Biological treatments are one of the medical breakthroughs in the twenty-first century. The initial enthusiasm pushed the field towards indiscriminatory use of cell therapy regardless of the pathophysiological particularities of underlying conditions. In the reparative and regenerative cardiovascular field, the results of the over two decades of research in cell-based therapies, although promising still could not be translated into clinical scenario. Now, when we identified possible deficiencies and try to rebuild its foundations rigorously on scientific evidence, development of potency assays for the potential therapeutic product is one of the steps which will bring our goal of clinical translation closer. Although, highly challenging, the potency tests for cell products are considered as a priority by the regulatory agencies. In this paper we describe the main characteristics and challenges for a cell therapy potency test focusing on the cardiovascular field. Moreover, we discuss different steps and types of assays that should be taken into consideration for an eventual potency test development by tying together two fundamental concepts: target disease and expected mechanism of action.

Graphical Abstract

Development of potency assays for cell-based products consists in understanding the pathophysiology of the disease, identifying potential mechanisms of action (MoA) to counteract it and finding the most suitable cell-based product that exhibits these MoA. When applied, the potency assay needs to correlate bioactivity of the product, via a measurement related to the MoA, with treatment efficacy. However, in the cardiovascular field, the process faces several challenges and high requirements.

中文翻译：

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心脏再生和修复医学中基于细胞的疗法的有效效力测定的基本需求。测试开发的实用方法

生物治疗是二十一世纪的医学突破之一。最初的热情推动该领域不分青红皂白地使用细胞疗法，而不管潜在疾病的病理生理学特殊性如何。在修复性和再生性心血管领域，基于细胞疗法的二十多年研究成果虽然前景广阔，但仍无法转化为临床应用。现在，当我们确定可能的缺陷并尝试在科学证据上严格重建其基础时，开发潜在治疗产品的效力测定是使我们的临床转化目标更接近的步骤之一。尽管非常具有挑战性，但监管机构将细胞产品的效力测试视为优先事项。在本文中，我们描述了针对心血管领域的细胞治疗效力测试的主要特征和挑战。此外，我们通过将两个基本概念联系在一起：目标疾病和预期的作用机制，讨论了最终效力测试开发应考虑的不同步骤和分析类型。

图形概要

开发基于细胞的产品的效力测定包括了解疾病的病理生理学，确定潜在的作用机制 (MoA) 以抵消它，并找到最合适的具有这些 MoA 的基于细胞的产品。应用时，效力测定需要通过与 MoA 相关的测量将产品的生物活性与治疗效果相关联。然而，在心血管领域，该过程面临着诸多挑战和高要求。