Do you remember the Ice Bucket Challenge? He ended up funding a new ALS drug

Picture by John Blanding/The Boston Globe via Getty Images

It’s 2014. Barack Obama is still president, nobody knows what an NFT is, and suddenly your social feeds are filled with videos of people having their friends and family dunk buckets of ice on them. and urging others to do the same, the suitor. has something to do with charity.

Turns out those ‘Ice Bucket Challenge’ videos – in which people said they’d donate money to a medical nonprofit and nominate others to do the challenge next – raised enough funds to help create a new treatment for amyotrophic lateral sclerosis. , better known as ALS or Lou Gehrig’s disease, according to a leading non-profit organization.

The ALS Association noted in its statement that since the viral sensation, which saw surrealist director David Lynch appointing none other than Russian President Vladimir Putin – remember, this was in 2014 – raised a whopping $2.2 million.

The ALS Association invested the money in the research and development of a drug known as AMX0035, trade name Relyvrio, which the Food and Drug Administration (FDA) just approved for consumer treatment before the completion of its phase 3 trials.

ALS is primarily a disease of motor function, neurons in the spine and brain slowly breaking down as a patient gradually loses motor function and eventually dies. Relivrio, according to Amylyx Pharmaceuticals, the company that makes the drug, “significantly” slows the loss of physical function in ALS patients. While not a cure, its best-case scenario is far better than the almost zero treatment options accessible to people living with the disease.

“This is a victory for the entire ALS community, which has come together to advocate for the speedy approval of AMX0035,” said ALS Association President and CEO, Calaneet Balas, in the press release. “We still have a lot of work to do to cure ALS, but this new treatment is an important step in that fight.”

While this is undeniably huge news for people with ALS, there are reviews who believe the eight years between fundraising sensation and drug approval are rushed.

As NPR Noted Following the FDA vote of Relyvrio last week, the drug was approved after a single study with fewer than 200 patients was conducted by the company Amylyx Pharmaceuticals. This goes against the typical FDA drug approval process, which requires either two smaller independent studies showing the same results, or a larger study with many more patients than the 167 in the AMX0035 research.

“I totally understand why people are trying to find a way to convey this to patients,” said Dr. David Rind, chief medical officer of the nonprofit Institute for Clinical and Economic Review. NPR. “There’s just a general concern out there that maybe the lawsuit is bogus.”

Moreover, its manufacturer admitted after its approval that it costs $158,000 per yearand that it should only extend the lives of ALS patients by about six months.

After posting this story, an Amylyx spokesperson reached out to say that despite the listed price, the company is working with insurers to provide the cheapest drug possible, and justified the cost by saying it fund the development of future treatments. .

“We believe this award balances the needs and contributions of the ALS community, will be supported by insurers, allows Amylyx to maintain programs to ensure anyone who can benefit from RELYVRIO can access it, and allows us to continue our mission – to do it all. we can help people with ALS today and continue to invest in new treatments so that ALS first becomes a manageable chronic disease and ultimately is cured,” said the doorstep. -speak in a press release.

The FDA said in its vote to approve Relyvrio that it was important to get it out quickly, rather than wait for another study’s scheduled end date in 2024.

“Given the serious and life-threatening nature of ALS and the significant unmet need,” the agency wrote in a benefit-risk assessment“This level of uncertainty is acceptable in this case.”

In other words, a lot of unprecedented things are happening with AMXo035’s endorsement, and only time will tell if it deserves its hype.

If nothing else, it offers patients suffering from one of the most grueling degenerative diseases a ray of hope – and the comfort of knowing that millions of people care enough to donate money and throw ice cream on their heads for the benefit of their cause.

Updated to add a statement from Amylyx.

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