TY - JOUR

T1 - Clinical translation of pluripotent stem cell-based therapies

T2 - successes and challenges

AU - Christiansen, Josefine Rågård

AU - Kirkeby, Agnete

N1 - © 2024. Published by The Company of Biologists Ltd.

PY - 2024

Y1 - 2024

N2 - 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.

AB - 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.

KW - Humans

KW - Pluripotent Stem Cells

KW - Cell Line

KW - Cell Differentiation

KW - Cell- and Tissue-Based Therapy

KW - Stem Cell Research

U2 - 10.1242/dev.202067

DO - 10.1242/dev.202067

M3 - Journal article

C2 - 38564308

VL - 151

JO - Development

JF - Development

SN - 0950-1991

IS - 7

M1 - dev202067

ER -