Blood stem cell research could transform bone marrow transplants

Melbourne researchers have made a world-first breakthrough in creating blood stem cells that are highly similar to those in the human body, a discovery that could soon lead to personalised treatments for children with leukaemia and bone marrow failure diseases.

The study was led by the Murdoch Children's Research Institute (MCRI), Nature Biotechnologyhas overcome a major hurdle in human production. Blood Stem CellsCan produce red blood cells White blood cells And the platelets are very similar to those in human fetuses.

MCRI Associate Professor Elizabeth Ng said the team had made an important discovery in the development of human blood stem cells, and that these lab-grown cells could become blood stem cells or Bone marrow transplantation.

“The ability to take any cell from a patient, reprogram it into a stem cell and then turn it into a specifically matched blood cell for transplant would have a profound impact on the lives of these vulnerable patients,” she said.

“Prior to this study, we developed human blood. Stem Cells “It has not been possible to develop stem cells in the lab that can be transplanted into animal models of bone marrow failure that are unable to make healthy blood cells. We have developed a workflow to create transplantable blood stem cells that closely resemble human fetal stem cells.”

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

In this study, immunocompromised mice were injected with lab-created human blood stem cells and found that the blood stem cells transformed into functional bone marrow at levels similar to those seen in umbilical cord blood cell transplants, a proven benchmark for success.

The study also found that lab-grown stem cells could be frozen before being transplanted into mice, mimicking the process of storing donor blood stem cells before they are transplanted into patients.

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

“Red blood cells are essential for oxygen transport, white blood cells play a role in immune defense, and platelets cause blood clotting to stop bleeding,” he said. Understanding how these cells develop and function is like solving a complex puzzle.

“Perfecting stem cell methods that mimic the development of normal blood stem cells in the body will help us understand and develop personalized treatments for a variety of blood disorders, including leukemia and bone marrow failure.”

MCRI's Professor Andrew Elefanti said blood stem cell transplants are often a vital part of life-saving treatment for children with blood disorders, but not every child can find an ideal donor match.

“If the transplanted donor's immune cells are incompatible, they can attack the recipient's own tissues, leading to severe illness or death,” he said.

“Developing patient-specific customized blood stem cells could help prevent these complications, address donor shortages, and, together with genome editing, correct the underlying causes of blood disorders.”

Professor Elefanti said the next step, probably five years from now, would be to conduct phase one clinical trials to test the safety of these lab-grown plants. blood Human cells.

Professor Elefanti, Professor Stanley and Associate Professor Ng are also principal investigators at the Melbourne-based Novo Nordisk Foundation Centre for Stem Cell Medicine (reNEW).

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 findings.

More information:
Long-term transplantation of multilineage hematopoietic cells differentiated from human induced pluripotent stem cells, Nature Biotechnology (2024). DOI: 10.1038/s41587-024-02360-7

Source
Murdoch Children's Research Institute

Citation: Blood stem cell research could change bone marrow transplants (September 2, 2024) Retrieved September 2, 2024 from https://medicalxpress.com/news/2024-09-blood-stem-cell-bone-marrow.html

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