ATLANTA (February 28, 2020) – Clinicians and researchers at Children’s Healthcare of Atlanta, the University of Maryland Medical Center and the University of Miami will lead a novel clinical trial that applies stem cell therapies to pediatric heart defects.
In this groundbreaking trial, surgeons at Children’s will implant patient-derived stem cells into the hearts of babies with hypoplastic left heart syndrome (HLHS) while they are undergoing surgical repair. The trial, funded by The Marcus Foundation, received clearance from the FDA last summer and is in the early stages of enrollment of newly delivered babies diagnosed with HLHS.
The trial, called Autologous Cardiac Stem Cell Injection in Patients with Hypoplastic Left Heart Syndrome: An open label pilot study (CHILD), is one of three stem cell clinical trials for HLHS in the country and will be led by William Mahle, MD, Chief of Cardiac Services at Children’s; Michael Davis, PhD, Director of the Children’s Heart Research and Outcomes (HeRO) Center and Professor of Biomedical Engineering at the Georgia Institute of Technology and Emory University; Sunjay Kaushal, MD, PhD, Professor of Surgery at the University of Maryland School of Medicine and Director of Pediatric and Adult Congenital Cardiac Surgery at the University of Maryland Medical Center; and Joshua Hare, MD, Director of the Interdisciplinary Stem Cell Institute at the University of Miami Miller School of Medicine.
Babies born with HLHS do not have a functioning left side of the heart and require surgery shortly after birth. They undergo three surgeries over the course of three years to redirect blood flow through the right side of the heart. In these babies, the single ventricle, doing the work of two ventricles, is under a lot of stress and can fail.
“For a baby with a congenital heart defect like HLHS, we can try to strengthen the heart and prevent deterioration of its existing tissues while the baby is young and the heart is still developing,” Davis said.
According to Kaushal, who has conducted extensive research to analyze whether donor stem cells can strengthen the pumping ability of HLHS hearts, “This new study may open even more possibilities for babies with HLHS.”
During the trial, a patient’s own stem cells will be injected into the heart with the intention of fortifying the working right ventricle, preventing heart failure, and, hopefully, preventing or delaying the need for a heart transplant. The cells are collected during the baby’s first surgery within the first few days of life. They are then implanted into the heart three to five months later during the second of the three HLHS surgeries.
“Congenital heart disease is the world’s most common birth defect, affecting nearly nine in every 1,000 babies born,” Davis said. “At Children’s, we have a large volume of young cardiac patients with these congenital issues. Our center is dedicated to developing new technologies that could impact how we care for these patients.”
Davis and his lab pioneered the advancement of stem cell research and its application in clinical trials, predictive medicine models, and 3D printing technology. He developed the concept of using stem cells to potentially treat congenital heart defects several years ago when studying small amounts of cardiac tissue routinely removed from children during bypass surgery. His team of researchers found that the young cells had reparative qualities and released healing proteins when injected into damaged tissue. Davis also recently published a paper showing that these same cells could be 3D printed into cardiac patches; he hopes to move this toward the clinic as well.
"We are so excited by the groundbreaking use of stem cell therapies that Dr. Davis is leading in his lab,” said Lucky Jain, MD, Chief Academic Officer at Children’s and Chair of the Department of Pediatrics at the Emory School of Medicine. “Once again we are seeing the power of our partnership with the country’s leading research institutions and its potential impact on kids with complex medical conditions. This is a great example of how research can create more effective care at the bedside and why our collaboration with Georgia Tech and Emory is so important for the future of pediatric care.”
In the future, Davis and his research partners hope to gather quantitative data on the behavior of stem cells to create a large data repository of cell signals. By studying the signals, otherwise known as protein secretions of the cell, the team aims to determine how effective certain cells are in treating diseases.
“The goal is to identify the best characteristics of different cells and which diseases they target,” Davis said. “This clinical trial could be an early step in creating personalized approaches to stem cell therapy for every patient.”