Leigh syndrome is a rare and severe neurological disorder that typically becomes apparent in the first year of life. It is characterized by progressive neuromuscular decline and usually results in death within two to three years, often from respiratory failure. A small number of patients don’t develop symptoms until adulthood or have symptoms that develop more slowly. There are currently no approved treatments for the disease.
Increasing evidence suggests that cannabis-derived compounds may have therapeutic value for treating some neurodegenerative disorders. CBD has been gaining attention in particular due to its few side effects and broad therapeutic properties- including anti-inflammatory, antioxidant, neuroprotective, and anticonvulsant effects.
In the current study, researchers observed the effects of CBD administration on two different mouse models of Leigh Syndrome and fibroblasts from children with the disease. They found that CBD exerts multiple effects on cells, including activating a protein inside the nucleus called PPARγ which is known to be altered by the condition, and which regulates the expression of many genes involved in the immune response, oxidation, and mitochondrial function. CBD was further shown to increase the expression of the metallothionein protein which enhances its antioxidant response.
In mouse models, the researchers found that CBD improved neuropathology in affected brain areas, breathing abnormalities and social deficits. It also delayed motor decline and signs of neurodegeneration and was linked to significantly longer lifespans. CBD further improved antioxidant processes in fibroblasts from patients with the condition.
"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", said study author Dr. Albert Quintana, lecturer in the Department of Cellular Biology, Physiology and Immunology at the Universitat Autonoma de Barcelona, in a press release.
“CBD has already been approved by the US regulatory agency FDA for the treatment of other rare paediatric diseases. We hope all of this will help in the translation of our results to clinical practices”, said lead study author Dr. Emma Puighermanal, researcher at the Universitat Autonoma de Barcelona, in a press release.
Sources: EurekAlert, Nature Communications