A new ‘pre-trial’ platform involving University of Sheffield researchers could dramatically speed up the search for treatments for the most common form of Motor Neuron Disease.
EXPERTS-ALS is designed to screen candidate drugs in people with Amyotrophic Lateral Sclerosis (ALS) faster, and with far fewer participants, than a traditional clinical trial, which typically requires participants to take a drug or placebo for at least 12 months before its effectiveness can be judged.
Instead, the platform looks for an early signal that a drug may be slowing disease progression, by tracking blood levels of a biomarker called neurofilament light chain (NFL), potentially saving years spent on drugs that don’t work.
The first two drugs tested on the platform, metformin and nifedipine, did not lower NFL levels in the roughly 30 participants involved, each of whom took one of the drugs for 18 to 24 weeks.
Fast-tracking efficacy
Despite the disappointing result for those two drugs, researchers say it demonstrates the platform itself works: it’s designed to rapidly tell the difference between promising and ineffective drugs, rejecting the latter sooner while fast-tracking anything showing more efficacy straight to larger trials. The approach relies on Bayesian modelling, a statistical method 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 the trial’s chief investigators, said: “These results mark a very important advance in speeding up clinical trials.”
The project was coordinated by the Sheffield Clinical Trials Unit, Sheffield Teaching Hospitals NHS Foundation Trust and the Sheffield Biomedical Research Centre, and funded by the National Institute for Health and Care Research, with support from the Motor Neurone Disease Association, My Name’s Doddie Foundation, LifeArc and the Darby Rimmer Foundation.
