Taking a selection of drugs, known as “dual potency retinoids” through pre-clinical trials to determine their potential to treat MND.

Prof Peter McCaffery

Principal Investigator: Prof Peter McCaffery

Lead Institution: University of Aberdeen

MND Association Funding: £241,796

Funding dates: April 2022 - March 2025

About the project

Deficiency of the retinoic acid receptor (RAR) system, that occurs in the central nervous system, has been thought to be a signature of MND. A group of drugs have been developed called “dual potency retinoids” (RAR-Ms) which can help restore RAR function and have the potential to reverse abnormalities caused by SOD1 and C9orf72 mutations. This project will take selected RAR-Ms through pre-clinical trials (laboratory tests prior to testing in humans) to determine their potential to treat MND.

What could this mean for MND research?

Putting drugs through pre-clinical trials (laboratory tests prior to testing in humans) is a vital step on the way to the approval of a drug to treat MND. Putting this group of drugs through pre-clinical testing will allow the researchers to see which has the most promise to treat MND and could then be taken forward to test in humans.

Project code: 882-791

Animal research