Existing Drug Could Help Fight Duchenne Muscular Dystrophy

Scientists studying drug therapy for Duchenne muscular dystrophy (DMD) have found a drug that could restore the missing protein needed for proper muscle function.

Dantrolene, which is already approved by the U.S. Food & Drug Administration and being used in humans, boosts therapy currently being tested in clinical trials. The UCLA researchers hope the combination of these drugs will overcome the genetic mutations that cause DMD and help those affected lead a normal life.

“Dantrolene is such an attractive candidate to test in this disease as it’s already approved, has been used safely in humans for decades and we won’t have to go through the lengthy and costly drug development process,” said study senior author Dr. Stan Nelson, a professor of human genetics. “We were very pleased to find out that this drug seems to work synergistically with the drugs being tested now on boys with DMD.”

The study appeared in the December 12 journal Science Translational Medicine.

The research by Nelson and co-senior author Carrie Miceli, a professor of microbiology, immunology and molecular genetics, is driven by the diagnosis of their youngest son with DMD.

“We entered into this field because of the diagnosis of our son, but we hope our research can help many others,” Miceli said. “There are drugs that can help manage the symptoms of the disease, but nothing that changes its course dramatically. We’re trying to correct the defect that causes DMD with highly personalized genetic medicine.”

Going forward, Nelson and Miceli will further their research with the goal of translating their findings from the bench to the bedside. The pair has received a $6 million grant from the California Institute of Regenerative Medicine for further studies.