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A Translational Readiness Checklist for Stem-Cell–Based Trials in Movement Disorders: A Practical Roadmap
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DOI：10.1177/11795735261463495.png)
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Abstract
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<jats:p>Stem cell-based therapies for movement disorders show inconsistent translation due to variations in product characterisation, delivery, endpoints, safety, and follow-up. Regulatory guidance provides principles but lacks a unified trial framework. The objective was to create a practical framework that translates scientific, regulatory, manufacturing, ethical, and clinical expectations into an auditable pre–First Patient In (FPI) checklist for stem cell trials. A structured review of the literature, clinical trials, and guidance documents was conducted. Searches were performed in PubMed/MEDLINE, Embase, Google Scholar, and clinical trial registries, including ClinicalTrials.gov, Japan Registry, European Union Register, and Clinical Trials Registry–India, until January 2026. Key guidance documents from the FDA, EMA, ICH, ISSCR, and selected PMDA sources were reviewed. Recurring requirements related to nonclinical evidence, manufacturing quality, ethics, trial design, safety, and follow-up were identified and translated into auditable pre-FPI checklist items. A framework was developed, structured as a universal Core Gate plus three risk-proportionate modules: Module A for pluripotent-derived neural grafts, Module B for somatic neural grafts, and Module C for mesenchymal stromal cell/secretome approaches. The Core Gate outlines the minimum pre-FPI requirements across preclinical justification, GMP release documentation, regulatory and ethics readiness, patient selection, trial design, safety oversight, trial conduct, long-term follow-up, and transparency. The modules add platform-specific requirements according to biological and procedural risks, including tumourigenicity and genomic stability assessment, immunologic monitoring, and route-appropriate biodistribution and persistence expectations. Movement disorder-specific operational elements not defined in general guidance were also incorporated, including harmonised baseline phenotyping, disease-specific endpoint menus, practical imaging and biomarker options. This framework turns fragmented guidance into a practical, auditable pre-FPI roadmap for stem cell trials in movement disorders. By combining a universal Core Gate with risk-proportionate modules, it aims to reduce protocol heterogeneity, improve cross-trial comparability, strengthen regulatory planning, and support safer, interpretable clinical translation.</jats:p>
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