A Drug Unsuccessful at Treating Cancer May Successfully Treat DMD

A drug previously used in an attempt to treat cancer, now has the potential to be repurposed to treat Duchenne muscular dystrophy (DMD), said 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).

DMD is a degenerative muscle disease, caused by a faulty gene, with no known cure. Those with the disease, typically males, lack the protein dystrophin, which is a shock absorber for muscle cells. Without dystrophin, the muscles have minimal strength and are more susceptible to injury.

The cancer drug, SU9516, was originally intended to treat leukemia. However, when targeting DMD, the drug assists in the muscle repair process, helping to reinforce the muscle structure, rather than attempting to replace the faulty gene.

Juan Marugan, PhD, chief of NCATS Chemical Genomics Center Acting Branch, and Dean Burkin, PhD, of UNR professor of pharmacology, performed research that demonstrated the improved muscle function in DMD animal and laboratory models with the use of SU9516. Through experiments by the NCAT and UNR teams, the researchers discovered that an increase in the levels of α7β1 integrin could minimize DMD symptoms in the muscles. SU9516 increases production of α7β1 integrin and resulted in increased muscle function while slowing down the disease progression, according to research.

“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.”

Burke, of UNR, concludes that SU9516 will be able to work alone, or in combinations with other drugs to treat DMD and other muscle-damaging conditions. The NCATS and UNR teams hope to continue their research of SU9516 by making it safer and more specific for targeting DMD.

“Our findings open the door to develop new drug treatments for DMD,” Marugan said in a statement.