Novo Nordisk Foundation Center for Stem Cell Medicine (reNEW), University of Copenhagen, 2200 Copenhagen N, Denmark.
Department of Neuroscience, University of Copenhagen, 2200 Copenhagen N, Denmark.
Development. 2024 Apr 1;151(7). doi: 10.1242/dev.202067. Epub 2024 Apr 2.
The translational stem cell research field has progressed immensely in the past decade. Development and refinement of differentiation protocols now allows the generation of a range of cell types, such as pancreatic β-cells and dopaminergic neurons, from human pluripotent stem cells (hPSCs) in an efficient and good manufacturing practice-compliant fashion. This has led to the initiation of several clinical trials using hPSC-derived cells to replace lost or dysfunctional cells, demonstrating evidence of both safety and efficacy. Here, we highlight successes from some of the hPSC-based trials reporting early signs of efficacy and discuss common challenges in clinical translation of cell therapies.
在过去的十年中，转化干细胞研究领域取得了巨大的进展。分化方案的开发和完善现在使得能够以高效且符合良好生产规范的方式从人多能干细胞（hPSC）中产生多种细胞类型，如胰腺β细胞和多巴胺能神经元。这导致了使用 hPSC 衍生细胞来替代丢失或功能失调的细胞的几项临床试验的启动，证明了安全性和有效性的证据。在这里，我们重点介绍了一些基于 hPSC 的试验的成功案例，这些试验报告了早期疗效的迹象，并讨论了细胞治疗临床转化中的常见挑战。
