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Investigating metformin as a possible treatment for facioscapulohumeral muscular dystrophy (FSHD)

Professor Peter Zammit and his team are studying how the drug metformin affects muscle cells in people with FSHD, to investigate whether it could be used as a treatment.
Details
Principal Investigator
Professor Peter Zammit
Institute
Kings College London
Official title
Examining the function of metformin in skeletal muscle differentiation and muscular dystrophy
Duration
Two years
Total cost
£175,744
Conditions
Facioscapulohumeral muscular dystrophy (FSHD)
Year
2026

Background

Facioscapulohumeral muscular dystrophy (FSHD) is one of the most common inherited muscle conditions in adults. It causes muscles to become weaker over time, making everyday activities such as lifting the arms, climbing stairs and walking more difficult. Many people also experience pain, fatigue and a loss of independence. Although researchers know the genetic cause of FSHD, there are currently no treatments that can slow down or stop it. 

Recent research suggests that muscle cells in people with FSHD may struggle to produce and use energy efficiently and to repair themselves after damage. This may contribute to the gradual loss of muscle strength. 

The research team has found early evidence that metformin, a medicine that has been used to treat type 2 diabetes, may improve the growth and health of muscle cells, including cells from people with FSHD. This needs to be better understood to see if it could potentially be developed as a treatment for FSHD. 

Project aims

This project aims to investigate whether metformin could become a future treatment for FSHD. 

The researchers will study how metformin affects muscle cells from people with FSHD and will compare them with healthy muscle cells. They will investigate whether the drug helps muscle cells grow, produce energy more effectively and repair themselves after damage. 

This will provide important evidence about how metformin works in muscle and whether it has the potential to slow down muscle damage in FSHD. 

Why is this research important?

There are currently no treatments that slow the progression of FSHD, creating an urgent need for new therapies. 

Metformin is already being used by people as treatment for diabetes. This means it may be possible to test it in clinical trials for FSHD much sooner than a completely new medicine. This research will provide the evidence needed to determine whether metformin has the potential to become a treatment for FSHD. If successful, it could lead to new ways of preserving muscle strength, maintaining independence for longer and improving quality of life for people living with the FSHD. 

This research is investigating whether metformin could be a potential treatment for FSHD. At this stage, it is not known whether metformin is safe or effective for people living with FSHD, and we do not recommend that anyone takes or seeks out this medication to treat their condition. 

Even if the results of this research are promising, further clinical trials will be needed to test whether metformin is safe and effective in people with FSHD before it could be considered as a treatment. 

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