Cardiovascular diseases persist as a global health challenge that requires methodological innovation for effective drug development. Conventional pipelines relying on animal models suffer from high failure rates due to significant interspecies variation between humans and animal models. In response, the recently enacted Food and Drug Administration Modernization Act 2.0 encourages alternative approaches including induced pluripotent stem cells (iPSCs). Human iPSCs provide a patient-specific, precise, and screenable platform for drug testing, paving the way for cardiovascular precision medicine. This review discusses milestones in iPSC differentiation and their applications from disease modelling to drug discovery in cardiovascular medicine. It then explores challenges and emerging opportunities for the implementation of ‘clinical trials in-a-dish’. Concluding, this review proposes a framework for future clinical trial design with strategic incorporations of iPSC technology, microphysiological systems, clinical pan-omics, and artificial intelligence to improve success rates and advance cardiovascular healthcare.

中文翻译：

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心血管医学用培养皿中的临床试验


心血管疾病一直是一项全球健康挑战，需要方法创新才能进行有效的药物开发。由于人类和动物模型之间的显著物种间差异，依赖动物模型的传统管道故障率很高。作为回应，最近颁布的《食品和药物管理局现代化法案 2.0》鼓励采用包括诱导多能干细胞 （iPSC） 在内的替代方法。人类 iPSC 为药物检测提供了患者特异性、精确和可筛选的平台，为心血管精准医疗铺平了道路。本文讨论了 iPSC 分化的里程碑及其在心血管医学中从疾病建模到药物发现的应用。然后，它探讨了实施“培养皿内临床试验”的挑战和新出现的机遇。总之，本综述提出了未来临床试验设计的框架，战略性地结合了 iPSC 技术、微生理系统、临床泛组学和人工智能，以提高成功率并推进心血管医疗保健。