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Researchers from the Novo Nordisk Foundation Center for Stem Cell Medicine, reNEW, have created blood stem cells in the lab that closely resemble those in the human body.
This world-first breakthrough could lead to improved, personalised treatments for children with leukaemia and bone marrow failure disorders.
reNEW Principal Investigators Associate Professor Elizabeth Ng and Professor Andrew Elefanty, co-group leaders of the Blood Development group at the Murdoch Children’s Research Institute (MCRI), and Professor Ed Stanley, group leader of the MCRI Immune Development group, and their teams, have overcome a major hurdle for producing human blood stem cells in the lab.
The findings, which were published today in Nature Biotechnology , will pave the way for patient specific, lab grown cells to be used in blood stem cell and bone marrow transplants.
“The ability to take any cell from a patient, reprogram it into a stem cell and then turn these into specifically matched blood cells for transplantation will have a massive impact on these vulnerable patients’ lives,” said Ng.
“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.”
Currently, a blood stem cell transplant is often a key part of lifesaving treatment for childhood blood disorders. However, not all children can find a matched donor.
“Mismatched donor immune cells from the transplants can attack the recipient’s own tissues, leading to severe illness or death,” Elefanty said.
“Developing personalized, patient-specific blood stem cells will prevent these complications, address donor shortages and, alongside genome editing, help correct underlying causes of blood diseases.”
The next stage, likely in about five years, would be conducting a phase one clinical trial to test the safety of using these lab grown blood cells in humans.
