Motor neuron disease (MND) affects the brain and nerves, causing severe weakness, including of the breathing muscles. It is referred to in several countries outside the UK as amyotrophic lateral sclerosis or ALS, and this is also the scientific term used for it. It has a gradual onset and gets worse over time, paralysing the affected person and resulting in death often within three to five years. One in every 300 people will develop ALS.
There’s no cure for ALS and it significantly shortens life expectancy, but recent major advances in our understanding of the disease means there are now many potential targets for new treatments. A major barrier to developing new treatments for ALS is the capacity to deliver Phase III clinical trials for a growing pool of promising new drugs.
As part of multi-centre study, EXPERTS-ALS, our theme is helping to overcome this barrier, by ensuring that we design and deliver clinical trials that efficiently evaluate these new treatments and enable drugs with potential to become available to patients.
New approach to ALS trials
The multicentre study is taking place at Universities and BRCs across the UK, and is a component of the new UK MND Research Institute. It is taking a new approach, recruiting people with ALS to screen experimental drugs in a safe clinical environment, to identify which treatments are most likely to be successful.
The study is judging the biological effectiveness of a drug by its ability to lower levels of a protein in the blood called Neurofilament Light (NfL) which has become an established biomarker for ALS based on original research supported by NIHR Maudsley BRC and other studies.
Those drugs which reduce NfL are recommended for prioritisation in future studies, including Phase III trials.
EXPERTS-ALS is creating a bank with biological samples provided by each patient. It is building expertise across centres and clinical research networks, as well as capacity within the UK to conduct ALS research.
Our theme for ALS is drawing on expertise from our Experimental Medicine and Novel Therapeutics theme and our Trials, Genomics and Prediction theme and industry partnerships will be supported by our Centre for Innovative Therapeutics.
Collaborators
