Amyotrophic Lateral Sclerosis (ALS) is a neurodegenerative disease that in advance stages results in loss of control over voluntary muscles, dramatically affecting people's ability to perform basic everyday actions such as walking, speaking, swallowing and eventually breathing. The disease has no cure to date.
The trial's results, published in the Muscle and Nerve scientific journal and reported by Medical Express News, clearly point at the benefits that patients with ALS may experience when taking an oral drug called AMX0035, a combination of sodium phenylbutyrate and taurursodiol that targets oxidative stress in nerve cells and helps prevent neurodegeneration.
AMX0035 is an experimental neuroprotective therapy developed by Amylyx Pharmaceuticals. A recent study including 137 participants with ALS showed that AMX0035 slowed ALS disease progression over six months, improving patients' ability to perform daily tasks for a longer period of time before the disease progressed.
Participants who completed that initial study were invited to enroll in another long-term trial that aimed at determining the long-term efficiency and safety of the drug.
The median overall survival for all CENTAUR study participants being administered AMX0035 was 6.5 months longer than the group originally randomized to placebo.
"These findings are an important step forward because, in this trial, early treatment with AMX0035 was associated with longer survival in people with ALS," said Sabrina Paganoni, MD, Ph.D., researcher at the Healey & AMG Center for ALS."These results provide substantial evidence supporting the role of AMX0035 for the treatment of ALS. Next steps will depend on ongoing discussions with regulatory agencies," she added.
Merit Cudkowicz, MD, director of the Healey & AMG Center for ALS at MGH, added that this study "is one of the first studies to show effect on both function and survival. We are hopeful that this is just the beginning of many new treatments for ALS."