On June 21, during a conference given for World Amyotrophic Lateral Sclerosis (ALS) Day, David Devos, neurologist, professor of pharmacology and researcher in neuroscience at Lille University Hospital, presented, with his team, preclinical results[1] of a treatment path aimed at considerably extending the life expectancy of patients with ALS, or Lou Gehrig’s disease[2] (cf. Charcot’s disease: the Senate must examine a bill to improve care).
Improve patient life expectancy
Every year, nearly 1,600 new cases are diagnosed in France and every day, three people die from it. The life expectancy of patients with this disease is three to five years after the onset of symptoms. For the moment, a treatment, riluzole, can slow the progression of the disease thanks to a ” life gain of 3 months or more », « which is insignificant when you have 3 years left to live ” comments David Devos (see Charcot’s disease: a new treatment approved at European level).
The new technique he has developed uses biotherapy to inject the contents of blood platelets, stripped of their external membrane and from healthy donors, directly into the brains of patients (see Charcot’s disease: combining “technicality and humanity” instead of legalizing euthanasia). They then diffuse into the nervous system, and release neurotrophic factors involved in the growth and survival of neurons. The treatment thus slows down the destruction of motor neurons essential for muscle control.
This approach is original. because it exploits the potential of platelets to repair tissues “, explains Sévérine Boillée, researcher at Inserm, head of the team Causes of ALS and mechanisms of motor neuron degeneration at the Brain Institute, which was not involved in the study. The other strong point is that a local injection into the central nervous system allows maximum benefit from the effects of neurotrophic factors. ” she continues.
Towards clinical trials on humans
Preclinical results demonstrate an increase of almost 130% in the life expectancy of sick mice compared to untreated mice. In view of this success, clinical trials should begin in 12 patients in Lille in 2027. Concretely, we will place, under the skin of the patients’ abdomen, a pump which contains the treatment and which is connected to a catheter inserted into the brain tissue, the role of which is to ensure the delivery of platelet derivatives inside the brain. “, explains Professor Devos. The researchers will also have to identify blood biomarkers or biomarkers from medical imaging that will make it possible to evaluate and quantify the effectiveness of the treatment on the progression of symptoms.
Despite the encouraging results on rodents, “ preclinical results in no way guarantee such efficacy in humans, nor even an absence of undesirable side effects “, warns Sévérine Boillée. A brain infection due to contaminated blood, or a risk of coagulation could prove dangerous for the patient. However, the various preliminary stages of detoxification of platelets in the laboratory should make it possible to avoid these risks (see Brain implant: a new test in a patient suffering from Charcot’s disease).
[1] Whole and fractionated human platelet lysate biomaterials-based biotherapy induces strong neuroprotection in experimental models of amyotrophic lateral sclerosis, Flore Gouel, Kelly Timmerman, Philippe Gosset, Cedric Raoul, Mary Dutheil, Aurélie Jonneaux, Guillaume Garçon, Caroline Moreau, Veronique Danel-Brunaud, James Duce, Thierry Burnouf, Jean-Christophe Devedjian, David Devos, https://doi.org/10.1016/j.biomaterials.2021.121311
[2] Lou Gehrig’s disease or ALS is an incurable neurodegenerative disease caused by the progressive destruction of motor neurons that control the muscles of voluntary movement, speech, swallowing and breathing.
Source: Le Figaro, Elisa Doré (06/26/2024) – Photo: iStock
