New programme to move potential MND drugs into clinical trials faster

New programme to move potential MND drugs into clinical trials faster

A ground-breaking experimental medicine programme that will enable researchers to more rapidly screen potential drugs in people with motor neuron disease (MND, also known as ALS), is announced today on Global MND 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 MND Research Institute (UK MND RI). EXPERTS-ALS is led by Professor Martin Turner at the University of Oxford and UK MND RI co-director Professor Chris McDermott at the University of Sheffield. It involves 11 MND 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 MND researchers.

MND 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 MND 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 MND having only modest effects.

Professor Christopher McDermott, co-lead of EXPERTS-ALS, co-director of the new UK MND Research Institute, Professor of Translational Neurology at the University of Sheffield and Honorary Consultant Neurologist at Sheffield Teaching Hospitals NHS Foundation Trust, said: "MND 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 MND."

Potential drugs for MND 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 MND. 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 MND-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 MND research. Each centre will bring its expertise to help us learn from every person with MND that participates in the programme, to increase our understanding of the causes of MND 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 MND who led the United2EndMND campaign, the UK MND Charities and wider UK MND 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 MND.”

Professor Ammar Al-Chalabi, co-Director of the UK MND 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 MND treatments in people and is a superb example of how scientists from different institutions working together in the new UK MND 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 MND RI is planning to deliver.”

Lee Millard, who is living with MND and is also a co-applicant on the grant said: “EXPERTS-ALS will help us find drugs that could help people with MND faster than ever before. Time is one commodity MND 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 MND trials."

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 MND Association, My Name’5 Doddie Foundation, MND Scotland, and medical research charity LifeArc intend to provide additional support to extend the study to 5 years and support additional lab research.

If you are a patient and want to find out more about the study you can do so by clicking here. 

About the UK MND Research Institute

The UK Motor Neuron Disease Institute (UK MND RI) is a national network of MND centres accelerating the search for a cure for MND. Researchers across the UK are carrying out world-leading MND research in a coordinated way. The UK MND RI is funded by grants from the MND Association, My Name’5 Doddie Foundation, MND Scotland, LifeArc, Medical Research Council and National Institute for Health and Care Research. It is co-led by Professor Ammar Al-Chalabi of King’s College London and Professor Chris McDermott of the University of Sheffield.

About the University of Oxford’s Nuffield Department of Clinical Neurosciences

The University of Oxford’s Nuffield Department of Clinical Neurosciences (NDCN) is a leading centre for research and treatment in disorders of the nervous system. Its laboratories will house and run the state-of-the-art analysis equipment for EXPERTS-ALS. Leading trial designers in Oxford Univresity’s Nuffield Department of Population Health are providing the statistical modelling expertise.

About the University of Sheffield

The University of Sheffield is a leading Russell Group university, with a world-class reputation. Over 30,000 students from 150 countries study at Sheffield. In a truly global community, they learn alongside over 1,500 of the world’s leading academics.

Sheffield’s world-shaping research feeds into its excellent education. Students learn at the leading edge of discovery from researchers who are tackling today’s biggest global challenges. 

Driven by outstanding people, staff and students share a commitment to changing the world for the better, through the power and application of ideas and knowledge.

From the first documented use of penicillin as a therapy in 1930, to building Europe’s largest research-led manufacturing cluster, Sheffield’s inventive spirit and top quality research environment sets it apart. 

Current research partners include Boeing, Rolls-Royce, Unilever, AstraZeneca, GlaxoSmithKline, Siemens and Airbus, as well as many government agencies and charitable foundations. 

Sheffield’s Students’ Union has won the Whatuni Student Choice Award for Best Students’ Union for five consecutive years. Students can choose from 350 societies and clubs, or join over 2,000 volunteers. 

Over 300,000 Sheffield alumni from 205 different countries make a significant influence across the world, with six Nobel Prize winners included amongst former staff and students.

The Sheffield Clinical Trials Research Unit (CTRU) is CTRU is a UKCRC Registered Clinical Trials Unit providing high quality support in running clinical trials in line with international regulatory standards, from the planning stages right through to analysis and reporting.

About the Sheffield Teaching Hospitals NHS Foundation Trust

Sheffield Teaching Hospitals is one of the UK’s largest NHS Foundation Trusts and one of the largest and busiest teaching hospitals. We have over 18,500 staff caring for over two million patients each year at our five hospitals and in the local community:

·         The Royal Hallamshire Hospital

·         The Northern General Hospital

·         Charles Clifford Dental Hospital

·         Weston Park Cancer Hospital

·         Jessop Wing Maternity Hospital

·         Over 40 community health care hubs

We offer a full range of local hospital and community health services for people in Sheffield as well as specialist hospital services to patients from further afield in our many specialist centres.

The Trust is a recognised leader in medical research for bone, cardiac, endocrine, gastrointestinal, neurosciences and long-term conditions such as diabetes and lung disease. We also play a key role in the training and education of medical, nursing and dental students with our academic partners, including the University of Sheffield and Sheffield Hallam.

The Trust hosts a number of national projects including Devices for Dignity as well as being a partner in the South Yorkshire and Bassetlaw Integrated Care System and the National Centre for Sports and Exercise Medicine.