The EXPErimental medicine Route To Success in ALS (EXPERTS-ALS) Investigators are seeking partnerships with ALS academics and Industry around the world.


EXPERTS-ALS is a drug de-risking and prioritisation platform designed to more quickly identify therapeutic candidates with human biomarker signals of likely clinical benefit.


EXPERTS-ALS is a flagship initiative of the UK Motor Neuron Disease Research Institute, and is funded by the National Institute for Health and Care Research (NIHR) with generous financial support from major UK MND charities. The programme brings together major UK centres of MND translational research excellence (NIHR Biomedical Research Centres and Dementia Research Institute Centres).


There are three workstreams:


EXPERTS-ALS uses a cutting-edge Bayesian modelling of serum neurofilament light chain (NFL), an established surrogate marker of disease progression rate in ALS. Our model allows IMP to be given on an open-label basis to an expected group size of 30-850 people with ALS in each one of three drug arms running concurrently at any time. NFL is measured at the start and at regular intervals to a maximum of 6 months for any individual participant, with a continuous Bayesian modelling to determine the study endpoints, and an outcome expected within 6-9 months per IMP.


Within our model, we consider a reduction in mean NFL of 30% to indicate a drug likely to have a clinically meaningful disease-slowing benefit in ALS, to be prioritised for definitive Phase 3 placebo-controlled study. If NFL levels do not change significantly in the model, we conclude that this reflects a much smaller (or no) disease-slowing effect and declare a ‘futility’ outcome, with a correspondingly lower priority status. Our independent Data & Safety Monitoring Committee will recommend stopping any IMP showing a consistent group mean rise in NFL levels from baseline.


A range of nested biomarker development studies are funded across the recruiting centres, using the clinically well-characterised participants and with a range of biofluid samples and some neuroimaging data being routinely collected. The platform will generate a powerful longitudinal dataset for biomarker development beyond NFL, and the possibility to focus on the molecular basis for any apparent ‘responder’ and ‘non-responder’ individuals within each IMP group.


The platform is fully funded until 2028. The initial IMPs are repurposed licensed drugs, selected by expert panel consensus based on preclinical data. We now wish to begin to select IMP for subsequent inclusion on the EXPERTS-ALS platform. We are interested in hearing from potential academic and industry partners around the world who are interested in potentially testing their IMPs in the EXPERTS platform. We envisage collaborations will take on different forms and we will be flexible. If you are interested in discussing further, please use the contact details below.


If you are interested in becoming a partner, please get in touch by visiting our contact page