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Updated: Aug 7, 2026
Generation and Expansion of Human Cardiomyocytes from Patient Peripheral Blood Mononuclear Cells
Published on: February 12, 2021
Clinical landscape of human pluripotent stem cell-derived cardiomyocyte therapy
Byron W H Mui1, Evgenios Neofytou1, Joseph C Wu2
1Stanford Cardiovascular Institute, Stanford University School of Medicine, Stanford, CA 94305, USA; Department of Medicine, Division of Cardiovascular Medicine, Stanford University School of Medicine, Stanford, CA 94305, USA.
Insights
Human pluripotent stem cell (hPSC) technology offers a promising regenerative approach for heart failure by providing functional cardiomyocytes. Current clinical trials are evaluating the safety and efficacy of hPSC-derived products for cardiac repair.
Area of Science:
- Regenerative Medicine
- Cardiology
- Stem Cell Biology
Background:
- Heart failure affects 60 million people globally, with current therapies unable to restore lost cardiomyocytes.
- Human pluripotent stem cell (hPSC) technology presents a scalable solution for generating functional human cardiomyocytes to address cardiac regenerative limitations.
Purpose of the Study:
- To review the rationale for cell-based therapy in heart failure with reduced ejection fraction.
- To examine preclinical data for various hPSC-derived cardiac product formats.
- To update on the status and outcomes of clinical trials using hPSC-based therapies.
Main Methods:
- Literature review of pathophysiological rationale for cell-based therapy.
- Analysis of preclinical studies on hPSC-derived cell suspensions, epicardial sheets, engineered heart muscle, and cardiac spheroids.
- Summary of ongoing and completed first-in-human clinical trials.
Main Results:
- hPSC technology provides a scalable source of cardiomyocytes, crucial for cardiac regeneration.
- Different hPSC-derived product formats (cell suspensions, epicardial sheets, engineered heart muscle, cardiac spheroids) have unique translational considerations.
- Early clinical trials are assessing the safety, feasibility, and preliminary efficacy of these regenerative approaches.
Conclusions:
- hPSC-based therapies are advancing towards clinical application for heart failure.
- Further research and clinical evaluation are necessary to optimize hPSC-derived products for cardiac repair.
- Cell-based therapies hold significant potential to improve outcomes for patients with heart failure.
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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