Trial reportStem cell research & therapy2026
Safety and preliminary efficacy of autologous bone marrow-derived mesenchymal stem cell transplantation in hereditary cerebellar ataxia: phase I/IIa clinical trial.
Trial report in Stem cell research & therapy, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.
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18 authors.
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Abstract
backgroundHereditary cerebellar ataxias (HCA) encompass a spectrum of pathological conditions affecting the cerebellum. Currently, there is growing interest in the potential role of mesenchymal stem cells (MSCs) as an investigational therapeutic approach for this condition. Hence, the objective of this single-center, open-label, phase I/IIa clinical trial was to assess the safety and exploratory clinical and biomarker changes following a single intrathecal injection of autologous bone marrow-derived mesenchymal stem cells (BM-MSCs) in HCA.
methodsTen confirmed patients with HCA entered the study and underwent a single dose of (1 × 10
resultsNo severe adverse effects were observed following the cell transplantation procedure. A decreasing trend in SARA score was observed, with a statistically significant difference at month 6 compared with baseline. Except for one patient, GAD-65 antibody levels remained within the normal range in all patients. In one patient with markedly elevated baseline GAD-65 titers, serum and CSF GAD-65 levels decreased from 814 IU/mL and 781 IU/mL, respectively, to values within the normal range after 3 months. Significant changes were observed in selected inflammatory biomarkers, including decreased serum IL-6 at month 3, decreased CSF TNF-alpha at months 1 and 3, and increased serum IL-10 during follow-up. CSF IL-6 did not show a significant decrease.
conclusionThe findings support the short-term safety and tolerability of a single intrathecal dose of autologous BM-MSCs in this small HCA cohort. The study provides preliminary signals of possible clinical and biomarker changes; however, efficacy cannot be established due to the uncontrolled design, small sample size, disease heterogeneity, and short follow-up. Larger randomized controlled trials are required to validate these exploratory findings.
trial registrationThis clinical trial was registered with the Iranian Registry of Clinical Trials (ID: IRCT20160809029275N3).
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