Doing Whatever it Takes to Find New Treatments & A Cure
We are currently providing funding for 155 active projects in 12 countries around the world, representing a current commitment of $52 million. This is in the context of a total commitment of over $116 million expended on research since 2014.
When that commitment to research leads to promising new treatments, we immediately begin the work of making those treatments available to the community. Throughout 2021, we continued to work tirelessly to bring AMX0035, a promising new drug shown to extend life and slow disease progression, to people living with ALS. And we’ve never been closer. Amylyx recently filed a New Drug Application with the FDA for approval of AMX0035.
From our We Can’t Wait Action Meeting that brought the voice of the ALS community directly to the FDA, to our recent call for the agency to conduct a priority review of Amylyx’s New Drug Application, we have led the way to bring this promising treatment to market as quickly as possible.
Listen to the team at Amylyx talk about their journey from the dorm room to the FDA – and the support The ALS Association provided along the way.
That’s just one illustration of the robust ALS drug development pipeline. Other promising developments in 2021 from research we supported includes:
- Identifying the first compound (NU-9) that eliminates the ongoing degeneration of diseased upper motor neurons, a key contributor to ALS.
- The announcement by Ionis Pharmaceuticals of FUSION, of a Phase 3 clinical trial of ION363 (also known as jacifusen), a novel antisense oligonucleotide (ASO) for ALS patients with a mutation in the fused in sarcoma (FUS) gene
Our advocacy for swift approval for AMX0035 is just part of our efforts to bring greater speed and effectiveness to the agency.
We continue to advocate for a series of policy actions to help the FDA accelerate development and delivery of effective therapies. We are also asking Congress for $50 million for the FDA to fund clinical research to benefit people with ALS and for Congress to fund a study by the National Academies of Science and Medicine to help coordinate the entire ALS research agenda. In addition, the House and Senate this year appropriated $1 million for a study to identify and recommend actions for the public, private, and nonprofit sectors to make ALS a livable disease and the House approved $5 million in new funding for the Orphan Products Grants Program (OPGP) at FDA at our request. This program can fund ALS clinical trials and invest in regulatory science to speed the approval of ALS treatments.
We have also led the fight for enactment of ACT for ALS, legislation that supports access to experimental treatments and provides the FDA additional research capacity and public private partnerships to speed development and approval of new treatments. The bill was recently signed into law.
To learn more about our efforts to move ACT for ALS through Congress, check out this discussion with the bill’s primary sponsor in the House, Rep. Mike Quigley of Illinois.
This year we launched a new grant funding program to support early stage clinical trials. In addition, our Milton Safenowitz Postdoctoral Fellowship Program welcomed four new fellows to the program that is the only program of its kind specifically funding early ALS postdoctoral fellows eagerly understanding the disease biology and searching for a cure. Our Lawrence & Isabel Barnett Drug Development Program awarded close to $3M to 6 projects to invest in bringing potential treatments into clinical trials.