This world-first breakthrough could bring better personalized treatment to children with leukemia and bone marrow failure diseases.

Researchers have created hematopoietic stem cells very similar to the human body in the laboratory, a breakthrough in the world's first. 

The study was led by the Murdoch Children's Research Institute (MCRI) in Melbourne, Australia, and the results were published in the journal Nature Biotechnology.

The breakthrough overcomes a major obstacle to the creation of human hematopoietic stem cells, which can produce red blood cells, white blood cells and platelets very similar to those produced in human embryos. This work provides important clues on how to generate human hematopoietic stem cells in culture dishes that can rebuild the entire blood system.

Hematopoietic differentiation of iPS cells in vitro

Data from the research team shows that during stem cell culture, the precise and timed addition and removal of specific growth factors plays a decisive role in this process.

Professor Constanze Bonifer, emeritus professor of experimental hematology at the University of Birmingham and co-author of the study, said: "This is a remarkable discovery that may significantly improve future treatments for leukemia and bone marrow failure diseases.

Dr Elizabeth Ng, associate professor at the Melbourne Medical Research Center who led the research, said: "The ability to take any cell from a patient, reprogram it into stem cells, and then convert it into specifically matched blood cells for transplantation will have a huge impact on the lives of these fragile patients. "

Professor Elizabeth Ng

Prior to this study, it was impossible to grow human hematopoietic stem cells in the laboratory and transplant them into animal models where bone marrow was unable to produce healthy blood cells.

The research has developed a process to create transplantable hematopoietic stem cells that are very similar to those found in human embryos.

Importantly, these human cells can be manufactured at the size and purity required for clinical use.

In the study, immunodeficient mice were injected with the artificial hematopoietic stem cells, and the study found that the hematopoietic stem cells formed levels of functional bone marrow similar to those in cord blood cell transplants, which has proven to be a sign of success.

The study also found that laboratory-grown stem cells can be frozen before being successfully transplanted into mice, thus simulating the preservation process of donor hematopoietic stem cells before being transplanted into patients.

Professor Andrew Elivanti of Melbourne Medical Center said that although hematopoietic stem cell transplants are often a key part of saving lives in children with blood diseases, not all children can find an ideal match. Mismatched donor immune cells during the transplant can attack the recipient's own tissue, causing serious disease or death. The development of personalized, patient-specific hematopoietic stem cells will prevent these complications, solve the donor shortage problem, and work with genome editing, Help correct the root cause of blood disorders.

The next phase is a phase 1 clinical trial funded by the government to test the safety of using these laboratory-grown blood cells in humans.