Cardiac stem cell therapy for cardiac repair.

opinion statementThe discovery of adult cardiac stem cells (CSCs) and their potential to restore functional cardiac tissue has fueled unprecedented interest in recent years. Indeed, stem-cell-based therapies have the potential to transform the treatment and prognosis of heart failure, for they have the potential to eliminate the underlying cause of the disease by reconstituting the damaged heart with functional cardiac cells. Over the last decade, several independent laboratories have demonstrated the utility of c-kit+/Lin- resident CSCs in alleviating left ventricular dysfunction and remodeling in animal models of acute and chronic myocardial infarction. Recently, the first clinical trial of autologous CSCs for treatment of heart failure resulting from ischemic heart disease (Stem Cell Infusion in Patients with Ischemic cardiOmyopathy [SCIPIO]) has been conducted, and the interim results are quite promising. In this phase I trial, no adverse effects attributable to the CSC treatment have been noted, and CSC-treated patients showed a significant improvement in ejection fraction at 1 year (+13.7 absolute units versus baseline), accompanied by a 30.2 % reduction in infarct size. Moreover, the CSC-induced enhancement in cardiac structure and function was associated with a significant improvement in the New York Heart Association (NYHA) functional class and in the quality of life, as measured by the Minnesota Living with Heart failure Questionnaire. These results are exciting and warrant larger, phase II studies. However, CSC therapy for cardiac repair is still in its infancy, and many hurdles need to be overcome to further enhance the therapeutic efficacy of CSCs.