What’s new on the horizon for scleroderma?
NIH-supported experts review stem cell transplantations, anti-fibrosis drugs, and more
There may not be a cure yet, but many scleroderma experts are optimistic about ongoing research into new treatments.
We asked John Varga, M.D., professor of medicine and director of the Northwestern University Scleroderma Program, and Carol Feghali-Bostwick, Ph.D., a scleroderma patient herself and a professor of medicine at the Medical University of South Carolina, to discuss potential new advancements. Both researchers work with NIH's National Institute of Arthritis and Musculoskeletal and Skin Diseases.
"Stem cell transplants could be a promising treatment for those with very severe scleroderma." - John Varga, M.D.
There are major lung studies looking at drugs that can halt the development of thick scarring tissue, called fibrosis, that can occur in systemic scleroderma, as well as other lung diseases like asthma and chronic obstructive pulmonary disease, Dr. Feghali-Bostwick says.
"My team and I also have used NIH funding to identify an essential peptide—a small piece of protein—that seems to be able to stop and possibly reverse the formation of fibrosis in mouse and human tissue. We are now researching how this peptide works, with hopes of testing it in a clinical trial," she adds.
More drugs for rare diseases
Many immunomodulatory drugs, which can regulate the immune system, are in clinical trials. And a number of recent studies have found that drugs that block an inflammation-causing protein in lupus and similar diseases may also work for scleroderma.
Stem cell transplants
There is a lot of interest in a recent NIH-funded study that found that stem cell transplants for people with severe scleroderma could have long-term, beneficial results, says Dr. Varga. In the study, chemotherapy and radiation were used to wipe out the person's immune system, and then the person's own stem cells were used to rebuild a new system. But Dr. Varga and others warn that a stem cell transplant is not appropriate for every patient and can involve dangerous side effects. "But it could be a promising treatment for those with very severe scleroderma," he says.