Researchers identify promising gene therapy for DMD

CureDuchenne, the Orange County non-profit focused on finding a cure for Duchenne muscular dystrophy (DMD) is one step closer to that goal through the work of Dr. Jeff Chamberlain.

“The focus of the trial will be on how the patient's immune system will respond to the gene delivery”

Dr. Chamberlain, of the University of Washington, in partnership with Dr. Stephen Tapscott, of the Fred Hutchinson Cancer Research Center, has identified a promising gene therapy that could lead to a treatment for DMD. The method delivers a new gene to the body's muscles to replace the defective, disease-causing gene.

As the leading genetic killer of young boys, DMD affects more than 1 in 3,500 boys worldwide and is the most common and lethal form of Muscular Dystrophy. Boys with DMD are usually diagnosed by the age of five, in a wheelchair by age 12, and may be completely paralyzed by their late teens. Historically, most patients with DMD do not live to see adulthood. Duchenne can occur in any family, from any race and from any background.

Debra and Paul Miller, co-founders of CureDuchenne will allocate the $250,000 raised during last weekend's 2010 Climb to CureDuchenne: Pick Your Peak, where teams across the country summitted mountains from coast to coast to raise awareness for DMD, along with the $165,000 raised by Mack and Sally Brown of Austin, TX during CureDuchenne's annual "Dealing for Duchenne" fundraiser, to fund Drs. Chamberlain and Tapscott's pre-clinical research.

"The focus of the trial will be on how the patient's immune system will respond to the gene delivery," said Dr. Chamberlain. Immune related issues have emerged as a major limitation on applying gene therapy, and the Seattle team has identified methods that appear to overcome the immune issues that have hampered many previous attempts at gene therapy. Another arm of this research will study the delivery of another gene, Utrophin, through AAV delivery. "I believe we're on the precipice of an amazing breakthrough with this research. We're seeing more and more that in order to get this kind of research to the human clinical trial stage, the funding has to come from non-profit organizations like CureDuchenne. Without their support of our work, we'd never have gotten this far," Chamberlain continued.

If successful, the studies will lead to clinical trials aimed at improving strength in patients with DMD. CureDuchenne is playing a key role in funding not only the pre-clinical research but in providing funds to obtain regulatory approval from the FDA, which regulates all gene therapy and drug trials.

"This is what we're all about," said CureDuchenne co-founder Debra Miller. "CureDuchenne is dedicated to focusing on research that is en route to the human clinical trial phase. It's this level of research that has true potential to save this generation of boys."

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