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Updated: Apr 25, 2026

Pluripotent Stem Cell Derived Cardiac Cells for Myocardial Repair
Published on: February 3, 2017
Translating stem cell research to cardiac disease therapies: pitfalls and prospects for improvement
Michael R Rosen1, Robert J Myerburg2, Darrel P Francis3
1Departments of Pharmacology and Pediatrics, Columbia University Medical Center, New York, New York.
Abstract:
Over the past 2 decades, there have been numerous stem cell studies focused on cardiac diseases, ranging from proof-of-concept to phase 2 trials. This series of papers focuses on the legacy of these studies and the outlook for future treatment of cardiac diseases with stem cell therapies. The first section by Drs. Rosen and Myerburg is an independent review that analyzes the basic science and translational strategies supporting the rapid advance of stem cell technology to the clinic, the philosophies behind them, trial designs, and means for going forward that may impact favorably on progress. The second and third sections were collected as responses to the initial section of this review. The commentary by Drs. Francis and Cole discusses the review by Drs. Rosen and Myerburg and details how trial outcomes can be affected by noise, poor trial design (particularly the absence of blinding), and normal human tendencies toward optimism and denial. The final, independent paper by Dr. Marbán takes a different perspective concerning the potential for positive impact of stem cell research applied to heart disease and future prospects for its clinical application. (Compiled by the JACC editors).
Insights
This review examines the legacy and future of stem cell therapies for cardiac diseases. It analyzes basic science, translational strategies, and clinical trial designs, considering their impact on patient outcomes.
Area of Science:
- Cardiology
- Regenerative Medicine
- Translational Science
Background:
- Extensive stem cell research for cardiac diseases over two decades.
- Focus on proof-of-concept to phase 2 clinical trials.
- Need to evaluate the legacy and future of these therapies.
Purpose of the Study:
- Analyze the scientific and translational progress of stem cell therapies for heart disease.
- Critically assess clinical trial designs and their outcomes.
- Discuss future prospects for stem cell application in cardiology.
Main Methods:
- Independent review of basic science and translational strategies.
- Analysis of clinical trial philosophies and designs.
- Commentary and response to initial review, addressing trial limitations.
Main Results:
- Stem cell technology has advanced rapidly towards clinical application.
- Trial outcomes can be influenced by design flaws, lack of blinding, and human bias.
- Differing perspectives exist on the potential positive impact and future clinical application.
Conclusions:
- Critical evaluation of stem cell research in cardiology is essential.
- Improved trial design and bias mitigation are crucial for accurate outcome assessment.
- Future progress depends on robust scientific evidence and strategic clinical development.
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