Daily administration of cannabidiol (CBD) has demonstrated the ability to substantially extend lifespan and alleviate symptoms in animal models of Leigh syndrome, offering new hope for those affected by this severe and often fatal mitochondrial disease. The study, published in Nature Communications, was conducted by researchers at the Universitat Autònoma de Barcelona’s (UAB) Institut de Neurociències.
Leigh syndrome is a rare genetic condition primarily affecting children, characterized by a progressive decline in motor and cognitive functions and a shortened lifespan. The disease impairs organs and tissues requiring high energy, such as the nervous system and muscles, due to mitochondrial dysfunction.
CBD's Mechanism of Action
The research highlights that CBD acts on multiple levels within the cell to enhance mitochondrial function. Specifically, CBD activates a protein within the cell nucleus called PPARγ. This protein regulates the expression of various genes involved in mitochondrial function, immune responses, and oxidative stress—all affected in Leigh syndrome. By increasing the expression of proteins like metallothionein, CBD enhances the cell’s capacity to counteract oxidative damage, a key feature of mitochondrial diseases.
Impact on Animal Models
In animal models, CBD treatment exhibited significant improvements in brain function, including better regulation of breathing and motor skills. The treated mice also showed fewer neurodegenerative signs, delayed motor decline, and improved overall neurological health. Notably, mice receiving CBD lived longer compared to those without treatment, indicating CBD's potential to extend life expectancy in Leigh syndrome.
On a cellular level, fibroblasts from patients treated with CBD showed enhanced antioxidant processes, further supporting its therapeutic potential. According to Dr. Albert Quintana, one of the lead researchers, "This study provides compelling evidence that CBD could be a truly promising treatment for patients with Leigh syndrome, especially given its safety and well-tolerated profile."
Regulatory and Clinical Prospects
The European Medicines Agency (EMA) granted CBD orphan drug designation for the treatment of Leigh syndrome a year ago. This designation provides financial incentives and reduced development costs, crucial for advancing treatments for rare diseases with limited market demand. Given the existing FDA approval of CBD for other pediatric diseases, the research team is optimistic that these findings will expedite the path toward clinical trials for Leigh syndrome.
