Motor neurone diseases are a group of progressive neurological disorders characterised by the death of motor neurons in cortical and medullary areas, leading to a progressive loss of motor strength, muscular atrophy and, consequently, an inability to live a normal life (moving around, communicating, breathing, etc.). Amyotrophic Lateral Sclerosis (ALS) is one of these diseases. Between 6,000 and 7,000 patients suffer from ALS in France, over 80% of whom are monitored in ALS centres. There are an estimated 1,600 new cases each year in the country.
The median survival time after diagnosis is two years, and there is currently only one treatment approved by the European Medicines Agency (EMA), namely Riluzole, for its effect in slowing down the progression of ALS. Two other treatments can be prescribed: Edaravone in injectable form can be prescribed under an exemption on an individual basis and Tofersen can be used for genetic forms with mutation of the SOD1 gene following a multidisciplinary consultation meeting based on the medical record.
It is essential to carry out therapeutic trials and multi-centre clinical and pathophysiological studies to improve our knowledge of these diseases and identify therapeutic targets, with the aim of finding effective treatments.
The ACT4ALS-MND (Alliance on Clinical Trials for ALS-MND) clinical research network was created in March 2020 on the initiative of the FilSLAN healthcare network dedicated to ALS and motor neurone diseases, to encourage the participation of French centres and patient enrolment in clinical trials. While the aim of FilSLAN is to harmonise care practices and bring together the players involved in monitoring patients suffering from ALS, the ACT4ALS-MND network has the same objectives on the clinical research side.
In January 2022, the ACT4ALS-MND network was awarded accreditation by F-CRIN (the French Clinical Research Infrastructure Network), which is a platform for supporting and structuring national clinical research, expertise and investigation networks on targeted medical themes.
ACT4ALS-MND aims to be the single point of contact for clinical research into ALS and MND in France by supporting project sponsors in setting up their studies and centralising information on trials being carried out in France's ALS expert centres. This information is distributed to all members of the network, giving them a clearer overall picture of clinical research at national and international levels.
To this end, the network has deployed four complementary missions to support ALS-MND clinical research stakeholders:
Map of the ALS expert centers
The network is structured around 10 Reference Centres for Rare Diseases (CR-MR) and 12 Resource and Skills Centres for Rare Diseases (CRC-MR).
