The famous visionary English physician William Harvey wrote in 1651 how our blood contains all the secrets of life.
“And thus I conclude that the blood lives and nourishes itself and in no way depends on any other part of the body to be superior or superior to it,” he said. wrote “So that by it we may understand not only the causes of ordinary life … but also of Long or short lifeof sleeping and waking, of skill, of strength and so on.”
Dr. Kevin Watt, team leader in the Heart Regeneration and Disease Laboratory at the Murdoch Children’s Research Institute (MCRI) in Melbourne, Australia, understands this concept deeply.
Stem cell research is showing new possibilities for treating pediatric heart disease
He lives it every day, as he and his fellow researchers study and reprogram the blood’s ability to treat disease, specifically Heart failure in children.
Building 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 colleagues have taken this work several steps further.
They used small molecules to transform these new stem cells from the blood into heart cells.
Tiny heart organoids are created in the lab – which can then be injected into the failing hearts of children.
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Relying on the philanthropy of the Murdoch Institute, 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.”
Clinical trials in humans are due to begin soon, and as Dr. Watt told me in an interview from Australia, “huge sheets of heart tissue will be sutured into a failing heart.”
As well as congenital heart failure Side effects of chemotherapy Children will be the target of this miracle therapy. Millions of children around the world suffer from these conditions every day.
Some chemotherapy (anthracyclines) have a higher risk of heart failure – up to 15 percent – and these treatments may be useful to protect the heart, Watt said.
“Heart failure remains an urgent, unmet medical challenge worldwide. While we have made significant progress over the decades in the management of this disease, we have no cure for this devastating condition,” Watt said. There is a lack of targeted therapies.”
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He added, “More than 500,000 children worldwide live with advanced heart failure. Transplantation is required. The focus of our research is to develop new treatments that can change the lives of children with heart failure.”
To achieve this goal, he said, “We use a technology called induced pluripotent stem cells, where we can transform blood or skin cells from heart failure patients into stem cells that we then transplant into the heart. can turn into cells of … The patient’s heart is stitched to help it pump.”
The target cells in the blood are called peripheral blood mononuclear cells (PBMCs).
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“They are pushed earlier in time before differentiating into heart or kidney cells,” he said.
They can then be advanced to become healthy heart cells or mutations – or correct other abnormalities.
While in the team Murdoch Children’s Research Institute Creating heart cells from blood stem cells for clinical use, it is exploring new drugs to directly treat heart failure using these stem cells.
“Using stem cells from patients with chemo-induced heart failure, we are actively developing new drugs and cell-based therapies that we believe will be game-changing,” Watt said. . Patients’ lives With these conditions … our research group has pioneered ways to transform these stem cells into tiny heart tissues that can be used to model a dish in the disease, to develop new therapeutic drugs. New targets can be identified.”
These treatments are personalized 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.
This is one The Christmas Miracle Which relies on the kind of philanthropy that MCRI is famous for managing.
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“Human support plays an important role in accelerating the development of these new, transformative therapies,” Watt said, “and this support will be essential as we continue to develop stem cell-based therapies for heart failure for every child.” Work to bring treatment to those who need it. ”
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