Building a therapeutic pipeline to deliver personalised blood stem cell treatments

The Challenge

Each year, about 160 people in Australia (mostly children and adolescents) are diagnosed with bone marrow failure syndromes. These conditions often require bone marrow transplants, which depend on finding a suitable donor. Without a match, patients face limited treatment options and potentially life-threatening complications.

Project Overview

The Blood Development group at MCRI is using induced pluripotent stem cells (iPSCs) to generate hematopoietic stem cells, termed iHSCs, in the laboratory. These cells are the building blocks of blood, capable of developing into all types of blood cells.

In the project Hearts & Minds is directly supporting, the team is:

• Optimising protocols to guide iPSCs into forming functional iHSCs.
• Testing their functionality in laboratory settings.
• Using the iHSC platform to model and study stem cell defects in blood diseases, such as bone marrow failure syndromes and leukaemia.

The ultimate goal is to produce personalised blood stem cells from a patient’s own cells for bone marrow replacement. A clinical-grade iHSC product could revolutionise transplant therapy, offering safer, more effective treatments for patients with blood diseases.

The vision is clear: to harness stem cell technology to replace or reduce the need for matched-donor bone marrow transplants, improving outcomes and access for patients worldwide.

‍Funding support from Hearts and Minds Investments, as nominated by Core Fund Manager, Munro Partners.

This content was last updated in August 2025, for further information and updates, visit MCRI.