What does the drug do?

Metformin is an existing drug which is currently used in the treatment of diabetes to control blood sugar levels. It is now being investigated as a potential treatment for MND in those who have the C9orf72 mutation. In mouse models of MND, metformin has been found to reduce the number of toxic proteins (repeat-associated non-AUG or RAN proteins) that are made due to the C9orf72 mutation, accumulate in brain cells and improve disease features. Metformin works by blocking a key pathway (protein kinase R pathway) that is essential to the production of the toxic proteins.

Phase 2

As metformin is already known to be a safe and well tolerated drug, a phase 1 study into the use for MND was not needed. A phase 2 study assessing the safety, tolerability and efficacy of the drug in those with MND is currently being conducted. This open label trial in the U.S. is recruiting 18 participants with C9orf72 associated MND who will receive treatment with metformin for 24 weeks. You can read more about the trial here.

This trial is recruiting in the US only.

Latest News


The trial opened for recruitment. 

Last updated: 21/12/2022