- The EXPErimental medicine Route To Success in ALS (EXPERTS-ALS) project aims to rapidly screen potential drugs for evidence that they can slow down damage to the nervous system occurring in people with motor neuron disease
- The results from the EXPERTS study will allow scientists to better prioritise which drugs should go forward for evaluation in larger placebo-controlled trials
- Although EXPERTS on its own cannot confirm long-term benefits of a drug, this approach provides a way to ensure only the most promising drugs proceed to larger, more intensive phase 3 trials
- Led by Professor Chris McDermott from the University of Sheffield and Professor Martin Turner from the University of Oxford, the study involves 11 MND centres across the UK
A groundbreaking drug screening programme is set to accelerate the development of new MND treatments.
The EXPErimental medicine Route To Success in ALS (EXPERTS-ALS) project is a pioneering study which aims to rapidly screen potential drugs for evidence that they can slow down damage to the nervous system occurring in people motor neuron disease, also known as Amyotrophic lateral sclerosis (ALS).
Led by Professor Chris McDermott from the University of Sheffield and Professor Martin Turner from the University of Oxford, the study involves 11 MND centres across the UK and is designed to continually test new drug candidates, with initial funding to cover the next three years.
Candidate drugs will have their potential effectiveness assessed by looking at the blood levels of a protein called neurofilament. Neurofilament levels rise with the emergence of symptoms in people with ALS and are expected to lower when effective disease-slowing therapy is given.
The results from the EXPERTS study will allow scientists to better prioritise which drugs should go forward for evaluation in larger placebo-controlled trials.
The first participant was recruited in November 2024 and is being assigned to one of the first three drugs under evaluation. EXPERTS-ALS is being sponsored by Sheffield Teaching Hospitals NHS Foundation Trust.
A new approach to ALS drug development
EXPERTS-ALS fills a previous gap in the way drugs come to trial, assessing the biological effect on a blood marker of a candidate treatment more quickly. This determines if a drug shows stronger promise of human benefit and so whether it should be prioritised for further study in a large trial.
Although EXPERTS cannot confirm long-term benefits of a drug on its own, this approach provides a way to ensure only the most promising drugs proceed to larger, more intensive phase 3 trials.
Professor Chris McDermott from the University of Sheffield, who is also co-director of the UK MND Research Institute, explained: “EXPERTS-ALS is a vital step forward in ALS drug development, enabling us to rapidly screen multiple candidate therapies and focus only on those showing real promise.
“This approach accelerates research and offers patients earlier opportunities to contribute to a drug screening programme that can help to shape future treatments for all those living with ALS. We’re very optimistic that this study will set a new standard in the care of those diagnosed with ALS, and help bring us closer to meaningful therapies more quickly.”
What this means for people with ALS
The launch of EXPERTS-ALS is an important milestone for ALS research in the UK, accelerating the speed with which drugs are screened to increase the likelihood that those drugs which reach the larger Phase 3 study have a stronger human evidence base and so stand a better chance of success.
Accelerating the drug screening process offers new hope for patients and families affected by this devastating disease.
Patient representative Lee Millard, co-founder of United2endMND, highlighted the study's importance: “EXPERTS-ALS represents a much-needed shift in ALS research, allowing us to focus on identifying up-and-coming treatments more efficiently.
“For too long, the process has been slowed by trials based on evidence that didn’t always translate into meaningful patient outcomes. This project gives me hope that we’ll see fewer but better phase 3 trials, prioritising quality over quantity and ensuring the most promising drugs have the best chance of success.”
To learn more about EXPERTS-ALS, visit its dedicated website or watch a video discussion with our study leads.