Dr. Kevin Watt, team leader of the Heart Regeneration and Disease Laboratory at the Murdoch Children's Research Institute (MCRI) in Melbourne, Australia, has been doing something amazing. He and his fellow researchers are exploring the potential of reprograming blood to treat diseases, particularly heart failure in children.
Building on Nobel Prize winner Dr. Shinya Yamanaka's discovery that specialized cells can be reprogrammed into immature stem cells, Watt and his collaborators have further advanced this research.
They have used small molecules to convert new blood stem cells into heart cells.
Small heart organoids are developed in the lab for injection into failing hearts of children. Funded by the Murdoch Institute, this research has shown effectiveness in mice, pigs, and sheep. Human trials are on their way.
Dr. Watt said, "The vision of our research is to develop new therapies that can transform the lives of children with heart failure." explaining that "large sheets of heart tissue will be stitched into the failing heart."
This innovative therapy will focus on congenital heart failure and the side effects of chemotherapy in children -- conditions that affect millions worldwide. These are children already fighting for survival that suffer serious side effects from the drugs used to help them.
Dr. Watt highlighted that certain chemotherapy agents, like anthracyclines, can lead to heart failure in up to 15% of cases, making this treatment particularly important for heart protection.
Dr. Watt warned, "Heart failure remains an urgent, unmet clinical challenge across the world. While we have made significant advances over several decades in managing the disease, we lack targeted therapies to treat these devastating conditions."
He noted that certain chemotherapy (anthracyclines) have a higher risk of heart failure.
Dr. Watt added, "More than 500,000 children around the world live with advanced heart failure that requires transplantation. The vision of our research is to develop new therapies that can transform the lives of children with heart failure."
To achieve this, he explained, "We use a technology called induced pluripotent stem cells, where we can convert blood or skin cells of patients with heart failure into stem cells that we then turn into heart cells … or even make engineered heart tissues that can be stitched onto the patient's heart to help it pump."
The cells targeted in the blood are known as peripheral blood mononuclear cells (PBMCs).
Simply put, they are "pushed back in time to an earlier time before they became differentiated into heart or kidney cells."
Then, they can be pushed forward to become healthy heart cells, or mutations — or other abnormalities can be corrected.
While the Murdoch Children's Research Institute team is making heart cells from stem cells in the blood for clinical use, they are also using them to figure out new drugs to treat heart failure directly.
Dr. Watt again explains further, "Using stem cells from patients with heart failure caused by chemo, we are actively developing new drugs and cell-based treatments that we believe will transform the lives of patients with these conditions … Our research group has pioneered methods to turn these stem cells into miniature heart tissues that can be used to model disease-in-a-dish, to identify new drug targets for the development of new therapies."
These treatments are tailored to individual needs and are quite expensive, but they are also very effective.
Correcting heart failure in young children is only a few years away from becoming a reality.
This research miracle depends entirely on the philanthropic support MCRI is known for.
"Philanthropic support plays a critical role in accelerating the development of these new, transformative treatments," said Watt, "and this support will be essential as we work toward bringing stem cell-based precision therapies for heart failure to every child who needs it."
Hopefully and prayerfully, in a few short years, we will begin to see far more heart-related miracles. In the meantime, supporting research programs like this is an excellent way to have a life-saving impact.
Sources:
https://www.mcri.edu.au/research/research-areas/stem-cell-medicine/heart-regeneration
https://pmc.ncbi.nlm.nih.gov/articles/PMC10094119/
https://www.cancer.gov/publications/dictionaries/cancer-terms/def/anthracycline
https://pmc.ncbi.nlm.nih.gov/articles/PMC4673925/
