Muscle Regeneration: ECM/MBV

Budget

US$18,160

Lay Abstract

Test period:

November 2023 to January 2024

Investigator:

University of Pittsburgh: Stephen Badylak, PhD & George Hussey, 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 ECM/MBV Muscle Regeneration Project

Dr. Badylak, at the University of Pittsburgh, has been successful at regenerating muscle in wounded soldiers using ECM (extra cellular matrix).

Based on dish work on muscle cells from FSHD patients, Dr. Badylak believes that ECM, in combination with an enhancer called MBV (matrix bound vesticules), may work in FSHD.