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

Source: PubMed Central Open Access, NCBI / U.S. National Library of Medicine

Stem Cells Translational MedicineLast synced 9/6/2026Status: syncedPMID: 42697696 pmidDOI: 10.1093/stcltm/szag077

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. Graphical Abstract Graphical Abstract Graphical abstract showing stem cell-based therapy in epilepsy. Recent preclinical and clinical studies on stem cell therapy in epilepsy probe neurodegenerative and regenerative mechanisms of action underlying the disease and its treatment. Whereas impaired neurogenesis, aberrant inflam

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. Graphical Abstract Graphical Abstract Graphical abstract showing stem cell-based therapy in epilepsy. Recent preclinical and clinical studies on stem cell therapy in epilepsy probe neurodegenerative and regenerative mechanisms of action underlying the disease and its treatment. Whereas impaired neurogenesis, aberrant inflammation, GABAergic cell death, neural circuit damage, and extracellular vesicle loss accompany epilepsy, stem cell therapy, especially when optimized under novel genetic engineering, patient-derived brain organoids, and drug-resistant disease models, improves safety and efficacy of the treatment by enhancing neurogenesis, dampening inflammation, rescuing GABAergic cells, reconstructing neural circuits, and promoting extracellular vesicle secretion. http://www.w3.org/1999/xlink float portrait szag077f1.webp float szag077-F1 portrait graphical

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