Cannabidiol shows promise in combating motor and cognitive decline effects associated with the disease.
A recent study led by researchers at the Universitat Autònoma de Barcelona’s (UAB) Institut de Neurociències offers new hope for those affected by Leigh syndrome, a severe and often fatal mitochondrial disease. Published in the journal Nature Communications, the study reveals that daily administration of cannabidiol (CBD) has the ability to substantially extend lifespan and alleviates symptoms in animal models of Leigh syndrome. CBD, widely known for its antioxidant, anti-inflammatory, and anticonvulsant properties, has already been approved by the U.S. Food and Drug Administration (FDA) for use in treating other rare pediatric diseases.
Leigh syndrome is a rare genetic condition that primarily affects children. It is characterized by a progressive decrease in motor and cognitive functioning, along with a short lifespace. The disease mostly affects organs and tissues that require high energy, such as the nervous system and muscles, due to mitochondrial dysfunction. Mitochondria are responsible for producing energy in cells, and when they fail to function properly, as in Leigh syndrome, the consequences are devastating.
The research team, led by Drs. Emma Puighermanal and Albert Quintana from the UAB’s Laboratory of Mitochondrial Neuropathology, has been studying Leigh syndrome for years. Their goal has been to better understand how mitochondrial dysfunction occurs in the disease and to identify possible therapies that could mitigate its effects.
One of the key findings in the study is that CBD acts on several levels within the cell to improve mitochondrial function. Specifically, the researchers found that CBD activates a protein within the cell nucleus called PPARγ. This protein regulates the expression of a wide range of genes involved in mitochondrial function, immune responses, and oxidative stress, all of which are affected in Leigh syndrome. By increasing the expression of certain proteins like metallothionein, CBD enhances the cell’s ability to counteract oxidative damage, a hallmark of mitochondrial diseases.
In the animal models, CBD treatment showed profound effects. Mice treated with CBD demonstrated improvements in brain function, including better regulation of breathing and motor skills. They also exhibited fewer neurodegenerative signs, delayed motor decline, and overall better neurological health. Mice receiving the treatment lived longer compared to those who did not, suggesting that CBD has the potential to extend life expectancy in Leigh syndrome. On the cellular level, fibroblasts from patients treated with CBD showed enhanced antioxidant processes, further confirming its therapeutic potential.
“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,” said Dr. Albert Quintana, one of the study’s lead researchers.
The European Medicines Agency (EMA) granted CBD orphan drug designation for the treatment of Leigh syndrome a year ago. This designation is given to drugs that show potential in treating rare diseases, offering several benefits like financial incentives and reduced development costs, which are crucial for advancing treatments for conditions with limited market demand. With FDA approval of CBD already in place for other pediatric diseases, the team is optimistic that these new findings will help accelerate the path toward clinical trials for Leigh syndrome.
Sources:
Cannabidiol shown to extend lifespan and improve symptoms in Leigh syndrome models
Cannabidiol ameliorates mitochondrial disease via PPARγ activation in preclinical models
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