From Viral Video to Magic Medicine

Before completing its ongoing phase III trial, Amylyx Pharma’s AMX0035 combination therapy for amyotrophic lateral sclerosis (ALS) has received approval from the FDA (1). The drug will be marketed as Relyvrio. Believe it or not, the roots of its success lie in the viral ALS Ice Bucket Challenge. 

Unless you are a very young reader (welcome!) or locked yourself away from all forms of media in 2014, you’ll remember the Ice Bucket Challenge. To take part, the challenger would receive a bucket of ice cold water over the head before challenging a friend or colleague, with participants and challengers donating money to charity and/or urging others to do the same or join the Challenge.

Just as the trend was achieving liftoff, ALS activists Pat Quinn and Pete Frates launched the more specific ALS Ice Bucket Challenge – a move that captured the moment, eventually raising over $220 million in donations for ALS research worldwide in 2014. The funding was certainly boosted thanks to celebrity participants that included Justin Timberlake, Homer Simpson, pre-presidential Donald Trump, and cult film director David Lynch (who, perhaps ahead of his time, nominated Vladimir Putin).

The lion’s share of money raised by the Challenge went to America’s ALS Association. In June 2016, the Association committed $750,000 of this money to fund a pilot clinical trial of AMX0035, a combination therapy for ALS. The Association then gave $1.46 million to the Northeast ALS Consortium to fund a phase II trial of AMX0035.

The Association kept on pushing in 2020, securing more than 50,000 signatures to call on the FDA to approve AMX0035. In 2021, they kept the pressure up, holding discussions with the FDA and a special “We Can’t Wait” meeting, where people with ALS spoke directly to the regulators.

In the phase II trial that preceded the approval, the drug was found to slow the loss of physical function in people with ALS and potentially extend their survival. These positive results – combined with the fact that Amylyx has chosen to keep the price below the most recently FDA-approved ALS treatment – appear to suggest that all those buckets of ice-cold water might just pay off for patients.