- EXPERTS-ALS is a 'pre-trial' platform to screen candidate drugs in people living with the most common form of Motor Neuron Disease faster and with a smaller sample size than in traditional clinical trials
- The first two drugs have been tested on the platform, with researchers saying the findings could transform the way treatments are tested
- Traditional trials see participants take a drug or placebo for at least 12 months before researchers can determine whether it is effective
- Rather than immediately committing hundreds of people to a trial lasting a year or more, EXPERT-ALS looks for an indication that a drug may be slowing the progression of the disease, potentially saving years of fruitless research
A gamechanging trial design could mean faster results in the race to find effective treatments for Motor Neuron Disease.
The first EXPERTS-ALS study has shown that promising drugs can be trialled on patients with Amyotrophic Lateral Sclerosis (ALS), the most common form of Motor Neuron Disease, quickly and at a fraction of the size and cost of conventional drug trials.
A University of Sheffield researcher says the findings could transform the way ALS treatments are tested, allowing ineffective drugs to be rejected sooner while treatments that have shown more efficacy are fast tracked to larger trials.
EXPERTS-ALS is a 'pre-trial' platform to screen candidate drugs in people living with ALS, looking for early signals of disease-slowing such as lowering of the blood levels of a protein called neurofilament light chain (NFL), a biomarker associated with nerve cell damage.
The first drugs tested on the platform, metformin and nifedipine, were not able to lower NFL levels.
Around 30 participants were involved in the trial, each taking one of the drugs for approximately 18-24 weeks, despite the small number of participants and some dropouts there were no overall changes in NFL levels.
While this sounds disappointing, the result is still important because it demonstrates that EXPERT-ALS can rapidly tell the difference between promising and ineffective drugs, which could save years of painstaking research, only for drugs to be found ineffective. For people living with ALS time is of the essence.
The platform can work quickly with a relatively small sample size thanks to a revolutionary statistical approach called Bayesian modelling, pioneered at the University of Oxford.
Professor Chris McDermott, Professor of Translational Neurology, at the University of Sheffield's School of Medicine and Population Health and one of EXPERT-ALS' Chief Investigator's, said: "We share the disappointment that neither metformin or nifedipine were able to lower neurofilament light chain levels in EXPERTS-ALS.
"However, EXPERTS-ALS is the first platform of its kind and this result, at this stage of the study, has exceeded our expectations of how it might perform.
"These results mark a very important advance in speeding up clinical trials that improves the prospects for finding effective drugs for ALS."
This vital work comes from a major collaboration between centres across the country, coordinated by the Sheffield Clinical Trials Unit, Sheffield Teaching Hospitals NHS Foundation Trust, and the Sheffield Biomedical Research Centre - part of the National Institute for Health and Care Research (NIHR).
The Project was funded by the NIHR, with support from the Motor Neurone Disease Association, My Name's Doddie Foundation, Life Arc and the Darby Rimmer Foundation.
