Melbourne researchers have achieved a world first: they have succeeded in creating blood stem cells that closely resemble human tissue. The discovery could also lead to personalised therapies for children suffering from bone marrow failure syndromes and leukaemia.
The study, led by the Murdoch Children’s Research Institute (MCRI) and published in Nature Biotechnology, has successfully overcome a significant hurdle in the production of human blood stem cells, which are capable of generating red and white blood cells and platelets very similar to those seen in human embryos.
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MCRI Associate Professor Elizabeth Ng said the team had made a significant discovery in the development of human blood stem cells, paving the way for these lab-grown cells to be used in blood and bone marrow stem cell transplants.
“The ability to take any cell from a patient, reprogram it to become a stem cell and then convert those cells into blood cells specifically matched for transplantation will have a huge impact on the lives of these vulnerable patients,” he said.
“Prior to this study, it had not been possible to develop human blood stem cells in the laboratory that could be transplanted into an animal model with bone marrow failure to produce healthy blood cells. We have developed a workflow that has created transplantable blood stem cells that closely mirror those of the human embryo.
“Importantly, these human cells can be created at the scale and purity required for clinical use.”
In the study, immunodeficient mice were injected with laboratory-modified human blood stem cells. The blood stem cells were found to develop into functional bone marrow at levels similar to those seen in umbilical cord blood cell transplants, a proven benchmark for success.
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The research also found that lab-grown stem cells could be frozen before being successfully transplanted into mice. This mimicked the process of preserving donor blood stem cells before transplanting them into patients.
MCRI Professor Ed Stanley said the findings could lead to new treatment options for a range of blood disorders.
“Red blood cells are vital for oxygen transport and white blood cells are our immune defence, while platelets trigger clotting to stop bleeding,” he explained. Understanding how these cells develop and function is like solving a complex puzzle.
“By perfecting stem cell methods that mimic the development of normal blood stem cells found in our bodies, we can understand and develop personalized treatments for a variety of blood diseases, including leukemias and bone marrow failure.”
MCRI Professor Andrew Elefanty said that while a blood stem cell transplant was often a key part of life-saving treatment for childhood blood disorders, not all children were able to find an ideally matched donor.
“Immune cells from the unmatched transplant donor can attack the recipient’s own tissues, causing serious illness or death,” he explained.
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“Developing personalized, patient-specific blood stem cells will prevent these complications, address the donor shortage, and, together with genome editing, help correct the underlying causes of blood diseases.”
Professor Elefanty said the next stage, likely in about five years with government funding, would be to conduct a phase one clinical trial to test the safety of using these lab-grown blood cells in humans.
Riya was diagnosed at age 11 with aplastic anemia, a rare and serious blood disorder in which the body stops producing enough new blood cells.
Riya’s family, including her parents Sonali and Gaurav Mahajan, were in India at the time when she began feeling fatigued, rapidly lost weight and developed bruises on her thighs.
“We took Riya for a simple blood test, the first one she had ever had. But as soon as the results came, we were told to rush her to the emergency room as she had very few red blood cells and platelets,” Sonali said.
“Riya was originally diagnosed with leukaemia because the symptoms are very similar to aplastic anaemia. When we finally got the diagnosis, it was a total shock and a disease we had never heard of before.
“Doctors told us he had bone marrow failure and began requiring regular platelet and blood transfusions to increase his blood cell count.”
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Sonali said the family had already planned to return to Australia for Riya’s secondary education, but the diagnosis accelerated the return.
“Once they were able to stabilise her, they gave us two days to get her to Australia for hospitalisation,” he said.
“As soon as we got off the plane we went straight to the Royal Children’s Hospital. Within a few days Riya started therapy but never really responded to the medication.
“A bone marrow transplant was ultimately recommended due to the number of transfusions he needed and concerns about possible long-term complications.”
Sonali said that for six months they struggled to find a perfectly matched donor and were losing hope. Despite the donor being a 50/50 match, Sonali, on the advice of a specialist, became her daughter’s donor.
Following the bone marrow transplant in June last year, Riya remained in hospital for three months, where she experienced minor complications.
Without a matching donor, Riya’s platelet count took longer to return to normal, she required longer immunosuppressive therapy, and she was more susceptible to infections. Riya only recently started receiving the vaccine again.
“She had a weakened immune system for a long time after the transplant, but fortunately, once she was discharged from the hospital, she did not need another transplant,” Sonali said.
Riya, 14, said that after a few painful years she was now feeling well, taking hydrotherapy classes and was happy to be back at school with her friends.
Sonali said the new MCRI-led research on blood stem cells was a remarkable achievement.
“This research will be a blessing to many families,” he said. “The fact that there may one day be specific treatments for children with leukemia and bone marrow failure disorders is life-changing.” (ANI)
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