Clinical Translation of Pluripotent Stem Cell-based Therapies: Successes and Challenges
Overview
Affiliations
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.
Espana-Lopez A, Fernandez-Valades R, Cubiles E, Garzon I, Martin-Piedra M, Carriel V BMJ Open. 2024; 14(12):e093491.
PMID: 39638596 PMC: 11624797. DOI: 10.1136/bmjopen-2024-093491.