Three Brown Alumni Go Beyond Ice Buckets in Search for ALS Cure

It sounds like a movie: Three college buddies are hanging out in their dorm room, and they vow to cure a terminal disease. They pore over medical journals. They bat ideas back and forth. They have a “lightbulb moment.” And from these humble origins, they set up their own lab in a tireless quest to defeat amyotrophic lateral sclerosis, or ALS...

And yet, that’s more or less how Amylyx Pharmaceuticals, Inc. was started, right on Brown University’s campus.

“It is true that the science that drives Amylyx today was born in a dorm room,” says Josh Cohen, co-founder and co-owner of the Boston-based start-up. Cohen was housemates with Kent Leslie and in the same fraternity as Justin Klee, and all three were science majors. “During my junior year, I began looking into neurodegenerative diseases, which are notoriously difficult to treat. For weeks, I stayed in my dorm room and read countless papers on neurodegenerative diseases and found one common thread: neuronal death. We asked ourselves one key question: How and why do neurons die?”

The question wasn’t born in a vacuum. ALS, commonly known as Lou Gehrig’s disease, affects the brain and spinal cord, and symptoms include weakened muscles, impaired limbs, twitching, breathing difficulties, and slurred speech. About 5,000 people are diagnosed with ALS each year, and there is no known treatment that can reverse its progress. Advocates earned global attention in 2014 with the viral “Ice Bucket Challenge.”

“We [Klee and Cohen] both have relatives who lived with neurodegenerative diseases,” says Klee, “and over the last eight years, we have met incredible people living with ALS and Alzheimer’s disease. The people, their families and caregivers, and the advocates, make this endeavor personal. They inspire us every day to work harder and faster to bring potential new treatments forward.”

Amylyx is headquartered in Cambridge, MA, and the company is currently focused on AMX0035, an experimental drug. Developing pharmaceuticals is a slow and careful process, but the team recently published its findings in the New England Journal of Medicine. In summary: “AMX0035 is the first investigational therapy to demonstrate statistically significant benefit on this prespecified primary outcome in people with ALS since approved therapy Edaravone,” and it “showed numerical benefits on secondary outcomes including measures of muscle strength, breathing, and hospitalizations.”

“AMX0035 is not a cure for ALS,” clarifies Leslie, “but we are applying insights from AMX0035 and our platform to drive toward a cure in the future. We are and will continue to be relentless in helping patients and families affected by ALS, a disease left unanswered for too long.”

In the hypothetical movie, the three friends, wearing lab coats, celebrate their victory by shrieking “Eureka!” and hurling papers into the air. The reality is less dramatic, but possibly more fruitful. They have won accolades from Business Insider and an Xconomy Young Innovator Award, among other plaudits, and they are already expanding the breadth of their clinical trials.

“We are actively discussing next steps with global regulatory authorities like the FDA to AMX0035 to people living with ALS as fast as possible,” says Cohen. “We are also evaluating AMX0035 in Alzheimer’s disease, in a study called PEGASUS. These preliminary Alzheimer’s disease trial results are expected in the first half of the year. We are excited to be able to share the update with the community, once available.”