Stem cells: will they cure pediatric heart failure?

The purpose of this review is to provide an overview of the state of cardiac regenerative medicine, including the unique opportunities and challenges in its application to pediatric patients.

There has been a rapid proliferation of clinical studies using stem cells in adults with heart failure, yet little convincing evidence of clinically significant improvement. Readers will develop an understanding of the current limitations of stem cell treatments and the challenges to be overcome before they can achieve successful clinical translation.

Clinical trials in cardiac regeneration using stem cells are advancing rapidly despite clear knowledge of mechanism and rigorous evidence in animal models. The potential for cardiac regeneration in children may be greater than in adults, given the smaller degree of scar present in nonischemic heart disease and the greater potential of the younger heart for repair. However, similar to adult trials, there has yet to be convincing evidence of a positive effect in pediatric patients, and rigorous controlled studies are still lacking. There is still much biology to be learned in cardiac regeneration; future clinical trials in children should be based on solid evidence in animal models of both efficacy and safety.