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Breakthrough in Blood Stem Cell Research Offers Hope for Personalized Treatments
Researchers in Melbourne have achieved a significant milestone by developing blood stem cells that closely replicate those naturally found in the human body. This groundbreaking advancement holds the promise of facilitating personalized treatment options for children suffering from leukaemia and disorders related to bone marrow failure.
The study, conducted by the Murdoch Children’s Research Institute (MCRI) and published in Nature Biotechnology, has successfully addressed a major challenge in the generation of human blood stem cells. These cells are essential for producing red blood cells, white blood cells, and platelets, making them crucial for various health functions.
According to MCRI Associate Professor Elizabeth Ng, this research marks a pivotal development in the field of human blood stem cell research, with potential implications for blood stem cell transplants and therapies for bone marrow disorders. She highlighted the transformative impact of being able to take any patient cell, reprogram it into a stem cell, and subsequently generate specifically matched blood cells for transplantation.
“Before this breakthrough, it was impossible to cultivate human blood stem cells in a lab for transplant into animal models of bone marrow failure, which would produce healthy blood cells. We have now established a procedure that creates transplantable blood stem cells which closely resemble those in human embryos,” Ng stated. She also emphasized the scalability and purity of these cells, making them suitable for clinical applications.
In their experimental study, immune-deficient mice were given injections of lab-engineered human blood stem cells. Results indicated that these stem cells could effectively function as bone marrow, achieving levels akin to those seen with umbilical cord blood cell transplants, a recognized standard for success.
The research further demonstrated that the engineered stem cells could be cryopreserved before being successfully introduced into the mice, mirroring the process used to store donor blood stem cells prior to transplantation.
MCRI Professor Ed Stanley pointed out that this research could pave the way for innovative treatments for various blood disorders. He explained the crucial roles that red blood cells play in oxygen transport, white blood cells in immune response, and platelets in blood clotting. “Understanding the development and function of these key cells is akin to piecing together a complicated puzzle,” Stanley remarked.
“By refining stem cell techniques that align with the development of normal blood stem cells, we can gain deeper insights into and formulate personalized treatments for numerous blood-related diseases, including leukaemias and bone marrow failure,” he added.
MCRI Professor Andrew Elefanty noted the significance of blood stem cell transplants in treating childhood blood disorders. However, he pointed out the challenge presented when suitable matched donors are not available. “Mismatched donor immune cells can initiate a harmful response against the recipient’s own tissues, leading to severe complications or even fatal outcomes,” he explained.
Elefanty emphasized that creating personalized, patient-specific blood stem cells would mitigate these complications, alleviate donor shortages, and, in conjunction with genome editing, could help rectify the underlying causes of blood diseases.
Looking forward, Professor Elefanty indicated that the next phase, anticipated within five years pending government funding, involves launching a phase one clinical trial to assess the safety of employing these lab-grown blood cells in human patients.
Alongside Professors Elefanty and Stanley, Associate Professor Ng is also a Principal Investigator at the Melbourne branch of the Novo Nordisk Foundation Center for Stem Cell Medicine (reNEW), an international collaborative focused on advancing stem cell therapies. The research received contributions from scholars across institutions including the University of Melbourne, Peter MacCallum Cancer Centre, University of California Los Angeles, University College London, and University of Birmingham, underscoring a robust collective effort in the field.
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