New advances in the treatment of ALS: Genethon’s DNA bank involved in the trial

Promising results from the European clinical trial MIROCALS (Modifying Immune Responses and Outcomes in ALS) have been published in The Lancet, opening up new prospects for the treatment of Amyotrophic Lateral Sclerosis (ALS)

This pioneering trial, sponsored by the University Hospital of Nîmes (France), and coordinated by Dr Gilbert Bensimon, University Hospital of Nîmes, Sorbonne University of Paris and Assistance Publique Hôpitaux de Paris, (France) and Professor Professor Peter Nigel Leigh, Brighton and Sussex Medical School, Universities of Brighton and Sussex (UK), assessed the effectiveness and safety of low-dose interleukin-2 (IL2 LD). It benefited from the expertise of Genethon’s DNA bank, a key partner in this collaboration.

Between 2017 and 2019, the MIROCALS trial recruited 220 participants newly diagnosed with ALS. These participants were initially treated with riluzole, the standard treatment for ALS, before being randomized to receive either IL2 LD or placebo for 18 months. The double-blind trial demonstrated that IL2 LD is well tolerated and revealed a statistically significant survival benefit in about 80% of the study participants who had lower levels of phosphorylated heavy-chain neurofilaments (pNFH) in cerebrospinal fluid. In these people, the risk of death at the end of the study was reduced by over 40%.

Genethon’s DNA bank played a key role in this study:

• Providing a high-quality service by contributing its expertise in the processing and storage of samples from 220 patients during the trial. 
The processing of these samples (plasma, cells, cerebrospinal fluid) and the extraction of genomic DNA from the blood of study patients thanks to the implementation of collection and transport procedures.
• By guaranteeing the safety and traceability of over 53,500 samples stored in liquid nitrogen or at -80°C.

“From the outset, Genethon’s bank has been committed to providing high-quality service to the scientific community and meeting the expectations of patients and their families.
I’m very proud to have contributed to this project. The idea for this clinical trial was born some fifteen years ago in the mind of Professor Bensimon, and it is thanks to his determination and perseverance that it has been taken to the European level.
The Genethon Bank also believed in this project from the outset and has never ceased to support Professor Bensimon throughout these years. I would also like to thank the AFM-Téléthon for its confidence and unfailing financial commitment,” emphasized Safaa SAKER, DNA Bank Manager at Genethon.

Although IL2LD is not currently approved for the treatment of ALS, these encouraging results justify further development of IL2 LD and larger trials to confirm its efficacy.