La génétique de la neurodégénérescence liée à l'âge


Alex Parker

Centre de recherche du CHUM


Domaine : neurosciences, santé mentale et toxicomanies

Programme chercheurs-boursiers - Junior 2

Concours 2014-2015

Amyotrophic lateral sclerosis (ALS) is a relentless late-onset disease affecting motor neurons causing fatal paralysis anywhere from 6 months to 6 years after onset. No effective treatments, let alone cures, are available. Advances in genetics have discovered many new genes that are responsible for the inherited version of ALS. With all of this new information comes the need to make new models to better understand disease mechanisms and for drug discovery. Making new ALS models in mice will take many years, and industrial drug discovery approaches can only be set up once disease mechanisms are known.
To meet the urgent needs of ALS patients we use the model organism Caenorhabditis elegans with its powerful genetics and rapid methods to model human neurodegenerative diseases. We created new worm models for various forms of ALS and we used these animals to discover disease mechanisms. Importantly, we also use these animals to discover drugs that may be clinically relevant to ALS patients. We discovered a group of FDA approved drugs that rescued motor neuron degeneration in C. elegans. In collaboration with local and national ALS researchers and clinicians we have validated one drug in higher animals and a clinical trial in ALS patients is underway. Our work suggests that many of the genetic and cellular mechanisms of ALS may overlap with other neurological diseases. Thus, we will use our simple genetic models and continue to discover new disease mechanisms and therapies for ALS and other late-onset neurodegenerative disorders.