A new study has demonstrated how daily administration of cannabidiol (CBD), can extend lifespan and improve symptoms associated with Leigh syndrome.
Leigh syndrome is a rare and severe mitochondrial disease affecting around one in 40,000 children and young people. There are currently no approved treatments for the condition, which is characterised by a progressive decline in cognitive and motor functions and premature death.
Dr Emma Puighermanal and Dr Albert Quintana from the Laboratory of Mitochondrial Neuropathology of the Institut de Neurociències at the Universitat Autònoma de Barcelona (INc-UAB), have spent years studying the disease to find therapies capable of reverting it.
Their latest research published in Nature Communications, demonstrates that daily administration of CBD is a promising treatment option. Through its multiple action, CBD was found to provide antioxidant, anti-inflammatory and anticonvulsant effects, which improve symptoms and help recover cell functions in patients.
Promising results in cell and animal studies
The study was conducted with two different Leigh syndrome mouse models, as well as with fibroblast cells from patients.
Looking closer at the results reveals that CBD acts at many levels within the cell, including activating a protein inside the cell nucleus known as PPARγ. This protein regulates the expression of many genes involved in the immune response, oxidation and mitochondrial function, and has been seen to be altered by the disease. CBD also increases the expression of the metallothionein protein, which enhances its antioxidant response.
In the animal models, CBD administration improved neuropathology in the affected brain regions, breathing abnormalities and social deficits, and also delayed motor decline and neurodegenerative signs.
In addition, mice receiving treatment lived significantly longer than those with no treatment. In the fibroblast cells from patients, CBD improved their antioxidant processes.
“The benefits we observed, together with CBD’s safe and well-tolerated profile, show it to be a truly promising treatment for patients with Leigh syndrome,” explains Dr Albert Quintana, researcher at the INc-UAB and lecturer in the Department of Cellular Biology, Physiology and Immunology at the UAB.
Orphan drug designation
The next stage is to advance towards the use of CBD in clinical practice. Last year the researchers obtained an orphan drug designation for CBD in the treatment of Leigh syndrome from the European Medicines Agency. This entails many benefits such as advice and assistance in protocols, market exclusivity and reduced costs in developing the drug.
“CBD has already been approved by the US regulatory agency FDA for the treatment of other rare paediatric diseases,” adds Dr Emma Puighermanal, researcher at the INc-UAB and lead author of the article.
“We hope all of this will help in the translation of our results to clinical practices.”
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