An investigational drug for treating amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig's disease, has been shown to slow the progression of the disease, according to a study published in the New England Journal of Medicine. According to the researchers, these results provide hope for a future treatment for ALS, which is a fatal disease that currently has no cure.

The oral medication tested in the trial, AMX0035, is a combination of sodium phenylbutyrate and taurursodiol. Each of these 2 drugs contained within AMX0035 target a different cell component critical to protecting against nerve damage.

During the CENTAUR trial, the researchers randomized 137 patients with ALS into groups of those receiving AMX0035 and those receiving the placebo. The researchers then observed the patients over 6 months and found that those given AMX0035 had better outcomes than those given the placebo. This assessment was based on patients’ responses to the ALS Functional Rating Scale (ALSFRS-R), a questionnaire that evaluates activities of daily living, such as ability to walk, hold a pen, or swallow food.

"The participants treated with AMX0035 demonstrated a significant slowing of ALS disease progression as measured by the ALSFRS-R. This is a milestone in our fight against ALS," said Sabrina Paganoni, MD, PhD, principal investigator of the CENTAUR study, investigator at the Healey & AMG Center for ALS at MGH, and assistant professor of PM&R at Harvard Medical School (HMS) and Spaulding Rehabilitation Hospital, in a press release.

Additionally, senior author Merit Cudkowicz, MD, director of the Healey & AMG Center for ALS at MGH, chief of Neurology at MGH, and the Julieanne Dorn Professor of Neurology at HMS, explained in the press release that through collaboration with others, the authors of the study were able to investigate this novel approach to the problem of motor nerve cell dysfunction.

“With guidance from our team and in collaboration with our colleagues in the Northeast ALS Consortium (NEALS), Mass General Biostats and the Barrows Neurological Institute, the clinical trial moved forward quickly and carefully,” Cudkowicz said in the release. “We are proud of this important study. We are also very thankful to the participants and their families for their key role in advancing research."

Justin Klee, one of the co-founders and co-CEOs of Amylyx Pharmaceuticals, the company that manufactures AMX0035, noted that patients with ALS and their families do not have time to wait.

"People with ALS progressively lose their ability to function and care for themselves, so we want to do everything we can to help them slow down this devastating disease,” Klee said in the press release. “We will be working with the FDA to determine next steps and the path for patients to gain access to AMX0035. We'll continue to share our plans with the community as they develop."

REFERENCE
Investigational ALS drug generates promising clinical trial results. Boston, MA: Massachusetts General Hospital; September 2, 2020. eurekalert.org/pub_releases/2020-09/mgh-nad090220.php. Accessed September 14, 2020.