LOS ANGELES May 30, 2013 Newport Beach-based nonprofit Coalition Duchenne has awarded a $150,000 grant to a Cedars-Sinai Heart Institute team investigating whether an experimental cardiac stem cell treatment could be used to treat Duchenne muscular dystrophy patients who have developed heart disease.
Coalition Duchenne is led by Catherine Jayasuriya, a mother whose 20-year-old son, Dusty Brandom, has cardiomyopathy associated with Duchenne muscular dystrophy. She was inspired to underwrite cardiac stem cell research at Cedars-Sinai after reading about a successful clinical trial led by Eduardo Marbn, MD, PhD, director of the Cedars-Sinai Heart Institute and the Mark S. Siegel Family Professor.
The experimental stem cell therapy, developed by Marbn, is the only treatment shown in clinical trials to regenerate healthy heart muscle. In the clinical trial, patients underwent biopsies during which doctors removed a piece of heart muscle about the size of half a raisin. The heart tissue was then used to grow specialized heart stem cells, which then were injected back into the patient's heart. Results published in The Lancet showed that patients experienced an average 50 percent reduction in muscle damaged by heart attack.
"I immediately sensed the potential for applying this rapidly evolving treatment to Duchenne," said Jayasuriya. "I made it my personal quest to help get this kind of therapy for Duchenne patients."
Jayasuriya's commitment was further cemented when she discovered that Ron Victor, MD, associate director of the Cedars-Sinai Heart Institute, has been working with Duchenne patients as part of his investigation of the cardiac benefits of sildenafil (Viagra) and tadalafil (Cialis).
"We know that boys with Duchenne are born with a small scar in the base of their heart," said Victor, the Burns and Allen Chair in Cardiology Research at the Cedars-Sinai Heart Institute. "The damage to hearts in boys w
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Cedars-Sinai Medical Center