June 7, 2018

SPOTLIGHT

Guest Director’s Letter: Integrating the Patient’s Voice in Drug Development and Regulatory Decision Making

Dear Colleagues:

It is my pleasure to introduce Theresa M. Mullin, Ph.D., Associate Director for Strategic Initiatives at the FDA’s Center for Drug Evaluation and Research (CDER). Dr. Mullin leads the Patient-Focused Drug Development initiative, which includes work related to the FDA Reauthorization Act and implementation of the 21st Century Cures Act. She also oversees CDER's International Program.

Read more.

Image: Theresa M. Mullin, Ph.D.

 

 

NEWS

Fiscal Year (FY) 2018 Funding Plan

The NIAMS Fiscal Year (FY) 2018 Funding Plan has been updated. In addition, the FY 2019 Congressional Justification has been posted.

New Clues to the Causes of Lupus

Insights from two recent studies suggest novel avenues for treating the autoimmune disease systemic lupus erythematosus (SLE). The NIAMS supported the pair of studies that sought to better understand why the immune system mistakenly attacks its own tissues and organs, as happens in SLE. The work could possibly impact other autoimmune diseases as well.
 
Image: Kidney blood vessels (red) in a mouse model of lupus show signs of damage, as evidenced by immune cells (dark purple/light pink) collecting around them [upper panel]. This effect is eliminated in IL-23-insensitive mice [lower panel].

Photo credit: Vasileios Kyttaris, M.D., Beth Israel Deaconess Medical Center

 

 

Early Antiretroviral Therapy in HIV Patients Tied to Bone Mineral Density Loss

An international research team funded by the NIAMS and the National Institute of Allergy and Infectious Diseases (NIAID) found that antiretroviral treatment (ART) for HIV-positive patients is associated with a greater loss of bone mineral density than for those who delay ART.

NIH Researchers Crack Mystery Behind Rare Bone Disorder: Study Finds Gene Mutations That Cause “Dripping Candle Wax” Bone Disease

Melorheostosis, also called “dripping candle wax” disease, is a rare bone condition where excess bone formation can look like dripping candle wax on x-rays. Intramural researchers from the NIAMS and the Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) discovered a genetic basis for the disease and uncovered new information about metabolic pathways of normal bone.
 
Image: An x-ray image of a patient with melorheostosis shows excess bone formation, likened to dripping candle wax.
 
Photo credit: NIH

Optimizing Steroid Treatment for Duchenne Muscular Dystrophy

Research funded in part by the NIAMS has revealed insights into glucocorticoid steroid treatment for Duchenne muscular dystrophy (DMD). One study determined the mechanisms of how glucocorticoid steroids improve muscle repair and function in mouse models of acute muscle injury and muscular dystrophy. A second study found that in patients with DMD, long-term glucocorticoid steroid treatment delayed loss of muscle strength and function and decreased risk of death.
 
Image: Images of mouse muscle repair without (top) and with (bottom) treatment with the glucocorticoid steroid prednisone. Red indicates muscle injury, which is reduced by prednisone. Green shows repair cap (scab) forming over injury. The repair forms more quickly with glucocorticoid steroid treatment.
 
Photo credit: Elizabeth M. McNally, M.D., Ph.D., Northwestern University

 

 

Gut Microbe Drives Autoimmunity

With support from the NIAMS and other Institutes at the National Institutes of Health (NIH), researchers found that a certain gut microbe can trigger autoimmune disease in mice that are prone to such disease. The microbe can breach the intestinal lining and move into the bloodstream and other organs. In limited testing in people, they identified the same microbe in the livers of people with autoimmune diseases.
 
Image: The bacterium E. gallinarum (shown in orange) was found in liver tissue.

Photo credit: Martin Kriegel Lab, Yale School of Medicine

Dietary Fiber Protects Obese Mice From Arthritis

A NIAMS-funded study found that in obese mice, boosting the growth of certain gut bacteria with a fiber known as oligofructose may reduce osteoarthritis.
 
