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Drug Appears Promising for Muscular Dystrophies

Last Updated: June 15, 2009.

The selective cyclophilin inhibitor D-MeAla3-EtVal4-cyclosporin -- related to cyclosporin A -- may offer a useful treatment for collagen VI muscular dystrophies, according to research published online June 10 in the British Journal of Pharmacology.

MONDAY, June 15 (HealthDay News) -- The selective cyclophilin inhibitor D-MeAla3-EtVal4-cyclosporin (Debio 025) -- related to cyclosporin A (CsA) -- may offer a useful treatment for collagen VI muscular dystrophies, according to research published online June 10 in the British Journal of Pharmacology.

Tania Tiepolo, of the University of Padova in Italy, and colleagues assessed the effects of Debio 025 in a mouse model of muscular dystrophy due to defects of collagen VI. Mice received the treatment or vehicle intraperitoneally for five days.

The researchers found that treated mice showed a decreased number of abnormal muscle fibers, and the treatment prevented mitochondrial dysfunction and normalized apoptotic rates and ultrastructural lesions of the mice. Debio 025 was as effective as CsA in the model, but it doesn't inhibit calcineurin. As a result, the researchers state that it wouldn't expose patients to immunosuppression.

"Taken together, our findings have major implications for Ullrich Congenital Muscular Dystrophy (UCMD) patients, whose treatment with CsA has proven beneficial. UCMD is a chronic muscle wasting disease involving the diaphragm, and respiratory failure is a common complication which is worsened by pulmonary infections. Long-term treatment with CsA must therefore be carefully weighed against the risks of immunosuppression, which may favor life-threatening infections," the authors conclude.

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