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Pre-clinical drug discovery against hexanucleotide repeat-containing C9orf72 toxicity using Caenorhabditis elegans and patient-derived induced motor neurons

Principal Investigator:
  • Prateep Pakavathkumar,

    Centre Hospitalier de l'Université de Montréal

  • ALS Society of Canada

Project Overview

A few years ago, Dr. Parker and researchers in his lab genetically engineered worms that mimicked human ALS through motor neuron degeneration and paralysis. He then tested them with a battery of potential treatments to see if they could find one that would have a positive effect on motor neuron health and function. Through this work, they identified the drug pimozide as having a potential impact on functionality in worm models and investigators are now studying the effectiveness of pimozide in a Phase 2 clinical trial in eight locations across Canada.

In this project, Dr. Pakavathkumar will use a similar approach to screen potential therapies using new worms that have been modified in C9ORF72, which is the most common genetic cause of ALS in humans. If he finds promising candidates, he will validate the therapies in further lab experiments using motor neurons derived from people with the C9orf72 form of ALS. Any therapies that continue to show potential will be shared with other researchers to test them in zebrafish and mouse models with the hope of eventually moving them into human clinical trials.