A new type of gene therapy doubled the life of mice with a laboratory form of Lou Gehrig’s disease and researchers said they are the planning to test the technique on human patients.
In a study published this week in the journal Science, researchers at the Salk Institute for Biological Studies in La Jolla, Calf. and Johns Hopkins University in Baltimore report that injecting mice with a gene that makes a nerve cell stimulating protein delayed symptoms of Lou Gehrig’s disease and extended the life span in laboratory mice.
The gene makes a molecule called insulin like growth factor, or IGF-1, a type of protein that has produced marginal results against Lou Gehrig’s disease in human clinical trials.
By delivering the IGF-1 gene directly into muscle tissue, the researches found that nerve cells were preserved and muscle wasting was reduced in mice with a laboratory form of the disease.
Lou Gehrig’s disease, or amyotrophic lateral sclerosis, causes the gradual death of nerve cells that control muscle movement, resulting eventually in paralysis and death. Its cause is unknown and there currently is no cure. ALS attracted worldwide attention when New York Yankee first baseman Lou Gehrig announced that he had the disease in 1939, two years before his death. ALS affects about 30,000 Americans.
More study is needed before the gene therapy can be used in humans, but researchers reported they were in the planning stages for human trials of the treatment.
The research was conducted by Fred H. Gage, Brian K. Kaspar and Nushin Sherkat of Salk, and Jeronia Llado and Jeffrey D. Rothstein of Hopkins.