New ALS Drug Can Extend Patient Survival, Study Finds

• A potential medication recently shown to slow the progression of ALS has demonstrated the potential to prolong patient survival
• Drug targets oxidative stress within nerve cells’ mitochondria and protein-processing endoplasmic reticulum to help prevent neurodegeneration
• Patients who recieved this drug lived for a median of 6.5 months longer than those originally randomized to receive the placebo

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A potential medication for the neurodegenerative disease amyotrophic lateral sclerosis (ALS), has now shown the ability to extend patients’ survival by several months.

These findings are a result of a clinical trial conducted by Sean M. Healey & AMG Center for ALS at Massachusetts General Hospital (MGH) and Amylyx Pharmaceuticals, Inc., the company that manufactures the medication.

The study recently appeared in Muscle & Nerve journal. The study was led by Sabrina Paganoni, an investigator at the Healey & AMG Center for ALS and assistant professor of Physical Medicine and Rehabilitation at Harvard Medical School and Spaulding Rehabilitation Hospital.

What is ALS?
ALS or Lou Gehrig’s disease is a nervous system disease that weakens muscles and impacts physical function. In this disease, nerve cells break down reducing functionality in the muscles that they supply. Moreover, this dreadful disease doesn’t have any cure.

The current drug which is basically a combination of drugs is called AMX0035. The drug is a combination of sodium phenylbutyrate and taurursodiol which target oxidative stress within nerve cells’ energy-producing mitochondria and protein-processing endoplasmic reticulum to prevent neurodegeneration.

The team conducted a trial called CENTAUR where in 137 patients participated. They found that participants originally randomized to receive AMX0035 lived for a median of 6.5 months longer than those originally randomized to receive the 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.

“This is one of the first studies to show the effect on both function and survival. We are hopeful that this is just the beginning of many new treatments for ALS.” said Merit Cudkowicz, senior author of the study.

Journal Reference:
Sabrina Paganoni, Suzanne Hendrix, Samuel P. Dickson, Newman Knowlton, Eric A. Macklin, James D. Berry, Michael A. Elliott, Samuel Maiser, Chafic Karam, James B. Caress, Margaret Ayo Owegi, Adam Quick, James Wymer, Stephen A. Goutman, Daragh Heitzman, Terry D. Heiman‐Patterson, Carlayne E. Jackson, Colin Quinn, Jeffrey D. Rothstein, Edward J. Kasarskis, Jonathan Katz, Liberty Jenkins, Shafeeq Ladha, Timothy M. Miller, Stephen N. Scelsa, Tuan H. Vu, Christina N. Fournier, Jonathan D. Glass, Kristin M. Johnson, Andrea Swenson, Namita A. Goyal, Gary L. Pattee, Patricia L. Andres, Suma Babu, Marianne Chase, Derek Dagostino, Meghan Hall, Gale Kittle, Matthew Eydinov, Michelle McGovern, Joseph Ostrow, Lindsay Pothier, Rebecca Randall, Jeremy M. Shefner, Alexander V. Sherman, Maria E. St. Pierre, Eric Tustison, Prasha Vigneswaran, Jason Walker, Hong Yu, James Chan, Janet Wittes, Zi‐Fan Yu, Joshua Cohen, Justin Klee, Kent Leslie, Rudolph E. Tanzi, Walter Gilbert, Patrick D. Yeramian, David Schoenfeld, Merit E. Cudkowicz. Long‐Term Survival of Participants in the CENTAUR Trial of Sodium Phenylbutyrate‐Taurursodiol in ALS. Muscle & Nerve, 2020; DOI: 10.1002/mus.27091

Press Release: Massachusetts General Hospital