New Gene Therapy for Lou Gehrig's Disease

Using a virus as a drug delivery system, scientists delayed physical deterioration and prolonged the lives of mice with amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig's disease. This fatal, neurological disease is characterized by the degeneration of spinal and brainstem motor neurons, leading to atrophy of limb, head-and-neck, and respiratory muscles. Currently, there is no effective therapy.

Brian Kaspar and colleagues describe their findings in the 8 August 2003 issue of the journal Science. They injected an adeno-associated viral vector, carrying proteins that promote the survival of neurons into the limb muscles of mice. When delivered to the cell bodies of the neurons, these proteins, called "insulin growth factor-1," delayed disease progression and prolonged survival of the mice.

The authors report that their results demonstrate substantial behavioral, functional, and pathological improvements in a clinically relevant mouse model of a motor neuron disease. A clinical trial testing this approach is being designed.

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More information is available from the journal Science: www.sciencemag.org/feature/data/als/.

—Daniel Kane

7 August 2003