| Principal Investigator: | Prof. Dame Pamela Shaw | |
| Co-Investigators: | Prof. Guillaume Hautebergue Prof. Mimoun Azzouz Dr Jonathon Foley Dr Amanda Heslegrave |
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| Lead Institution: | University of Sheffield | |
| Funding awarded: | £598,880 | |
| Duration | 18 months | |
| Completion date: | 31st August 2026 | |
About the project
ALS/MND is a progressive neurodegenerative condition where the motor neurons, enabling movement through our muscles, die, causing disability in walking, talking, eating, and breathing. Average life expectancy is 2-3 years from symptom onset. Treatments to slow the loss of motor neurons are urgently needed.
An expanded section of DNA in the C9orf72 gene causes ALS/MND in 10% of cases. The expansion varies in size and produces 5 different toxic proteins that injure motor neurons to varying degrees, though their relative impacts are incompletely understood. Crucible is developing a potential treatment that stops toxic protein production and has plans to evaluate this in clinical trials. Using samples donated by patients and clinical data, we aim to develop tests to reliably measure the toxic proteins as biomarkers to rapidly demonstrate treatment effects and reduced motor neuron injury, aiming to accelerate the development of new treatments for people with C9orf72-MND.
