CAMBRIDGE, MASS. (WHDH) - A new treatment that researchers have labeled as a breakthrough may slow the progression of amyotrophic lateral sclerosis, or ALS, according to a recent study funded in part by the Ice Bucket Challenge.
The CENTAUR trial was conducted across 25 top medical centers in the United States through the Northeast ALS consortium and showed that treatment with AMX0035 was well tolerated and decreased the rate of decline among people with ALS, Cambridge-based Amylyx Pharmaceuticals, Inc. announced Wednesday.
A total of 89 patients with ALS received AMX0035, while 48 patients with ALS were given a placebo over 24 weeks as part of the randomized, double-blind trial.
Those who received AMX0035 scored higher on the Revised ALS Functional Rating Scale — a 48-point questionnaire measuring daily functions such as the ability to walk, dress independently, self-feed, speak and breathe.
“The data published (Wednesday) in the New England Journal of Medicine show that AMX0035 demonstrated a clinically meaningful benefit and a favorable safety profile for people living with ALS,” said Josh Cohen, Co-CEO, Chairman and Co-Founder at Amylyx. “This development is a breakthrough for the ALS community and we are working collaboratively and expeditiously with agencies worldwide to bring this potential new treatment option forward.”
Donors of the ALS Ice Bucket Challenge helped fund this trial.
“We are grateful to all the Ice Bucket Challenge donors whose contributions helped make this trial possible,” Calaneet Balas, President and CEO of The ALS Association, said. “The data published (Wednesday) makes a clear and compelling case that AMX0035 should be made available to people with ALS as soon as possible. We look forward to working with Amylyx, the FDA, and the entire community to help make that happen.”
Patients who completed the CENTAUR trial were given the option to enroll in an open-label extension study and receive AMX0035 long-term. A total of 92 percent of the participants elected to enroll in the study.
Results from this study and a long-term survival analysis on the AMX0035 and placebo groups are expected to be shared in the coming months.
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