Cord Blood & Hypoplastic Left Heart Syndrome

Hypoplastic Left Heart Syndrome and Cord Blood

What is Hypoplastic Left Heart Syndrome (HLHS)?

Hypoplastic Left Heart Syndrome (HLHS) is a severe congenital heart defect in which the left side of the heart is underdeveloped. As a result, the baby doesn’t receive enough oxygen-rich blood, forcing them to undergo three surgeries early in life to reconstruct the heart.


Cord Blood’s Role in Clinical Trials For HLHS

Results were recently released for The Mayo Clinic’s Phase I Clinical Trial using cord blood in children with HLHS. The study was designed to establish safety and feasibility of using a baby’s own cord blood cells to help improve heart function in those with HLHS. 

Ten newborns with HLHS will participate in the trial. Following birth, their umbilical cord blood will be collected, processed and cryopreserved (all by the Mayo Clinic) until needed. During the baby’s second surgery — typically performed at 4 to 6 months of age — the stem cells from the cord blood will be injected into the heart muscle.

Phase I Clinical Trial Results

HLHS & Cord Blood

Researchers found that cord blood injections in conjunction with surgery were both safe and feasible for the infant participants. Importantly, none of the children were found to have any significant safety concerns at six months post-surgery. 

“Our hopes are that this groundbreaking research will lead to stem cell therapy strengthening these babies’ hearts while delaying or even preventing the need for a heart transplant in some,” says Harold Burkhart, M.D., a pediatric cardiothoracic surgeon at OU Medicine in Oklahoma and first author on the paper. 

Details were published in The Journal of Thoracic and Cardiovascular Surgery. Read the Mayo Clinic's press release and watch one family’s story here.

Next Steps: Phase II Clinical Trial Planning Underway

Planning for Phase II of this Clinical Trial is underway at seven hospitals across the country through the Mayo Clinic’s HLHS Consortium. The study will include fifty infant participants, which will further test safety and examine the effectiveness of using cord blood to improve heart function. Please note that this trial is only available to those who have stored with the Mayo Clinic’s facility for this specific purpose.

Cord Blood and Ongoing Regenerative Medicine Research

The therapeutic potential of cord blood continues to grow.

As research continues to try to harness the healing potential of cord blood stem cells in new ways, we continue to be encouraged and excited. As with all advancements in medicine, discoveries are the result of dedicated researchers and time. Other areas of cord blood use for regenerative medicine clinical trials include:

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Cerebral Palsy 
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