The Todd and Karen Wanek Family Program for Hypoplastic Left Heart Syndrome (HLHS) is a research program with the goal of delaying or preventing heart failure for individuals with HLHS.

To learn more or to participate, email HLHS@mayo.edu.

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Mar 15, 2016

Japanese Cell Therapy Study Demonstrates Impressive Results in the HLHS Field

By Suzanne R. Ferguson, @suzannerferguson

In the first clinical study of its kind, researchers at Okayama University Hospital in Japan have published groundbreaking results using stem cells in HLHS children. The researchers published two papers—one with patient results at 18 months and then again at 3 years. (Read the full studies here and here). The study is not only the first to demonstrate safety and successful use of stem cells in HLHS children, but it is also the first clinical trial to use stem cells in congenital heart disease children. It has the potential to dramatically impact future treatment for HLHS children. ekg The Study

The study included a total of 14 HLHS patients: 7 patients received cells originating from cardiac tissue, and 7 patients received no cells, as the control. The cardiac tissue cells were first extracted during each individual’s surgery. Next, the cells were processed and then injected into the heart just 33 days after the procedure. The patients each received 300,000 cells per kilogram during the injection.

The Results

The results published by the team reveal both the safety and preliminary effectiveness of injecting cells into the hearts of HLHS patients. Not only is this study truly pioneering in the HLHS field, but also in the congenital heart disease world as a whole. There have been no other studies that have examined the safety or effectiveness of using cell-based treatments in the pediatric population. The landmark paper has also demonstrated that the younger the patients are, the better their outcomes are with cell-based treatments.

Currently, the same researchers from Okayama University Hospital are focused on another clinical trial looking into the overall efficacy of these cells. Establishing the efficacy of this procedure will bring the HLHS world one step closer to finding new treatment options for this disease.

Our Work

Japan is not alone! We are currently conducting our own clinical trials using cell-based therapies in our work here at Mayo. In fact, some of our work includes collecting the cells from the child’s cord blood, processing them, and then injecting the cells into the right ventricle during the Glenn procedure. We have published preliminary results from our first case study, which can be found here. For more information on the work we are doing, please contact us at HLHS@mayo.edu.

Congratulations to the Okayama University Hospital for their promising work in the HLHS field and we look forward to what the near future holds for cell-based treatments!


The Todd and Karen Wanek Family Program for Hypoplastic Left Heart Syndrome (HLHS) is a collaborative network of specialists bonded by the vision of delaying or preventing heart failure for individuals affected by congenital heart defects including HLHS. The specialized team is addressing the various aspects of these defects by using research and clinical strategies ranging from basic science, to diagnostic imaging to regenerative therapies.
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