Scientists at the Salk Institute in La Jolla, California, report in the August 8 issue of Science that a novel gene therapy approach prolonged survival and slowed disease progression in mice with ALS (amyotrophic lateral sclerosis), also known as Lou Gehrig's disease. ALS is a fatal neuromuscular disease characterized by the degeneration of spinal and brainstem motor neurons, leading to atrophy of limb, head-and-neck, and respiratory muscles. Plans for the first ALS patient trial utilizing this approach are underway.

Fred H. Gage, Brian Kaspar, and colleagues, injected an adeno-associated viral vector (AAV) to express a protein that promotes the survival of neurons into the limb muscles of mice. When delivered to the cell bodies of the neurons, this gene, called insulin-like growth factor-1 (IGF1), delayed disease progression and prolonged survival. The authors report that their results demonstrate substantial behavioral, functional, and pathological improvements in a clinically relevant mouse model of a motor neuron disease.

Says Fred Gage, "Adeno-associated viruses are retrogradely transported from the synapses in the muscle to the nucleus of the motor neuron inside the spinal cord, emphasizing the importance of basic science in developing rational therapies for disease."

Dr. Gage approached Project A.L.S. with the idea of trying this gene therapy approach in ALS a year-and-half ago. When data from the initial ALS studies proved promising, Project A.L.S. put Gage and Brian Kaspar together with Jeffrey D. Rothstein, an ALS researcher and clinician at Johns Hopkins University. The teaming of Gage with Rothstein is a prime example of the Project A.L.S. strategy: uniting the best basic scientists with those who can help translate that science into clinical application.

Adds Gage, "Project ALS has been impressive in recognizing and supporting fundamental science in what is now called translational science."

"I am proud
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Contact: Pat Harrington
HSPRNYC@aol.com
212-581-0407
Harrington Communications
7-Aug-2003

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