FDA Approves ALS Drug That Internet Sensation ‘Ice Bucket Challenge’ Helped Fund

Ice, Ice Baby. It’s no secret that the Ice Bucket Challenge, born in the era of the Harlem Shake and Gangnam Style, froze the internet.

The wildly successful awareness campaign started by two patients saw participants from Bill Gates and Oprah Winfrey to Mark Zuckerberg, Taylor Swift, Lady Gaga, David Beckham, and Justin Timberlake, who all paid it forward by either having a bucket of iced water dumped over their heads and nominating a friend, or by making a donation of US$100 to the ALS Association.

ALS, short for amyotrophic lateral sclerosis and also known as Lou Gehrig’s disease, is a neurodegenerative condition that gradually damages nerve cells in charge of voluntary movements, leaving the patient unable to move, breathe, speak, or eat at the end of their life. The disease is currently incurable. Most people with the condition—about 6,000 in the US—succumb to it within two to five years of a diagnosis.

Pat Quinn and Pete Frates started the Ice Bucket Challenge in 2014 to educate the public about their illness. Evidently, it worked: The ALS Association has now reported that US$115 million was raised from over 17 million videos of the viral social media trend. US$2.2 million of those donations funded the development of Relyvrio, an ALS drug that was just approved by the US Food and Drug Administration (FDA) on September 29.

“We are thinking of Pat Quinn and Pete Frates, who championed the Ice Bucket Challenge and changed the trajectory of ALS forever,” says Calaneet Balas, president and CEO of the ALS Association, in a press statement. Frates and Quinn passed away in 2019 and 2020 respectively.

While Relyvrio doesn’t cure ALS, it slows down the rate of the neurodegenerative disease taking over the body. A study of 137 patients suggests that the drug can extend a patient’s life by five or six months.

Six months added to a person’s life could mean that they’d be able to be part of another milestone, like their child’s graduation or a wedding, notes Balas, as cited by NPR.

Balas says the FDA green-lighting the treatment 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,” Balas adds.

However, the true effectiveness of Relyvrio—developed by Amylyx Pharmaceuticals and a combination of two drugs—has been contested by medical experts, including the FDA’s own scientists.

Critics point out the trial’s small sample, which is less significant than the few hundreds normally expected of FDA studies. The Peripheral and Central Nervous System Drugs Advisory also concluded in March that the Relyvrio research did not offer “substantial evidence” of the drug’s efficacy, NPR reports.

Dr David Rind, chief medical officer of the Institute for Clinical and Economic Review, argues that Relyvrio’s creators should be able to prove that the drug works with a larger trial if it can indeed prolong lives by five to six months.

Further, being treated with Relyvrio isn’t cheap. It’s expected that the drug will cost about US$158,000 a year. Seeing as how its success rate isn’t a surety, Dr Rind is asking that the price of the drug be lowered for the time being.

A larger trial for Relyvrio is in the works in Phoenix, and its findings will only be concluded over a year later. Amylyx’s founders reassure skeptics that if the results aren’t as positive as the first study, they will make the responsible decision of removing the treatment from the market.

[via Mashable and NPR, cover photo 114610756 © Salajean | Dreamstime.com]