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A Translational Readiness Checklist for Stem-Cell-Based Trials in Movement Disorders: A Practical Roadmap
Sahar Hemeda1,2, József Janszky1,3, Dávid Pintér1,3
1Department of Neurology, Medical School, University of Pécs, Pécs, Hungary.
Journal of Central Nervous System Disease
|July 22, 2026
Summary
A new framework standardizes stem cell therapy trials for movement disorders. This auditable checklist ensures product characterization, safety, and regulatory compliance for better clinical translation.
Area of Science:
- Regenerative Medicine
- Neuroscience
- Clinical Trial Design
Background:
- Stem cell therapies for movement disorders face challenges in translation due to inconsistent product characterization, delivery, endpoints, safety, and follow-up protocols.
- Existing regulatory guidance offers principles but lacks a unified framework for conducting clinical trials.
Purpose of the Study:
- To develop a practical, auditable framework and pre-First Patient In (FPI) checklist for stem cell trials in movement disorders.
- The framework integrates scientific, regulatory, manufacturing, ethical, and clinical expectations to streamline trial preparation.
Main Methods:
- A structured literature review of scientific publications, clinical trials, and regulatory guidance documents was conducted.
- Searches encompassed major databases and clinical trial registries up to January 2026, alongside reviews of key international regulatory and scientific body guidelines.
- Identified requirements were synthesized into a universal Core Gate checklist with risk-proportionate, platform-specific modules (A, B, C).
Main Results:
- A novel framework was developed, featuring a universal Core Gate for minimum pre-FPI requirements and three risk-proportionate modules for specific stem cell types (pluripotent-derived neural grafts, somatic neural grafts, MSC/secretome).
- The framework incorporates movement disorder-specific operational elements, including harmonized phenotyping, disease-specific endpoints, and practical imaging/biomarker options.
- The resulting auditable checklist addresses preclinical justification, manufacturing quality, ethics, trial design, safety, and long-term follow-up.
Conclusions:
- This framework transforms fragmented guidance into a cohesive roadmap for stem cell trials in movement disorders.
- It aims to reduce protocol heterogeneity, enhance cross-trial comparability, improve regulatory planning, and support safer, more interpretable clinical translation of these therapies.
