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Ambitious research project trials promising new therapy for motor neurone disease

Researchers at King's College London have been awarded a grant as part of an ambitious European research project aimed at finding a new treatment for motor neurone disease (MND), which is testing a molecule that occurs naturally in our bodies and helps to regulate our immune system. 

The project, Modifying Immune Response and Outcomes in Amyotrophic Lateral Sclerosis (MIROCALS), is being headed up Dr Gilbert Bensimon, University Hospital, Nimes, France with the clinical trials being led by Professor Nigel Leigh at Brighton and Sussex Medical School. 

In May, MIROCALS was awarded €5.98 million by the European Commission Directorate-General for Research and Innovation, under the EU Horizon 2020 Scheme. Additional support for the Clinical Centres has been awarded by the French Health Ministry Programme de Recherche Clinique (PHRC) in France and is under consideration by the MND Association in the UK. 

Currently, a low dose of the molecule, interleukin-2 (IL-2), is being developed for the treatment of conditions affecting the immune system, including diabetes, arthritis, liver disease, and the complications of treating leukaemia and other cancers with stem cells. While IL-2 has been used for many years at high dose to treat cancer, it is much safer – but still effective – when used at low doses in these immune disorders, as it can damp down harmful immune responses. 

“Our main objective is to achieve a breakthrough in the treatment of MND by significantly slowing the progress of the disease through a low dose of IL-2,” says Dr Gilbert Bensimon, University Hospital, Nimes, France, who is project leader of Modifying Immune Response and Outcomes in Amyotrophic Lateral Sclerosis (MIROCALS). 

To date, only one drug – riluzole – has been shown to slow the advance of MND, but its impact on the quality of life of people with the illness is marginal. Many other drugs have been tested but have failed. 

Professor Nigel Leigh, co-lead and chief investigator for the clinical trial, of Brighton and Sussex Medical School, says: “We are delighted to be collaborating with world-leading research groups in biomarker development, immunology, genetics and gene expression on this project. This collaboration will allow us to research a number of factors that may affect MND. Taken together, these analyses should allow us to ‘individualise’ responses to treatment that may be revealed during the study.” 

Dr Tim Tree, the Programme of Infection & Immunity at King's College London and is involved in the MIROCALS project said 

"Immunological and genetic studies at King's will help us understand why, or in whom, IL-2 treatment was able to slow down disease progression in patients. By identifying biomarkers of disease progression and response to treatment, these studies offer the potential to personalise therapies and may even reveal new targets which can be exploited to stop this this devastating disease". 

Project planning will start in September this year and researchers intend to recruit the first patients into the trial by September 2016. They aim to complete the study in 2019. In the meantime, the team is working on the essential groundwork for MIROCALS, including a small pilot study in France.