Letter From Dr. Stephen I. Katz: From Basic Science to Clinical Breakthroughs—the NIAMS Intramural Research Program
The first NIAMS Advisory Council meeting of the year gives the Institute an opportunity to share progress with members about the activities of the NIAMS Intramural Research Program (IRP). The annual presentations from NIAMS Scientific Director John J. O’Shea, M.D., and Clinical Director Richard Siegel, M.D., Ph.D., are a special highlight for me. These talks illustrate how the IRP exemplifies the best qualities of the National Institutes of Health (NIH) Intramural Research Program—outstanding basic science discovery, translational and clinical research that bridges the bench and bedside to improve patients’ lives and building the next generation of scientists and clinicians.
Image: Stephen I. Katz, M.D., Ph.D.
The NIAMS is currently operating under a Continuing Resolution through March 23, 2018. The funding plan for research and training grants represents the most current information available. However, many factors can affect the operating policies, and they are subject to change.
New clinical trial findings show that a therapeutic regimen involving transplantation of a person’s own blood-forming stem cells can improve survival and quality of life for people with severe scleroderma, a life-threatening autoimmune disease.
Continuing its commitment to support translational research, the NIAMS announced five Centers of Research Translation (CORT) (P50) awards. CORTs foster the application of basic research to human diseases within the NIAMS mission. Synergistic teams of scientists carry out these programs to address significant translational research challenges.
The NIAMS announced five new Core Centers for Clinical Research (CCCR) (P30) awards. The CCCRs provide avenues to advance the methodological sciences that support clinical research within and across the NIAMS' scientific portfolio. The overall goal of the CCCRs is to develop and apply methods, metrics and outcome measures that address existing and emerging clinical research needs to advance the prevention, diagnosis and treatment of musculoskeletal, rheumatologic and skin diseases.
NIH Director Francis S. Collins, M.D., Ph.D., shares the story of a 15-year-old named Aaron who came to the NIH in 2015 with severe muscle weakness. Andrew Mammen, M.D., Ph.D., leader of the NIAMS’ Muscle Disease Unit, tested Aaron and confirmed a diagnosis of a rare autoimmune disease called anti-HMGCR myopathy. To see what happened, watch Aaron’s video.
In this NIH Director’s Blog post, Dr. Collins features research supported in part by the NIAMS at the University of California, San Francisco. The findings may offer a potentially important “unifying theory” for the genetics of obesity.
Image: Mouse neurons (purple), with their nuclei (blue) and primary cilia (green).
Photo credit: Yi Wang, Ph.D., Vaisse Lab, University of California, San Francisco.
NIAMS-supported researchers at the Massachusetts Institute of Technology developed and photographed this mini-brain organoid. The research is part of the larger Tissue Chip for Drug Screening program funded by the NIH and the Defense Advanced Research Projects Agency.
Image: A mini-brain organoid, the center consisting of a clump of neuronal bodies (magenta) surrounded by an intricate network of branching extensions (green) through which these cells relay information.
Photo credit: Collin Edington, Ph.D., and Iris Lee, Department of Biomedical Engineering, Massachusetts Institute of Technology.
With support from the NIAMS and other NIH Institutes, scientists are making progress in methods to make stem cell donation easier for donors and more successful for people who receive stem cell transplants.
Image: Study co-authors Jonathan Hoggatt, Ph.D., (r) and Bin-Kuan Chou, Ph.D., (l) look through a microscope at a patient’s mobilized stem cells.
Photo credit: Lee Hopkins, OLP Creative.
The NIH lists research accomplishments made by NIH-supported scientists in 2017. This NIAMS-supported research was cited as one of several noteworthy advances in basic research.
Mechanisms of Age-Related Bone Loss: Bone is continually broken down and renewed. Osteoblasts, the cells that build bone, are derived from stem cells that can also form other types of cells, including fat cells. Older adults have fewer osteoblasts and more fat cells in their bone marrow than younger people. Experiments in mice revealed the mechanisms that cause stem cells to create fat cells instead of bone-producing cells. These findings may help explain why bones become weaker in older adults.
Image: A bone from a control mouse, top, compared to one from a Cbfβ-deficient mouse, bottom. The right column shows higher magnifications of the highlighted areas on the left. The Cbfβ-deficient images show reduced bone density and more white, round adipocytes.
Photo credit: University of Alabama at Birmingham, PNAS (Proceedings of the National Academy of Sciences).
FDA Approves First Drug for Eosinophilic Granulomatosis With Polyangiitis, a Rare Disease Formerly Known as the Churg-Strauss Syndrome
The U.S. Food and Drug Administration (FDA) expanded the approved use of Nucala (mepolizumab) to treat adult patients with eosinophilic granulomatosis with polyangiitis, a rare autoimmune disease that causes vasculitis, an inflammation in the wall of blood vessels of the body.
Osteochondroretricular stem cells (red) are a newly identified type of bone stem cell that appears to be vital to skeletal development. Research on these stem cells may lead to treatments for osteoarthritis, osteoporosis and fractures.
Photo credit: Timothy C. Wang, M.D., Columbia University.
On December 6-7, 2017, the NIH and the FDA hosted a public Regenerative Medicine Innovation Workshop that brought together key stakeholders to explore the state of regenerative medicine science involving adult stem cells, with a focus on approaches to the development of safe and effective products. The workshop agenda [PDF – 278 KB] and speaker biographies [PDF – 2.5 MB] are also available.
A video recording of the February 7 NIAMS Advisory Council Meeting is available. The next NIAMS Advisory Council Meeting will be on June 12, 2018.
The NIH’s Wednesday Afternoon Lecture Series offers weekly lectures every Wednesday at 3:00 p.m. in Masur Auditorium, Building 10, NIH Campus. Renowned scientists from around the globe present research on a variety of topics. The lectures are Continuing Medical Education-certified, open to the public and available live via webcast.
March 7, 2018
Navdeep S. Chandel, Ph.D., Northwestern University
“Mitochondria Control of Physiology and Disease: Beyond ATP”
March 28, 2018
Frederic Geissmann, M.D., Ph.D., Memorial Sloan Kettering Cancer Center
“Of Bones, Brain and Fat: Macrophages and the Control of Homeostasis”
NIH Science Lectures and Events Available via Internet
The NIH hosts a number of science seminars and events that are available online through real-time streaming video (videocast). The NIH calendar notes these videocast events with a video icon .