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Game-changing Stem Cell Trial for Children with Congenital Heart Disease

July 06, 2021

Phase IIb stem cell trial aims to improve outcomes for children with hypoplastic left heart syndrome

 

A recently launched Phase IIb randomized clinical trial, led by Sunjay Kaushal, MD, PhD, at Ann & Robert H. Lurie Children’s Hospital of Chicago, uses donor stem cells injected into the patient’s heart during surgery, which is expected to improve right ventricular function of children born with a congenital heart disease called hypoplastic left heart syndrome. Based on a decade of Dr. Kaushal’s pioneering lab research, clinical experience in adults and positive safety results in the Phase I trial, this stem cell-based strategy promises to extend life expectancy for these children.

Hypoplastic left heart syndrome is one of the most complex forms of congenital heart disease, with a reported incidence of 0.2 per 1000 live births. These patients show a serious lack of left sided heart structure development. Once a universally fatal diagnosis, dramatic improvements in three staged surgeries now allow the single right ventricle of the heart to functionally support the circulation. Despite these strides in medical care, these surgeries are not curative. The right ventricle tends to become enlarged and not work properly, leading to heart failure.

The mortality rate of these infants remains as high as 25 to 35 percent during the first year of life. Those surviving childhood are likely to progress towards heart transplantation. However, survival after heart transplantation in these patients is poor and innovative treatments are desperately needed.

“We anticipate that the injected stem cells during the second surgery that infants with hypoplastic left heart syndrome undergo at around 6 months of age will keep their right ventricle from getting too large and so improve their heart function,” said Dr. Kaushal, lead Principal Investigator and Division Head of Cardiovascular-Thoracic Surgery at Lurie Children’s, as well as Professor of Surgery at Northwestern University Feinberg School of Medicine. “Our preliminary results are striking, and we expect that this treatment will be potentially a game-changer, potentially delaying or preventing the need for heart transplantation and helping these children live longer lives.”

Thirty-eight patients will be enrolled at seven clinical sites. The study is randomized so that during the second surgery, half the patients will receive the stem cell treatment and the other half will receive standard of care. The two groups will receive the same care thereafter and researchers will assess the right ventricle performance. Patients will be followed for one year.

“We foresee that many of the lessons learned developing and translating this stem cell therapy to the clinic will apply as we expand its use to improve outcomes for children with other types of congenital heart disease,” said Dr. Kaushal, who also holds the A.C. Buehler Professorship in Surgery.

The clinical trial is sponsored by the National Heart Lung and Blood Institute. Dr. Kaushal and colleagues hope to receive full FDA approval for this stem cell therapy in five to six years.

Research at Ann & Robert H. Lurie Children’s Hospital of Chicago is conducted through the Stanley Manne Children’s Research Institute. The Manne Research Institute is focused on improving child health, transforming pediatric medicine and ensuring healthier futures through the relentless pursuit of knowledge. Lurie Children’s is ranked as one of the nation’s top children’s hospitals by U.S. News & World Report. It is the pediatric training ground for Northwestern University Feinberg School of Medicine. Last year, the hospital served more than 220,000 children from 48 states and 49 countries.