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Experimental ALS drugs have shown potential to prolong patient survival




Experimental drugs recently shown to slow the progression of the neurodegenerative disease amyotrophic lateral sclerosis (ALS), or Lugeric’s disease, now show the potential to prolong the survival of patients. The findings are from Sean M. Healey & AMG Center for ALS at Massachusetts General Hospital (MGH) and Amylyx Pharmaceuticals, Inc., which manufactures pharmaceuticals. It was obtained from clinical trials conducted by researchers in Japan. Amylyx has developed AMX0035, an ongoing neuroprotective therapy evaluated in the CENTAUR trial and designed to reduce motor neuron death and dysfunction.

ALS, a non-healing degenerative state, attacks nerve cells in the brain and spinal cord, gradually affecting an individual’s ability to move, speak, eat, and even breathe.New results reported in the journal Muscles and nervesProvides additional evidence of the benefits that patients with ALS may experience when taking an oral drug called AMX0035, which is a combination of sodium phenylbutyrate and taurulusodiol. These components help prevent neurodegeneration by targeting oxidative stress in nerve cell energy-producing mitochondria and protein-treated vesicles.

In the CENTAUR trial, 137 ALS participants were randomized 2: 1 to receive AMX0035 or placebo. Recently, researchers have demonstrated that AMX0035 slows the progression of ALS disease for six months and affects activities of daily living such as walking, talking, using equipment, and swallowing food in patients. Patients who completed CENTAUR were eligible to participate in an open-label extension (all patients underwent AMX0035) aimed at assessing long-term safety. Effectiveness of medicine.

The investigator’s nearly three-year survival analysis included all participants enrolled in the CENTAUR, regardless of whether they continued long-term treatment with AMX0035 in an open-label extension. The team found that the first randomized participant to receive AMX0035 lived 6.5 months longer by median than the first randomized participant to receive the placebo.

These findings are an important step forward in this study, as early treatment with AMX0035 was associated with longer survival in ALS patients. These results provide substantive evidence supporting the role of AMX0035 in the treatment of ALS. The next step depends on ongoing discussions with regulators. “

Sabrina Paganoni, MD, PhD, Research Leader, Researcher at ALS Healy & AMG Center, Assistant Professor of Physical Therapy and Rehabilitation at Harvard Medical School and Spaulding Rehabilitation Hospital

Senior authors Merit Kudkowicz, MD, Director of the Healey & AMG Center for ALS at MGH, Dean of Neurology at MGH, and Professor of Neurology at Harvard Medical School, Julieanne Dorn, added: For both function and survival. We hope this is just the beginning 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-taurulusodiol in ALS. Muscles and nerves.


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