Clinical landscape of human pluripotent stem cell-derived cardiomyocyte therapy
- 1Stanford Cardiovascular Institute, Stanford University School of Medicine, Stanford, CA 94305, USA
- 2Department of Medicine, Division of Cardiovascular Medicine, Stanford University School of Medicine, Stanford, CA 94305, USA
- 3Greenstone Biosciences, Palo Alto, CA 94304, USA
Abstract
Heart failure is a major clinical and economic burden that afflicts 60 million individuals worldwide. Guideline-directed medical therapies can slow disease progression, but they cannot restore the loss of cardiomyocytes. Over the past two decades, human pluripotent stem cell (hPSC)-based technology has emerged as a leading approach to overcome limited cardiac regenerative capacity, offering a scalable source of functional human cardiomyocytes. The field is now at a pivotal translational stage, as advances in differentiation and tissue engineering have enabled hPSC-based products to enter first-in-human clinical trials. In this review, we summarize the pathophysiological rationale for cell-based therapy in heart failure with reduced ejection fraction. Then, we examine the preclinical foundations of distinct hPSC-derived product formats, including cell suspensions, epicardial sheets, engineered heart muscle, and cardiac spheroids, each with distinct tradeoffs and translational considerations. We conclude by providing updates on ongoing and recently completed clinical trials, evaluating their safety, feasibility, and preliminary efficacy outcomes.
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BibTeX
@article{mui2026clinical,
title = {{Clinical landscape of human pluripotent stem cell-derived cardiomyocyte therapy}},
author = {Byron W.H. Mui and Evgenios Neofytou and Joseph C. Wu},
journal = {Cell Reports Medicine},
year = {2026},
month = aug,
doi = {10.1016/j.xcrm.2026.102962},
url = {https://doi.org/10.1016/j.xcrm.2026.102962},
note = {CC BY-NC-ND 4.0},
}
RIS
TY - JOUR
TI - Clinical landscape of human pluripotent stem cell-derived cardiomyocyte therapy
AU - Byron W.H. Mui
AU - Evgenios Neofytou
AU - Joseph C. Wu
PY - 2026
DA - 2026/08/05
JO - Cell Reports Medicine
DO - 10.1016/j.xcrm.2026.102962
UR - https://doi.org/10.1016/j.xcrm.2026.102962
AB - Heart failure is a major clinical and economic burden that afflicts 60 million individuals worldwide. Guideline-directed medical therapies can slow disease progression, but they cannot restore the loss of cardiomyocytes. Over the past two decades, human pluripotent stem cell (hPSC)-based technology has emerged as a leading approach to overcome limited cardiac regenerative capacity, offering a scalable source of functional human cardiomyocytes. The field is now at a pivotal translational stage, as advances in differentiation and tissue engineering have enabled hPSC-based products to enter first-in-human clinical trials. In this review, we summarize the pathophysiological rationale for cell-based therapy in heart failure with reduced ejection fraction. Then, we examine the preclinical foundations of distinct hPSC-derived product formats, including cell suspensions, epicardial sheets, engineered heart muscle, and cardiac spheroids, each with distinct tradeoffs and translational considerations. We conclude by providing updates on ongoing and recently completed clinical trials, evaluating their safety, feasibility, and preliminary efficacy outcomes.
SN - 2666-3791
ER -