Clinical trial reports six and a half month longer survival in patients who were treated with new treatment for ALS.

Amyotrophic lateral sclerosis or ALS is a progressive nervous system disorder that affects nerve cells in the brain and spinal cord, causing muscle weakness and loss of control. It is also known as Lou Gehrig’s disease after the baseball player was diagnosed with it. ALS usually begins with muscle twitching and weakness in a limb or slurred speech. Eventually, the disease affects control of the muscles needed to move, speak, eat, and breathe.

A clinical trial – called the CENTAUR trial – has reported up to six and a half month longer survival in patients who were treated with the experimental treatment, AMX0035.

The investigators, led by Sabrina Paganoni, MD, Ph.D., of the Healey & AMG Center for ALS at Massachusetts General Hospital, noted that these data, combined with the study’s previously reported results, suggest that the therapy provides patients both functional and survival benefits.

These new findings are significant, as there are only two available ALS treatments, riluzole and edaravone. As of yet, only riluzole had been shown to prolong survival. The effects of edaravone on survival are unknown.

In the randomized trial, included individuals were randomized in two-to-one fashion to AMX0035 (3-g PB and 1-g TURSO per sachet) or matching placebo, administered twice daily by mouth or feeding tube for a planned duration of six months. Additionally, patients eligible for the open-label extension (OLE) were given the option to enroll in an OLE and receive the active drug for around thirty months. In total, ninety-two percent of the study population enrolled. The team evaluated the long-term safety and efficacy of the new ALS treatment during the follow-up.

The analysis included 135 patients randomized in the trial, with median overall survival being 25 months for those treated with AMX0035 compared with a median survival of 18.5 months in the placebo group, equating to a forty-four percent lower risk of death.

Nearly three-year survival analysis incorporated all participants who enrolled in CENTAUR, whether they continued long-term treatment with AMX0035 in the open-label extension or not. The team found that AMX0035 treatment resulted in a 6.5-month longer median survival as compared to a placebo. These results support that AMX0035 has both functional and long-term survival benefits.

Reference: Paganoni S., Hendrix S., Dickson SP., Knowlton N., Macklin EA., Berry JD., Cudkowicz M.E. Long-Term Survival of Participants in the CENTAUR Trail of Sodium Phenylbutyrate-Taurursodiol in ALS. Muscle Nerve. 2020 Oct 16. doi: 10.1002/mus.27091.