Candidate drug selected for testing on EXPERTS-ALS clinical platform trial

A promising compound, developed by the company Raya Therapeutic, has been selected to be tested in a platform trial, part-funded by My Name’5 Doddie Foundation. 

The chosen compound will be tested within EXPERTS-ALS, a platform that aims to accelerate the process of identifying promising treatments, by focusing on candidate drugs being tested at speed and at scale.

This candidate drug has shown encouraging results in laboratory models of amyotrophic lateral sclerosis (ALS), the most common type of motor neuron disease (MND). It has already been tested in a Phase 2 trial for another condition, and so its safety is well understood. 

The trial measures how effective compounds are by monitoring levels of a protein called neurofilament light chain (Nfl) over a three to six month period. If Nfl levels in the blood reduce, it suggests a slowing of neuron damage. When this happens, treatments will be prioritised to move from EXPERTS-ALS to larger Randomized Controlled Trials (RCTs).

More about EXPERTS-ALS

The EXPErimental medicine Route to Success in ALS (EXPERTS-ALS) platform is led by Professor Chris McDermott at the University of Sheffield and Professor Martin Turner at the University of Oxford. It involves 11 clinical centres around the UK and is sponsored by Sheffield Teaching Hospitals NHS Foundation Trust. 

It is a flagship study within the broader portfolio of the UK MND Research Institute and is funded by the National Institute for Health and Care Research (NIHR) and Biomedical Research Centre Capacity Building funding schemes. 

My Name’5 Doddie Foundation, MND Association, the Darby Rimmer Foundation, and LifeArc are providing additional support. You can read more about EXPERTS-ALS here, and watch a video with the study leads Professors Chris McDermott and Martin Turner, who explain the study here.