Stem cell-based therapeutics in epilepsy: current advances and future prospects

Abstract

Abstract Epilepsy represents as one of the most prevalent and serious neurological disorders, with approximately one-third of patients resistant to conventional treatments. Current pharmacologic therapies primarily suppress seizures without targeting the underlying epileptogenesis, resulting in unchanged long-term prognosis. At the same time, epilepsy surgery remains underutilized due to the requirement for extensive evaluations and specialized expertise. Stem cell-based therapy has emerged as a promising avenue, offering the potential to address both the neurological and neuropsychiatric aspects. This review summarizes recent preclinical and clinical studies on stem cell therapy in epilepsy, with a focus on their mechanisms of action, including direct engraftment, restoration of normal neurogenesis, and immunomodulation via bystander effect. Advances in genetic engineering, extracellular vesicle-based therapy, and patient-derived brain organoids offer more personalized treatment strategies. Although stem cell therapy holds significant promise as a treatment for epilepsy, further extensive research is required to establish its clinical safety and efficacy.