UM Leading Stem Cell Trial to Help Tiniest Heart Patients

Even with a trio of extensive surgeries over their first three years of life, just over half of children with hypoplastic left heart syndrome (HLHS) die by the time they’re five years old. But pediatric cardiac experts at the University of Maryland Medical Center (UMMC) are hoping an innovative clinical trial using stem cells to regenerate heart tissue — one of the first such efforts in the world — will offer these tiny patients and their families hope for a longer and stronger life.

Led by Sunjay Kaushal, MD, PhD, director of pediatric cardiac surgery at the University of Maryland Children’s Hospital, the phase 1 trial will gather 30 patients at UMMC over the next three to five years. Twenty of the patients — all born with this congenital heart condition — will be treated with stem cells in addition to standard treatment, while 10 patients will receive only standard therapy.

Affecting about 960 newborns in the United States each year, HLHS is a birth defect hindering normal blood flow through the heart. With the left side of the heart severely underdeveloped, the right side is tasked with pumping blood both into the lungs and into the rest of the body — responsibilities normally shared by both sides. Three surgeries are typically performed in these patients in their first three years to normalize blood flow, but about 45% die by age 5 from heart failure.

“We’re hoping this could be a game-changer for these patients in order to help them live longer, have a better quality of life and continue to have robust function of their hearts,” says Dr. Kaushal, also an associate professor of surgery at the University of Maryland School of Medicine. “It should improve their long-term outcomes.”

Rapid Results Possible

About four or five children come to UMMC each year for treatment of HLHS, often diagnosed while their mothers are still pregnant or just after birth using an echocardiogram. Dramatically smaller or nonexistent left heart chambers and valves are a hallmark of the condition, which requires early intervention to stabilize patients.

A series of cardiac surgeries are typically performed on these patients at two weeks, four months and three years of age. For this trial, children randomized for the additional treatment will receive stem cells injected into the area around their heart during their second surgery. It’s hoped that the stem cells — derived from tissue-compatible donors and supplied by sponsor University of Miami — will improve heart muscle function.

“We’ll know within the first year after that surgery if the stem cells will improve function of the right ventricle,” Dr. Kaushal explains. “Since this is a Phase 1 trial, we’ll be looking at the safety and feasibility of stem cell treatment in HLHS babies, but also preliminarily examine whether the right ventricle function improves over time.”

Leading Stem Cell Efforts in Congenital Heart Disease

UMMC’s effort represents one of the first clinical trials in the country examining whether stem cell therapy might be effective in congenital heart disease patients and will make University of Maryland a referral center for this type of intervention beyond HLHS, Dr. Kaushal says.

Stem cell therapy in patients with HLHS has been studied at only a handful of healthcare facilities around the world, including Japan. As the only site for this particular study in the United States, “it’s paramount to make sure these trials are well-executed and that they reach the safety and efficacy parameters set forth in the trial,” Dr. Kaushal says.

He adds, “We hope this will lay further groundwork for these therapies with these children and others with heart failure.”

For more information, please contact Dr. Kaushal at 410-328-5842.

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