Motor neuron disease (MND) Research

Targeting TDP-43

Pre-clinical

Assessing the dosing and effectiveness of novel compounds targeting TDP-43 pathology

Professor Mark Baker, Newcastle University

£166,666.67* | July 2023 - June 2026

Summary: Approximately 97% of people living with MND exhibit the toxic build-up and aggregation of a protein called TDP-43 in their motor neurons. This pathology has been shown to contribute to the death of motor neurons via several mechanisms including the trigger of toxic pathways and the spread of toxic clumps of TDP-43 to otherwise healthy motor neurons. Professor Baker is investigating two groups of compounds that have shown promise in reducing TDP-43 toxicity in cellular models of disease, and is now testing them in animal models of disease.

Why this research is important: TDP-43 pathology is a major hallmark of MND. If high quality pre-clinical data from animal models of disease suggests that these new compounds are safe and effective, it would position them for human clinical trials. If successful, these treatments could potentially be used to treat the majority of people living with MND.

To find out more about TDP-43, check out our infographic.

*This project is part of the Motor Neuron Disease Translational Research Fund and is co-funded by LifeArc and MND Association.

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