Dissecting immune dysregulation in acquired bone marrow failure syndromes to identify new therapeutic leads. 

2022-2025 (Fellowship): Dissecting immune dysregulation in acquired Bone Marrow Failure Syndromes to identify new therapeutic leads. Associate Professor Rachel Koldej, ACRF Translational Research Laboratory, Melbourne Health.

Bone Marrow Failure Syndromes are a group of disorders where low white blood cell and platelet counts lead to increased risk of infection and bleeding. With an overall mortality rate of 50-70%, new treatment options to improve outcomes for Bone Marrow Failure Syndromes are urgently required. The only current treatment option for these conditions is the transplant of new bone marrow from a healthy donor. However, the new bone marrow will not always engraft in patients. Dr Koldej’s research seeks to understand why.

The goal of this fellowship is to utilise an innovative new technique called Digital Spatial Profiling to understand how the bone marrow in Bone Marrow Failure Syndrome patients differ, and how this may contribute to poor outcomes post-transplant.  In early studies, Dr Koldej found many patients with Bone Marrow Failure Syndromes or poor transplant graft outcome share an immune dysregulation and/or inflammation of the bone marrow. In this fellowship, Dr Koldej investigates how these changes may impact bone marrow failure and develop an assay system for testing of new therapeutic drugs using preclinical models. Outcomes of this fellowship will provide new insights on how Bone Marrow Failure Syndromes may develop, and the potential use of therapeutics that target dysregulated inflammation in alleviating Bone Marrow Failure Syndromes and transplant outcomes in patients.