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Genetic Answer to Lou Gehrig's Disease

Gene therapy shows promise in treating degenerative condition

(HealthDay is the new name for HealthScoutNews.)

THURSDAY, Aug. 7, 2003 (HealthDayNews) -- A method of gene therapy postpones the symptoms of Lou Gehrig's disease and nearly doubles the lifespan of mice with the condition.

So finds a study in the Aug. 8 issue of Science.

This is the first study to show this amount of recovery after the paralyzing and ultimately fatal nervous system disease begins. It may help scientists develop a new gene-based treatment for Lou Gehrig's disease, which affects more than 30,000 Americans.

In this study, led by the Salk Institute in La Jolla, Calif., researchers injected a gene that produces the nerve cell growth-stimulating protein called insulin like growth factor-1 (IGF-1) into the muscles of mice with the disease.

The study found the injected IGF-1 preserved nerve cells, reduced muscle wasting and increased the lifespans of the treated mice.

The researchers demonstrated that IGF-1 triggers a molecular pathway that seems to preserve motor nerve function in the mice. When the receptor for IGF-1 is activated, an enzyme called Akt has a phosphate molecule added to it.

This results in activation of the Akt enzyme, which helps block programmed cell death, called apoptosis.

Lou Gehrig's disease, also known as amyotrophic lateral sclerosis (ALS), is marked by the degradation of nerve cells that control muscle movement. It attacks these motor nerve cells in the brain and spinal cord, leading to total paralysis and death. The cause is unknown.

More information

Here's where you can learn more about ALS.

SOURCE: Salk Institute, news release, Aug. 7, 2003
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