Treating Huntington’s disease. This is the mission entrusted to the researchers of Insertion, from the University of Grenoble Alpes and from the CHU Grenoble Alpes to the Grenoble Institute of Neurosciences. In fact, they work on the trail of a first “neuroprotective” treatment (protection of neurons). One molecule, ML348, is already showing promising results in mice and is undergoing preclinical evaluation.
A rare inherited neurodegenerative disease, Huntington’s disease leads to the degeneration of neurons involved in motor, cognitive and psychiatric functions. Although about 10% of patients report it before the age of 20, it generally begins in people between the ages of 30 and 50. Cognitive and psychiatric disturbances and uncontrolled movements worsen until the patient’s death, approximately twenty years later.
Drawing of boys with Huntington’s chorea (St. Vitus dance)
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In France, 18,000 people are affected. Nearly 6,000 already have symptoms and 12,000 are carriers of the mutated gene. Current treatments are symptomatic and alleviate some ailments but do not change the course of the disease, which inevitably ends with the patient’s death.
Huntington’s disease is caused by an abnormality in the gene that codes for the huntingtin protein. This leads in particular to a reduction in the transport of an important molecule, BDNF, between two areas of the brain which are the cortex and the striatum. A reduced influx that decreases the lifespan of neurons and the connection of these brain regions.
