Salk news: gene therapy for Lou Gehrig's disease

La Jolla, Calif. -- A unique gene therapy method postpones the symptoms and nearly doubles the life span in a mouse animal model of Lou Gehrig's disease, a research team led by the Salk Institute has found.

The findings are the first to show this degree of recovery after the paralyzing and ultimately fatal nervous system disorder begins and may lead eventually to a new, gene-based treatment for the disease that affects more than 30,000 Americans. The study appears in the August 8 issue of the journal Science.

Fred H. Gage, professor of genetics, Salk research fellow Brian Kaspar, Jeffrey Rothstein, professor of neurology at Johns Hopkins University, and their colleagues found that injecting a gene that produces the nerve cell growth-stimulating protein, insulin like growth factor-1 (IGF-1), into muscles resulted in longer life spans, preserved nerve cells and reduced muscle wasting.

Lou Gehrig's disease, known as amyotrophic lateral sclerosis (ALS), is marked by the degradation of nerve cells that control muscle movement. It quickly attacks these motor nerve cells in the brain and spinal cord, resulting eventually in total paralysis and death. Its cause is unknown. While the disease was first identified in the 19th century, it gained international attention in 1939 when baseball great Lou Gehrig announced he had ALS and retired from the New York Yankees. He died two years later.

"IGF-1 protein has been used in clinical trials for a while, with marginal results," said Gage. "The biggest challenge has been to deliver the protein across the blood-brain barrier into the central nervous system. By injecting our viral vector into muscles, the gene could then deliver the protein into nerve cells that controlled the muscle, resulting in the preservation of those nerve cells that would otherwise have succumbed more quickly to ALS."

"ALS is a terrible disease, and patients have few choices for therapy today," said Rothstein, also profes

Contact: Andrew Porterfield
858-453-4100 x1340
Salk Institute

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