Professor Kevin Talbot, University of Oxford & Professor Tom Gillingwater, University of Edinburgh
£507,751.76 | August 2021 - March 2025
Summary: It has been suggested that diminished energy production within motor neurons is one reason that these cells die in people living with MND. Previous research funded by the Foundation and MND Scotland showed that a treatment called terazosin, that boosts energy production in cells, improved motor function and survival of animal models of MND, and protected against the death of motor neurons.
TRUST is an experimental medicine study, looking at the effects of terazosin in 50 people living with MND. Participants are being recruited in Oxford and biosamples are being analysed in Edinburgh to assess the neuroprotective effects of the treatment. Terazosin is already licensed for the treatment of hypertension and enlarged prostate in the UK so this is a “repurposing” study.
Why this research is important: Experimental medicine approaches enable researchers to demonstrate proof-of-concept that a treatment may have an effect, in a relatively short time period compared to larger clinical trials, and without the need for a placebo) group. If this study demonstrates that terazosin may have a protective effect on motor neurons, it can be rapidly prioritised for larger clinical trials to test the effectiveness of the treatment in slowing the progression of disease. The design of the TRUST study has been fundamental to providing the framework of the multi-million pound EXPERTS-ALS platform that is due to launch in 2024.
Results of the pre-clincial study that led to this research were published in EBioMedicine in 2022.
To find out more about terazosin, check out our infographic.