Clinical Trial: Could Cord Blood Stem Cells Help Treat Heart Defects?

Building a bigger, better, stronger heart for children born with congenital heart defects (CHDs) could one day include stem cells as a treatment option. CHDs affect nearly 1% of―or about 40,000―births per year in the United States.[1] About 25% of babies with a CHD have a critical CHD. Infants with critical CHDs generally need surgery or other procedures in their first year of life.[2]

A new FDA-approved study is focusing on the potential of stem cell therapy for pediatric patients at the Mayo Clinic. The trial is designed for children born with a severe congenital heart defect called hypoplastic left heart syndrome (HLHS), where the left side of the heart is underdeveloped. As a result, the baby’s body doesn’t receive enough oxygen, which could lead to heart failure. The current treatment option for HLHS is multiple heart surgeries. [3]

Now, 10 newborn patients with HLHS can participate in the Mayo Clinic study, which will monitor the safety of using stem cells derived from their own umbilical cord blood. The study will also investigate if there is improved cardiac function. As part of the study, the cord blood is collected at the child’s birth and processed at the Mayo Clinic so that the stem cells are collected and stored, then re-injected into the right side of the heart during the child’s second surgery. The long term hope is that the stem cells might help the existing heart get stronger, or even delay the time to transplant.[4]

This initial study aims to enroll 10 patients, and the first case report has already been published. [5]

Here’s hoping for a successful outcome!


[1] Hoffman JL, Kaplan S. The incidence of congenital heart disease. J Am Coll Cardiol. 2002;39(12):1890-1900. Reller MD, Strickland MJ, Riehle-Colarusso T, Mahle WT, Correa A. Prevalence of congenital heart defects in Atlanta, 1998-2005. J Pediatr. 2008;153:807-13.

[2] Oster ME, Lee KA, Honein MA, Riehle-Colarusso T, Shin M, Correa A. Temportall trends in survival among infants with critical congenital heart defects. Pediatrics. 2013 May;131(5):e1502-8.doi: 10.1542/peds.2012-3435. Epub 2013 Apr 22.



[5] Burkhart HM, Qureshi MY, Peral SC, O’Leary PW, Olson TM, Cetta F, Nelson TJ, and the Wanek Program Clinical Pipeline Group, Regenerative therapy for hypoplastic left heart syndrome: First report of intraoperative intramyocardial injection of autologous umbilical cord blood-derived cells, The Journal of Thoracic and Cardiovascular Surgery (2014), doi: 10.1016/ j.jtcvs.2014.10.093.


Leave a Reply

Your email address will not be published. Required fields are marked *