Researchers have achieved a groundbreaking advancement by successfully creating blood stem cells that are very similar to those found in the human body. This important discovery could lead to tailored treatments for children suffering from leukemia and disorders related to bone marrow failure.
Scientists in Melbourne have made a historic achievement by developing blood stem cells that closely mimic those present in humans. This development holds promise for personalized therapies aimed at treating children diagnosed with leukemia and bone marrow failure conditions.
The research was spearheaded by the Murdoch Children’s Research Institute (MCRI) and published in Nature Biotechnology. The team has successfully navigated a major challenge in creating human blood stem cells capable of forming red blood cells, white blood cells, and platelets that closely resemble those in human embryos.
Associate Professor Elizabeth Ng from MCRI stated that the team’s findings mark a significant milestone in the realm of human blood stem cell development, setting the stage for these lab-designed cells to be used in blood stem cell and bone marrow transplants.
“The capability to take any patient cell, reprogram it into a stem cell, and then convert those into specially matched blood cells for transplantation will greatly enhance the lives of these vulnerable patients,” she commented.
“Before our study, it was not possible to develop human blood stem cells in the lab that could be successfully transplanted into an animal model of bone marrow failure to produce healthy blood cells. We have now established a procedure that successfully creates transplantable blood stem cells mirroring those in human embryos.
“Crucially, these human cells can be generated in the necessary quantity and purity for clinical applications.”
During the study, immune-deficient mice were injected with the human blood stem cells engineered in the lab. The results showed that these blood stem cells developed into functional bone marrow at a level comparable to that seen in umbilical cord blood cell transplants, which serves as a reliable measure of success.
The study further indicated that these lab-grown stem cells could be frozen prior to being effectively transplanted into the mice. This process resembles the storage of donor blood stem cells before transplantation into patients.
Professor Ed Stanley from MCRI expressed that these findings could pave the way for new treatment alternatives for various blood disorders.
“Red blood cells are essential for transporting oxygen, while white blood cells serve as our immune defense, and platelets are crucial for clotting and preventing bleeding,” he explained. “Grasping how these cells develop and operate is akin to piecing together a complex puzzle.”
“By refining stem cell techniques that imitate the normal development of blood stem cells within our bodies, we can better understand and create personalized treatments for a variety of blood diseases, including leukemia and bone marrow failure.”
Professor Andrew Elefanty from MCRI noted that although blood stem cell transplants are often crucial for lifesaving treatments of childhood blood disorders, not every child has access to a well-matched donor.
“Donor immune cells that are not optimally matched can attack the recipient’s own tissues, leading to severe complications or even death,” he said.
“The creation of personalized, patient-specific blood stem cells will help avoid these complications, resolve donor shortages, and combined with genome editing, address the root causes of blood diseases.”
Professor Elefanty mentioned that the next step, expected to take place in about five years with government support, will involve conducting a phase one clinical trial to evaluate the safety of these lab-grown blood cells in humans.
Professors Elefanty, Stanley, and Associate Professor Ng are also Principal Investigators at the Melbourne branch of the Novo Nordisk Foundation Center for Stem Cell Medicine (reNEW), a global initiative dedicated to advancing future stem cell-based therapies.
Researchers from the University of Melbourne, Peter MacCallum Cancer Centre, University of California Los Angeles, University College London, and the University of Birmingham were also instrumental in this study.
