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The Golden West Chapter of The ALS Association helped fuel a significant number of research innovations, advancements in drug development and clinical trials, and new collaborations designed to accelerate the pace of discovery. We are excited to share some important milestones and promising progress in the search for effective treatments and cures for ALS, made possible by your support.
The ALS Association is providing funding for 155 active projects in 12 countries around the world, representing a current commitment of $52 million. The ALS Association has invested $116 million in ALS research since the 2014 ALS Ice Bucket Challenge.
Researchers funded by The ALS Association identified the first compound (NU-9) that eliminates the ongoing degeneration of diseased upper motor neurons, a key contributor to ALS.
The Golden West Chapter launched our first Industry Partner Consortium, with long time supporters CNS and Cytokinetics. These industry partners are deeply committed to supporting the ALS community, scientific discovery, collaboration, and investment in therapies for neurodegenerative diseases including ALS.
Ionis Pharmaceuticals announced a pivotal patient trial of antisense therapy (ASO) following aggressive joint research efforts with The ALS Association and Project ALS. Their Phase 3 clinical trial of ION363 (also known as jacifusen), targets patients with a mutation in the fused in sarcoma (FUS) gene, a rare and aggressive form of ALS.
California Institute for Regenerative Medicine (CIRM) awarded $11.99 million to fund clinical research on a combined cell and gene therapy approach as a treatment for ALS. The trial builds upon the first ALS trial supported by CIRM, conducted by Clive Svendsen, PhD, Executive Director of the Cedars-Sinai Regenerative Medicine Institute and the Chair of the California ALS Research Network.
Our Chapter’s 2021 Commitment to Cures honoree, Amylyx Pharmaceuticals, Inc, announced that the AMX0035 clinical trial, funded by The ALS Association, demonstrated statistically significant treatment benefits for people with ALS in clinical trials. Amylyx recently filed a New Drug Application with the FDA for approval of AMX0035.
The HEALEY ALS Platform Trial completed enrollment for three investigational treatments. The ALS Association helped with the launch of this first-ever major initiative, by investing in this ground-breaking trial approach that will test multiple therapies for efficacy at the same time, providing time and cost savings and increasing patient access.
Biogen, our Chapter’s 2020 Commitment to Cures honoree, shared topline results from their Phase 3 study (ATLAS) and open-label extension for Tofersen, focused on SOD1-gene mutation.
Members of the Golden West Chapter leadership team have been appointed to several volunteer roles within CIRM. These include serving as a part of a clinical trial advisory panel and on the Independent Citizens’ Oversight Committee that provides recommendations regarding CIRM funding, ethical standards, and research facilities.
The Golden West Chapter hosted the 11th annual California ALS Research Summit, bringing together world renowned researchers, investigators, clinicians, biotech companies, government representatives, and advocates in ALS and related fields. The outcomes of their efforts are presented to the public at our online educational “Ask Me” event.