The Allogeneic Heart Stem Cell Trial in Hypoplastic Left Heart Syndrome Patients

DESCRIPTION provided by applicant One of the most complex forms of congenital heart disease hypoplastic left heart syndrome HLHS affects an average of in every newborns with a mortality rate of up to percent during the first year of life The overall goal f our research is to develop safe and effective ways to improve short term and long term survival rates in babies born with HLHS We aim to introduce a novel cell based therapeutic strategy in the three stage surgical procedure that HLHS patients typically undergo Our hypothesis is that injected allogeneic cardiosphere derived cells CDCs will boost and regenerate the myocardium of the single right ventricle in HLHS patients improving right ventricular function and thus short and long term clinical outcomes The aims of this Phase I clinical trials are to
evaluate the feasibility and safety of direct intra coronary injection of allogeneic CDCs in these HLHS patients at the time of cauterization for their second operation This proposal will translate
a series of basic and pre clinical observations into a potentially paradigm shifting therapy to meet an unmet medical need in HLHS patients In addition this study will bring a deeper insight into the biology of stem cells in human patients which have never been studied before in pediatric congenital heart patients

PUBLIC HEALTH RELEVANCE Hypoplastic left heart syndrome is one of the most complex forms of congenital heart disease with a reported incidence of of children born with congenital heart disease Despite these strides in medical care during the last twenty years the mortality rate of these infants remain at during the first year of life because mostly of right ventricle dysfunction This proposal will translate a series of basic and pre clinical observations into a potentially paradigm shifting cell based therapy to improve the right ventricular function and thus improve clinical outcomes