After Phyl Gerber was diagnosed with amyotrophic lateral sclerosis in 2017, she went through stages of grief: anger, sadness, denial. Then her innate optimism kicked in.
“There was exciting research being done,” she recalls telling herself. “It all sounded so hopeful for the immediate future—I would be cured!”
ALS is a rare neurodegenerative disease that progressively robs patients of their ability to move, speak, eat, and breathe. Few people live longer than five years after diagnosis. When Gerber, 66, an athletic outdoorswoman all of her life—and a potter, gardener, wife, mother, and grandmother—learned of a six-month study for a new ALS drug, she “jumped on the opportunity to be a part of it.”
Gerber, of Amherst, MA, began taking the drug, AMX0035, in August 2017, and says that while her symptoms continued to progress, that progression slowed during the phase two, double-blind, placebo-controlled trial.
Thus when she learned, in December, that the study achieved its primary outcome—a statistically significant slowing of disease progression—she was “pleased but not surprised.” To the ALS world, though, the news was a revelation; the FDA has approved just two treatments for the disease, and there is no cure. Though the manufacturer, Cambridge, MA-based Amylyx Pharmaceuticals, wasn’t yet sharing data, any good news was cause to celebrate.
Perhaps even more surprising than an apparent success in this notoriously difficult field were the two people behind the new drug. Not yet 30 years old, they conceived of AMX0035 and founded Amylyx in 2013, when they were still undergraduates at Brown.