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

*Supported by Robert Barr's Charitable Trust and Santa Barbara Heights Charitable Trust.

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