Researchers at Melbourne’s Murdoch Children’s Research Institute (MCRI) achieved an advancement in the creation of lab-grown blood stem cells that closely resemble those found in the human body. The pioneering work, published in Nature Biotechnology, could revolutionize personalized treatments for children with leukemia and bone marrow failure disorders.
Associate Professor Elizabeth Ng from MCRI highlighted the significance of this discovery, stating that the ability to reprogram a patient’s cells into stem cells and then generate specifically matched blood cells for transplantation could dramatically improve outcomes for vulnerable patients.
The study involved injecting lab-engineered human blood stem cells into immune-deficient mice, resulting in functional bone marrow similar to that produced by umbilical cord blood transplants—a key benchmark of success. Moreover, these lab-grown cells can be produced at the scale and purity required for clinical applications and can be frozen for future use, mimicking the preservation of donor blood stem cells.
Professor Ed Stanley emphasized that this breakthrough offers new avenues for treating various blood disorders, as it enables a deeper understanding of how blood cells develop and function. This knowledge could lead to personalized therapies for conditions such as leukemia and bone marrow failure.
Professor Andrew Elefanty pointed out the potential to overcome the challenges of donor shortages and mismatched transplants, which can cause severe complications. The development of personalized blood stem cells could prevent these issues and, when combined with genome editing, address the root causes of blood diseases.
As reported by medicalxpress.com, the research team, including contributors from the University of Melbourne, Peter MacCallum Cancer Center, and international institutions, anticipates beginning a Phase I clinical trial to test the safety of these lab-grown cells in humans within the next five years.