#‘Ice Bucket Challenge’ pays off in promising new ALS treatment

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Peter Frates, the former Boston College baseball captain who died of ALS in 2019, pictured participating in the Ice Bucket Challenge with his family in 2018.
Boston Globe via Getty Images

Internet cynics are eating their words over news that a viral stunt actually raised more than $200 million for medical research — and prompted a promising new treatment for amyotrophic lateral sclerosis, or ALS.

The 2018 Ice Bucket Challenge — which actually launched in 2015 but took years to go viral — featured citizens and celebs alike enduring showers of ice on video in exchange for donations. Using the money raised, scientists recently developed a potentially breakthrough medication — sodium phenylbutyrate–taurursodiol — for ALS.

While the experimental therapy is not a cure, it would help slow the progression of ALS — a disease marked by the rapid deterioration of nerve cells, according to a new study published in the New England Journal of Medicine. Those who live with the disease suffer a gradual loss of the ability to control their muscles, impacting speech, diet and even breathing.

“Patients keep telling me their No. 1 goal is to be able to retain physical function for as long as possible,” said Dr. Sabrina Paganoni, the study’s lead author and a neuromuscular specialist at Massachusetts General Hospital’s Sean M. Healey & AMG Center for ALS. She told NBC News, “They want to be able to continue to walk and to use their hands.”

The rare disease, which effects some 20,000 Americans, according to the ALS Association, usually appears during middle age, between 40 and 70. Life expectancy upon diagnosis is just two to five years for most, although a few, including the late physicist Stephen Hawking, have lived more years, even decades, with the debilitating disease.

The new research study recruited 137 ALS patients who had to see symptoms only within the past 18 months. Approximately two-thirds (89 volunteers) received the new drug, while the rest were administered a placebo.

After a period of time, patients were surveyed on a scale of 0 to 48, denoting their degree of disability.

“By the time they entered the trial, on average, patients had already lost 12 points. Their baseline score was about 36, on average,” Paganoni said. “Each question addresses a specific domain of function and is scored on a scale from 0 to 4.”

During the six-month study, considered a “Phase 2” trial (out of three) by the Food and Drug Administration, researchers found that patients who received the tested medication lost 2.32 points less on average than those on the placebo. This suggests a 25% improved outcome for those on the new drug.

“A 2- to 3-point change can mean the difference between being able to do an activity independently or with an assistance device,” said Paganoni, who hopes the discovery could add to the myriad medications required to make ALS more manageable. Meanwhile, researchers will continue to track the progress of volunteers, some of whom have decided to continue or switch to the pilot treatment after the study results showed promise.

“The Ice Bucket Challenge was an important turning point in the fight against ALS,” Paganoni added. “It put ALS on the map and raised awareness of the disease and attracted more investigators and investment to the research.”

The ALS Association is now pushing for wider availability of the drug even before a final human trial, which would last much longer and enlist more participants before the Food and Drug Administration makes a call.

“It’s very unusual for an ALS clinical trial to hit its primary endpoint, so we’re very excited about it,” Neil Thakur, chief mission officer for the ALS Association, told NBC News. “It’s the difference between being able to feed oneself versus being fed or needing versus not needing a wheelchair.”