Fighting to Get Effective Treatments to People with ALS

This year we continued to lead the global search for treatments and a cure as the largest philanthropic funder of ALS research around the world. Since January 1, 2020, The ALS Association has funded 46 research projects around the world. Here are a few examples of advancements in our research program.
Research funded by the Association led to the development of a promising new drug from Amylyx Pharmaceuticals. Clinical testing of AMX0035 showed it to be safe and effective at slowing the progression of ALS and helping people live longer. This research was one of the first projects to be funded with monies raised through the ALS Ice Bucket Challenge.
Due to the safety and effectiveness of AMX0035, The ALS Association, in collaboration with I AM ALS, collected more than 50,000 signatures on a petition we delivered to the Food and Drug Administration calling on the FDA and Amylyx to expeditiously work together to make sure AMX0035 is made available for people with ALS as quickly as possible.
Our $3 million commitment to help set up the first-ever ALS platform trial at the Sean M. Healey & AMG Center for ALS is accelerating the rate at which we can test and evaluate treatments. Even in the face of the pandemic, the Healey platform trial is already enrolling clinical trial participants. The project is on track to have 50 sites up and running by the end of December, with 100 people fully enrolled in the study by July.
Our partner Biogen published promising results from its phase 1–2 Trial of Antisense Oligonucleotide Tofersen for SOD1 ALS and began enrolling participants for their Phase 3 Valor study. Biogen also announced that there is an open-label extension available in the study, more promising news and hope for people living with ALS.