Cannabidiol as a treatment for mitochondrial disease
Insitut de Neurociències, Universitat Autònoma de Barcelona, Spain
Deficits in mitochondrial function lead to debilitating and generally fatal pathologies known as Mitochondrial Disease (MD), affecting 1:5000 births. MD are neuromuscular diseases that mostly affect children, leading to severe multiorgan failure and death. There is no cure for mitochondrial disease and the existing treatments are ineffective. Current treatments are limited to palliative care and vitamins or other supplements without clear indication of their effectiveness.
Recently, Quintana’s team identified that daily administration of cannabidiol (CBD), a drug extracted from the cannabis plant, restores the deficits and extends the lifespan in an in vivo model of MD. Importantly, CBD is neuroprotective, anti-inflammatory, safe and without any psychoactive properties, and has already been approved by the Food and Drug Administration (FDA), indicating the promising potential of this drug as a treatment of MD.
Hence, MitoCBD aims at further demonstrating the increased effectiveness of CBD compared to other available options by generating more data in preclinical models of mitochondrial disease, with the overarching goal of bringing a much needed treatment for these devastating orphan diseases.