important in regulating the human immune system, and they hypothesized that blocking the proteins might protect against the damaging inflammation of rheumatoid arthritis (RA) and certain other autoimmune diseases. After many years of collaborative research, a new class of drugs targeting Janus kinases exists. Tofacitinib is a member of this new class, and is the first new drug in more than a decade that can be taken as a pill, rather than an injection, to slow or halt RA joint damage. Many clinical studies are now being pursued to explore the use of this drug in the treatment of psoriasis and many other inflammatory diseases.
The second FDA approval was for the new use of an established drug to treat a rare genetic disease in children. Neonatal-onset multisystem inflammatory disease (NOMID) is a rare, debilitating disease that strikes within the first weeks of life. If left untreated, children with NOMID may develop hearing and vision loss, cognitive impairment, and physical disability. Previous work by NIAMS IRP clinical researcher Raphaela Goldbach-Mansky, M.D., M.H.S., Acting Chief of the Translational Autoinflammatory Disease Section, showed that the symptoms of NOMID were facilitated through the immune system’s interleukin-1 (IL-1) signaling pathway, and that blocking IL-1 with the FDA-approved RA drug anakinra relieved symptoms of NOMID. Recently, Dr. Goldbach-Mansky and her team conducted a successful clinical trial demonstrating that anakinra not only improved the signs and symptoms of NOMID, but also worked over the long-term to stop the progression of organ damage. Based on the trial’s results, anakinra has become the first FDA-approved treatment for NOMID.
NIAMS IRP researchers are frequently recognized for their outstanding contributions to their fields. Recently, Paul H. Plotz, M.D., Scientist Emeritus, was awarded the 2013 Presidential Gold Medal from the American College of Rheumatology (ACR). The medal is the highest award the ACR can bestow and recognizes "outstanding achievements in rheumatology over an entire career." Dr. Plotz has been with NIH for nearly 40 years, and his pioneering work in myositis, and more recently Pompe Syndrome, is a model for moving basic research from bench to beside, and back again.
NIAMS also enhanced our IRP’s expertise in rheumatology with the addition of two key staff members over the past year. James D. Katz, M.D., is the new Director of the Rheumatology Fellowship and Training Branch overseeing the Rheumatology Fellowship Training Program. He is also the Director of the intramural NIAMS Community Research and Care Branch. Dr. Katz has published studies on decision-making skills in physician trainees and has received numerous honors and awards for his clinical expertise, teaching, and mentoring — skills that will be essential in his new position at NIAMS. I am also pleased to report that Mariana Kaplan, M.D., was appointed as Chief of the newly established intramural Systemic Autoimmunity Branch. Dr. Kaplan is a rheumatologist and most recently was a Professor of Internal Medicine in the Division of Rheumatology, Department of Internal Medicine, at the University of Michigan, where she held several active NIH grants. Her branch will combine natural history and treatment studies with basic investigations to uncover the underlying causes of rheumatic diseases, with an emphasis on systemic lupus erythematosus and other systemic autoimmune diseases affecting adults.
NIAMS’ IRP is a dynamic program and is conducting outstanding work that improves the lives of people with musculoskeletal, rheumatic, and skin diseases. I encourage you to connect with them through the NIAMS IRP Facebook page to stay up-to-date on the latest research. You can also connect to the Rheumatology Fellowship program through their Facebook page or Twitter feed.