UCL and UCLH have been awarded over £1million to look at how potential new treatments for motor neurone disease (MND) work in the body, and who responds to treatment.
It is part of a big national drive funded by the National Institute for Health and Care Research (NIHR) Efficacy and Mechanism Evaluation program and the UCLH Biomedical Research Centre (BRC) to tackle the debilitating disease.
There are currently no effective therapies for MND – also known as Amyotrophic Lateral Sclerosis (ALS). Patients face a rapidly progressing paralysis that spreads around the body. This robs them of the ability to walk, eat, talk and eventually breathe. Around half of those diagnosed with MND will die within two years. The condition affects around 330,000 people around the world.
In an effort to identify effective treatments for the condition, the NIHR and the UCLH BRC are investing £8million in a programme of research called EXPERT-ALS.
The research programme aims to identify and prioritise potential new treatments for MND and run clinical trials of potential new treatments.
As part of this programme of research, UCL will lead on work to understand precisely how new treatments work in the body, who responds to the treatments, and why treatments fail and on the capacity building for the training and clinical trial infrastructure supporting innovative research in ALS.
How the research will work
Around 700 people with MND will be recruited to take part in the EXPERT-ALS research from 11 centres in the UK. Over five years, between 9 and 12 potential new treatments will be trialled as part of the research.
Patients taking part will be assigned to one of three drugs. They will know what they are receiving and there is no placebo. They will take the drug for a maximum of 6 months, with the possibility of continuing the drug after the trial or, if preferred, of rejoining the trial with a different drug.
The prioritisation of a treatment after 6 months will be based on the effect on a blood biomarker, called neurofilament light chain, which has been developed and validated by many of the EXPERT Team over the years. Lead researcher at UCL Prof Andrea Malaspina has made a major contribution to the establishment of this biomarker, to its measurement and to its implementation in clinical studies.
The research team then aim to take forward the most promising drugs into a large phase 3 (advanced stage) trial.
The UCL component of the research
In work led by UCL, every patient who takes part in the research will provide additional biological samples which will enable the research team to create a bank of samples they can analyse. Further tests will be conducted to obtain a molecular signature in biofluids from individuals that appear to respond or not respond to the trialled medication. This will give the researchers clues on novel biomarkers and therapeutics for ALS and how to initially stratify patients based on the likelihood of response to a treatment.
Prof Malaspina, Professor of Neurology and the Clinical Academic Lead of the Queen Square Motor Neurone Disease Centre, Institute of Neurology, UCL, said: “This research will enable us to learn from every patient, understand why drugs are working and uncover new insights into ALS. It will increase capacity in the UK to carry out research in MND, and help us find effective treatments faster.”
In addition to the NIHR funding, patient charities MND Association, My Name’5 Doddie Foundation, MND Scotland, and medical research charity LifeArc intend to provide additional support.
Professor Lucy Chappell, NIHR Chief Executive, said: “Motor neurone disease is debilitating. This new funding demonstrates our commitment to helping the UK’s world-leading scientists to test promising new drugs, and confirms the Government’s commitment to MND research. Our investment will also help train and support the next generation of MND scientists to tackle this complex illness.
“Research is the best opportunity we have of finding new insights and ways to treat MND and improve the lives of everyone impacted by the disease.”
Image credit: Dr_Microbe / Adobe Stock
