CNS neuroscience & therapeutics · 2026
Mesenchymal Stem Cell-Based Therapy for Cerebellar Ataxia: From Bench to Bedside.
СтатьяОбзорНазвание и аннотация: Перевод в очереди
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Suk Kyoungho, Lee Ho-Won, Kim Sang Ryong
Аннотация
INTRODUCTION: Cerebellar ataxia (CA) encompasses hereditary and acquired disorders unified by Purkinje cell loss and neuroinflammation, for which no disease-modifying therapy exists. Human mesenchymal stem cells (hMSCs) offer multimodal neuroprotection through paracrine secretion of neurotrophic factors and immunomodulatory mediators.
METHODS: We reviewed preclinical and clinical evidence for hMSC therapy across multiple CA etiologies, integrating findings from neuroinflammatory, toxic/developmental, and genetic mouse models alongside published clinical trials and case reports. A systematic literature search was conducted in PubMed/MEDLINE, Embase, and the Cochrane Library (search period: 2000-2026) using the following key terms: "mesenchymal stem cell" AND "cerebellar ataxia"; "MSC" AND "spinocerebellar ataxia"; "hMSC" AND "Purkinje cell"; "stem cell therapy" AND "ataxia". Inclusion criteria encompassed: peer-reviewed original research articles and reviews in English; in vivo animal model studies; clinical trials, case series, and case reports. Studies addressing non-CA neurological conditions without CA-relevant data were excluded.
RESULTS: hMSC transplantation consistently improved motor function, preserved Purkinje cell integrity, and attenuated neuroinflammation across LPS-induced, Ara-C-induced, and SCA2 transgenic models. A critical observation is that MSCs from CA patients exhibit markedly reduced anti-inflammatory secretome capacity compared with healthy-donor MSCs, justifying an allogeneic strategy. Therapeutic efficacy was maintained even after symptom onset in the SCA2 model. A published case report demonstrated safety and preliminary functional benefit of intrathecal allogeneic bone marrow-derived MSCs in a sporadic adult-onset ataxia patient.
CONCLUSIONS: hMSC therapy targets convergent CA pathomechanisms-microglial suppression, neurotrophin restoration, and Purkinje cell preservation-through a paracrine rather than cell-replacement mechanism. Post-symptomatic efficacy and an emerging clinical evidence base support advancing toward placebo-controlled randomized trials.
METHODS: We reviewed preclinical and clinical evidence for hMSC therapy across multiple CA etiologies, integrating findings from neuroinflammatory, toxic/developmental, and genetic mouse models alongside published clinical trials and case reports. A systematic literature search was conducted in PubMed/MEDLINE, Embase, and the Cochrane Library (search period: 2000-2026) using the following key terms: "mesenchymal stem cell" AND "cerebellar ataxia"; "MSC" AND "spinocerebellar ataxia"; "hMSC" AND "Purkinje cell"; "stem cell therapy" AND "ataxia". Inclusion criteria encompassed: peer-reviewed original research articles and reviews in English; in vivo animal model studies; clinical trials, case series, and case reports. Studies addressing non-CA neurological conditions without CA-relevant data were excluded.
RESULTS: hMSC transplantation consistently improved motor function, preserved Purkinje cell integrity, and attenuated neuroinflammation across LPS-induced, Ara-C-induced, and SCA2 transgenic models. A critical observation is that MSCs from CA patients exhibit markedly reduced anti-inflammatory secretome capacity compared with healthy-donor MSCs, justifying an allogeneic strategy. Therapeutic efficacy was maintained even after symptom onset in the SCA2 model. A published case report demonstrated safety and preliminary functional benefit of intrathecal allogeneic bone marrow-derived MSCs in a sporadic adult-onset ataxia patient.
CONCLUSIONS: hMSC therapy targets convergent CA pathomechanisms-microglial suppression, neurotrophin restoration, and Purkinje cell preservation-through a paracrine rather than cell-replacement mechanism. Post-symptomatic efficacy and an emerging clinical evidence base support advancing toward placebo-controlled randomized trials.