Massachusetts Scientists Use MicroRNA To Treat ALS For First Time

BOSTON (CBS) – Scientists in Massachusetts have made a potential breakthrough in ALS research.

In 1993, Dr. Robert Brown and his team at UMass discovered the first gene linked to familial ALS. His team, working with Massachusetts General Hospital, has now safely treated two people using synthetic microRNA for the first time. The microRNA suppressed and silenced the gene that caused ALS.

Brown said microRNA is an exciting and powerful method to target ALS cases caused by gene mutations. "This is very hopeful for a couple of reasons," he said. "First of all, we're essentially at the dawn of a new era using biologics not just small molecules like aspirin or antibiotics, but things like proteins, antibodies and DNA to manipulate genes in the nervous system. I think this has bearing on many, many disorders and is quite hopeful, not just for familial diseases but also some of the nonfamilial forms of these diseases.

The next step is to test this discovery in a placebo-controlled trial.