A bucket of ice-cold water rains down on your head, while friends challenge you and video this frigid maneuver to post to social media. The shock … the cold … the soaking.

Who doesn’t remember the Ice Bucket Challenge of 2014, which raised $115 million dollars in donations for ALS? The unique fund-raising challenge has helped bring researchers closer to breakthroughs in diagnosing, treating and eventually curing amyotrophic lateral sclerosis, or Lou Gehrig's disease, a degenerative disorder with no known cure.

There are currently only two approved drugs to treat ALS, a progressive disease of the nervous system that affects nerve cells in the brain and spinal cord, resulting in loss of muscle control and affecting a person’s ability to speak, move, eat and breathe. One drug, riluzole, has been available for 25 years and works to extend lifespan by an average of a few months. The second drug, approved in 2017, is edaravone, which has been shown to help patients function longer into their disease.

“[Edaravone's] effect is relatively modest, but it's supposed to slow down disease progression for those who are in the early stages of the disease,” social worker Leigh Stephens, a psychosocial counselor at the ALS Society of Quebec, told Medical Daily.

Now, research is underway on third drug, AMX0035, an investigational, neuroprotective therapy designed to reduce the death and dysfunction of motor neurons.

AMX0035 is in Phase 2 of a clinical trial of people who have the disease. The study is conducted by the Sean M. Healey & AMG Center for ALS at Massachusetts General Hospital, and Amylyx Pharmaceuticals, Inc., the company that manufactures the medication. Results show promise in slowing the progression of ALS and prolonging patient survival.

“Ever since the Ice Bucket Challenge of 2014, research in ALS has been booming,” Ms. Stephens said. “Families are sometimes frustrated by the time required to see the fruit of this labor.”

What is AMX0035?

Researchers looked at 137 participants aged 18-80 who were symptomatic with the disease for less than 18 months. They randomly received either AMX0035 or a placebo over six months.

Patients who completed the trial were then eligible for an open-label extension, where all patients received AMX0035 to further test its long-term efficacy. The results of the phase 2 efficacy study of AMX0035 were reported in the journal Muscle and Nerve .

The study aimed to measure a change on the ALSFRS-R (the revised ALS functional rating scale), compared with the placebo group. Researchers also measured muscle strength, vital lung capacity, need for tracheostomy, hospitalizations and survival. About 5% of participants experienced diarrhea and abdominal pain, the most common side effects, which lessened with time. Ninety-eight out of the 137 participants completed the trial.

AMX0035 isn’t completely new. It is a combination of two drugs already in use, sodium phenylbutyrate and taurursodiol, that target oxidative stress in nerve cells’ mitochondria to help prevent neurodegeneration.

Results Offer Hope

The researchers found AMX0035 slowed ALS disease progression over six months and had a positive impact on daily life, such as walking, talking, using utensils and swallowing food. Participants who were randomized to receive the medication also lived an average of 6.5 months longer than those who received the placebo.

Study researchers consider the findings an important step in both treating and eventually curing ALS, as AMX0035 is associated with both longer survival and improved functionality.

“[This research] is bringing hope to many, if for no other reason than to buy time to increase the chances of still being alive for other advancements in disease treatment and, hopefully, cure,” Ms. Stephens said. “It may encourage others to access a subsequent trial.”

Typically, drugs become available after phase 3 trials. There are some 57 phase 3 trials ongoing for ALS. Some are currently recruiting participants, while others are not. Find more information here.

Jennifer Nelson is a health writer based in Florida who also writes about health and wellness for AARP, PBS’ Next Avenue, Shondaland, and others.