Metformin

What is the drug thought to do in the body?

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 changes in the C9orf72 gene. In models of MND, metformin was able to reduce the number of toxic proteins that are made due to the C9orf72 gene change. Metformin works by blocking a key pathway (protein kinase R pathway) that is essential to the production of these toxic proteins.

Phase 2

Current status: Collecting data

Primary objective: To investigate the safety of metformin 

As metformin is already known to be a safe and well tolerated drug, a phase 1 study was not needed. The phase 2 study is investigating the safety, tolerability and efficacy of metformin in 18 people with C9orf72 MND over 24 weeks. This is an open-label trial, which means it is not being compared to a placebo group. You can read more about the trial here.

This trial is recruiting in the US only.

Last updated: 21/08/2024