Cannabidiol demonstrated to alleviate symptoms of Leigh syndrome

Medical Xpress / Autonomous University of Barcelona / Nature Communications ^ | Sept. 6, 2024 | Emma Puighermanal et al

[ConservativeMind]

A study demonstrates in animal models how daily administration of cannabidiol (CBD), a substance obtained from the cannabis plant, extends lifespan and improves symptoms associated with Leigh syndrome.

This severe mitochondrial disease affecting children is characterized by a progressive decline in cognitive and motor functions and premature death. The research group also demonstrated in both mice and fibroblasts from children with the disease that CBD improves cellular function.

Researchers demonstrated that daily administration of CBD is a promising treatment option. Through its multiple actions, it provides antioxidant, anti-inflammatory and anticonvulsant effects, which improve the symptomatology and help recover cell functions in patients. The study was conducted with two different Leigh syndrome mouse models, as well as with fibroblast cells from patients.

The results revealed 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. Moreover, CBD increases the expression of the metallothionein protein, which enhances its antioxidant response.

In animal models, cannabidiol 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 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.

"CBD has already been approved by the US regulatory agency FDA for the treatment of other rare pediatric diseases. We hope all of this will help in the translation of our results to clinical practices," concludes Dr. Emma Puighermanal.

[ConservativeMind]

This should already be available as an option in the US, due to FDA approval of CBDs for kids.

[ConservativeMind]

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[Oystir]

It’s a mouse study. Not a human trial.