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New therapy for motor neurone disease

BSMS > About BSMS > News > 2015 > Ambitious research project trials promising new therapy for motor neurone disease

Ambitious research project trials promising new therapy for motor neurone disease

An ambitious European research project is testing a molecule that could help slow the progression of motor neurone disease (MND).

The Modifying Immune Response and Outcomes in Amyotrophic Lateral Sclerosis (MIROCALS) project, which is co-lead by Prof Nigel Leigh, aims to achieve a breakthrough in the treatment of MND through a low dose of a molecule called interleukin-2 (IL-2).

IL-2 occurs naturally in our bodies and helps to regulate our immune system. Low doses of IL-2 increases the number of immune cells known as Regulatory T-cells ('Tregs') in the body, and previous research has shown that there is a strong relationship between the number of these cells and the progression of MND.

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.

Prof Nigel Leigh, who is also chief investigator for the clinical trial, said: "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."

The research is being undertaken by a partnership between leading medical researchers in France, UK, Italy, and Sweden, the Motor Neurone Disease Association of England Wales and Northern Ireland, and the pharmaceutical industry.

"Our main aim 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 MIROCALS. Currently, a low dose of the IL-2 is being developed for the treatment of a number of conditions affecting the immune system, including diabetes, arthritis, liver disease, and the complications of treating leukaemia and other cancers with stem cells.

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.

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.

Prof Nigel Leigh, together with Dr Majid Hafezparast and Dr Sarah Newbury, was recently awarded a £120,405 grant from the Motor Neurone Disease Association for a separate study. 'Identification of non-coding RNA biomarkers from serum and cerebrospinal fluid for use as biomarkers in Amyotrophic Lateral Sclerosis' aims to identify markers in the progression of MND, in order to help earlier diagnosis and identify responses to potential new treatments.