Stem Cells: Clinical Trials Results The End of the Beginning or the Beginning of the End?

Atta      Behfar; Ruben      Crespo-Diaz; Timothy      J. Nelson; Andre      Terzic; Bernard      J. Gersh

Abstract

With increasing focus on the advance towards curative solutions, it is hard not to be excited by the potential of stem cell-based therapy. Application of the stem cell paradigm to cardiovascular medicine has fostered the evolution of novel approaches aimed at reversing injury caused by ischemic and non-ischemic cardiomyopathy. The feasibility and safety of stem cell use has been established in over 3, 000 patients with either recent myocardial infarction or chronic organ failure. Nonetheless, the efficacy of stem cell therapy continues to remain in question. Initial clinical trials have focused on evaluation of multiple adult stem cell phenotypes in their unaltered, naïve state as a “first generation” resource for repair. Though significant strides in perfecting delivery of these biologics to the diseased heart have been achieved, the benefits with regard to myocardial functional recovery have been modest at best. One approach towards optimizing outcome may lie upon preemptive guidance of stem cells down the pathway of myocyte regeneration. As seen with pharmacotherapeutics in the last century, successful translation of “second generation” biotherapeutics in the 21st century will require close integration of a community of practice and science to ensure broad application of this emerging technology in the treatment of heart disease.

Keywords: Regenerative medicine, Cell therapy, Heart Disease, Myoblast, Bone marrow mononuclear cell, Endothelial progenitor, Mesenchymal stem cell, Induced pluripotency stem cell.