小児心不全に対する再生医療からのアプローチ

抄録

In July 2010, the domestic law for organ transplants was revised to enable children under 15-year of age to donate their organs if the parents had given consent in the case of brain death. After this revision, the substantial number of pediatric heart transplantations was expected to increase; however only 7 cases were performed during these 5 years. The problems related to the shortage of organ donors and long waiting times for orthotopic heart transplantations have not yet been resolved. As a result, new treatment strategies including innovative mechanical devices and regenerative therapies for pediatric heart failure, are critically needed. Accumulated evidence of cardiac regeneration strategies using various types of somatic stem cells have revealed the efficacy of stem cell therapies for heart failure. Initial results of the Transcoronary Infusion of Cardiac Progenitor cell infusion in patients with single ventricle physiology (TICAP) phase I study (NCT01273857) conducted at Okayama University (2011-2013) have shown that intracoronary infusion of cardiosphere-derived cells (CDCs) following staged palliation was feasible and safe to treat children with hypoplastic left heart syndrome. Currently, a randomized phase II trial (Cardiac Progenitor Cell Infusion to treat Univentricular Heart Disease: PERSEUS, NCT01829750) is ongoing at our institution to verify the therapeutic efficacy of CDC infusion in patients with univentricular heart diseases. From 2016, we will conduct a countrywide sponsor-initiated multicenter clinical trial to construct this therapeutic strategy for children with heart failure as an insurance coverage treatment. In this review, we discuss the problems involved with end-stage pediatric heart failure and the current status of stem cell therapy for heart failure in children.