Researchers at the Murdoch Children’s Research Institute (MCRI) are developing a groundbreaking stem cell therapy that could soon help children with heart failure.
Building on the pioneering work of Japanese scientist Dr. Shinya Yamanaka, this approach has already shown promise in animal studies and could soon offer lifesaving treatments for young patients in need.
Under the leadership of Dr. Kevin Watt, the Heart Regeneration and Disease Laboratory at MCRI is focusing on “induced pluripotent stem cells.”
As Dr. Watt 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.”
He further noted that his team is reprogramming peripheral blood mononuclear cells (PBMCs) to “push them back in time”—effectively returning them to an earlier developmental stage, before they first differentiated into specific cell types.
By adding small molecules to these newly produced stem cells, the researchers can produce heart cells that may help with tissue replacement or repair. The process has already shown effectiveness in mice, pigs, and sheep.
“Clinical trials in humans will be starting soon,” Dr. Watt said, expressing optimism about the treatment’s potential. “Large sheets of heart tissue will be stitched into the failing heart.”
According to Dr. Watt, heart failure remains “an urgent, unmet clinical challenge across the world,” especially in children who suffer from congenital heart conditions or experience cardiac damage as a result of chemotherapy treatments. “While we have made significant advances over several decades in managing the disease, we lack targeted therapies to treat these devastating conditions,” he added.
An estimated 500,000 children worldwide are currently living with advanced heart failure that requires transplantation, Dr. Watt said.
He stressed that MCRI’s research vision is “to develop new therapies that can transform the lives of children with heart failure.” Looking ahead, his team is also using patient-derived stem cells to test new drugs that could prevent or treat heart failure, expanding options for children facing difficult diagnoses.
The costs of these personalized treatments are high, yet philanthropic support continues to fuel these efforts.
“Philanthropic support plays a critical role in accelerating the development of these new, transformative treatments,” Dr. Watt explained, “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.”
With promising studies already underway, this cutting-edge research is offering renewed hope for families around the globe — and could soon deliver a lifesaving treatment option for children who need it most.