Institute of Medical Biology, A*STAR, Singapore.
BioDrugs. 2010 Apr 1;24(2):99-108. doi: 10.2165/11532270-000000000-00000.
The need for new and improved pharmacotherapies in medicine, high late-stage compound attrition in drug discovery, and upcoming patent expirations is driving interest by the pharmaceutical industry in pluripotent stem cells for in vitro modeling and early-stage testing of toxicity and target engagement. In particular, human embryonic and induced pluripotent stem cells represent potentially cost-effective and accessible sources of organ-specific cells that foretell in vivo human tissue response to new chemical entities. Here we consider the potential of these cells as novel tools for drug development, including toxicity screening and metabolic profiling. We hold that despite various challenges to translating proof-of-concept screening platforms to industrial use, the promise of research is considerable, and close to being realized.
医学领域需要新的和改进的药物疗法，药物发现的后期化合物淘汰率高，以及即将到来的专利到期，这促使制药行业对多能干细胞产生兴趣，用于体外建模和早期毒性和靶点结合测试。特别是，人类胚胎和诱导多能干细胞代表了潜在的具有成本效益和可及性的器官特异性细胞来源，可以预测新化学实体对体内人类组织的反应。在这里，我们考虑这些细胞作为药物开发的新工具的潜力，包括毒性筛选和代谢谱分析。我们认为，尽管将概念验证筛选平台转化为工业用途存在各种挑战，但研究的前景是相当可观的，并且即将实现。
