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$2.5 million Defense Department grant funds gene therapy study for Lou Gehrig's disease
Date:4/1/2014

tory failure when the diaphragm muscles become incapacitated," said Svendsen, who has studied ALS for more than a decade and whose team will perform the leg muscle studies while Suzuki's group conducts the respiratory muscle experiments.

The Department of Defense grant will support all animal studies needed to take these experiments from the bench side to the bedside leading to the filing of an Investigational New Drug application with the Food and Drug Administration. The IND process is a regulatory step between preclinical study and human clinical trials. If approved, Cedars-Sinai's large ALS Program, led by Robert H. Baloh, MD, PhD, has a clinical trials group in place to move this research into the clinic.

"Muscle is much more readily accessible for treatment and biopsy than brain tissue or the spinal cord, which makes this strategy very appealing," Svendsen said. "Our previous findings provide strong evidence that the viral vector can transfer the GDNF protein into muscle, and we have shown that GDNF delivered to muscle may slow disease progression. We appreciate the Department of Defense's interest and funding, which will make it possible for us to further this exciting research."

Svendsen's research group in 2012 received a $17.8 million grant from the California Institute for Regenerative Medicine to develop stem cell treatments for patients with ALS based on injections of stem cells releasing GDNF into the spinal cord.


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Contact: Sandy Van
sandy@prpacific.com
808-526-1708
Cedars-Sinai Medical Center
Source:Eurekalert

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