Stem cell therapy can correct heart failure in children

The famous visionary English physician William Harvey wrote in 1651 how our blood contains all the secrets of life.

“And so I conclude that the blood lives and feeds on itself and in no way depends on any other part of the body as before it or more excellent,” he wrote. “So that from this we may perceive the causes not only of life in general … but also of longer or shorter life, of sleep and waking, of skill, of strength, and so forth.”

Dr. Kevin Watt, team leader of the Heart Regeneration and Disease Laboratory at the Murdoch Children’s Research Institute (MCRI) in Melbourne, Australia, understands this concept deeply.

He lives it every day as he and his fellow researchers study and reprogram blood’s potential to treat disease, especially heart failure in children.

Based on the work of Dr. Shinya Yamanaka of Japan, who discovered that specialized cells can be reprogrammed back into immature stem cells, Watt and his collaborators have taken this work a few steps further.

They used small molecules to turn these new blood stem cells into heart cells.

Tiny heart organoids are being developed in the lab – which can then be injected into the failing hearts of children.

Backed by philanthropic support from the Murdoch Institute, the work is progressing rapidly and has already been shown to be effective in mice, pigs and sheep.

“The vision of our research is to develop new therapies that can transform the lives of children with heart failure.”

Clinical trials in humans will begin soon, and as Dr. Watt in an interview from Australia, “Large sheets of heart tissue will be sewn into the failing heart.”

Congenital heart failure as well as the side effects of chemotherapy in children will be the targets of this miracle therapy. Millions of children around the world suffer from these conditions every day.

Watt said certain chemotherapy (anthracyclines) have a higher risk of heart failure — up to 15% of cases — and this treatment may be helpful in protecting the heart.

Watt said, “Heart failure remains an urgent and unmet clinical challenge worldwide. While we have made significant advances over several decades in disease management, we lack targeted therapies to treat these devastating conditions.”

He added, “More than 500,000 children worldwide are living with advanced heart failure that requires a transplant. The vision of our research is to develop new therapies that can transform the lives of children with heart failure.”

To achieve this, he said, “we use a technology called induced pluripotent stem cells, where we can convert blood or skin cells from heart failure patients into stem cells that we then turn into heart cells… or even make engineered heart tissue that can be sewn into a patient’s heart to help it pump.”

The target cells in the blood are known as peripheral blood mononuclear cells (PBMCs).

They “go back in time to an earlier time before they differentiated into heart or kidney cells,” he said.

Then they can be pushed forward to become healthy heart cells or mutations – or other abnormalities can be corrected.

While the team at the Murdoch Children’s Research Institute is making heart cells from blood stem cells for clinical use, it is also using these stem cells to find new drugs to directly treat heart failure.

Said Watt, “Using stem cells from patients with chemotherapy-induced heart failure, 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 created methods to turn these source cells into miniature heart tissue that can be used to model disease in a dish, to identify new drug targets for the development of new therapies.

These treatments are customized and very expensive, but they are also very effective.

Correcting heart failure in young children is only a few years away from becoming a reality.

It’s a Christmas miracle that relies on the kind of philanthropic support MCRI is famous for hosting.

“Philanthropic support plays a critical role in accelerating the development of these transformative new treatments,” said Watt, “and this support will be essential as we work toward bringing precision stem cell-based therapies for heart failure to every child that needs it.”