Research conducted by Murdoch Children’s Research Institute (MCRI) and published in Biotechnology of naturehas overcome a major obstacle to producing human blood stem cells that can create red blood cells, white blood cells, and platelets that closely match those in the human embryo.

MCRI associate professor Elizabeth Ng said the team had made a significant discovery in the development of human blood stem cells, which paves 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 into a stem cell and then transform it into specially matched blood cells for transplantation will have a huge impact on the lives of these vulnerable patients,” she said.

“Before this study, it was not possible to engineer human blood stem cells in the laboratory that could be transplanted into an animal model of bone marrow failure to produce healthy blood cells. We developed a workflow that created transplantable blood stem cells that closely mirrored those in the human embryo.

“Importantly, these human cells can be created at the scale and purity required for clinical applications.”

In a study of immunodeficient mice, lab-engineered human blood stem cells were injected. They found that the blood stem cells became functional bone marrow at a similar level to that seen in cord blood cell transplants, a proven benchmark.

Studies have also shown that lab-grown stem cells can be frozen before being successfully transplanted into mice, mimicking the process of preserving donor blood stem cells before being transplanted into patients.

Professor Ed Stanley, from MCRI, said the findings could lead to new treatment options for a range of blood diseases.

“Red blood cells are essential for transporting oxygen, and white blood cells are our immune defense, while platelets cause blood to clot to stop bleeding,” he said. Understanding how these cells develop and function is like unraveling a complex puzzle.

“By improving 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 range of blood diseases, including leukemias and bone marrow failure.”

Professor Andrew Elefanty, from the MCRI, said that while blood stem cell transplantation is often a key part of life-saving treatment for children with blood diseases, not all children find a perfectly matched donor.

“Incompatible donor immune cells from the transplant can attack the recipient’s own tissues, causing severe illness or death,” he said.

“Developing personalized, patient-specific blood stem cells will prevent these complications, address the donor shortage and, alongside genome editing, help correct the underlying causes of blood diseases.”

Professor Elefanty said the next step, likely to happen in about five years with government funding, would be to conduct phase one clinical trials to test the safety of using lab-grown blood cells in humans.

Professor Elefanty, Professor Stanley and Assistant Professor Ng are also Principal Investigators at the Melbourne branch of the Novo Nordisk Foundation Centre for Stem Cell Medicine (reNEW), a global consortium that aims to pave the way for future stem cell-based treatments.

Scientists from the University of Melbourne, Peter MacCallum Cancer Centre, University of California, Los Angeles, University College London and the University of Birmingham also contributed to the findings.