The famous English physician William Harvey wrote in 1651 about how our blood contains all the secrets of life.
“I thus conclude,” he wrote, “that the blood lives and nourishes itself and is in no way dependent on any other part of the body as being older or better than it.” “So that from this we can understand not only the causes of life in general… but also its causes Longer or shorter life“In sleeping and waking, in skill, in strength, and so on.”
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.
Stem cell research shows new possibilities for treating heart disease in children
He lives it every day, as he and his fellow researchers study and reprogram the potential of blood to specifically treat diseases Heart failure in children.
Building on the work of Dr. Shinya Yamanaka of Japan, who discovered that specialized cells could be reprogrammed back into immature stem cells, Watt and his collaborators took this work several steps further.
They used small molecules to convert these new stem cells from the blood into heart cells.
Tiny heart organoids are developed in the laboratory, which can then be injected into children's failing hearts.
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Relying on 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 treatments that can change the lives of children with heart failure.”
Human clinical trials will begin soon, and as Dr. Watt told me in an interview from Australia, “large sheets of heart tissue will be sewn onto the failing heart.”
Congenital heart failure as well Side effects of chemotherapy In children they will be targets of this miracle treatment. Millions of children around the world suffer from these conditions every day.
Watt said that some types of chemotherapy (anthracyclines) increase the risk of heart failure – up to 15% of cases – and this treatment may be useful for protecting the heart.
“Heart failure remains an urgent, unaddressed clinical challenge worldwide. While we have made significant progress over several decades in managing the disease, we lack targeted therapies to treat these devastating conditions,” Watt said.
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He added: “More than 500,000 children around the world live with advanced heart failure Requires transplantation. “The vision of our research is to develop new treatments that can change 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 the blood or skin cells of patients with heart failure into stem cells that we then turn into heart cells… or even make engineered heart tissue that can be sewn on.” The patient's heart helps it pump blood.
The target cells in the blood are known as peripheral blood mononuclear cells (PBMCs).
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“They were traced back in time to an earlier time before they differentiated into heart or kidney cells,” he said.
They can then be pushed forward into healthy heart cells or mutants — or other abnormalities can be corrected.
While the team was in Murdoch Children's Research Institute He manufactures heart cells from blood stem cells for clinical use, and he also uses these stem cells to discover new drugs to directly treat heart failure.
“Using stem cells from patients with chemotherapy-induced heart failure, we are actively developing new drugs and cell-based therapies that we believe will be life-changing,” Watt said. Patients' lives Given these conditions… our research group has pioneered ways to transform these stem 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 treatments.
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 Which depends on the type of charitable support MCRI is known for arranging.
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“Philanthropic support plays a critical role in accelerating the development of these transformative new therapies, and this support will be essential as we work to bring precision stem cell-based treatments for heart failure to every child who needs them,” Watt said.
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