On any given day, the stem cells located in our muscles are busy rebuilding and repairing the damage caused by exercise or injury. Under normal circumstances, these muscle stem cells are able to keep up with the task, allowing normal muscle function. However, in muscular dystrophies, a mutation causes the formation of weaker muscle fibres, leading to a much higher number of injury sites, along with chronic inflammation, such that the muscle stem cells become incapable of regenerating the tissue.
This has a severe impact on the quality of life for individuals with muscular dystrophy, and despite progress in learning more about the genetics of these diseases along with improved physical aids to help with mobility, there are still few treatments available to patients.
But knowing impaired stem cells play a role in these diseases provides an opportunity for regenerative medicine, and Dr. Florian Bentzinger, who recently established a new lab at the Université de Sherbrooke, has been pursuing this challenge. His research is currently focused on a small circulating hormone that is highly efficient at stimulating muscle stem cells to multiply and form new tissue.
With support from an Impact grant from the Stem Cell Network, Bentzinger and collaborators will test top candidate pharmaceuticals for their ability to activate muscle stem cells in models of muscular dystrophy. A lot of important information can be gained from this work about the fundamental properties of muscle stem cells that will be of great help to future regenerative therapies.
This line of research is well positioned for success at SCN’s newest institutional partner, the Université de Sherbrooke, which is known for its expertise in medicinal chemistry and its ability to form industry partnerships that can take therapeutic findings to market.
Florian Bentzinger’s project is part of the 2018-19 Impact Research Agreement Program providing $1.4M for 14 national projects that span clinical translation, commercialization and public policy. A total of 59 investigators (20 Principal and 39 Collaborators) at 18 institutions and 54+ trainees are engaged.