Muscle Regeneration: SATELLOS

Budget

US$17,300

Lay Abstract

Test period:

December 2023 – March 2024

Investigator:

Satellos: Phil Lambert, PhD

Myologica: Ramzi Khairallah, PhD

Lay abstract

Muscle Regeneration Projects

We don’t have a cure for FSHD – yet. But that shouldn’t stop us from working on muscle regeneration. Ideally, we would like to have a way to stop FSHD – and get our muscles back too.

So, in parallel to all the good work being done by Solve FSHD and others to find a cure for FSHD – the FSHD Canada Foundation is working closely with the SMA Foundation on muscle regeneration. The SMA Foundation are screening potential muscle regeneration compounds on SMA-mice at Myologica – a lab that specializes in using mice for muscle regeneration testing.

Myologica also has a colony of FSHD-mice. These mice will be used for testing compounds that may work for muscle regeneration in FSHD patients. Some of these compounds have shown encouraging results on SMA-mice. And most of the compounds selected are already being used in other diseases and should, therefore, have a faster track to regulatory approval.

Each test is relatively inexpensive – and short. The idea is that with enough “shots on goal” – we will have a good chance of at least one of them working.

The Satellos Muscle Regeneration Project

This study will evaluate the effect of enhancing muscle regeneration using a small molecule inhibitor of AAK1 (provided by Satellos Bioscience Inc.) in a model of FSHD.  FSHD mice will be treated for up to 4 weeks with an orally available small molecule inhibitor of AAK1, followed by a non-invasive evaluation of muscle force.