Projects
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Identifying therapeutics which can be repurposed for the treatment of Myelodysplastic Syndromes and other bone marrow failure disorders.

2018-2021 (Grant-in-Aid): Identifying therapeutics which can be repurposed for the treatment of Myelodysplastic Syndromes and other bone marrow failure disorders. Associate Professor Amee George, The Australian National University.

There are currently limited treatment options for patients with Bone Marrow Failure Syndromes. This study aims to identify known drugs that could be repurposed to treat Diamond Blackfan Anaemia (an inherited Bone Marrow Failure Syndrome) and/or myelodysplasia (a bone marrow failure condition associated with malignancy), both with limited current treatment options.

Libraries of 4169 and 3500 FDA approved compounds are available for this study. To identify new drugs that may potentially delay Diamond Blackfan Anaemia progression, library compounds that modify the p53 pathway have already been screened (the protein p53 was chosen as when activated it is thought to contribute to the red blood cell failure). A second screen for treatment of myelodysplasia, will select for drugs that impact myelodysplasia cell survival or differentiation in culture. Top hits from both screens will then be validated in patient bone marrow cultures before confirmation in preclinical models.

The overall goal of this research is to identify drugs already used clinically which could be potentially repurposed for the treatment of bone marrow failure syndrome caused by Diamond Blackfan Anaemia and/or myelodysplasia as a way to fast-track availability of new therapeutics to patients. 

Related Projects

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 ...

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Discovering new genes and mutations that cause failure of bone marrow neutrophil production. 

2018-2020 (Grant in aid): Discovering new genes and mutations that cause failure of bone marrow neutrophil production. Professor Graham Lieschke, Australian Regenerative Medicine Institute/Monash University and The Royal Melbourne Hospital. ...

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Influences of clonal haematopoiesis in allogeneic bone marrow transplantation.

2018-2020 (Grant in aid): Influences of clonal haematopoiesis in allogeneic bone marrow transplantation. Dr Paul Yeh and Professor Mark Dawson, The University of Melbourne. Dr Paul Yeh’s research focusses on using genetic testing to study clonal ...

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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 ...

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