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New hope for people affected by Charcot Disease. A team from Lille is betting on the natural repair capacity of blood platelets to repair the neurons destroyed by the disease. A promising avenue of research
June 21, 2024 is World Amyotrophic Lateral Sclerosis (ALS) Day, better known as Charcot disease, which affects 500,000 patients worldwide. This disease was publicized when famous people, such as astrophysicist Stephen Hawking, suffered from it and when the Ice Bucket Challenge brought it out of anonymity. Every day in France, 4 people die from it.
June 21, Amyotrophic Lateral Sclerosis Day
Amyotrophic Lateral Sclerosis (ALS) is a neurodegenerative disease characterized by the progressive death of motor neurons, which control, among other things, walking, speaking, swallowing and breathing. This loss of motor neurons leads to muscular atrophy and progressive paralysis of patients down to their respiratory functions, leading irremediably to their death. Life expectancy after diagnosis is generally a few years. Currently, only one treatment, riluzole, is offered with modest results.
Much research is underway to combat this terrible disease. And one of these avenues developed by teams from Lille University Hospital appears promising.
Naturally repair neurons thanks to blood platelets
The role of blood platelets in repair and healing is commonly accepted but has never been tested on neurons. This is the idea of neurologist and pharmacologist David Devos from the Lille Neurosciences & Cognition laboratory and his colleague Professor Thierry Burnouf (Taipei Medical University Taiwan), a specialist in blood and platelets. And the first results at the preclinical stage have proven them right, including a 130% extension of life expectancy in an animal model of the disease.
So that these results could lead to treatment, the inventors patented this platelet-based biotherapy and created the start-up InVenis Biotherapies. Faced with this new therapeutic hope, the SecretGift University Hospital Research (RHU) project received as part of the France 2030 Plan no less than €8.4 million over 5 years, with the aim of developing a new treatment for patients with ALS, via the injection of platelet derivatives into their brain using a pump placed in the abdomen.
A bet whose outcome is still far away
Before such a still distant realization, it will be necessary to go through several stages, all potentially eliminatory: toxicological tests to demonstrate the safety of the treatment, determine the effective doses, find reliable indicators (biomarkers, imaging, etc.) to monitor the evolution of the disease and thus measure the effectiveness of the treatment. These tests will be carried out at the Lille University Hospital.
“To finally hope to change the course of the disease, we are proposing a completely new therapeutic strategy: on the one hand using our powerful natural repair system contained in blood platelets, and on the other hand providing in complete safety and with optimal power this repair directly in the brain. A bit like a stem cell transplant, but in a more controlled and continuous manner. This is SECRET GIFT” declares Professor David Devos.