Grant funds cardiac stem cell research for Duchenne muscular dystrophy-related heart disease

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 Marbán, MD, PhD, director of the Cedars-Sinai Heart Institute and the Mark S. Siegel Family Professor.

The experimental stem cell therapy, developed by Marbán, 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 with Duchenne increases over time. If we can use stem cells to slow or stop heart damage, it could help stall progression of the disease."

The first step in the study is to examine the effect of injecting cardiac stem cells into the hearts of mice with Duchenne. If the data is positive, the experimental treatment could be rapidly approved for use in humans with Duchenne because of cardiac stem cell treatments have been approved for other patient populations, including those with advanced heart disease.

"Each year, 20,000 boys are born with Duchenne," Jayasuriya said, who founded Coalition Duchenne in 2010 to raise global awareness for Duchenne muscular dystrophy, fund research and find a cure for Duchenne. "Many do not live into their 20s and we lose many to cardiac issues. We need to focus on changing the course of the disease. We hope that working with cardiac stem cells is one way we will eventually change that outcome."

Duchenne muscular dystrophy is a progressive muscle-wasting disease and the most common fatal disease that affects children. Duchenne occurs in one in 3,500 male births, across all races, cultures and countries. Duchenne is caused by a defect in the gene that produces the protein dystrophin, which helps connect the muscle fiber to the cell membranes. Without dystrophin, muscle cells become unstable, are weakened and lose their functionality. Life expectancy of boys and young men with Duchenne ranges from the mid-teens to the mid-20s. Their minds are unaffected.