First Drug For Scleroderma Treatment Maybe on Horizon

Until now, no drug has been found to be effective for treating scleroderma. Results of a new study to be presented at the annual meeting of the American College of Rheumatology in Philadelphia on October 18 may change that.

Approximately 300,000 people in the United States have scleroderma, a chronic connective tissue disease in which hardening of the skin is one of the most obvious signs. Scleroderma can also affect other areas of the body, including the blood vessels, lungs, kidneys, heart, and gastrointestinal tract. The disease can range in severity from mild to life-threatening, which depends on where and to what extent the disease develops.

Scleroderma affects women about four times more than men, and it typically first appears between the ages of 30 and 50. No cure has been found, and treatment consists of medications to relieve symptoms that can range from heartburn to itchy skin, dry mouth, skin fibrosis, pulmonary fibrosis (lung disease), depression, joint and muscle pain, and Raynaud’s syndrome.

The potential new treatment for scleroderma is Gleevec (imitinib mesylate), a drug that is currently approved to treat certain types of cancer. In the study, which was conducted by investigators at Hospital for Special Surgery, 30 patients with severe scleroderma were given 400 mg of Gleevec daily. Patients were evaluated every month for one year and monitored for three months after stopping the drug.

The investigators evaluated how much of the skin was affected by the disease and also measured lung function, as lung disease is the main cause of death among people with scleroderma. After one year of treatment, the patients had a 23 percent improvement in skin scores and improvements in lung function ranging from 9.6 to 11 percent. The improvement in lung function is especially important, as this aspect of scleroderma typically worsens over time. In this study, however, the patients improved in two vital lung tests: forced vital capacity and diffusion capacity.

Further studies of the use of Gleevec in the treatment of scleroderma need to be conducted. Part of that effort and other research of scleroderma may be facilitated by passage of the “Scleroderma Research and Awareness Act,” a bill (H.R. 2408) that was introduced to the United States Congress in May 2009 and that is still accepting sponsors.

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