Muscle Regeneration: EPIRIUM
Budget
US$21,860
Lay Abstract
Test period:
February 2024 – March 2024
Investigator:
Epirium: Micah Webster, 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 Epirium Muscle Regeneration Project
Prostaglandins, including PGE2, are powerful lipid signaling molecules important for tissue repair and function. The positive effects of PGE2 on muscle are counterbalanced by an enzyme, 15-PGDH, which makes PGE2 non-functional. Blocking 15-PGDH in mice promotes the positive effects of PGE2 and increases muscle stem cell proliferation, improves mitochondrial function, and increases strength. These results suggest an opportunity in leveraging 15-PGDH inhibition to address muscle weakness in FSHD.
Epirium is developing MF-300, a novel orally bioavailable small molecule inhibitor of 15-PGDH that demonstrates significant improvements in muscle strength in multiple mouse models of neuromuscular disease. Orally administered MF-300 will be tested in FSHD mice for improvements on muscle quality and strength.