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<p>Spinocerebellar degenerations (SCDs) are progressive hereditary and selected sporadic cerebellar neurodegenerative disorders with limited disease-modifying options. Cell-based therapies particularly, mesenchymal stromal/stem cells (MSCs), neural stem/progenitor cells, olfactory ensheathing cells (OECs), cord blood mononuclear cells (CBMCs), and hematopoietic stem cell transplantation (HSCT) are biologically plausible candidates, but their clinical efficacy and safety remain uncertain. We searched MEDLINE/PubMed, Embase, Scopus, Web of Science, and Cochrane Library from inception to 10 October 2025 (no language restrictions). The OSF-registered protocol prespecified eligibility, outcomes, and design-specific risk-of-bias (RoB) tools, with harmonized overall RoB judgments. We included human interventional or observational reports of cell-based or cell-derived products in hereditary ataxias and selected cerebellar-predominant degenerative syndromes within the OPCA/MSA-C spectrum, and extracted clinical efficacy, safety, and follow-up. Of 603 records screened, 17 reports met the protocol-defined inclusion criteria (15 full texts; 2 abstracts; 2006–2025). Most were small, uncontrolled case series, predominantly from China. Cell types: MSCs (9/17), HSCT in ataxia-telangiectasia (3/17), OECs (3/17), and others (neural stem cells [NSCs], CBMCs; 2/17). Routes were intrathecal and/or intravenous for MSC/CBMC; OEC/NSC were intracerebral/intraspinal; HSCT was delivered intravenously. Primary follow-up was typically 1–6 months. Safety was generally acceptable for MSC/CBMC/OEC with no treatment-related deaths and mostly transient post-procedural symptoms; HSCT exhibited regimen-related toxicities consistent with conditioning intensity. Efficacy signals were modest and often transient: short-term International Cooperative Ataxia Rating Scale (ICARS) improvements were reported across multiple series. Cell-based therapies for SCDs show biological plausibility and generally reassuring short-term safety, but current human evidence, dominated by uncontrolled, high-bias studies does not establish efficacy or durability. Priority should shift to rigorously controlled trials, standardized good manufacturing practice (GMP)-characterized products, blinded centralized ratings, careful control of co-interventions, and objective biomarkers.</p>

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Cell-Based Therapies for Spinocerebellar Degenerations: A Systematic Review of Human Clinical Evidence

  • 期刊论文

  • 发表日期：

    2026年4月13日

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  • Hamid Khalesi,
  • Mehran Beiraghi Toosi,
  • Arshia Khalesi,
  • Alireza Khalesi,
  • Arash Ziaee,
  • Javad Akhondian

摘要

Spinocerebellar degenerations (SCDs) are progressive hereditary and selected sporadic cerebellar neurodegenerative disorders with limited disease-modifying options. Cell-based therapies particularly, mesenchymal stromal/stem cells (MSCs), neural stem/progenitor cells, olfactory ensheathing cells (OECs), cord blood mononuclear cells (CBMCs), and hematopoietic stem cell transplantation (HSCT) are biologically plausible candidates, but their clinical efficacy and safety remain uncertain. We searched MEDLINE/PubMed, Embase, Scopus, Web of Science, and Cochrane Library from inception to 10 October 2025 (no language restrictions). The OSF-registered protocol prespecified eligibility, outcomes, and design-specific risk-of-bias (RoB) tools, with harmonized overall RoB judgments. We included human interventional or observational reports of cell-based or cell-derived products in hereditary ataxias and selected cerebellar-predominant degenerative syndromes within the OPCA/MSA-C spectrum, and extracted clinical efficacy, safety, and follow-up. Of 603 records screened, 17 reports met the protocol-defined inclusion criteria (15 full texts; 2 abstracts; 2006–2025). Most were small, uncontrolled case series, predominantly from China. Cell types: MSCs (9/17), HSCT in ataxia-telangiectasia (3/17), OECs (3/17), and others (neural stem cells [NSCs], CBMCs; 2/17). Routes were intrathecal and/or intravenous for MSC/CBMC; OEC/NSC were intracerebral/intraspinal; HSCT was delivered intravenously. Primary follow-up was typically 1–6 months. Safety was generally acceptable for MSC/CBMC/OEC with no treatment-related deaths and mostly transient post-procedural symptoms; HSCT exhibited regimen-related toxicities consistent with conditioning intensity. Efficacy signals were modest and often transient: short-term International Cooperative Ataxia Rating Scale (ICARS) improvements were reported across multiple series. Cell-based therapies for SCDs show biological plausibility and generally reassuring short-term safety, but current human evidence, dominated by uncontrolled, high-bias studies does not establish efficacy or durability. Priority should shift to rigorously controlled trials, standardized good manufacturing practice (GMP)-characterized products, blinded centralized ratings, careful control of co-interventions, and objective biomarkers.