It is my great pleasure to highlight the Accelerating Medicines Partnership (AMP) in rheumatoid arthritis (RA) and systemic lupus erythematosus (SLE) program in this month’s letter, since this partnership is fundamentally changing the way we go after drug targets for these diseases. Collaboration has been the hallmark of this effort, and all of our partners are to be commended for their commitment to this vision. The investigators in the AMP RA/SLE Network are studying cells from the joints of RA patients and the kidneys of patients with lupus nephritis. They are adapting cutting-edge technologies to allow those cells to be analyzed individually, one by one, using high throughput.
Image: Robert H. Carter, M.D., NIAMS Deputy Director.
Investigators funded in part by the NIAMS have identified a chemical compound that increases bone mass in mice. The compound, which blocks parathyroid hormone signaling, offers a promising new approach for treating osteoporosis and related conditions.
Researchers at the National Institutes of Health's (NIH) National Center for Advancing Translational Sciences (NCATS) and at the University of Nevada, Reno School of Medicine (UNR Med) have demonstrated that a drug, known as SU9516 and originally targeted unsuccessfully to treat cancer, may have new life as a potential treatment for Duchenne muscular dystrophy. The UNR Med research was supported in part by grants from the NIAMS.
Image: Diaphragm muscle from a SU9516-treated dystrophin deficient mouse showing nuclei (blue), myofibers (outlined in red) and regenerating muscle fibers (green).
Photo credit: Burkin Lab at the University of Nevada, Reno School of Medicine.
Each year, more than 16,000 babies around the world are born with ichthyoses, and more than 50 gene mutations have been identified as responsible for various types and subtypes of the disease. The NIH Director's Blog highlights how a research team—funded in part by the NIAMS—has found another genetic cause, one that has important implications for treatment.
Photo credit: Keith Choate, M.D., Ph.D., Yale University School of Medicine, New Haven, CT.
Scientists Identify Single-Gene Mutations That Lead to Atopic Dermatitis: NIH-Supported Research Suggests Potential Treatment Strategy
Researchers have identified mutations in a gene called CARD11 that lead to atopic dermatitis, or eczema. Scientists from the National Institute of Allergy and Infectious Diseases (NIAID) and other institutions discovered the mutations in four unrelated families with severe atopic dermatitis and studied the resulting cell-signaling defects that contribute to allergic disease. Their findings also suggest that some of these defects potentially could be corrected by supplementation with the amino acid glutamine.
Image: Atopic dermatitis is an inflammatory skin condition that affects an estimated 30 percent of the U.S. population, mostly children and adolescents.
Photo credit: National Institute of Allergy and Infectious Diseases.
Research advances are resulting in improved treatments for psoriasis. NIAMS-supported researcher James T. Elder, M.D., Ph.D., from the Department of Dermatology at the University of Michigan, shares his insights.
Image: James T. Elder, M.D., Ph.D., Department of Dermatology, University of Michigan.
Brian LaFoy has not allowed psoriasis to slow down his active lifestyle. He plays soccer and ice hockey, and enjoys snowboarding, wakeboarding and scuba diving. LaFoy is active with the National Psoriasis Foundation and is a national mentor for psoriasis patients.
Image: Brian LaFoy and family.
The NIH Loan Repayment Programs (LRPs) are designed to recruit and retain qualified health professionals into biomedical or biobehavioral research careers. They repay up to $35,000 annually of a researcher's educational debt in return for a commitment to engage in NIH mission-relevant research. There are five LRPs for health professionals outside of the NIH (extramural). For a comprehensive summary of the programs and eligibility requirements, please visit the Frequently Asked Questions webpage. New award and renewal award applications for the 2017 Extramural LRPs are due between September 1 and November 15, 2017.
Immature muscle cells fuse together during development to form long muscle fibers with many nuclei. To identify factors involved in the fusion process, scientists studied fibroblasts—cells that don’t normally fuse. As shown in the microscopic image, adding a gene that makes a protein named myomerger to fibroblasts causes them to fuse together in flower-like clumps of fluorescently stained cell nuclei. The protein works in tandem with another protein, called myomaker, to cause fusion. By gaining a better understanding of the processes involved in muscle development and regeneration, the research may help lead to new therapies for muscle disorders.
Photo credit: Malgorzata Quinn, Ph.D., and Douglas Millay, Ph.D., Cincinnati Children's Hospital Medical Center Heart Institute.
The NIAMS Advisory Council Meeting will be held September 6, 2017, in Building 31, 6th Floor, C Wing, Conference Room 6, NIH Campus. A meeting agenda will be posted as soon as it is available. The Council meeting will be available for live viewing via the NIH videocasting service as well. The June 21 NIAMS Advisory Council archived videocast is available in the Past Events section of the NIH Videocasting website.
The Science of Caregiving: Bringing Voices Together
August 7–8, 2017
Natcher Building (Building 45), NIH Campus, Bethesda, Maryland
Additional information is available here.
Discovery Channel Documentary: “First in Human: The Trials of Building 10”
August 10, 2017, 9 p.m. ET/PT
Premieres on the Discovery Channel
Details available here.
FDA Public Meeting: Patient-Focused Drug Development for Alopecia Areata
September 11, 2017
1 p.m. to 5 p.m.
FDA White Oak Campus, Silver Spring, Maryland
Additional information is available here.
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 .