Last updated on April 17, 2014 at 7:39 EDT

Promising New Research May Have Impact on Scleroderma Population

May 30, 2012

Key Peptide that Blocks Skin, Lung Fibrosis Could Improve Mortality Rates

DANVERS, Mass., May 30, 2012 /PRNewswire-USNewswire/ — A member of the Scleroderma Foundation’s Board of Directors has led a team of researchers who have identified a peptide that can block fibrosis of the skin and lungs, according to a report published today. “Lung fibrosis currently is the number one cause of death in patients with scleroderma,” said the report’s senior author Carol A. Feghali-Bostwick, Ph.D. “Identifying a way to stop this process from happening could have enormous impact on mortality and quality of life.” Dr. Feghali-Bostwick is vice chair of the Scleroderma Foundation’s Board of Directors and chair of the Foundation’s Research Committee.

In the research reported today in Science Translational Medicine, investigators were looking into agents that promote fibrosis when, by happenstance, they discovered the peptide, a stretch of amino acids representing a small piece of a protein that stopped fibrosis in mice.

Tissue fibrosis contributes to 45 percent of all deaths in developed countries, according to Dr. Feghali-Bostwick. To determine if the peptide, called E4, is relevant for human disease, investigators tested it in human skin samples maintained in the laboratory. The results indicated that the peptide was successful at reducing fibrosis in human skin. This suggests that the peptide has a high likelihood of being effective in humans with fibrosis. “Identification of a molecule that stops fibrosis in both skin and lung brings us closer to a possible treatment for fibrosis. Currently, the only therapy for lung fibrosis is transplantation,” she said.

These findings emphasize the importance of supporting research on scleroderma and related diseases. “This should be a call-to-action as to why more dollars for scleroderma research is essential,” said Joseph P. Camerino, Ph.D., chair of the Foundation’s Board of Directors. “Many investigators who have been previously funded by the Foundation have become leaders in their field. This indicates the importance of investing in research for scleroderma. It ensures that the research will continue so we can find the cause and a cure for this devastating disease.” Early in her career, the Scleroderma Foundation supported Dr. Feghali-Bostwick. In 1999, she received the Foundation’s New Investigator Grant to study twins with scleroderma.

“This new data highlights the rapid progress being made in scleroderma research,” said John Varga, M.D., director of the Scleroderma Program and professor of medicine at Northwestern University’s Feinberg School of Medicine, and also chair of the Scleroderma Foundation’s Medical Advisory Board. “These are exciting findings, but we need to remember that the new compound is not the cure. Rather, it is a piece to the puzzle that will eventually lead us to find the cure.”

Dr. Feghali-Bostwick is an associate professor, Division of Pulmonary, Allergy and Critical Care Medicine, and co-director of the Scleroderma Center at University of Pittsburgh School of Medicine.

Other co-authors of the study include Yukie Yamaguchi, M.D., Ph.D., Takahisa Takihara, M.D., Roger A. Chambers, B.S., and Kristen L. Veraldi, M.D., Ph.D., of the Division of Pulmonary, Allergy and Critical Care Medicine, the Department of Pathology, and the Scleroderma Center, University of Pittsburgh School of Medicine; and Adriana T. Larregina, M.D., Ph.D., of the Departments of Dermatology and Immunology, University of Pittsburgh School of Medicine, and the McGowan Institute for Regenerative Medicine.

About Scleroderma
Scleroderma is a chronic, often progressive, autoimmune disease in which the immune system attacks its own body.

Scleroderma means “hard skin.” It can cause a thickening and tightening of the skin. In some cases, it causes serious damage to internal organs including the lungs, heart, kidneys, esophagus and gastrointestinal tract. As scarring, or sclerosis, of these organs and organ systems progress, they work less effectively, and can lead to organ failure and death.

Some medications and treatments can help with certain symptoms, but there is no cure for scleroderma. The disease affects about 300,000 people across the U.S. In comparison, approximately the same number of people are affected by multiple sclerosis.

About the Scleroderma Foundation
The Scleroderma Foundation is the national organization for people with scleroderma, their families and friends. It was formed Jan. 1, 1998, by a merger between the United Scleroderma Foundation and the Scleroderma Federation.

Today, the Scleroderma Foundation is headquartered in Danvers, Mass. It is a 501(c)(3) non-profit organization dedicated to serving the interests of people living with scleroderma. The Foundation has a network of 23 chapters and 168 support groups committed to carrying out the three-fold mission of support, education and research. The Scleroderma Foundation makes available at least $1 million per year in new research grants to find the cause, better treatments, and a cure for scleroderma.

For more information about the Scleroderma Foundation, visit www.scleroderma.org or call (800) 722-HOPE (4673). You also can find the Foundation on Facebook (www.facebook.com/sclerodermaus) and Twitter (@scleroderma).

For more information, visit the Science Translational Medicine website at http://stm.sciencemag.org

Editor’s Note: To arrange an interview with Dr. Feghali-Bostwick, please contact Christina Relacion at (800) 722-4673, Ext. 243, or email crelacion@scleroderma.org.

SOURCE Scleroderma Foundation

Source: PR Newswire