Stem Cell Therapy For Congenital Heart Disease: Toward Translation
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AbstractStem cell therapy has the optimistic goal of regenerating the myocardium as defined by regrowth of lost or destroyed myocardium. As applied to patients with heart failure, many confuse or limit the regenerative definition to just improving myocardial function and/or decreasing myocardial scar formation which may not be the most important clinical outcome to achieve in this promising field of molecular medicine. Many different stem cell-based therapies have been tested and have demonstrated a safe and feasible profile in adult patients with heart failure but with varied efficacious endpoints reported. While not been achieved as of yet, the encompassing goal to regenerate the heart is still believed to be within reach using these cell-based therapies in adult patients with heart failure as the first generation therapies are now being tested in different phases of clinical trials. Similar efforts to foster the translation of stem cell therapy to children with heart failure have, however, been limited. With this review, we aim to summarize the findings from preclinical models and clinical experience to date that have focused on the evaluation of stem cell therapy in children with heart failure. Finally, we present methodological considerations pertinent to the design of a stem cell-based trial for children with heart failure since they represent a population of patients with very different sets of issues when compared to adult patients. As has been taught by many learned clinicians, children are not small adults!