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Gene therapy and stem cell therapy for cardiovascular diseases today: a model for translational research

Nature Clinical Practice Cardiovascular Medicine volume 4, pages S1–S8 (2007)Cite this article

Abstract

Clinical trials looking at ways to promote myocardial regeneration have reported that the administered therapies have either neutral effects or modest benefits of questionable impact. These somewhat disappointing results should emphasize the need for translational research, with bidirectional feedback between the basic research laboratory and the clinical arena. Such a translational pathway is illustrated by the quest to find an effective therapy for restenosis, which culminated in the development of sirolimus. At this point a move away from the bedside and a return to the bench seems necessary to better understand the mechanisms of action of progenitor cells and stimulating factors. Without such basic knowledge research might be prematurely discouraged and the opportunity to fully understand the true potential of cardiovascular regenerative therapy might be missed.

Key Points

  • Recent myocardial regeneration trials have reported no or questionable beneficial effects

  • Interpretation of these studies must be cautious owing to the complexity of the factors involved (cell lineages, growth factors, timing of therapy, measured surrogate endpoints, etc.)

  • Lack of meaningful benefits in preliminary studies should not be necessarily considered as a failure of the therapy

  • Extensive work in the basic research laboratory seems now appropriate to elucidate the most adequate targets, and to understand the true mechanisms of action of different types of regenerative therapy

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Figure 1: Diagrammatic representation of the complexity of different factors (genes, growth factors and cyclins) involved in cell proliferation
Figure 2: Combined PET and multidetector-row CT imaging of a patient with diabetes from the FREEDOM trial
Figure 3: Myocardial regeneration trials: myocardial components, progenitor cell lineages, diseases, surrogate endpoints and imaging modalities

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Authors and Affiliations

  1. V Fuster is Professor of Medicine and Cardiology, and Director, and J Sanz is Assistant Professor of Medicine/Cardiology, at the Zena and Michael A Wiener Cardiovascular Institute and The Marie-Josee and Henry R Kravis Center for Cardiovascular Health, Mount Sinai School of Medicine, New York, USA.,

    Valentin Fuster & Javier Sanz

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  1. Valentin Fuster
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  2. Javier Sanz
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Correspondence to Valentin Fuster.

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The authors declare no competing financial interests.

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Fuster, V., Sanz, J. Gene therapy and stem cell therapy for cardiovascular diseases today: a model for translational research. Nat Rev Cardiol 4 (Suppl 1), S1–S8 (2007). https://doi.org/10.1038/ncpcardio0737

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  • DOI: https://doi.org/10.1038/ncpcardio0737

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