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Muscle-targeted therapy for ALS

Principal Investigator:
  • Blair Leavitt, University of British Columbia
  • ALS Society of Canada

Project Overview

In ALS, the disease is characterized by degeneration of motor neurons and an inability of the brain to signal the muscles to move, resulting in paralysis. Over the past decade, it has become clear that motor neurons are not the only cell type involved in the disease process and attempting to maintain proper functioning of cells that interact with motor neurons has become a strong target for therapy. Dr. Blair Leavitt, professor at the University of British Columbia aims to target muscle health as a potential treatment for ALS. It is known that a specific type of muscle called fast muscle fibers are more vulnerable to becoming paralyzed in ALS while those termed slow fibers are more spared. In this Discovery Grant, Dr. Leavitt will attempt to covert fast muscle fibers into slow fibers using specialized substances called antisense oligonucleotides (ASOs) to reduce the levels of a factor called MyoD in the muscle. He hypothesizes that this might slow down the progression of ALS in mice and if it does, current clinical trials using approved ASOs for other treatments in humans suggests that it would hold great potential to begin human ALS trials in the near future.