In the US, approximately 1,000 babies per year are born with hypoplastic left heart syndrome and currently have a mortality rate of about 33% within the first year of life. Despite surgical advancements, about half of those children make it to their 5th birthday. The poor clinical outcomes are predominantly due to right ventricular dysfunction leading to heart failure. Presently, heart transplantation is the only clinically viable option even though there is not a significant improvement in survival rates. Innovative, adjunctive therapies to regenerate and remodel the right ventricle have become a crucial, yet unmet medical need in these challenging patients.

After allogeneic mesenchymal stem cells (Allo-MSCs) therapy was proven to be safe and effective in animal studies and adults with severe heart conditions, Dr. Sunjay Kaushal, associate professor of surgery and co-director of the Children's Heart Program at the University of Maryland Children's Hospital, initiated a Phase I/IIb clinical trial to test the therapeutic effects of the allogeneic mesenchymal stem cells in children with HLHS. The Allo-MSCs are directly injected into the right ventricular myocardium during the second of 3 standard operations (Glenn procedure/Bidirectional Cavopulmonary Anastomosis)—when the baby is about 4 months of age. This trial is intended to address the remaining obstacles of long-term cardiac function in HLHS patients. We propose that a stem cell-based therapy for these patients may prevent right heart failure and therefore improve survival outcomes and reduce the need for transplantation.

To date, all currently enrolled patients are showing early signs of safety and feasibility. The UMCH Children's Heart Research Lab is one of the only labs in the world to begin testing these stem cells clinically in children. This trial will be testing a total of 30 patients, and after proving that this stem cell therapy works in strengthening the heart function, we will be able to treat many other children with severe heart conditions.

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In a first-in-children randomized clinical study, medical researchers at the University of Maryland School of Medicine (UM SOM) and the Interdisciplinary Stem Cell Institute (ISCI) at the University of Miami Miller School of Medicine have begun testing to see whether adult stem cells derived from bone marrow benefit children with the congenital heart defect hypoplastic left heart syndrome (HLHS).

Read more about this treatment in First U.S. Babies Treated In Unique Study of Adult Stem Cell Therapy for Congenital Heart Disease

About The University of Maryland Children's Heart Research Lab

The University of Maryland Children's Heart Program Laboratory researchers are interested in studying the biology of resident cardiac stem cells in children. The lab's findings first described the presence of cardiac stem cells in the myocardium of congenital heart disease patients and showed that, when isolated and expanded, these injected cells can regenerate. Learn more about UMCH Heart Research Lab.

About The University of Maryland Children's Hospital (UMCH)

The children's heart program specializes in providing continuum of care for babies born with heart conditions from birth all the way through childhood and into adulthood. UMCH has 145 total licensed beds; 306 employees/staff; 120+ attending physicians/faculty, 87 resident physicians; 4,451 patient admissions; 17,213 ER visits; 1,078 inpatient surgical cases; 2,233 outpatient surgical cases and 45,055 outpatient visits. Learn more about UMCH.

Call 410-328-3778 to refer a patient or learn more at Stem Cell Research – HLHS.