Promising Experimental ALS Drug

Patients who received the drug in the closely monitored clinical trial are reported to have retained a higher level of certain motor functions than those on placebo. The drug is being developed at the biotech Amylyx who released the outlines of the data in December 2019, but this new report details how effective the drug is in slowing the progression of the disease. 

Currently, there is no cure for ALS and only a couple treatment to help manage the symptoms, any additional treatments would be a welcomed addition to those affected by the disease. While the researchers are hopeful, and accompanying editorial in NEJM called this promising data from the Phase ⅔ study only “tantalizing”, saying the benefit appeared to be modest, and that a Phase 3 trial is important to validate the study conclusions. 

“We’re thrilled to be part of the charge to change the care for patients with ALS — a disease that’s been unanswered for far too long,” Justin Klee, a co-founder and co-CEO of Amylyx, said in an interview. “We hope that we and everyone else in this field can change the face of this horrible disease.” 

Though the exact cause for amyotrophic lateral sclerosis/Lou Gehrig’s disease is unknown, motor neurons begin to stop functioning and die off which stops the transmission of commands to the rest of the body. 10% of the cases have clear genetic roots, but the remaining cases have no clear cause. This disease follows a similar course in those affected with patients experiencing muscle stiffening and over time with progression the patient loses the ability to move, speak, eat, and even breathe.

This double-blinded, randomized and controlled study monitored 137 ALS patients over the course of 6 months. Patients had particularly fast progressing illness, and for every 1 patient receiving placebo 2 were given the drug which is a combination of sodium phenylbutrate and taurursodiol. Outcomes were measured on the ALS Functional Rating Scale. 

On this 48 point scale, those taking the drug experienced their condition decline on average 2.9 points less over 6 months than those on placebo. While outcomes varied, overall most saw some improvement in fine motor function, without seeing improvements in their ability to breathe. Most began the study at 36 points, the placebo group dropped to 26 by the end of 6 months and those in the drug group scored 29 points, according to Sabrina Paganoni, who is an ALS researcher at Harvard Medical School and the principal investigator of the trial. 

“Even a small change in a couple of points can mean a large change in what daily life looks like,” Paganoni said. “A two-point change could mean the difference between eating successfully or requiring a feeding tube — or between walking and using a wheelchair.”