Targeting the endocannabinoid system to treat epilepsy in Niemann Pick disease type C
Awardee: Lola Ledesma
Institution: Centro Biologia Molecular Severo Ochoa
Grant Amount: $40,140.00
Funding Period: February 1, 2024 - January 31, 2025
Summary:
Pharmacoresistent epilepsy is frequently found in NPC patients. This condition impact heavily in the quality of life of the patients and their families. Several studies have reported the ability of cannabinoids to reduce the frequency of seizures with a safe profile even in pediatric population. We have also shown the downregulation of the endocannabinoid system in the brain of an NPC mouse model and patient. We thus propose the enhancement of the endocannabinoid system as anti-epileptic treatment in the disease. We will assess the efficacy and safety of this strategy in a mouse model for NPC using oral administration of exogenous cannabinoids or of an inhibitor of the endocannabinoid degrading enzyme FAAH.