An investigational drug for ALS
An experimental drug that was recently shown to slow the development of the neurodegenerative disease of amyotrophic lateral sclerosis (ALS), or Lou Gehrig’s disease, has now shown the potential to prolong the survival of sufferers as well. The results come from a clinical trial conducted by researchers at Sean M. Healey & AMG Middle for SLA at the Massachusetts General Clinic (MGH) and Amylyx Prescription drugs, Inc., the company that makes the drug. Amylyx developed AMX0035, the experimental neuroprotective therapy evaluated in the CENTAUR trial and designed to reduce motor neuron death and dysfunction.
ALS, a no-cure degenerative disease, attacks nerve cells in the brain and spinal cord to gradually affect an individual’s ability to move, speak, eat, and even breathe. The new findings, reported in the journal Muscle mass and Nerve, provide further evidence of the benefits people with ALS may experience when taking the oral medication called AMX0035, which is a combination of sodium phenylbutyrate and taurursodiol. These components target oxidative worry in the energy-producing mitochondria of nerve cells and the protein-processing endoplasmic reticulum to help prevent neurodegeneration.
In the CENTAUR trial, 137 individuals with ALS were randomized two-to-one to receive AMX0035 or placebo. Recently, researchers have shown that AMX0035 slows the progression of ALS over six months, with impacts on various activities of daily living such as a client’s ability to walk, talk, use utensils or swallow food. . Individuals who completed CENTAUR were eligible to participate in an open label extension (in which all patients received AMX0035) to assess the safety and extensive term efficacy of the drug.
The investigator’s nearly three-year survival analysis included all individuals who enrolled in CENTAUR, whether or not they continued long-term treatment with AMX0035 in the open-label extension. The team found that individuals initially randomized to receive AMX0035 lived a median of 6.5 months longer than those initially randomized to receive placebo.
“These results are a self-critical step forward, in this trial, early treatment with AMX0035 was associated with longer-over-longer survival in people with ALS,” said study leader Sabrina Paganoni, MD, PhD, researcher at Healey & AMG Middle for SLA Assistant Professor of Physical Medicine and Rehabilitation at Harvard Health care University and Spaulding Rehabilitation Hospital. “These results provide substantial evidence supporting the role of AMX0035 in the treatment of ALS. Next steps will depend on ongoing discussions with regulatory agencies. ”
Blocking molecules involved in resistance to ALS drugs may improve the work of ALS therapeutics, suggesting that re-evaluation of drugs that appeared to have failed may be appropriate
(Philadelphia) – Amyotrophic lateral sclerosis (ALS) is a neurodegenerative disease that primarily kills motor neurons, leading to paralysis and death within 2 to 5 years of diagnosis. ALS currently has no cure. Despite promising early-stage research, the majority of drugs in development for ALS have failed. Now, researchers have discovered a possible explanation. In a study published Nov. 20 in the Annals of Clinical and Translational Neurology, researchers show that the brain machinery for emitting toxins is intensified in ALS patients, and that this machinery also throws out (by pumps) a drug intended to treat ALS, which decreases the therapeutic effectiveness of the drug. Work has shown that when these pumps are blocked, the drug becomes more effective at slowing disease progression in mouse models.
Lead author Advantage Cudkowicz, MD, director of the Healey & AMG Center for ALS at the MGH, chief of neurology at the MGH, and Julieanne Dorn professor of neurology at Harvard Health care University, added: “ This is one of the first studies to show the effect on both function and survival. We hope this is just the start of many new treatments for ALS. “