The regenerative medicine team in the Todd and Karen Wanek Family Program for HLHS at Mayo Clinic focuses on creating patient-specific stem cell-based interventions for HLHS.
Today's standard treatment for HLHS entails a three-stage surgery that enables the right side of the patient's heart to pump blood to the entire body.
While the current treatment is effective, it does not eliminate the risk that the patient will eventually need a heart transplant. Because the right side of the heart is not meant to support the entire body, the strain on the heart can lead to declines in its pumping ability over time, making a transplant necessary.
Regenerative medicine may help scientists restore the right side of the heart's pumping ability when it begins to decline, or may even help prevent the decline altogether, eliminating the need for a heart transplant. Using different types of stem cells from various sources — including cells from the patient's own body — regenerative medicine may be able to replace or regenerate defective heart tissue, leaving new, healthy tissue in its place.
Regenerative medicine researchers in the program aim to discover the safest and most effective regenerative therapies for HLHS and make them clinically available.
The Todd and Karen Wanek Family Program for HLHS' regenerative medicine team works to determine from where and from whom therapeutic stem cells can originate and when best to deliver the cells to patients. The team's research strategies include:
- Tissue and biomarker profiling. Researchers collect surgically discarded tissue from children with HLHS and skin biopsies from their unaffected parents. The researchers use the samples to create reprogrammed, pluripotent cell lines capable of differentiating into patient-specific cardiac tissue. The team then searches at the molecular level for differences between bioengineered cardiac tissue from children with HLHS and similar tissue from their unaffected parents.
Development of a preclinical model. Before promising stem cell-based regenerative therapies for HLHS can be offered in clinical trials, researchers must evaluate efficacy in preclinical models.
Program investigators are working to create a preclinical model of HLHS in which the right ventricle is overworked — just like in people with HLHS — to assess the effectiveness of emerging regenerative methodologies in repairing damaged cardiac tissue.
- Umbilical cord blood. Program researchers are studying whether people with HLHS could benefit from direct myocardial injection of stem cells from umbilical cord blood during HLHS-related surgical procedures.