Safety & therapeutic potential of metformin for C9orf72 ALS
Description: A repeat expansion in the C9orf72 gene is the most common cause of familial and sporadic amyotrophic lateral sclerosis (ALS). While there are no current effective treatment strategies for ALS, reducing the levels of toxic proteins produced from these repeat expansions has been shown to offer improvements in a number of pre-clinical models. The Ranum lab has shown that metformin, a widely used and well-tolerated type-2 diabetes drug inhibits a key pathway important for the production of these toxic proteins. Treatment of a C9-ALS mouse model with metformin decreased toxic protein levels, improved behavior, and increased motor neuron survival. This proposal seeks to complete a small-scale human phase II clinical trial (NCT04220021) to assess the safety and potential efficacy of metformin for the treatment of C9-ALS. The results of this trial will be analyzed and if successful, will enable the transition to a large multi-center follow-up study. This project has the potential to rapidly move a well-tolerated FDA approved drug into the clinic as a safe, low-cost treatment strategy for the most common genetic cause of both ALS and FTD.
Funder: ALS Association
Amount: $500,000
Funding type: award
Start date: 2022-12-01
End date: 2024-11-30
Investigators: Laura Ranum, Stephanie Gray, Tiffany Schmidt, James Wymer, Emily Plowman, Connie Philebaum, Deborah Morrison, John Cleary
Institutions: University of Florida, FL - 109/109 - University of Florida
Subfield: Neurology
Field: Medicine
Domain: Health Sciences
Funded outputs count: 0
Funder award ID: 23-TC-644
OpenAlex ID: G2875050848