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基于细胞的骨修复治疗性药物:保持简单?

Cell based advanced therapeutic medicinal products for bone repair: Keep it simple?

机构信息

Prometheus, Division of Skeletal Tissue Engineering, KU Leuven, Leuven, Belgium; Skeletal Biology and Engineering Research Center, Department of Development and Regeneration, KU Leuven, Leuven, Belgium.

Prometheus, Division of Skeletal Tissue Engineering, KU Leuven, Leuven, Belgium; Biomechanics Research Unit, University of Liege, Liege, Belgium; Biomechanics Section, KU Leuven, Leuven, Belgium.

出版信息

Adv Drug Deliv Rev. 2015 Apr;84:30-44. doi: 10.1016/j.addr.2014.10.025. Epub 2014 Nov 1.

Abstract

The development of cell based advanced therapeutic medicinal products (ATMPs) for bone repair has been expected to revolutionize the health care system for the clinical treatment of bone defects. Despite this great promise, the clinical outcomes of the few cell based ATMPs that have been translated into clinical treatments have been far from impressive. In part, the clinical outcomes have been hampered because of the simplicity of the first wave of products. In response the field has set-out and amassed a plethora of complexities to alleviate the simplicity induced limitations. Many of these potential second wave products have remained "stuck" in the development pipeline. This is due to a number of reasons including the lack of a regulatory framework that has been evolving in the last years and the shortage of enabling technologies for industrial manufacturing to deal with these novel complexities. In this review, we reflect on the current ATMPs and give special attention to novel approaches that are able to provide complexity to ATMPs in a straightforward manner. Moreover, we discuss the potential tools able to produce or predict 'goldilocks' ATMPs, which are neither too simple nor too complex.

摘要

基于细胞的先进治疗性药物产品(ATMPs)的开发有望为骨修复的医疗保健系统带来革命性变化,从而临床治疗骨缺损。尽管有如此巨大的前景,但已经转化为临床治疗的少数基于细胞的 ATMP 的临床结果远非令人印象深刻。部分原因是由于第一批产品的简单性,临床结果受到了阻碍。为了应对这一情况,该领域已经提出并积累了大量的复杂性,以减轻由简单性引起的限制。其中许多潜在的第二代产品仍滞留在开发渠道中。这是由于多种原因造成的,包括过去几年中不断发展的监管框架的缺乏,以及缺乏用于工业制造的使能技术来应对这些新的复杂性。在这篇综述中,我们反思了当前的 ATMP,并特别关注那些能够以简单直接的方式为 ATMP 提供复杂性的新方法。此外,我们还讨论了能够生产或预测“金发姑娘”ATMP 的潜在工具,这些工具既不太简单也不太复杂。

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