Professor Nigel Leigh from BSMS

A group of researchers led by a professor at Brighton and Sussex Medical School, and backed by the University of Sussex, has signed an exclusive licence agreement with ILTOO Pharma in Paris, France to enable them to develop a potential treatment for amyotrophic lateral sclerosis (ALS).

ALS is the most common type of Motor Neurone Disease. MND affects up to 5000 people in the UK at any time and is a progressive disease for which there is no cure. The prospect of a treatment which could extend and improve patients’ lives could offer a ray of hope for many people.

The group of researchers is called the MIROCALS consortium (Modifying Immune Responses and Outcomes in ALS). The worldwide licensing agreement means ILTOO Pharma can use the data from the research to develop low dose interleukin-2 (IL-2) as a treatment.

IL-2 is a molecule that helps to regulate the human immune system. It is hoped that this project will contribute to enhancing quality of life and care for people with ALS and provide a robust model for other industry partners to encourage investment in ALS and other neurodegenerative diseases.

Professor Nigel Leigh, Chief Investigator and co-coordinator of the study, and Professor of Neurology at Brighton and Sussex Medical School, a joint partnership between the Universities of Sussex and Brighton, said: “Without the altruism and commitment of all the people with ALS who volunteered to join the trial, MIROCALS could not have succeeded. We must now do our best to translate our findings into benefit for all people with ALS, for which collaboration with ILTOO Pharma is essential.

"The study incorporates a novel approach to clinical trials in ALS/MND, integrating blood and spinal fluid markers of disease activity and progression with clinical measures approved by Regulatory agencies such as the MHRA,EMA, and FDA. The MIROCALS Consortium has brought together clinicians and scientists from four European countries in a mutual commitment to find a new treatment for this devastating disese. We also have a unique biobank and clinical databse to explore new avenues of research. We are confident that ILTOO will now do its best to develop low dose IL2 as a potential treatment for ALS/MND.

“The real heroes of this story are, of course, all the altruistic people with MND who volunteered to take part knowing they might be allocated to placebo, and that they were committing to three lumbar punctures, many blood tests and frequent visits to hospital. In the end, we hope that low dose IL2 will become a new treatment for ALS.”

Dr Gilbert Bensimon, Project Coordinator, from the Centre Hospitalier Universitaire de Nîmes, said: “We believe our trial results show that IL-2 treatment has the potential to positively impact the health of people affected by this devastating condition. We welcome this agreement as a critical step toward making this promising therapy accessible for the whole ALS community.”

The University of Sussex is one of the major stakeholders in the MIROCALS Consortium, with the Research and Enterprise Services at the university supporting the study, along with colleagues from the Innovation and Business Partnerships team.

The results of the MIROCALS Trial were presented at the 33rd International Symposium on ALS/MND in 2022 by Dr Bensimon and Professor Leigh. The study started in 2017 with 220 volunteers with MND taking part in a randomised double-blind trial comparing a low dose of the molecule in IL2 against a dummy (placebo) injection. People with ALS were recruited into MIROCALS in 17 ALS Centres; seven in the UK and 10 in France.

The MIROCALS study was funded by The European Commission H2020 programme, the Motor Neurone Disease Association (MNDA) and the French Health Ministry with significant contributions from The MyName’5 Doddie Foundation and ALS-related medical charities in France.

Find out more about MIROCALS:
www.mirocals.eu/
www.mndassociation.org/research/clinical-trials/mirocals