下一代疗法:利用 iPSC、基因组学、人工智能和培养皿中的临床试验进行开创性药物发现。
Next-Gen Therapeutics: Pioneering Drug Discovery with iPSCs, Genomics, AI, and Clinical Trials in a Dish.
发表日期:2024 Sep 16
作者:
Zehra Yildirim, Kyle Swanson, Xuekun Wu, James Zou, Joseph Wu
来源:
Annual Review of Pharmacology and Toxicology
摘要:
在药物发现的高风险领域,从实验室到临床的过程受到高达 92% 的失败率的阻碍,这主要是由于临床试验中不可预测的毒性和治疗效果不足。 FDA 现代化法案 2.0 预示着一种变革性的方法,提倡将替代方法整合到传统动物测试中,包括采用人类诱导多能干细胞 (iPSC) 衍生的类器官的细胞分析和器官芯片技术,与复杂的人工智能 (AI) 方法相结合。我们的综述探讨了 iPSC 衍生临床试验在专为心血管疾病研究而设计的培养皿模型中的创新能力。我们还强调了 iPSC 技术与人工智能的整合如何能够加速识别可行的候选治疗药物、简化药物筛选并为更加个性化的医疗铺平道路。通过此,我们全面概述了研究界和制药行业正在引导的 iPSC 和人工智能应用的当前形势和未来影响。
In the high-stakes arena of drug discovery, the journey from bench to bedside is hindered by a daunting 92% failure rate, primarily due to unpredicted toxicities and inadequate therapeutic efficacy in clinical trials. The FDA Modernization Act 2.0 heralds a transformative approach, advocating for the integration of alternative methods to conventional animal testing, including cell-based assays that employ human induced pluripotent stem cell (iPSC)-derived organoids, and organ-on-a-chip technologies, in conjunction with sophisticated artificial intelligence (AI) methodologies. Our review explores the innovative capacity of iPSC-derived clinical trial in a dish models designed for cardiovascular disease research. We also highlight how integrating iPSC technology with AI can accelerate the identification of viable therapeutic candidates, streamline drug screening, and pave the way toward more personalized medicine. Through this, we provide a comprehensive overview of the current landscape and future implications of iPSC and AI applications being navigated by the research community and pharmaceutical industry.