Projects
decoration

Microenvironmental determinants of Aplastic Anaemia progression to MDS / AML.

2019-2021 (Grant-in-Aid): Microenvironmental determinants of Aplastic Anaemia progression to MDS / AML. Associate Professor Rachel Koldej, ACRF Translational Research Laboratory, Melbourne Health.

Aplastic Anaemia is a disorder where the body stops producing enough new blood cells. Approximately 15% of Aplastic Anaemia patients are also at risk of progressing to Myelodysplasia or Acute Myeloid Leukaemia (MDS/AML) malignancy. This study aims to improve the identification of those Aplastic Anaemia patients most at risk of malignant disease progression as a first step towards the design of novel therapeutic interventions.    

Genetic mutations associated with MDS/AML are observed in almost half of Aplastic Anaemia patients, potentially putting them at risk of disease progression. However, most of these patients do not progress to Myelodysplasia or Acute Myeloid Leukaemia. This suggests other factors, (such as the bone marrow microenvironment) may also play a role in enabling or hindering disease progression. The goal of this application is to identify these additional factors involved; then determine how they impact patient disease outcomes.

Methodology includes the monitoring of bone marrow trephines from patients over time using Nanostring Digital Spatial Profiling, a new technology that allows both spatial protein and gene expression data to be tracked on single slide.

The results of this study will lead to improved biomarkers to monitor patient risk, plus potential identification of additional pathways involved in disease progression that may be targeted for therapeutic intervention. 

Related Projects

Establishing an in vivo humanised mouse model for telomere related bone marrow failure syndromes.

2019-2021 (Grant-in-Aid): Establishing an in vivo humanised mouse model for telomere related Bone Marrow Failure Syndromes. Professor Tracy Bryan, Children’s Medical Research Institute. Human genetic information is packaged into discrete bundles ...

Read more

Towards targeted treatments for Fanconi Anaemia 

2017 – 2019 Fellowship / 2017 Grant-in-Aid, Towards targeted treatments for Fanconi Anaemia, Associate Professor Wayne Crismani, St Vincents Institute for Medical Research Fanconi Anaemia is an inherited disorder which can lead to bone ...

Read more

Outcomes of Haematopoietic Stem Cell Transplantation for Paediatric Patients with Severe Aplastic Anaemia (SAA) and Bone Marrow Failure (BMF) Syndromes.

2021-2022 (Grant-in-Aid): Outcomes of Haematopoietic Stem Cell Transplantation for Paediatric Patients with Severe Aplastic Anaemia (SAA) and Bone Marrow Failure (BMF) Syndromes. Dr Steven Keogh, Australasian Bone Marrow Transplant Recipient ...

Read more

Using induced pluripotent stem cells to find causes and cures for bone marrow failure in children and young adults

2018-2023 (Grant-in-Aid): Using induced pluripotent stem cells to find causes and cures for bone marrow failure in children and young adults. Professor Andrew Elefanty, Murdoch Children’s Research Institute. Bone Marrow Failure Syndromes may be ...

Read more

Sign up to be the first to hear about our upcoming News & Events