About FSHD Canada Foundation

FSHD clinical trials are coming to Canada – get registered here: Canadian Neuromuscular Disease Registry

Muscle Regeneration: ALKAHEST

Muscle Regeneration: ALKAHEST



Lay Abstract

Test period:

January 2023 – March 2024


Alkahest: Benson Lu, PhD & Annette Tennstaedt, 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 Alkahest Muscle Regeneration Project

Grifols is a global leader in producing human plasma-derived medicines for both common and rare diseases. Alkahest, a subsidiary of Grifols, is leveraging Grifols’s expertise in plasma manufacturing to explore therapeutic options for FSHD. Alkahest has identified several plasma protein fractions that have demonstrated promising results in enhancing recovery of muscle function in both mouse and cell models of neuromuscular diseases.  The primary objectives of this project are to investigate the efficacy of these plasma fractions in improving muscle functions in an FSHD mouse model and in cells from FSHD patients.

Aim 1: Evaluation of a Plasma Fraction in Improving Muscle Functions in FSHD mice

  • Funding for this project will support the in-life phase of an FSHD mouse model conducted at Myologica, a contract research organization specializing in testing therapeutics in mouse model of neuromuscular diseases.
  • The focus will be on assessing a promising plasma fraction manufactured by Grifols, in improving grip strength and muscle force. The study is expected to commence in January 2024 and will span a 2-month period. The funding will support animal experiments and personnel costs.

Aim 2: Evaluation of Plasma Fractions in Improving Cellular Defects in FSHD patient cells

  • Funding for this project will facilitate in vitro cell culture studies aimed at assessing the efficacy of plasma fractions in muscle cells derived from FSHD patients. The funding will support the initial phase of this endeavor, which involves establishing optimal conditions for the growth of patient cells and covering licensing fees.
  • The study is projected to commence in December 2023 and will span a 3-month period. Following the completion of this preliminary phase, we will establish clinically relevant endpoints including DUX4 gene expression and cell death, to validate the beneficial effects of plasma fractions.

The FSHD Canada Foundation acknowledges the generous support of the Wilson 5 Foundation towards the funding of this grant.

https://twitter.com/#!/FSHDCanada/ http://www.facebook.com/pages/FSHD-Canada/331378503625267 /contact Suite 201, 1100 1st St. SE Calgary, Alberta T2G 1B1 403.470.0141 neil.camarta@fshd.ca