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Researchers from the University of Nevada, Reno Aim to Repurpose Experimental Cancer Therapy to Treat Muscular Distrophy

Researchers at the National Institutes of Health’s National Center for Advancing Translational Sciences (NCATS) and the University of Nevada, Reno School of Medicine (UNR Med) have demonstrated that a drug originally targeted unsuccessfully to treat cancer may have new life as a potential treatment for Duchenne muscular dystrophy (DMD).

The candidate drug, SU9516, represents a different kind of approach for treating DMD, a degenerative muscle disease that usually begins in childhood and has no known cure. It is caused by a faulty gene that leads to progressive muscle weakness, with death often occurring around age 25. Rather than trying to fix or replace the broken gene, SU9516 ramps up the muscle repair process, helping reinforce muscle structure.

“Integrin stabilizes muscle structure, and helps stimulate muscle repair and regeneration,” Burkin said. “If we can artificially increase its production with drugs, we think it can help protect muscle cells from damage.”

Dean Burkin, Professor of Pharmacology, UNR
20170613 Diaphragm Muscle

Diaphragm muscle from SU9516 treated dystrophin
deficient mouse showing nuclei (blue), myofibers (outlined in red) and

regenerating muscle fibers (green).

Burkin lab at the University of Nevada, Reno School of Medicine

NCATS Chemical Genomics Center Acting Branch Chief Juan Marugan, Ph.D., and UNR Med Professor of Pharmacology Dean Burkin, Ph.D., led a team that screened more than 350,000 compounds to find SU9516, which hadbeen previously developed as a treatment for leukemia. The research demonstrated that this compound improved muscle function in both laboratory and animal DMD models. The results, published recently in Molecular Therapy, may provide a promising approach against the disorder and other muscle-wasting conditions.

The NCATS-UNR Med team plans to work with medicinal chemists to make the molecule more specific for DMD, while also removing the toxic anticancer components, creating a safer version with a goal of future testing in patients. 

The work was supported by NCATS through its intramural research program, the National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS) grants R01AR053697, R01AR064338 and R41AR067014, the National Institute of Neurological Disorders and Stroke (NINDS) grant NS058429, Cure CMD and Struggle Against Muscular Dystrophy.

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