UMass Medical Researchers on Cusp of ALS Breakthrough
NEC member UMass Medical School recently announced the discovery of a possible new breakthrough in the treatment of amyotrophic lateral sclerosis (ALS). So far, the gene therapy technique has been proven effective in animal experiments, and will be tested soon in clinical trials on patients.
This treatment, which involves using synthetic microRNAs to counteract proteins linked to the disease, would only work on the roughly 10 percent of patients who have the familial version of ALS. 25 years ago, the research team of Dr. Robert H. Brown Jr. located the first gene associated with ALS, a protein called SOD1. The possible cure includes using specially engineered microRNAs to switch off the mutation of SOD1 that causes the disease.
Christian Mueller, a faculty member at UMass who worked on the study, said, “We’ve moved it pretty quickly. It’s pretty unusual to get a project from conception to patients in such a short period of time.”
The NEC congratulates UMass on discovering this possible new treatment and commends them on their commitment to curing ALS.