Cedars-Sinai receives $750,000 grant from Parent Project Muscular Dystrophy for sildenafil/taladafil study

Grant Funds Work of Ronald G. Victor, M.D. and Cedars-Sinai Medical Center

Parent Project Muscular Dystrophy (PPMD) – the largest, most comprehensive  non-profit organization in the United States focused on finding a cure for Duchenne muscular dystrophy (Duchenne) – announced today that they will award Ronald G. Victor, M.D. of Cedars-Sinai Medical Center, a $750,000 grant to fund his study of phosphodiesterase inhibitors (sildenafil and taladafil) as a possible therapy for Duchenne.

Dr. Victor and his team at the Cedars-Sinai Heart Institute in Los Angeles discovered a defect in muscle blood flow in mdx mice and boys with Duchenne. Correcting this defect with phosphodiesterase inhibitors (sildenafil and taladafil) improves muscle and heart function in mdx mice. Dr. Victor will conduct a clinical research study to determine if these drugs can improve muscle blood flow in boys with Duchenne, with plans to continue on to a larger multi-center clinical outcomes trial examining both heart and muscle function.

PPMD President and CEO Pat Furlong said that the organization is pleased to support the work of Dr. Victor and Cedars-Sinai. "Parent Project Muscular Dystrophy launched a cardiac initiative at the beginning of this year so that potential treatments in Duchenne took into consideration the effect therapies may have on the heart. Dr. Victor and the wonderful people at Cedars-Sinai have not only taken into consideration cardiac issues in Duchenne, but are also combining existing FDA approved drugs for use as a potential therapy. We are excited by the potential benefit of Dr. Victor's work with sildenafil and taladafil, and continue to be grateful that leading institutions around the country, like Cedars-Sinai Medical Center, are working with this community to end Duchenne."

Dr. Victor, who serves as Associate Director of Clinical Research, Director of the Hypertension Center, and Burns and Allen Professor of Medicine at the Cedars-Sinai Heart Institute, said he was excited that his research team had been awarded the $750,000 grant from Parent Project Muscular Dystrophy.

"To work on a project you feel so passionately about for a community you feel so passionately about, is a reward in and of itself," Dr. Victor said.  "But to have an organization as well respected and trusted at an international level, like Parent Project Muscular Dystrophy, to support your work with such a substantial grant is truly overwhelming. I am so grateful to Pat Furlong for the contribution she continues to make to Duchenne research by supporting projects like mine. She inspires the people in our lab to work even harder to find a therapy that will stop the progression of this devastating disorder."  

Ms. Furlong explained that PPMD has a long history of supporting promising therapies and taking risk on research strategies that otherwise might not get the financial backing they need. "The parents that make up the Duchenne community all share one single goal, to end Duchenne. We take our responsibility to our families very seriously: to thoroughly vet and financially support any research project, any therapy that brings us one step closer to this goal. When we announce something like the cardiac initiative that we launched earlier this year, we put our money where our mouth is. This grant to Dr. Victor combined with our recent $250,000 grant to PTC Therapeutics, gives us a total research investment of $1 million in the first quarter of 2011. We will continue to fund hope for young men living with Duchenne, whenever the opportunity presents itself."

SOURCE Parent Project Muscular Dystrophy