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Funded Grants

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

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.

Principal Investigator

Prateep Pakavathkumar , Centre Hospitalier de l’Université de Montréal

Partners and Donors

ALS Society of Canada

Project Complete

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

  • Grant Type

    Capacity building grants

  • Area of research

    Neurodegeneration

  • Disease Area

    ALS

  • Competition

    ALS Canada – Brain Canada Trainee Program 2018

  • Province

    Québec

  • Start Date

    2018

  • Total Grant Amount

    $30,692

  • Health Canada Contribution

    $15,346

Contact Us

1200 McGill College Avenue
Suite 1600, Montreal, Quebec
H3B 4G7

+1 (514) 989-2989 info@braincanada.ca

Please note all online donations will receive an electronic tax receipt, issued by Brain Canada Foundation.

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The offices of Brain Canada Foundation are located on the traditional, ancestral territory of the Kanien'kehá:ka Peoples, a place which has long served as a site of meeting and exchange amongst nations. We honour and pay respect to elders past, present and emerging, and dedicate ourselves to moving forward in the spirit of partnership, collaboration, and reconciliation. In our work, we focus our efforts on the Truth and Reconciliation Commission’s Calls to Action, particularly those that pertain to improving health for Indigenous Peoples and that focus on advancing our own learning on Indigenous issues.

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