Development of RAR class ligands for treatment of Motor Neuron Disease

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

Peter McCaffrey, SULSA Executive Committee.

Lead 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 does this mean for people living with MND?

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.

Resources 

Want to find out more about the project? Check out the resources below:

Podcasts:

Vitamin A, Brain Shrinkage and Depression | Interviews | Naked Scientists 

Video:

A new cure for neurodegenerative disease? 

Project code: 882-791