The challenge
Each year in Australia, more than 2,000 people receive blood stem cell transplants to treat serious conditions including blood cancers, bone marrow disorders and immune diseases. For many patients, treatment depends on finding a closely matched donor. When no suitable match is available, the risks increase significantly, including serious and sometimes life-threatening complications. As a result, not every patient can access a safe transplant when they need one.
Riya’s experience reflects this challenge. She was diagnosed with severe aplastic anaemia at age 11. With no fully matched donor available, she received a partially matched transplant from her mother. While the transplant was successful, she experienced significant complications as a result of the imperfect match and spent three months in hospital.
Experiences like Riya’s are driving the work of the Blood Development Group, co-led by Professor Andrew Elefanty and Associate Professor Elizabeth Ng at Murdoch Children’s Research Institute. Researchers are developing blood stem cells from a patient’s own cells, which could allow patients to receive a transplant made specifically for them, without relying on donor matching.
The breakthrough
The team has demonstrated a pathway toward creating blood stem cells in the laboratory - cells capable of developing into the key types of blood cells the body needs, including infection-fighting immune cells and red blood cells.
In early studies, these lab-grown cells have successfully established and regenerated blood systems in pre-clinical models, demonstrating their potential for blood stem cell transplantation. Importantly, this early success has since been translated into a scalable process that has been assessed as suitable for clinical-grade manufacturing, bringing it one step closer to future patient treatments.
For a patient like Riya, this would mean a very different path, with a treatment built from her own cells, without the need for a donor search or the complications that come with a partial match.
The role of philanthropy
Hearts & Minds funding, as nominated by Munro Partners, has supported this work at a critical early stage, enabling the development and testing of laboratory-grown blood stem cells. This support has helped accelerate progress from concept toward validated early results.
This is where philanthropy can make a difference: flexible funding that enables researchers to pursue ambitious ideas, retain highly skilled staff, and maintain momentum between major grant funding cycles. Without it, promising early science often stalls before it can generate the evidence needed to scale.
What this could unlock
The technology developed by the Blood Development Group is already being used by research laboratories globally to accelerate studies into blood diseases and gene therapies. Its significance has been recognised through major publications, international collaborations, and a $35 million commercial licensing agreement with US biotechnology company Retro Biosciences (2025), validating both its scientific credibility and its clinical potential.
"This research brings us closer to a future where patients can receive blood stem cell transplants created from their own cells. By turning patients into their own perfect donor, we have the potential to transform treatment for blood diseases and dramatically improve outcomes."
A/Prof Elizabeth Ng
The next phase of the project will focus on translating this laboratory breakthrough into clinical trials and scalable manufacturing, bringing personalised blood stem cell transplants closer to patients like Riya and others still waiting for a match.
Funding support from Hearts & Minds, as nominated by Core Fund Manager, Munro Partners. This content was last updated in July 2026. For further information, visit Murdoch Children's Research Institute.
