The ALS Association commends the FDA for approving tofersen under the agency’s accelerated approval pathway for the treatment of people living with ALS connected to mutations in the SOD1 gene. This marks the first time the FDA has approved a treatment for ALS under accelerated approval and serves as a promising endorsement of the antisense technology that underlies tofersen. 

The U.S. Food and Drug Administration awarded $3.7 million to three ALS research projects as part of the implementation of the Accelerating Access to Critical Therapies for Amyotrophic Lateral Sclerosis Act (ACT for ALS). 

The ALS Association, the country’s largest nonprofit committed to making ALS livable and finding a cure, today celebrated the Food and Drug Administration’s (FDA) approval of AMX0035, a new treatment for people living with amyotrophic lateral sclerosis (ALS), a fatal neurodegenerative disease. The Association invested $2.2 million of funds raised through the 2014 ALS Ice Bucket Challenge into the development and trial of AMX0035, and led the years-long advocacy campaign that pushed the FDA to approve the treatment prior to completion of an ongoing phase 3 trial.

“We are grateful to Cytokinetics for their partnership and transparency, enabling the global research community to leverage these data in their ongoing analyses and research activities,” said Neil Thakur, Ph.D., Chief Mission Officer at The ALS Association.

The ALS Association announced today that it has awarded a $620,000 grant to the Packard Center for ALS Research at Johns Hopkins to support the Answer ALS Data Portal. Launched in January this year, the Data Portal provides open access to the world’s most comprehensive collection of ALS data.

Following Amylyx’s announcement that it intends to submit a New Drug Application (NDA) for AMX0035, The ALS Association today urges the Food and Drug Administration to approve the treatment for all people with ALS as soon as possible.

The ALS Association today announced that California’s legislature, in conjunction with the California Department of Public Health has approved $15 million over the next five years for the wraparound model of care and treatment of ALS in the state.

Following the lack of action by the FDA to expedite approval of AMX0035, an experimental treatment for ALS, The ALS Association today called on the agency to follow its own guidance and move with the urgency of its Canadian and European Union counterparts. Health Canada and the European Medicines Agency (EMA) are working with Amylyx, the company that makes AMX0035, to move the treatment toward approval.

The ALS Association, Muscular Dystrophy Association (MDA) and ALS Finding a Cure® (ALSFAC), provided an additional $1.1 million in supplementary funding to leading investigators at the Houston Methodist Neurological Institute and Massachusetts General Hospital for promising research into cell therapy that could slow the progression of amyotrophic lateral sclerosis (ALS).

Today, in response to the New England Journal of Medicine publication of encouraging clinical trial results showing that Amylyx Pharmaceuticals’ AMX0035 brought statistically significant benefit to people living with ALS, The ALS Association and I AM ALS immediately called on the drug company and the FDA to make the treatment widely available as soon as possible.

The ALS Association and I AM ALS announced today that the organizations have awarded a combined grant of $500,000 to BrainStorm Cell Therapeutics (NASDAQ: BCLI), a biotechnology company, to support an amyotrophic lateral sclerosis (ALS) biomarker research study. The grant will be used to draw insights from data and samples collected from patients enrolled in BrainStorm’s ongoing phase 3 clinical trial of its NurOwn® treatment, to further understanding of critical biomarkers associated with treatment response for people with ALS.

The ALS Association, in partnership with the American Brain Foundation and the American Academy of Neurology, has awarded the 2020 Sheila Essey Award for ALS research to Guy Rouleau, M.D., Ph.D., director of the Montreal Neurological Institute and Hospital and chair of the Department of Neurology and Neurosurgery at McGill University. The award recognizes significant research contributions in the search for the cause, prevention of and cure for amyotrophic lateral sclerosis (ALS). Since 1996, The ALS Association and the American Academy of Neurology have jointly chosen recipients of the award.

The ALS Association today announced a three-year, $652,543 commitment to support new collaborative initiatives that will encourage therapeutic pipeline development, improve ALS trial efficiency and quality, and enhance the patient experience with trial access, recruitment, and retention efforts. Activities will take place at the Sean M. Healey & AMG Center for ALS at Mass General in collaboration with the Barrow Neurological Institute and the Northeast ALS Consortium (NEALS).

The ALS Association today announced a $3 million commitment to the first platform trial for ALS, taking place at the Sean M. Healey & AMG Center for ALS at Mass General in collaboration with the Barrow Neurological Institute and the Northeast ALS Consortium (NEALS). A "platform trial" is a clinical trial in which multiple treatments are tested and evaluated simultaneously. The Association’s commitment is $1 million per year for 3 years.

The ALS Association is partnering with, and will be directly investing in, the ALS Investment Fund II to raise $100 million to accelerate investment in ALS research and development. This second venture fund with offices in Boston and Amsterdam, succeeds a fund that started in The Netherlands and raised $25 million in two-and-a-half years to invest in companies with ALS initiatives. 

An independent research organization reported that donations from the 2014 ALS Ice Bucket Challenge enabled The ALS Association to increase its annual funding for research around the world by 187 percent. During this time, ALS researchers made scientific advances, care for people living with ALS expanded and investment in disease research from the federal government grew.

The ALS Association, the only national nonprofit fighting amyotrophic lateral sclerosis (ALS) on every front, applauded the Food and Drug Administration’s (FDA) announcement today that it has approved Radicava (edaravone), the first new treatment approved specifically for ALS in 22 years. The FDA approved Radicava less than a year after Mitsubishi Tanabe Pharma Corporation submitted a New Drug Application. There are only two other approved treatments specifically for ALS, Riluzole and Tiglutik.