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Foundation Supporting ALS Pilot Clinical Trials

Brain illustration


1 July 2015. ALS Association is funding pilot studies with patients having amyotrophic lateral sclerosis to confirm biomarkers, or biochemical indicators, that improve responsiveness to therapies. The studies are being conducted by Neuraltus Pharmaceuticals Inc., a biopharmaceutical developer in Palo Alto, California, and University of Washington Medical Center in Seattle.

ALS, also known as Lou Gehrig’s disease, is a progressive neurological disease that attacks the nerve cells controlling voluntary muscles, such as those in the arms, legs, and face. In ALS, muscles gradually weaken and waste away, leading to individuals losing their strength and their ability to move their arms, legs, and body. When diaphragm and chest wall muscles fail, people lose the ability to breathe without ventilatory support, often leading to death from respiratory failure.

Neuraltus Pharmaceuticals develops treatments for neurodegenerative disorders, with its lead product code-named NP001 designed to regulate the inflammation of macrophages, white blood cells in the immune system that ingest dead or damaged cells. People with ALS are believed to have higher levels of inflamed macrophage activity that release factors in the central nervous system that damage motor neurons.

The new clinical trial aims to confirm the mechanism of NP001 in reducing biomarkers associated with inflammation: interleukin-18 and lipopolysaccharide. In an earlier trial, individuals with ALS taking higher doses of NP001 reported no progression of their symptoms during the 6 months they were taking the drug. People responding to NP001 had higher levels of interleukin-18 and lipopolysaccharide before the treatments that decreased after the treatments.

The new study is led by Robert Miller, director of Forbes Norris MDA/ALS Research Center at California Pacific Medical Center in San Francisco., who led the earlier trial. ALS Association is providing a grant of $1.5 million for the study, in addition to about $1.2 million from Neuraltus.

ALS Association with other funders are also supporting an 8-week study of the drug mexiletine, designed to treat irregular heart rhythms that acts by reducing hyperexcitability of motor neurons. The study, led by neuromuscular specialist Michael Weiss at University of Washington, will investigate the effect of mexiletine on hyperexcitability in the brain’s motor cortex, which controls movements. The trial is expected to measure markers of hyperexcitability with transcranial magnetic stimulation, a non-invasive way to determine hyperexcitability in the brain.

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