Development of new compounds to help target faulty SOD1 protein as a potential treatment of MND

Identifying and developing compounds which interact with and help remove toxic SOD1 proteins in MND.

Principal Investigator: Professor Samar Hasnain

Lead Institution: University of Liverpool

MND Association Funding: £282,191

Funding dates: October 2023 - September 2026

About the project

Mutations in the SOD1 gene have long been linked with MND and are the cause of around 2% of total cases of MND. The mutation causes the SOD1 protein to misfold and clump together in toxic aggregates within neurons, which contributes to their death. This project aims to identify and develop compounds which interact with two particular parts of the toxic SOD1 proteins and mark them for destruction by triggering the cell’s own protein breakdown process. These compounds only interact with toxic forms of the SOD1 protein so that any healthy SOD1 protein remains within the neurons to carry out it’s usual functions. The researchers will use high resolution microscopy to view the compounds interacting with toxic SOD1 and observe whether they are able to reduce the build-up of toxic SOD1 within cells.

What could this mean for MND research?

This project could help to discover and develop compounds that could be used as possible therapies to reduce toxic SOD1 in neurons. It may lead to identifying promising new treatments that can be further tested and developed for future testing in people with MND in clinical trials.

Project code: 897-791