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A ground-breaking experimental medicine programme that will enable researchers to more rapidly screen potential drugs in people with motor neuron disease (51��ݶ�, also known as ALS), is announced today on Global 51��ݶ� Awareness Day.

The EXPErimental medicine Route To Success, or EXPERTS-ALS programme will screen candidate drugs at a scale not seen before, identifying those that should be tested in larger clinical trials faster. Researchers hope eligible patients can begin taking part in the study in summer 2024.

This is a flagship programme of the new UK 51��ݶ� Research Institute (UK 51��ݶ� RI). EXPERTS-ALS is led by Professor Chris McDermott at the University of Sheffield and Professor Martin Turner at the University of Oxford and UK 51��ݶ� RI co-director. It involves 11 51��ݶ� centres around the UK and is being sponsored by Sheffield Teaching Hospitals NHS Foundation Trust. The programme will also help to develop the next generation of 51��ݶ� researchers.

51��ݶ� is a neurodegenerative disease affecting the nerve cells in the brain and spinal cord. People progressively lose voluntary movement and need complex care. Around half of those diagnosed with 51��ݶ� will die within two years. The condition affects around 330,000 people around the world and there are currently no treatments to cure it, with the few licensed drugs for 51��ݶ� having only modest effects.

Professor Christopher McDermott, co-lead of EXPERTS-ALS, co-director of the new UK 51��ݶ� Research Institute, Professor of Translational Neurology at the University of Sheffield and Honorary Consultant Neurologist at 51��ݶ� Teaching Hospitals NHS Foundation Trust, said: "51��ݶ� is a cruel and devastating disease and we need new approaches to identify more effective treatments to help patients. EXPERTS-ALS is a pioneering project to prioritise the drugs which have the best chance of success in halting the progression of this terrible degenerative disease. Over five years, we will be able to screen drugs faster, on a larger scale and identify which ones should proceed into phase 3 trials based on signals found in people living with 51��ݶ�."

Potential drugs for 51��ݶ� have to be tested in phase 3 clinical trials and this necessarily involves a placebo group to show if they independently benefit patients. Their success rate has been very low so far. This is partly because the drugs put forward for testing have often been chosen largely on data from laboratory studies, rather than from people living with 51��ݶ�. The EXPERTS-ALS programme will screen drugs in patients, looking for early signals of benefit found in blood tests such as lower levels of a protein called neurofilament light (NFL) which can indicate how advanced the condition is. A ‘go’ or ‘no-go’ decision can be reached within a few months and successful drugs prioritised for testing in the larger phase 3 trials, including the innovative 51��ݶ�-SMART platform, with a higher chance of a positive outcome.

Professor Martin Turner, co-lead of EXPERTS-ALS and Professor of Clinical Neurology & Neuroscience at the University of Oxford added: "The EXPERTS-ALS programme brings together leading scientists from the UK’s National Institute for Health & Care Research (NIHR) network of Biomedical Research Centres (BRCs), Dementia Research Institute and other centres of 51��ݶ� research. Each centre will bring its expertise to help us learn from every person with 51��ݶ� that participates in the programme, to increase our understanding of the causes of 51��ݶ� and speed up the process of finding effective treatments.”

“This innovative project to select drugs with the most potential is only possible because of the vision of those who are living with 51��ݶ� who led the United2End51��ݶ� campaign, the UK 51��ݶ� Charities and wider UK 51��ݶ� Research Institute partners and funders. We are grateful to the NIHR and Department of Health & Social Care (DHSC) in helping us to deliver an experimental medicine platform in 51��ݶ�.”

Professor Ammar Al-Chalabi, co-Director of the UK 51��ݶ� Research Institute and Professor of Neurology and Complex Disease Genetics at King’s College London, said: “EXPERTS-ALS is a groundbreaking programme to rapidly screen potential 51��ݶ� treatments in people and is a superb example of how scientists from different institutions working together in the new UK 51��ݶ� Research Institute can accelerate the search for a cure. We are looking forward to the release of the remaining funds from the £50 million committed by DHSC and the Department of Science, Innovation & Technology (DSIT) for the other research that the UK 51��ݶ� RI is planning to deliver.”

Lee Millard, who is living with 51��ݶ� and is also a co-applicant on the grant said: “EXPERTS-ALS will help us find drugs that could help people with 51��ݶ� faster than ever before. Time is one commodity 51��ݶ� patients don’t have much of. Because the platform will only select the drugs with the best chance of success to go on to larger trials, patients will spend more time on trials that are likely to help them. I am so excited by the prospect of this truly amazing science and delighted that the UK is leading the world in improving 51��ݶ� trials."

Professor Cindy Cooper, Director of Sheffield Clinical Trials Research Unit, said: “The Sheffield Clinical Trials Research Unit has a longstanding tradition in supporting high quality clinical trials in line with international regulatory standards, from the planning stages right through to analysis and reporting. We are delighted the early stages of funding for this groundbreaking platform have been announced, and look forward to working with our partners to deliver the programme for the benefit of patients.”

The DHSC through the NIHR has awarded £8 million to the project, which will fund the project for 3.5 years (subject to contract signing). Patient charities 51��ݶ� Association, My Name’5 Doddie Foundation, 51��ݶ� Scotland, and medical research charity LifeArc intend to provide additional support to extend the study to 5 years and support additional lab research.