The EXPErimental medicine Route To Success in ALS (EXPERTS-ALS) is designed to accelerate progress towards highly-effective drug therapies for amyotrophic lateral sclerosis (ALS, the commonest form of motor neurone disease, MND). The study will screen candidate drugs in those living with ALS, more quickly identifying those with signals of likely clinical benefit, that may then be prioritised for testing in larger placebo-controlled trials.
EXPERTS-ALS is led by Professor Chris McDermott at the University of Sheffield and Professor Martin Turner at the University of Oxford.
It involves 11 MND centres around the UK and is being sponsored by Sheffield Teaching Hospitals NHS Foundation Trust.
It is a flaghip study within the broader portfolio of the UK MND Research Institute.
Professor Christopher McDermott
University of Sheffield
"A new approach was needed to more rapidly identify effective treatments for those living with ALS. With a large team of leading researchers, we have designed EXPERTS-ALS to quickly identify human disease signals of likely clinical benefit from drugs given to people living with ALS. Over five years, we will be able to screen candidate drugs faster and on a larger scale, to prioritise those going on to the larger phase 3 clinical trials needed to prove long-term benefit. EXPERTS-ALS is a partnership with patients, funded by the UK's National Institute for Health & Care Research (NIHR), and several charities."
"EXPERTS-ALS brings together leading scientists from the NIHR network of Biomedical Research Centres (BRCs) & Dementia Research Institute (DRI). Each centre will bring its expertise to help us learn from every person with ALS that participates, to increase our understanding of the causes of ALS as well as speed up the process of finding effective treatments.”
Professor Martin Turner
University of Oxford
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). Dynamic Bayesian modelling allows a ‘go’ or ‘no-go’ decision to be reached within a few months in a small group of people. Successful drugs based on such human benefit signals can be prioritised for testing in larger phase 3 trials, with a higher chance of a positive outcome.
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