The researchers are so encouraged by the results of their experiments in mice that they are in the early planning stages of a clinical trial incorporating their findings in human patients with the disease.
Scleroderma is a chronic degenerative disease that afflicts more than 300,000 Americans, primarily women. The life-threatening disorder is marked by dramatic tissue damage including hardening of the skin, shrinking of muscles, and damage to organs and blood vessels. To date, physicians have been unable to determine what causes the disease, and the available few therapies, serve primarily to relieve symptoms, according to the researchers.
"These new insights are critical clues to understanding a dreadful disease that has so far been impenetrable in terms of what causes it, by what mechanisms it works and why patients get so sick," said cardiologist Pascal Goldschmidt, M.D., senior member of the research team and chairman of Duke's Department of Medicine.
The results of the Duke studies were published in the Nov. 1, 2005, edition of Public Library of Science Medicine. The research was supported by the Scleroderma Research Foundation, San Francisco.
"While we really don't understand what causes scleroderma, we suspect that it may be autoimmune in nature, or that the body's own immune system is involved," said Chunming Dong, M.D., lead author of the paper "Using a novel mouse model, we were able to get a much better understanding of possible mechanisms of the di
Contact: Richard Merritt
Duke University Medical Center