Image: A dietary fiber known as oligofructose protected obese mice from loss of cartilage in the knee joint. The joint of a control mouse, top, showed a large white area of missing tissue. Mice given oligofructose, bottom, had less tissue loss. 
 
Photo credit: Schott, et al., JCI Insight

 

 

Epstein-Barr Virus Protein Can “Switch on” Risk Genes for Autoimmune Diseases

Researchers found a mechanism that may explain why the Epstein-Barr virus is associated with certain autoimmune illnesses such as lupus.
 
Image: An electron microscopy image showing three Epstein-Barr virions.
 
Photo credit: NIAID

 

Early Stimulation Improves Performance of Bioengineered Human Heart Cells

Researchers at Columbia University, funded in part by the NIAMS, created a model of human adult-like heart muscle that can be used for drug testing.
 
Image: The iPS-derived cardiomyocytes have formed heart tissue that closely mimics human heart functionality at over four weeks of maturation.
 
Photo credit: Columbia University

 

 

All of Us Research Program Begins National Enrollment 

On May 6, the All of Us Research Program began national enrollment by inviting people ages 18 and older, regardless of health status, to join this momentous effort to advance individualized prevention, treatment and care for people of all backgrounds. Enrollment is open to all eligible adults who live in the United States. Enroll by visiting www.JoinAllofUs.org.

 

 

 

 

NIH Director’s Blog: Building Muscle in a Dish

NIH Director Francis S. Collins, M.D., Ph.D., highlights research supported by the NIAMS in this Director’s Blog. This image of skeletal muscle satellite cells was captured using a standard fluorescent microscope and was selected as a winner in the Federation of American Societies for Experimental Biology’s 2017 BioArt competition.
 
Image: Multinucleated muscle cells grown in culture.
 
Photo credit: Kevin Murach, Charlotte Peterson, and John McCarthy, University of Kentucky, Lexington

FDA Approves First Therapy for Rare Inherited Form of Rickets, X-Linked Hypophosphatemia

The FDA approved Crysvita (burosumab), the first drug approved to treat adults and children ages 1 year and older with x-linked hypophosphatemia, a rare, inherited form of rickets. NIAMS-supported research contributed to the development of this drug.

RESOURCE

Spotlight on Scientific Imagery: Stem Cells Healing a Skin Wound 

This microscopic image shows stem cells (stained green) migrating into a mouse skin wound to heal it. Cell nuclei are stained blue and the protein integrin alpha 5, a marker of migrating cells, is stained red. Researchers at The Rockefeller University have found that epithelial stem cells, which reside in a deep layer of the skin, “remember” wounds or other inflammatory events. This ability helps them respond faster to subsequent injuries.
 
Photo credit: Elaine Fuchs, Ph.D., Laboratory of Mammalian Cell Biology and Development, The Rockefeller University

 

We invite you to subscribe to the NIAMS Community Outreach Bulletin, which is an online digest designed to inform community advocates and health professionals about resources for diverse audiences on conditions of the bones, joints, muscles and skin and ways to stay healthy. The NIAMS also publishes the Honoring Health: Resources for American Indians and Alaska Natives e-newsletter, which is distributed three times per year and highlights a different health topic for each issue, along with helpful resources for community members and health professionals.

EVENTS

June NIAMS Advisory Council Meeting

The NIAMS Advisory Council Meeting will be held June 12, 2018, in Building 31, 6th Floor, C Wing, Conference Room 6, NIH Campus.  A meeting agenda is available. The Council meeting will be available for live viewing via the NIH videocasting service as well.

FDA Center for Biologics Evaluation and Research Science Symposium

June 25–26, 2018
9 a.m. to 3 p.m.
White Oak Campus, 10903 New Hampshire Avenue, Building 31, Great Room, Silver Spring, Maryland
Registration is required for in-person or webcast attendance.

FDA Public Meeting: Patient-Focused Drug Development for Chronic Pain

July 9, 2018
10 a.m. to 4 p.m.
White Oak Campus, 10903 New Hampshire Avenue, Building 31, Great Room, Silver Spring, Maryland
Registration is required for in-person or webcast attendance.

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 .

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