Patients who took [an experimental medication for ALS] — initially dreamed up over beers and obsessive internet searching in a Brown University dormitory — retained a higher level of certain motor functions than those given a placebo, according to the researchers’ study, published [September 2] in the New England Journal of Medicine. The company developing the drug, Cambridge, Mass.-based biotech Amylyx, released outlines of the data in December, but the new paper details how effective the treatment was in slowing progression of the disease.
While researchers involved in the study said it marked a watershed moment in the fight against ALS, an accompanying editorial in NEJM called the data from the Phase 2/3 study only “tantalizing.” It said the benefit appeared to be modest — and stressed a Phase 3 trial would be important to validate the conclusions.
Any additional treatments would be welcome by the ALS community. There is no cure for the disease, and only a couple of available treatments.
“When they first wrote to me in June 2013, they were just kids,” [Alzheimer’s researcher Rudy] Tanzi recalled. “I thought it was a pretty naive idea, to be honest, but thought I’d go through this exercise with them — throw them some hard-to-do science. And now here we are, today, with a paper coming out.”
