Dr. Merit Cudkowicz, director of the Sean M. Healey & AMG Center for ALS and chief of neurology at Massachusetts General Hospital, will never forget the very first person with ALS that she cared for.

“The first person that I ever took care of was a teacher in Connecticut, and she had [a] genetic form of [ALS], and an 8-year-old son,” Dr. Cudkowicz recalled. “I would go visit her in her home when she couldn't come in anymore. She taught me so much about being a doctor and about being a mom with a serious illness.”

At that time, back in the 1990s, the ALS research community was very small, and scientists were just starting to discover the first clues about how and why the disease develops.

“The first gene had been discovered for ALS, and everybody was talking about how this was going to change the world for ALS—that finally we had a clue in it,” Dr. Cudkowicz said, referring to the discovery of the SOD1 gene in 1993, the gene linked to her first patient’s disease. But the number of clinical trials testing treatments for ALS was minimal.

Today, the landscape of ALS research has changed significantly, with more than 40 genes linked to the disease and more than 70 interventional clinical trials currently recruiting people living with ALS—many with ties to Dr. Cudkowicz.

“The only way we're going to get drugs on the market for everybody is testing them … in well-done trials,” said Dr. Cudkowicz, who co-founded the Northeast ALS Consortium (NEALS), a group of more than 130 clinical trial sites in the United States, Canada, Europe, and the Middle East. Dr. Cudkowicz also launched the first platform trial in ALS in 2020, known as the HEALEY ALS Platform Trial, which tests multiple experimental treatments simultaneously.

To make ALS livable, we not only need to see more clinical trials but also make it easier for people with ALS to take part in research.

“It's real altruism for somebody to be in a trial, and we should do everything we can to make it as easy for them [to participate] as possible. And there are ways to do that,” Dr. Cudkowicz said.  “There's something called decentralized trials where you really bring the trial to the patients. It's a new idea in therapeutics in general, but we need to bring something like that to ALS.”

Initiatives supported by our Trial Capacity Awards also focus on eliminating barriers to clinical trial participation, helping translate promising laboratory science into new and better ways to treat and care for people living with ALS faster.

 For Dr. Cudkowicz, her sense of hope comes from the increased engagement in ALS research she’s witnessed over her career.

“What gives me hope for the future is the sheer number of people involved in the ALS fight and the different types, from the scientists to the physicians to the companies and the patients and the foundations. It feels like a big global family that's trying to fight together,” she said. “That's very different than 10 or 15 years ago. It was such a small community. So, the speed of progress is just going to go up because of the number of people involved.”

And just last year, Dr. Cudkowicz got to share news of that progress with a Connecticut family she made a promise to three decades before—a new treatment had just been approved by the FDA for ALS connected to mutations in the SOD1 gene.

“Last year, the world, the community of ALS found a treatment for [my first patient’s] form of ALS. So actually, the first person I called when I heard the results was her husband. … I've never forgotten her.”

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