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The Children's Heart Program at the University of Maryland Medical Center is leading a first-of-its-kind clinical trial that will use stem cells to treat hypoplastic left heart syndrome (HLHS), a congenital heart disease. HLHS leaves the left ventricle underdeveloped, and this procedure strengthens the function of the right side of the patient's heart.

Vision of the trial

Led by Sunjay Kaushal, MD, PhD, the HLHS Clinical Trial tests the effectiveness of allogeneic mesenchymal stem cells in conjunction with the second palliative surgery, the Glenn (bidirectional cavopulmonary anastomosis) operation. These cells have been shown to remarkably improve cardiac function in adult hearts but have never been tested on the pediatric population until now.

After performing rigorous studies, the Food and Drug Administration (FDA) granted approval for this trial.

In this Phase I trial, 30 HLHS patients will be enrolled at the time of the second palliative operation. The first 10 patients will receive the stem cells, and their results will be used to determine feasibility and safety.

After enrollment for the initial group is completed, the following 20 participants will be enrolled on a 1:1 basis, with 10 receiving the stem cell treatment and the other 10 being viewed as a control group (not receiving the stem cells).

The HLHS Clinical Trial will assess two major endpoints:

  • Primarily to look at the safety and feasibility of using stem cells to help HLHS patients
  • Secondarily to measure if the stem cells improve the functionality of the right ventricle

Treatment with allogeneic mesenchymal stem cells aims to strengthen the right ventricle, improving the function of the heart and thus the patient's quality of life. If the treatment is effective, this ground-breaking procedure could be applied to other complicated congenital heart diseases, such as dilated cardiomyopathy.

What are stem cells?

Stem cells are unspecialized cells that can regenerate through cell division and have the potential to develop into many different cell types under certain conditions, including those with a more specialized function such as cardiac muscle cells.

This trial uses allogeneic mesenchymal stem cells, which are from donated human bone marrow cells. These particular cells have the remarkable capability to escape immune response, are readily accessible, safe to use and proven to be effective in adult patients with heart conditions.

In this trial, the stem cells will be injected in the right ventricle during the second palliative surgery for HLHS patients. If effective, the cells will act as a biological factory, secreting proteins that will make the right side of the heart stronger.

Participation and Eligibility

To view eligibility requirements, visit the HLHS Clinical Trial’s page.

Call 410-328-3778 for participation information.

How you can help

Philanthropy allows us to advance cutting-edge research, teaching and patient care. To make a gift in support of this groundbreaking research, please visit the University of Maryland Medical System Foundation secure online giving page and select Pediatric Cardiovascular Research Fund in the dropdown menu.