Researchers in Melbourne have achieved a groundbreaking milestone: they have successfully created blood stem cells that closely resemble human tissue. This discovery may eventually lead to customized therapies for children suffering from bone marrow failure syndromes and leukemia.
Breakthrough Study Led by Murdoch Children’s Research Institute
The study, led by the Murdoch Children’s Research Institute (MCRI) and published in Nature Biotechnology, has overcome a major obstacle in the production of human blood stem cells. These cells are capable of generating red blood cells, white blood cells, and platelets that closely resemble those seen in human embryos.
MCRI Associate Professor Elizabeth Ng commented on the significance of the discovery, noting that it opens the door for lab-grown cells to be used in blood stem cell and bone marrow transplants.
“The ability to take any cell from a patient, reprogram it into a stem cell, and then develop these into specifically matched blood cells for transplantation will have a massive impact on the lives of vulnerable patients,” she said.
New Workflow for Creating Transplantable Blood Stem Cells
Before this study, developing human blood stem cells in the lab that could be transplanted into an animal model of bone marrow failure to produce healthy blood cells was not achievable. The team has now developed a workflow that creates transplantable blood stem cells that closely mirror those in human embryos.
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“Importantly, these human cells can be created at the scale and purity required for clinical use,” Ng added.
Successful Transplants in Animal Models
In the study, immune-deficient mice were injected with the lab-engineered human blood stem cells. The research found that these cells became functional bone marrow at levels similar to those seen in umbilical cord blood cell transplants, a proven benchmark of success.
The lab-grown stem cells were also found to be successfully frozen before transplantation, mimicking the preservation process of donor blood stem cells.
Potential Impact on Blood Disorders
MCRI Professor Ed Stanley highlighted the potential of these findings to lead to new treatment options for a range of blood disorders.
“Red blood cells are vital for oxygen transport, white blood cells are our immune defense, and platelets are crucial for clotting. Understanding how these cells develop and function is like decoding a complex puzzle,” Stanley said.
By perfecting stem cell methods that mimic normal blood stem cell development, researchers aim to develop personalized treatments for blood diseases, including leukemia and bone marrow failure.
Addressing Donor Shortages and Complications
MCRI Professor Andrew Elefanty emphasized that while blood stem cell transplants are often a key part of lifesaving treatment for childhood blood disorders, finding an ideally matched donor can be challenging.
“Mismatched donor immune cells from the transplant can attack the recipient’s own tissues, leading to severe illness or death,” Elefanty said. “Developing personalized, patient-specific blood stem cells will prevent these complications, address donor shortages, and, alongside genome editing, help correct underlying causes of blood diseases.”
The next stage of research, likely to begin in about five years with government funding, will involve a phase one clinical trial to test the safety of using these lab-grown blood cells in humans.
Personal Story: Riya’s Journey
Riya was diagnosed at the age of 11 with aplastic anemia, a rare and serious blood disorder where the body stops producing enough new blood cells. Her family, who were in India at the time, noticed symptoms including fatigue, weight loss, and bruising.
“We took Riya for a simple blood test, and when the results came in, we were rushed to the emergency department due to her dangerously low platelet and red blood cell counts,” said her mother, Sonali.
After being stabilized, Riya’s family returned to Australia for her high school education and medical treatment. Despite a challenging search for a perfectly matched donor, Sonali became a half-match donor for her daughter.
Following a bone marrow transplant in June last year, Riya experienced a prolonged hospital stay and minor complications. However, she has since been stable, and her condition has improved.
Sonali praised the MCRI-led research, saying, “This research will come as a blessing to many families. The possibility of targeted treatments for children with leukemia and bone marrow failure disorders is life-changing.”
(WITH INPUTS FROM ANI)
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