CBD Shows Promise for Leigh Syndrome
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A recent study published in Nature Communications has revealed promising results for cannabidiol (CBD) as a potential treatment for Leigh syndrome.
Researchers found that daily CBD administration in animal models significantly improved symptoms, extended lifespan, and reduced neuropathology.
The study, led by Drs. Emma Puighermanal and Albert Quintana, discovered that CBD increases the expression of metallothionein, a protein that enhances the body's antioxidant response. This is particularly important for Leigh syndrome, as oxidative stress is a key factor in the disease's progression.
In addition to improving symptoms and lifespan, CBD administration also delayed motor decline and neurodegenerative signs in animal models. Furthermore, the researchers observed that CBD enhanced antioxidant processes in fibroblast cells from patients with Leigh syndrome.
"The positive findings from this study, combined with CBD's safe and well-tolerated profile, make it a promising therapeutic option for Leigh syndrome," stated Dr. Albert Quintana.
The researchers have already obtained orphan drug designation for CBD from the European Medicines Agency, which provides various benefits, including reduced development costs. This, coupled with the FDA's approval of CBD for other rare pediatric diseases, brings hope for the translation of these findings into clinical practice.
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