MELBOURNE: Australian scientists have developed a stem cell technology that generated blood-forming cells in the laboratory and produced functional bone marrow after transplantation in mice, marking a significant advance for treatment research in leukemia, bone marrow failure and other serious blood diseases. The work was led by Murdoch Children’s Research Institute researchers in Melbourne, who used human induced pluripotent stem cells to create cells resembling early human blood stem cells, according to peer-reviewed findings and subsequent institute updates on the program.
The research addresses a longstanding challenge in regenerative medicine: creating transplantable human blood stem cells outside the body. In the study, scientists guided induced pluripotent stem cells through developmental stages that mimic early embryonic blood formation, generating CD34-positive cells that were frozen, thawed and then transplanted into immune-deficient mice. The recipient animals developed multilineage bone marrow engraftment, meaning the lab-made cells gave rise to multiple blood cell types, including cells involved in oxygen delivery, immune defense and clotting.
The peer-reviewed paper reported that cells derived from four independent stem cell lines produced bone marrow engraftment in 25% to 50% of recipient mice, at levels comparable to umbilical cord blood transplantation, an established medical benchmark. The results remain preclinical and do not constitute an approved therapy for patients, but they showed the cells could survive, function and be preserved before transplant. That combination is important for any effort to build a practical source of more closely matched blood stem cells for transplant medicine.
Preclinical transplant evidence
The technology is relevant to conditions in which patients need blood stem cell or bone marrow transplants, including leukemia and inherited or acquired marrow disorders. Current transplants often rely on a well-matched donor, and poor matches can trigger graft-versus-host disease, a serious immune complication. By starting with a patient’s own cells and reprogramming them into stem cells, researchers are working toward a way to produce better-matched transplant material while also creating a platform for studying how blood disorders begin and progress.
Murdoch Children’s Research Institute has said the lab-grown cells were created at a scale and purity aligned with clinical manufacturing requirements and were successfully cryopreserved before transplantation in mice. That storage step is significant because donor blood stem cells are routinely frozen before use in hospitals. The institute has linked the research to disorders such as aplastic anemia, leukemia and bone marrow failure syndromes, where shortages of ideal donors can complicate treatment and delay access to transplant procedures for children and adults.
Partnership expands development work
In May 2025, Murdoch Children’s Research Institute announced a research and commercial licensing agreement worth more than US$35 million with Retro Biosciences to further develop the platform. The deal licensed the blood stem cell discovery intellectual property to Retro Biosciences for therapeutic development, connecting the laboratory advance to a broader translational effort. The institute also said more than 90,000 blood stem cell transplants are performed worldwide each year, with more than 36,000 donor transplants used for leukemia or bone marrow failure syndromes.
Researchers 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 published study, underscoring the breadth of the collaboration behind the work. The technology remains in the research stage, with the strongest evidence so far coming from peer-reviewed mouse data rather than human trials. Even so, the findings mark a notable Australian advance in stem cell science aimed at blood diseases and blood cancers currently treated through donor-dependent transplant procedures – By Content Syndication Services.