Australian scientists say they are close to achieving clinically-usable laboratory-developed blood stem cells.
The researchers report that their stem cells “closely resemble” those that naturally develop in the human body. Their technology takes cells from the patient and reprogrammes them in to stem cells, then turns them into the haematopoietic cells needed for bone marrow transplantation.
In Nature Biotechnology, the researchers report testing their cells on immune deficient mice. According to the study, the cells became functional bone marrow, with multi-lineage engraftment, at a similar rate achieved by umbilical cord blood cell transplants.
The study also confirmed it was possible to freeze the cells before transplantation.
The researchers, from the Murdoch Children’s Research Institute, Melbourne, hope to begin clinical trials within five years and are hoping for funding from the Australian government. The project has also involved University College London and the University of Birmingham in the UK.
Study co-leader Professor Elizabeth Ng said: “Prior to this study, developing human blood stem cells in the lab that were capable of being transplanted into an animal model of bone marrow failure to make healthy blood cells had not been achievable.
"We have developed a workflow that has created transplantable blood stem cells that closely mirror those in the human embryo. Importantly, these human cells can be created at the scale and purity required for clinical use.”
Professor Ed Stanley said: “By perfecting stem cell methods that mimic the development of the normal blood stem cells found in our bodies we can understand and develop personalised treatments for a range of blood diseases, including leukaemias and bone marrow failure.”
Source:
Ng ES, Sarila G, Li JY, Edirisinghe HS, Saxena R, Sun S, Bruveris FF, Labonne T, Sleebs N, Maytum A, Yow RY, Inguanti C, Motazedian A, Calvanese V, Capellera-Garcia S, Ma F, Nim HT, Ramialison M, Bonifer C, Mikkola HKA, Stanley EG, Elefanty AG. (2024) “Long-term engrafting multilineage hematopoietic cells differentiated from human induced pluripotent stem cells.” Nature Biotechnology, 2 September 2024, doi: 10.1038/s41587-024-02360-7.
Link: https://www.nature.com/articles/s41587-024-02360-7
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