FDA approves ALS drug developed with funds from ice bucket challenge

Every year, around 5,000 people in the U.S. are diagnosed with ALS, also known as Lou Gehrig’s disease. There is no cure, and patients often die within two years. A social media movement several years ago brought ALS to the forefront, raising millions of dollars for research, and one of the drugs developed as a result has just been approved for use by the FDA.

In 2014, the ice bucket challenge was all over social media. People challenged their friends to dump ice water over their heads and donate to the ALS Association. A portion of the $115 million raised went to research and contributed to developing a new ALS drug.

Dr. Sabrina Paganoni, Ph.D., of Massachusetts General Hospital and Spaulding Rehabilitation Hospital, was the lead investigator in the clinical trial that led to the approval of this new therapy.

“The new drug is called AMX0035, and it’s a combination of two different drugs,” Paganoni said.

Now marketed as Relyvrio, the drug comes in a powder form that patients can drink or take by feeding tube. It works by slowing the loss of spinal and brain nerve cells.

“Things that we do every day that we take for granted, like talking or swallowing or walking or climbing stairs, people who took the drug did better on those functions for longer,” Paganoni said.

Patients taking the drug also lived, on average, five months longer, giving them some valuable time.

“People want to see their kids graduate from school, or they want to see their first child get married,” Paganoni said. “Really, five or six months could be the difference between making it to that event or not.”

One drawback is the cost: The medication costs around $158,000 annually. The company that developed the drug says the cost to patients will be lower because they anticipate insurance will cover it. This is the third FDA-approved drug for ALS and its symptoms.