A new study published in the journal Nature Communications reveals that cannabidiol (CBD) may offer a breakthrough in treating primary mitochondrial diseases (MD), a group of untreatable disorders caused by mutations in energy-producing genes.
The research specifically focuses on Leigh syndrome (LS), the most common pediatric MD, which is marked by progressive neuromuscular impairment and early death.
The study shows that daily CBD administration significantly extends lifespan and improves various symptoms in two mouse models of Leigh syndrome.
“CBD delays motor decline and neurodegenerative signs, improves social deficits and breathing abnormalities, decreases thermally induced seizures, and improves neuropathology in affected brain regions,” the abstract notes. This breakthrough suggests that CBD could offer a new therapeutic approach for patients suffering from this debilitating condition.
Researchers identified peroxisome proliferator-activated receptor gamma (PPARγ), a nuclear receptor, as a crucial mediator of CBD’s beneficial effects. PPARγ activation was found to improve cellular function in both mouse neurons and fibroblasts from LS patients. Additionally, the study highlights dysregulated PPARγ activity as a common feature in Leigh syndrome, further underscoring its importance in the disease’s progression.
Researchers note that this study provides the first evidence that CBD could be a viable treatment option, offering a new hope for patients and families dealing with mitochondrial disease.
The study’s full abstract can be found below:
Abstract
Mutations in mitochondrial energy-producing genes lead to a heterogeneous group of untreatable disorders known as primary mitochondrial diseases (MD). Leigh syndrome (LS) is the most common pediatric MD and is characterized by progressive neuromuscular affectation and premature death. Here, we show that daily cannabidiol (CBD) administration significantly extends lifespan and ameliorates pathology in two LS mouse models, and improves cellular function in fibroblasts from LS patients. CBD delays motor decline and neurodegenerative signs, improves social deficits and breathing abnormalities, decreases thermally induced seizures, and improves neuropathology in affected brain regions. Mechanistically, we identify peroxisome proliferator-activated receptor gamma (PPARγ) as a key nuclear receptor mediating CBD’s beneficial effects, while also providing proof of dysregulated PPARγ expression and activity as a common feature in both mouse neurons and fibroblasts from LS patients. Taken together, our results provide the first evidence for CBD as a potential treatment for LS.
