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Medical News & Perspectives
February 13, 2018

Simple Models and Ice Bucket Challenge Fuel Progress in ALS Treatment

JAMA. 2018;319(6):535-537. doi:10.1001/jama.2017.20704

Late last year, a phase 2 clinical trial began enrolling patients with amyotrophic lateral sclerosis (ALS) to see if a 50-year-old schizophrenia drug could curb their symptoms and slow progression. This promising drug candidate took an unusual path moving rapidly from studies in a tiny worm into early clinical trials thanks to an unusual collaboration and a splash of cold water.

The work started in the laboratories of neuroscientists Alex Parker, PhD, and Pierre Drapeau, PhD, at the University of Montréal Hospital Research Centre, who first identified and confirmed the therapeutic potential of the antipsychotic pimozide in 2 model organisms: a tiny worm called Caenorhabditis elegans and zebrafish. Five years ago at a conference, they shared their findings with Lawrence Korngut, MD, director of the Calgary ALS and Motor Neuron Disease Clinic and researcher at the Hotchkiss Brain Institute.