Treatment for Scleroderma?
A drug currently approved to treat cancer could provide the first treatment for scleroderma, according to researchers. Gleevec has shown to be effective in treating those with the chronic connective tissue disease.
"There has never been a drug that has been shown to be effective for this condition. I think there is a very good chance of Gleevec becoming a real treatment for a previously untreatable disease," Robert Spiera, M.D., an associate attending rheumatologist at Hospital for Special Surgery who led the study was quoted as saying.
Researchers enrolled 30 patients with diffuse scleroderma, a widespread severe form of the disease, and gave them 400 mg of Gleevec every day. To measure the effectiveness of the drug, researchers used a tool known as the Rodnan skin score; a measure of how much skin is affected by the disease. The investigators also measured lung function and diffusion capacity, a measurement of the lung's capacity to transfer gases. Lung disease is the main cause of mortality in scleroderma.
After one year, the investigators saw a 23 percent improvement in skin scores. They also saw an improvement in lung function. Patients on the treatment saw an 11 percent improvement in diffusion capacity scores.
"The lung function data was really exciting," Dr. Spiera said. "In patients with scleroderma, you usually see lung function tests getting worse over time, and if doctors try a therapy for a year and a patient doesn't get any worse, we get pretty excited. What is amazing to me in this study is that we actually saw improvements in both lung function tests."
Scleroderma affects not only the skin, but also underlying blood vessels, and often muscles and joints, as well as the gastrointestinal tract, kidneys, lungs and heart. According to the Scleroderma Foundation, 300,000 people have the disease in the US. The disease usually strikes in the prime of patients' lives, when they are 30-50 years old.