A year after the enormously popular “Ice Bucket Challenge” campaign had households across the country talking about ALS, scientists at UMass Medical School working toward a cure have made meaningful progress in their labs.
“There has been enormous progress and there are grounds, very legitimately, for hope and encouragement,” said Robert H. Brown Jr., DPhil, MD, the Leo P. and Theresa M. LaChance Chair in Medical Research, and chair and professor of neurology.
Amyotrophic lateral sclerosis (ALS) is a progressive, neurodegenerative disorder affecting the motor neurons in the central nervous system. The cause of most cases of ALS is not known. Approximately 10 percent of cases are inherited. Though investigators at UMMS and elsewhere have identified several genes shown to cause inherited or familial ALS, almost 50 percent of these cases have an unknown genetic cause. There are no significant treatments for the disease.
Dr. Brown’s lab is working to develop a therapy to silence genes that promote the ALS.
“In terms of therapy development, there has been real progress. Combining forces with the incredible staff in the gene therapy unit [at UMMS], it’s been possible to show that one can put gene silencing elements into the nervous system using viral delivery, and in so doing, turn off some of the toxic genes that promote the disease.”
So far, using animal models, this approach has led to increased survival. “The question is, can we take this most rapidly and far enough along to get it into humans and the answer is, ‘Yes, I think we can,’” said Brown.
Learn more about this and other promising developments in the fight against ALS in this video.
