Animal Trial of New Muscular Dystrophy Drug Yields Promising Results

A drug for treatment of some types of juvenile muscular dystrophy (MD) currently being tested in animals has been found to satisfactorily control the disease without causing unwanted immunosuppressive effects.

The drug, Debio 025 (D-MeAla3-EtVal4-cyclosporin), is a selective inhibitor of cyclophilin, a protein that binds to and suppresses the activity of the antirejection drug cyclosporin A (CsA).

Investigators from the University of Padova (Italy) worked with a line of mice that functioned as an MD model due to collagen VI defects. They reported in the June 10, 2009, online edition of the British Journal of Pharmacology that muscle cells taken from mice that had received the drug for five days showed decreases in numbers of abnormal muscle fibers similar to those reported in studies of treatment of MD with CsA. Debio 025, although related to CsA, did not target calcineurin, a primary signaling molecule in the pathway leading to transplantation rejection.

"Long-term treatment with CsA is risky because it suppresses the immune system, making patients more susceptible to life-threatening lung infections,” explained senior author Dr. Paolo Bernardi, professor of general pathology at the University of Padova. "Our findings suggest that Debio 025 may provide a safer alternative. This drug has no effects on the immune system and therefore could be used for prolonged periods of time without increasing risk of infection. We should be able to treat children affected by these forms of muscular dystrophy and possibly slow down or even stop the progression of the disease.”

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University of Padova