‘Ice Bucket Challenge’ funds FDA-approved ALS treatment

Ice Bucket Challenge

The US Food and Drug Administration (FDA) has granted approval to AMX0035, a new treatment for people living with amyotrophic lateral sclerosis (ALS).

Development of the drug was funded by $2.2 million from the ALS Association, raised by the 2014 social media craze the ‘Ice Bucket Challenge’. Another 40 potential new treatments are being investigated. 

The approval is the result of a long advocacy campaign to convince the FDA to approve the treatment prior to completion of an ongoing Phase III trial.  

“We thank the millions of people who donated, participated, and enabled us to invest in promising therapies like AMX0035 that will immediately help people living with ALS,” said Calaneet Balas, President and CEO of the ALS Association. 

“This is a victory for the entire ALS community, which came together to advocate for early approval of AMX0035. We still have a lot of work to do to cure ALS, but this new treatment is a significant step in that fight.” 

A years-long advocacy campaign 

In June 2016, the Association provided Amylyx, the producer of AMX0035, with a $750,000 grant for a clinical trial pilot. Then in July 2016, the Association provided the Northeast ALS Consortium (NEALS) with a $1.46 million grant to help pay for the Phase II clinical trial of AMX0035. 

In September 2020, the ALS Association and I AM ALS submitted over 50,000 signatures to the FDA calling on the agency to approve AMX0035. Following this, the Association held multiple meetings with FDA officials, including a public We Can’t Wait Action Meeting in May 2021.  

The Association also provided scientific and regulatory guidance and in 2022, drove over 14,000 individual emails to the FDA urging approval. 

Success of Ice Bucket Challenge 

In the summer of 2014, over 17 million people posted Ice Bucket Challenge videos to Facebook, including hundreds of celebrities, athletes, and politicians. The campaign, which was popularised by Pat Quinn and Pete Frates, two young men who were living with ALS, raised over $115 million for the ALS Association. Frates passed away in 2019 and Quinn passed away in 2020.  

Balas added: “The approval of AMX0035 is further proof of how the Ice Bucket Challenge dramatically accelerated the fight against ALS. Since then, we’ve seen new genes discovered, new assistive technology developed to help people living with ALS, and far more people living with ALS have access to care services than ever before.” 

Since the Ice Bucket Challenge, the ALS Association has spent over $127 million on ALS research and is currently funding 130 research projects in 12 countries. The Association is currently funding 40 potential treatments in the drug development pipeline.

Suggested Reading

Join FREE today and become a member
of Drug Discovery World

Membership includes:

  • Full access to the website including free and gated premium content in news, articles, business, regulatory, cancer research, intelligence and more.
  • Unlimited App access: current and archived digital issues of DDW magazine with search functionality, special in App only content and links to the latest industry news and information.
  • Weekly e-newsletter, a round-up of the most interesting and pertinent industry news and developments.
  • Whitepapers, eBooks and information from trusted third parties.
Join For Free