A new study suggests that the cancer drug imatinib mesylate (Gleevec) may benefit people with scleroderma, a chronic connective tissue disease.
No effective treatment currently exists for scleroderma, which affects the skin, blood vessels and often muscles and joints, as well as the gastrointestinal tract, kidneys, heart and lungs. About 300,000 people in the United States have scleroderma, which typically strikes people between the ages of 30 and 50, according to the Scleroderma Foundation.
Gleevec is currently approved in the United States to treat two types of cancer-chronic myeloid leukemia and gastrointestinal stromal tumor.
The study included 30 patients with diffuse scleroderma, a widespread, severe form of the disease. They took 400 milligrams of Gleevec a day and were evaluated monthly for 12 months during treatment and were seen for follow-up three months after they stopped taking the drug.
The researchers assessed the effectiveness of the drug treatment by using a tool called the modified Rodnan skin score, a measure of how much skin is affected by the disease.
"The skin score seems to be a very good marker of disease status and most scleroderma trials use this as an outcome measure," said study leader Robert Spiera, MD, an associate attending rheumatologist at the Hospital for Special Surgery and an associate professor at Weill Cornell Medical College. They also used two tests to measure patients' lung function. Lung disease is one of the main causes of death in scleroderma patients.
Interim findings showed a 23% improvement in skin scores and a 9.6% to 18% improvement in lung function tests.
"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."
The interim results were presented at the American College of Rheumatology annual meeting in Philadelphia. The study received funding and donated drugs from Novartis, which makes Gleevec.