Department of Environmental and Prevention Sciences, University of Ferrara, Ferrara, Italy.
Adv Exp Med Biol. 2023;1420:1-12. doi: 10.1007/978-3-031-30040-0_1.
Potency assays represent crucial experiments at the hub of the comprehensive complexity surrounding cell therapy. Moreover, numerous factors beyond biological and scientific considerations are involved in achieving successful potency assays that fulfil regulatory authority approval for a new advanced therapy medicinal product. Though this can mean a frustratingly long period of discovery and development, progress in cell therapy is nowadays proceeding remarkably quickly, assisted by the potency assay rigorously placing emphasis on the need to critically analyse the key factor/s responsible for the therapeutic mechanism of action. History has shown that it can take many decades for there to be an improved understanding of a mechanism of action. Yet the chasing of precise targets has revolutionised medicine, with no clearer example than approaches to viral pandemics. The centuries involved in the eradication of smallpox have paved the way for an unprecedented pace of vaccine development for the Covid-19 pandemic. Such extraordinary accomplishments foster encouragement that similarly for stem cell-based therapy, our scientific knowledge will continue to improve apace. This chapter focuses on the art of experimentation and discovery, introducing potency assay requisites and numerous factors that can influence potency assay outcomes. A comprehensive understanding of potency assays and their development can hasten the provision of new cell therapies to help resolve burdensome diseases of unmet medical need.
效力测定代表着细胞治疗领域综合复杂性的核心实验。此外，除了生物学和科学方面的考虑因素外，还有许多其他因素会影响到成功进行效力测定，从而获得新的先进治疗药物产品的监管机构批准。虽然这可能意味着需要经过一段令人沮丧的漫长发现和开发期，但如今细胞治疗的进展速度非常快，效力测定严格地强调需要对负责治疗作用机制的关键因素进行批判性分析，这为其提供了帮助。历史表明，要对作用机制有更深入的了解可能需要几十年的时间。然而，对精确靶点的追求彻底改变了医学，而在病毒大流行方面，没有比这更明显的例子了。在消灭天花的几个世纪里，为应对 COVID-19 大流行，疫苗的开发速度达到了前所未有的水平。这些非凡的成就鼓舞着人们相信，基于干细胞的治疗也将如此，我们的科学知识将继续快速提高。本章重点介绍实验和发现的艺术，介绍效力测定的要求以及可能影响效力测定结果的许多因素。全面了解效力测定及其发展可以加快提供新的细胞疗法，以帮助解决未满足医疗需求的负担性疾病。
