MIROCALS_H2020/PHRC-N/2014/GB-01_ID-IL-2_Neuro-Inflammation in ALS Pts
Efficacy and safety of low-dose IL-2 (ld-IL-2) as a Treg enhancer for controlling neuro-inflammation in newly diagnosed Amyotrophic Lateral Sclerosis (ALS) patients: A randomized, double-blind, placebo- controlled, phase-II Proof of Concept/ Proof of Mechanism Clinical Trial
Peter Nigel Leigh
CHU DE NIMES
PHRC-National 2014 grant, N°14-0077, 2014; The Motor Neurone Disease Association grant, N° 971-797
Duration of Study in the UK
2 years, 11 months, 30 days
Amyotrophic Lateral Sclerosis (ALS) is a devastating, disabling and rare disease which progressively breaks down and damages the nerve cells in the brain and spine. It affects approx 50,000 people in the EU at any time and approx 10,000 deaths each year. Some interventions are provided to manage the symptoms of this disease, but care is largely supportive and palliative. The cost to society and to individuals is significant; much of it is health care costs. There is a pressing need to find disease-modifying therapies that will slow disease progression, increase survival and better quality of life.
Riluzole is still the only agent shown to modify survival in ALS but its affects are modest. This tells us that modifying disease progression is a realistic goal in ALS.
Research has shown neuroinflammation significantly contributes to neuronal damage. It is thought if this neuroinflammation can be modified this targeted therapy would be very effective in ALS patients. Previous therapeutic attempts targeted the suppression of all immuno-inflammatory cells, but the risks of toxicity outweighed the benefits of drug effect.
Targeting specific regulatory T-cells called Tregs has shown to reduce neuroinflammation more safely and effectively and low dose IL-2 (ld-IL2) acts through these Tregs.
This study is a randomised, double-blind, placebo-controlled, parallel group, stratified study (country (UK vs France) and site of disease onset (limb vs bulbar)). Bulbar region is lower part of the skull. It investigates the efficacy & safety of low-dose IL2 therapy for 18 months as an add-on therapy with riluzole, and compares the efficacy and safety to riluzole plus placebo in recently diagnosed ALS patients.
The study will take place in 5 Hospitals in England (and 5 Hospitals in France) and will run for 36 months. They hope to recuit 72 patients in the UK from October 2016 (216 patients in total across France and UK).
London - Central Research Ethics Committee
Date of REC Opinion
25 Aug 2016
Further Information Favourable Opinion