NIH researchers look to retool experimental cancer therapy to treat muscular dystrophy

A team of researchers have identified a potential new use for a previously failed experimental cancer treatment, according to a study published in the journal Molecular Therapy

During the screening of more than 350,000 compounds for possible efficacy in the treatment of Duchenne muscular dystrophy, researchers with the National Institutes of Health's National Center for Advancing Translational Sciences and the University of Nevada, Reno School of Medicine identified SU9516, which had been previously used in the development of a leukemia treatment. In both laboratory and animal models, the compound generated significant improvements in muscle function.

The results of the study suggest the compound could one day be developed into a treatment for DMD and other muscle-wasting conditions.

"Our findings open the door to develop new drug treatments for DMD," said Juan Marugan, PhD, acting branch chief of NCATS' chemical genomics center.

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