Mesenchymal Stem Cells as a Biological Drug for Heart Disease: Where Are We With Cardiac Cell-Based Therapy?
Mesenchymal Stem Cells as a Biological Drug for Heart Disease: Where Are We With Cardiac Cell-Based Therapy?
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DOI：
10.1161/circresaha.117.306306
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发表时间：
2015-07-17
影响因子：
20.1
通讯作者：
Hare JM
中科院分区：
文献类型：
--
作者：
Sanina C;Hare JM
基于细胞的治疗代表了生物治疗发展的新一代。一种基于细胞的原型疗法，间充质干细胞（MSC），已经成功进入治疗心力衰竭的III期关键试验，这意味着来自I期和II期试验的充分的安全性和有效性数据。成功的三期试验可能导致一种用于再生医学的新生物疗法获得批准。干细胞或祖细胞用于治疗慢性疾病（包括心血管疾病）的研究正在进行中。对于一项处于早期阶段的颠覆性技术来说，早期的发现将该领域带到了一个重要而有争议的十字路口，这并不罕见。在很大程度上，基于围绕细胞疗法作用机制的分歧，一些作者呼吁对现有数据进行重新评估，而另一些人则得出结论，认为该领域“发展到临床实践的速度太快”，我们“需要回到板凳上”。然而，历史上有许多例子，包括阿司匹林和阿片类药物的著名例子，在完全了解其作用机制之前就被用于治疗。在这场关于细胞治疗作用机制的争论中，一个具有巨大前景的新兴研究领域需要得到强调。在这种情况下，MSCs的发展遵循临床前和临床发展的特征轨迹，并得到高度预测成功治疗结果的数据的支持。最重要的是，由于其同种异体的潜力，间充质干细胞可以被视为一种真正的细胞生物疗法，具有大批量质量控制生产和“现成”使用的能力。
Cell-based treatment represents a new generation in the evolution of biological therapeutics. A prototypic cell-based therapy, the mesenchymal stem cell (MSC), has successfully entered phase III pivotal trials for heart failure, signifying adequate enabling safety and efficacy data from phase I and II trials. Successful phase III trials can lead to approval of a new biologic therapy for regenerative medicine. The use of stem or progenitor cells therapeutically is under investigation for the treatment of chronic diseases, including cardiovascular pathologies. Not unusual for an early stage disruptive technology, early findings have led the field to an important and controversial cross-roads. Based in large part on disagreements surrounding mechanism of action of cell-based therapy, some authors have called for a reappraisal of the existing data, while others have concluded that the field has “evolved too quickly into clinical practice” and that we “need to go back to the bench”. However, there are many historical examples including the notable examples of aspirin and opioids that were employed therapeutically before the mechanisms of actions were fully understood. In the midst of this debate regarding mechanism of action of cell therapy, an emerging field of investigation that holds great promise needs to be highlighted. In this context, the development of MSCs has followed the characteristic trajectory of preclinical and clinical development, supported by data highly predictive of successful therapeutic outcome. Most importantly, because of its allogeneic potential, MSCs can be viewed as a true cellular biological therapy with the capacity for high volume quality-controlled production and “off the shelf” usage.