Cell therapies present an exciting potential but there is a long history of expensive translational failures in stroke research. Researchers engaged in cell therapy research would benefit from a practical framework that can help in planning research and development of investigational cell therapies into viable medical products. We developed a checklist using a mixed methodology approach to evaluate the impact of study design, regulatory policy, ethical, and health economic considerations for efficient implementation of early phase cell therapy studies. The checklist comprises a series of questions arranged under four domains: the first concerns study design such as characterization of target study population, trial design, endpoints and operational fit of dosage, time, and route of administration to target populations. A second domain addresses the data package required for regulatory approval relevant to the intended use (allogeneic/autologous; homologous/non-homologous; nature of cell processing). The third domain comprises patient involvement to ensure relevant data is collected via targeted study design. The final domain requires the team to determine the critical data elements that could be built into study design to enable health economic data collection to be started at an early phase of the study. The CT2S checklist can help to determine areas of expertise gaps and enable research groups to appropriately allocate resources for capacity building. Use of this checklist will allow identification of key areas where trial planning needs to be optimized, as well as helping to identify resources that need to be secured. The CT2S checklist can also serve as a general cell therapy research decision aid to improve research output and accelerate new cell therapy development.

中文翻译：

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脑卒中细胞治疗（CT2S）检查表的临床翻译—加速细胞治疗产品开发的实用工具

细胞疗法具有令人兴奋的潜力，但是在中风研究中长期存在昂贵的翻译失败的历史。从事细胞疗法研究的研究人员将从实用的框架中受益，该框架可以帮助计划将研究性细胞疗法研究和开发为可行的医疗产品。我们使用混合方法学方法开发了一份清单，以评估研究设计，监管政策，道德和健康经济因素对有效实施早期细胞疗法研究的影响。该清单包括在四个领域内排列的一系列问题：第一个涉及研究设计，例如目标研究人群的表征，试验设计，终点和剂量，时间以及对目标人群的给药途径的操作适应性。第二个域涉及与预期用途（同种/自体；同源/非同体；细胞加工的性质）相关的监管批准所需的数据包。第三领域包括患者参与，以确保通过目标研究设计收集相关数据。最后一个领域要求团队确定可以纳入研究设计的关键数据元素，以使健康经济数据收集能够在研究的早期阶段开始。CT2S清单可以帮助确定专业知识差距的领域，并使研究小组能够适当地分配资源以进行能力建设。使用此清单可以确定需要优化试验计划的关键领域，并有助于确定需要确保的资源。