Stem Cell Breakthroughs Poised to Revolutionize Transplant Procedures

A groundbreaking study conducted by the Murdoch Children’s Research Institute (MCRI) and published in Nature Biotechnology has successfully addressed a significant challenge in producing human blood stem cells that closely resemble those found in human embryos. These stem cells are capable of generating red blood cells, white blood cells, and platelets, essential components of the human circulatory system.

Associate Professor Elizabeth Ng of MCRI stated that the team made a vital discovery in the development of human blood stem cells, which could revolutionize blood stem cell and bone marrow transplant procedures. “The ability to reprogram any cell from a patient into a stem cell, and subsequently differentiate these into specifically matched blood cells for transplantation, will profoundly impact the lives of vulnerable patients,” she explained.

Previously, researchers faced difficulties in developing lab-grown human blood stem cells that could successfully be transplanted into animal models with bone marrow failure. The team has now established a workflow that generates transplantable blood stem cells that closely mimic those present in the human embryo. Importantly, these human cells can be produced in the scale and purity required for clinical applications.

In the study, immune-deficient mice were injected with the engineered human blood stem cells, which successfully established functional bone marrow, achieving results comparable to those seen in umbilical cord blood cell transplants—a proven benchmark of success. The research also demonstrated that these lab-grown stem cells could be frozen prior to transplantation, effectively replicating the preservation methods used for donor blood stem cells.

Professor Ed Stanley from MCRI emphasized the potential of these findings to create new treatment options for various blood disorders. He noted, “Red blood cells are essential for oxygen transport, white blood cells serve as our immune defense, and platelets facilitate clotting to prevent excessive bleeding. Understanding how these cells develop and function is akin to solving a complex puzzle. By refining stem cell methods that replicate the natural development of blood stem cells, we can advance personalized treatments for a range of blood diseases, including leukemias and bone marrow failures.”

Professor Andrew Elefanty added that while blood stem cell transplants are crucial for lifesaving treatments for childhood blood disorders, finding an optimally matched donor can be challenging. “Mismatched donor immune cells can attack the recipient’s tissues, leading to severe complications,” he cautioned. “Creating personalized, patient-specific blood stem cells can prevent these issues, mitigate donor shortages, and, when combined with genome editing, help address the underlying causes of blood diseases.”

Looking ahead, Professor Elefanty mentioned that the next phase—expected to begin in approximately five years with government funding—will involve conducting a phase one clinical trial to evaluate the safety of using these lab-grown blood cells in humans.

One inspiring case is that of Riya, who was diagnosed with aplastic anemia at the age of 11, a rare blood disorder in which the body fails to produce sufficient new blood cells. As a result, she required regular platelet and blood transfusions to maintain her blood cell count. A bone marrow transplant was recommended due to the frequency of her transfusions and the associated long-term risks. However, finding a perfectly matched donor proved difficult. Ultimately, Riya’s mother, despite being a half match, became her donor based on specialist guidance.

Professors Elefanty, Stanley, and Associate Professor Ng are also principal investigators at the Melbourne node of the Novo Nordisk Foundation Center for Stem Cell Medicine (reNEW), a global consortium focused on advancing stem cell-based therapies. Researchers from institutions including the University of Melbourne, Peter MacCallum Cancer Centre, UCLA, University College London, and the University of Birmingham contributed to these groundbreaking findings.