Lab-grown blood stem cells could be used for transplants

Researchers at the Murdoch Children’s Research Institute (MCRI) have overcome a major hurdle for producing blood stem cells — which can create red blood cells, white blood cells and platelets — that closely match those in the human embryo. The breakthrough paves the way for these lab-grown cells to be used in blood stem cell and bone marrow transplants for patients with a range of blood diseases.

“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 MCRI Associate Professor Elizabeth 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.

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

In the study, which was published in the journal Nature Biotechnology, immune-deficient mice were injected with the lab-engineered human blood stem cells. The blood stem cells became functional bone marrow at similar levels to that seen in umbilical cord blood cell transplants; a proven benchmark of success.

The research also found the lab-grown stem cells could be frozen prior to being successfully transplanted into the mice. This mimicked the preservation process of donor blood stem cells before being transplanted into patients.

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

“Red blood cells are vital for oxygen transport and white blood cells are our immune defence, while platelets cause clotting to stop us bleeding,” he 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 leukaemia and bone marrow failure.”

MCRI Professor Andrew Elefanty added that while a blood stem cell transplant is often a key part of lifesaving treatment for childhood blood disorders, not all children will find an ideally matched donor.

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

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

Elefanty said the next stage, likely in about five years with government funding, would be conducting a phase one clinical trial to test the safety of using these lab-grown blood cells in humans.

Image credit: iStock.com/Murat Photo