June 18, 2025

A study led by researchers from the University of Wisconsin—Madison and Mayo Clinic has demonstrated the potential of stem cell therapy in treating congenital heart defects. The study was supported by the Todd and Karen Wanek Family Program for Hypoplastic Left Heart Syndrome (HLHS) and an NIH Grant to the Wisconsin National Primate Research Center.

The research, published in Cell Transplantation, shows that heart muscle cells derived from human induced pluripotent stem cells can safely integrate into the hearts of monkeys with right ventricular pressure overload.

Key findings of the study include:

  • Successful integration of transplanted cells into the host myocardium
  • Feasibility and safety demonstrated in a nonhuman primate model
  • Potential to delay or prevent the need for heart transplants in patients with congenital heart defects

The research focused on right ventricular dysfunction, a condition that often affects children with congenital heart defects and can lead to heart failure. Current treatments, including surgical repair, are often temporary solutions, with many patients eventually requiring heart transplants.

Dr. Jodi Scholz, the study’s lead author, emphasized the critical need for alternative treatments, stating, “Stem cell treatments could someday delay or even prevent the need for heart transplants.”

While the study showed promising results, the researchers noted some instances of ventricular tachycardia in the animal subjects. However, these episodes resolved within 19 days.

This research represents a significant step towards clinical applications for congenital heart defects. The use of macaques in this study has been crucial in advancing stem cell therapies for various diseases, including heart disease, kidney disease, Parkinson’s disease, and eye diseases.

As heart disease remains the leading cause of death in the United States, this innovative approach to regenerating heart tissue offers hope for stronger, longer-lasting hearts across all age groups.

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