New programme to move potential MND drugs into clinical trials faster

A ground-breaking experimental medicine programme that will enable researchers to more rapidly screen potential drugs in people with motor neuron disease (MND), has been announced today on Global MND Awareness Day.

The ‘EXPErimental medicine Route To Success’ or ‘EXPERTS-ALS’ programme will screen candidate drugs at a scale not seen before, more quickly identifying drugs that should be tested in larger clinical trials. Researchers hope eligible patients can begin taking part in the study in summer 2024.

This is a flagship programme of the new UK MND Research Institute (UK MND RI), which has been established following the successful ‘United to End MND’ campaign, organised by a coalition of MND charities across the UK, including MND Scotland.

The campaign called on the UK Government to commit to investing £50 million into targeted MND research. Today’s £8 million announcement is a result of the first tranche of funding to come from that commitment.

EXPERTS-ALS is led by Professor Martin Turner at the University of Oxford and UK MND RI co-director Professor Chris McDermott at the University of Sheffield. It involves 11 MND centres across the UK, including Scotland, and is being sponsored by Sheffield Teaching Hospitals NHS Foundation Trust. The programme will also help to develop the next generation of MND researchers.

Dr Jane Haley, Director of Research for MND Scotland, said: “EXPERTS is an exciting new initiative that will help prioritise potential new drugs, on the basis of their effect in people, for taking into full phase three clinical trials, such as the MND-SMART trial that MND Scotland committed a further £2 million funding to this week.

“As the UK government starts to release the £50 million they have committed to MND research, this is a great example of how researchers across the UK can work in an integrated manner to accelerate the search for better treatments for people with MND.”

Professor Christopher McDermott, co-lead of EXPERTS-ALS, co-director of the new UK MND Research Institute, Professor of Translational Neurology at the University of Sheffield and Honorary Consultant Neurologist at Sheffield Teaching Hospitals NHS Foundation Trust, said: “MND is a cruel and devastating disease and we need new approaches to identify more effective treatments to help patients.

“EXPERTS-ALS is a pioneering project to prioritise the drugs which have the best chance of success in halting the progression of this terrible degenerative disease. Over five years, we will be able to screen drugs faster, on a larger scale and identify which ones should proceed into phase 3 trials based on signals found in people living with MND.”

The EXPERTS-ALS programme will screen drugs in patients, looking for early signals of benefit found in blood tests such as lower levels of a protein called neurofilament light (NFL) which can indicate how advanced the condition is. A ‘go’ or ‘no-go’ decision can be reached within a few months and successful drugs prioritised for testing in the larger phase 3 trials with a higher chance of a positive outcome.

The UK Government’s Department for Health and Social Care (DHSC), through the National Institute for Health and Care Research (NIHCR), has awarded £8 million to the project, which will fund the project for 3.5 years.

MND Scotland will continue working with charity partners across the UK to ensure that the remaining funding committed by the UK Government is delivered for targeted MND research.