The Ice Bucket Challenge Helped Fund a New FDA-Approved ALS Drug

The drug underwent a clinical study that was partly funded by the $115M raised for the ALS Association by the Ice Bucket Challenge.

Credit: Getty Images
Credit: Getty Images

If you dumped a bucket of ice water on your head in 2014, take a bow: You just helped a new treatment for ALS achieve FDA approval.

On September 29, the Food and Drug Administration (FDA) approved Relyvrio, a drug designed to treat amyotrophic lateral sclerosis (also known as ALS or Lou Gehrig’s disease). The approval comes after Relyvrio underwent a small Phase 2 clinical study that was partly funded by proceeds from the Ice Bucket Challenge, which raised about $115M for the ALS Association.

“This approval provides another important treatment option for ALS, a life-threatening disease that currently has no cure,” Dr. Billy Dunn, an FDA official said in a statement.

The newly authorized drug is meant to slow the progression of the disease’s symptoms, which include a loss of neurological and motor functions. However, despite the FDA’s approval, there is skepticism among many scientific experts about the drug’s effectiveness.

The study found that participants who took the drug lived longer and saw their symptoms progress more slowly. However, both internal FDA scientists and an external panel of experts have questioned the data’s persuasiveness. Typically, the FDA approves drugs after two large studies, per the AP.

The FDA’s decision to ultimately go forward with the approval is likely a result of persistent advocacy and lobbying from ALS patients and allied orgs. Currently, there is no known cure for ALS and very few available ways to treat it, with most of them being prohibitively expensive.

“I think [the approval] demonstrates the FDA’s ability to be facile and I think it demonstrates a lot of tenacity on the part of ALS patients and advocates,” said Dr. Catherine Lomen-Hoerth, an ALS specialist at the University of California San Francisco, via the AP. “The company really tried to do everything possible to get this potentially promising drug out to patients.”