Only two drugs have been FDA approved to treat amyotrophic lateral sclerosis (ALS) since 1995—and neither one has proven particularly effective in combating this mysterious degenerative disease. Now, an encouraging new treatment may exist in a phase 3 clinical trial of NurOwn, the first-ever stem cell therapy for ALS developed by BrainStorm Cell Therapeutics. And Massachusetts General Hospital (MGH) is one of six national sites testing the therapy in patients.

The trial is one example of research underway at MGH designed to shed light on the most intractable conditions with a history of eluding science. Despite the 100 other potential treatments researchers have recently tried, ALS patients continue to succumb to the disease within three to five years. And its rarity—occurring in just one to two new cases per 100,000 people each year—means less access to research funding. But investigators at MGH and the five other sites testing NurOwn are encouraged by the early-stage success of the drug, which modifies stem cells to secrete growth factors believed to promote motor neuron survival.

Merit Cudkowicz, MD, MSc, who runs the ALS program at MGH, says that the treatment seemed to stabilize some patients in its phase 2 trial, reducing brain inflammation that is implicated in the disease’s progression. For Richard P. Vinci, a Lehigh University professor and Reading, Mass. native receiving the treatment through the trial at MGH, that is reason enough for hope. “I have close friends who asked me what my bucket list is,” he says. “It’s not that I want to bungee jump in the Amazon…I’d like to see my kids graduate from high school.”

Read more about NurOwn and the trial underway at MGH in The Boston Globe

Tags: research and discovery