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Trial Underway Testing New ALS Treatment

Neurons

(commonfund.nih.gov)

8 August 2017. Patients in a clinical trial testing a treatment for amyotrophic lateral sclerosis, or ALS, began receiving their first doses administered by the study team. The intermediate-stage trial is conducted by Amylyx Pharmaceuticals Inc. in Cambridge, Massachusetts, developer of a therapy code-named AMX0035.

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, the 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, nor effective treatments for slowing progression of the disease.

Amylyx Pharmaceuticals is developing AMX0035 that aims to slow the disease’s progression by targeting two sources of stress on nerve cells believed to contribute to cell death and inflammation associated with ALS. One stress source is the mitochondria or energy center of the cell, while the second source is the endoplasmic reticulum, a network of sacs and tubes that takes up much of the internal space of the cell, and transfers molecules between the nucleus and the rest of the cell. The company says both of these stress pathways need to be addressed to treat neurodegenerative diseases such as ALS.

AMX0035, says Amylyx, is made of two approved compounds that together work on both of the stress sources: phenylbutyrate for mitochondrial stress and tauroursodeoxycholic acid for endoplasmic reticulum.  Both compounds were tested individually with ALS patients earlier, but AMX0035 is designed to combine and optimize their effects. The company claims preclinical studies conducted by contract research organizations and academic labs show AMX0035 blocks nerve cell death and inflammation in lab tests not representing any specific disorder, as well as simulated models of ALS.

The clinical trial is recruiting 132 individuals with ALS for a maximum of 18 months at 25 locations in the U.S. Participants are randomly assigned to receive either AMX0035 or a placebo on a 2-to-1 ratio, given twice a day for 24 weeks. The study team led by researchers from Massachusetts General Hospital in Boston are looking primarily for indicators of the treatment’s safety, such as incidence of adverse effects, as well as changes in participants’ scores on a standard rating scale of muscle activity and day-to-day functions among ALS patients. Participants are also rated on isometric limb strength, lung capacity, and chemical and imaging biomarkers of nerve cell death and integrity.

Participants receiving AMX0035 or the placebo may continue in an open-label or non-blinded clinical study after the initial 24 weeks of treatment. The organizations ALS Association and ALS Finding A Cure are providing nearly $3 million to help fund the trial, with the Northeast ALS Consortium and Leandro P. Rizzuto Foundation also taking part.

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