Trial ALS drug shows potential to drag out patient endurance

Trial ALS drug shows potential to drag out patient endurance


  • Post By : Kumar Jeetendra

  • Source: Massachusetts General Hospital

  • Date: 18 Oct,2020

An experimental medicine that was recently shown to slow the progression of the neurodegenerative disease amyotrophic lateral sclerosis (ALS), or Lou Gehrig’s disease, has now demonstrated the capacity to also prolong patient survival. The findings come from a clinical trial conducted by investigators at the Sean M. Healey & AMG Center for ALS at Massachusetts General Hospital (MGH) and Amylyx Pharmaceuticals, Inc., the company that produces the medication. Amylyx developed AMX0035, the investigational neuroprotective treatment evaluated in the CENTAUR trial and designed to reduce the death and dysfunction of motor neurons.

These findings are an important step forward because, in this trial, early treatment with AMX0035 was associated with longer survival in people with 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.”Sabrina Paganoni, MD, PhD, study’s leader, investigator at the Healey & AMG Center for ALS and assistant professor of Physical Medicine and Rehabilitation at Harvard Medical School and Spaulding Rehabilitation Hospital

ALS, a degenerative condition with no cure, attacks brain and spinal cord nerve cells to progressively influence individuals’ ability to move, speak, eat and even breathe. The new results, reported in the journal Muscle and Nerve, provide additional evidence of the benefits that patients with ALS may experience when taking the oral drug called AMX0035, which is a blend of sodium phenylbutyrate and taurursodiol. These elements target oxidative stress within nerve cells’ energy-producing mitochondria and protein-processing endoplasmic reticulum to help prevent neurodegeneration.

From the CENTAUR trial, 137 participants with ALS were randomized two-to-one to receive AMX0035 or placebo. Recently, researchers demonstrated that AMX0035 slowed ALS disease progression over six months, with impacts on several activities of daily living such as a patient’s ability to walk, talk, use utensils or consume food. Patients who completed CENTAUR were eligible to participate in an open-label expansion (where all patients received AMX0035) aimed at assessing the long-term safety and efficacy of the medication.

Investigators’ 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 group found that participants originally randomized to receive AMX0035 lived for a median of 6.5 months longer than those initially randomized to receive the placebo.

Senior author Merit Cudkowicz, MD, director of the Healey & AMG Center for ALS in MGH, chief of Neurology at MGH, as well as the Julieanne Dorn Professor of Neurology at Harvard Medical School, added:”This is among the first studies to show impact on both survival and function. We are optimistic that this is just the start of many new treatments for ALS.”

Journal reference:

Paganoni, S., et al. (2020) Long‐Term Survival of Participants in the CENTAUR Trial of Sodium Phenylbutyrate‐Taurursodiol in ALS. Muscle & Nerve.

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