Principal Investigator
Andrew Bateman
The Research Institute of the McGill University Health Centre
Co-investigator
Jay Penney
University of Prince Edward Island
Andrew Tasker
University of Prince Edward Island
Project of $881,122 over 3 years
- Supported by CQDM through:
Ministère de l’Économie, de l’Innovation et de l’Énergie du Québec (MEIE) - And by co-funding partners:
– Neurodyn Life Sciences Inc.
– Brain Canada Foundation
Challenge:
Amyotrophic lateral sclerosis (ALS) is a disease affecting the spinal cord motor neurons that control muscle movement. It currently has no cure and is invariably fatal. The objective of this project is to develop a therapy that slows, halts, or potentially functionally stabilizes ALS progression.
Solution:
In ALS, clumps of a molecule called TDP-43 form inside diseased motor neurons and contribute to their death. The research team proposes that the protein progranulin can counter TDP-43 toxicity and provide a route towards new treatments for ALS. Importantly, progranulin both boosts motor neuron survival and reduces inflammation in the ALS spinal cord, in part by regulating lysosomes, which remove harmful TDP-43 from the cell. Progranulin itself is processed by lysosomes into smaller biologically active units called granulins, but this may be impaired in ALS. To correct this defect, the research team will develop a suite of granulin combinations and test their protective activity on motor neuron survival and their effects on the inflammatory cells of the nervous system. The best granulin formulations will be then tested in TDP-43-ALS mice to measure motor function, motor neuron preservation and reduced inflammation compared against full-length progranulin.
Expected Achievements /Impacts:
As a result of the project, the team will deliver a robust, granulin-based formulation designed for an FDA Investigational New Drug submission and subsequent clinical development. This breakthrough has the potential to transform the lives of ALS patients, offering improved quality of life, extended survival, and even the possibility of disease stabilization. The project industrial partner, Neurodyn Life Sciences, will significantly enhance the progranulin program, bring this novel therapy into the clinics, while driving scalable growth and reinforcing Quebec’s position as a leader in life sciences innovation.
