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First Therapies Selected for Multi-Drug ALS Trial

Brain cell networks

(Gerd Altmann, Pixabay)

Updated 30 Sept. 2019. A clinical trial is getting underway to test five treatment candidates for amyotrophic lateral sclerosis, or ALS, beginning with the first three therapies. The study, known as a platform trial, is being conducted by the Healy Center for ALS at Massachusetts General Hospital in Boston.

ALS, also known as Lou Gehrig’s disease, is a progressive neurodegenerative disorder where neurons or nerve cells controlling muscles in the body begin to waste away, and can no longer send or receive signals from the brain or spinal cord. As the nerve cells stop functioning, muscles in the limbs, and later speech and breathing muscles, begin weakening and eventually stop functioning. Most people with the disease die of respiratory failure. There are currently no cures for ALS, and few effective treatments for slowing progression of the disease.

A platform clinical trial, also called an adaptive trial, is designed to test multiple treatments at a time. The studies use a common master plan or protocol that allow for adding new therapies or changing the study’s characteristics as conditions require. These trials also use Bayesian statistics, a branch of statistics using models built to accommodate adding new data and adjusting the model’s calculations as new data are added. Platform or adaptive trials are now used to test cancer therapies, with results from one of those trials reported by Science & Enterprise in September 2018.

Merit Cudkowicz, director of the ALS Center at Mass. General, says in a hospital statement, “This approach cuts the time to find an effective treatment in half, decreases costs by a third or more, and is supported by our patients, the FDA, ALS clinicians and scientists, and our pharma colleagues.”

In March 2019, the ALS Center issued an open call for new ALS therapies for testing in the trial. From 30 responses, the trial’s evaluation and advisory committee’s selected five drugs for inclusion. The study is expected to start with three drugs, then add two more soon after. The five drug candidates are:

Pridopidine, developed by Prilenia, a start-up enterprise in Herzliya, Israel. Pridopidine is a Sigma-1-receptor agonist that the company says regulates cellular processes important to neurodegenerative diseases. Prilenia says Pridopidine is being tested in preclinical studies and clinical trials for several neurodegenerative disorders, including ALS.

Zilucoplan, made by Ra Pharmaceuticals in Boston, a synthetic peptide developed to treat the autoimmune disorders generalized myasthenia gravis and  immune-mediated necrotizing myopathy, as well as ALS. The drug candidate is now in early- and mid-stage clinical trials for the two autoimmune diseases.

Verdiperstat, developed by Biohaven Pharmaceutical in New Haven, Connecticut. Verdiperstat, says Biohaven, inhibits the myeloperoxidase or MPO enzyme, considered a a key driver of oxidative and inflammatory processes. Higher MPO levels, says the company, are found in a number of brain disorders, and verdiperstat is found to reduce oxidative stress and inflammation, also implicated in the onset of ALS.

Also planned for testing are CNM-Au8 nanocrystalline gold being developed by Clene Nanomedicine in Salt Lake City, and IC14 immunotherapy, developed by Implicit Bioscience in Seattle. The drug candidates for immediate and later assessment in the study are still under consideration.

The trial is expected to evaluate promising biomarkers of ALS and incorporate new types of outcome measures. “This design,” adds Cudkowicz, “optimizes chances for people to receive active treatment, provides answers faster, and ensures we keep learning about the disease.”

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