Scleroderma

Gleevec® (imatinib mesylate, Novartis) is approved in the United States for the treatment of certain forms leukemias and solid tumors. The recent discovery that Gleevec® inhibits important cellular enzymes that control fibrosis led to studies to evaluate Gleevec® for the treatment of non-malignant diseases, including chronic graft-versus-host disease, lung fibrosis, pulmonary hypertension and systemic sclerosis. As of November 1, 2009 the Clinicaltrials.gov website lists 119 on-going treatment trials with Gleevec®, including six in systemic sclerosis.

Preliminary results from several small open-label clinical trials of Gleevec® in systemic sclerosis were presented at the Annual Meeting of the American College of Rheumatology in Philadelphia (October 18, 2009). While some of the studies report softening of the skin and stabilization of lung abnormalities, the results represent only interim analysis, and studies are still on-going. Side effects (not necessarily related to the study medication) including swelling and fluid retention, nausea, muscle aching and elevated muscle enzymes, and fatigue were common, and in some cases led to discontinuing the medication.

The efficacy and safety of Gleevec® for the treatment of systemic sclerosis are still not well understood, and the interim results from uncontrolled clinical trials are too preliminary for reliable conclusions. Although preclinical research indicates that Gleevec® might be effective for fibrosis, such results cannot be directly extrapolated into the clinical setting. Some people might respond to Gleevec® while others do not. The safety of Gleevec® in systemic sclerosis needs careful evaluation, as does the right dose and duration of treatment. At this time Gleevec® for the treatment for systemic sclerosis should be limited to research studies.

Article courtesy the Scleroderma Foundation.