Professor Michelle Haber

Michelle’s earlier studies were amongst the first characterising the complex molecular mechanisms underlying therapy-related drug resistance. She identified the relationship between high expression of multidrug transporter gene MRP1, the malignant phenotype of neuroblastoma and poor clinical outcome – the first definitive demonstration of clinical relevance of the MRP1 gene in human solid tumours. She subsequently established high-throughput chemical screening of small molecule libraries and developed novel MRP1 inhibitors for the treatment of neuroblastoma and other MRP1-assciated malignancies. This led to a $3.1M ACRF award to establish a Drug Discovery Centre for Childhood Cancer which is now developing a pipeline of potential new drugs for treating childhood and adult malignancies.

 Michelle pioneered polyamine inhibition therapy as a new approach to child cancer treatment, leading to one completed international trial in 15 children’s hospitals, one current trial open in 120 hospitals internationally, and two more being planned for neuroblastoma and for brain tumours. She and her colleagues identified targeting of the chromatin modifier FACT as a novel therapy for neuroblastoma, acute lymphoblastic leukaemia (ALL), and DIPG and based on these findings, a clinical trial using FACT inhibition, led by the Children’s Oncology Group, the largest children’s cancer study group in the world, will open this year. She was also a leader in the team developing OT82, a novel inhibitor of NAMPT and in developing NAMPT inhibitor therapy for paediatric ALL, with an adult Phase 1 open, and planning for a paediatric trial underway.

 She also played a major role in developing PCR-based technology to detect the presence of minimal residual disease (MRD) in bone marrow of children with ALL, after initial cancer treatment. The prognostic value of this assay was proved in several national trials, with a large international clinical trial of 1100 children, resulting in a doubling of survival rates for children at high risk of treatment failure from <40% to >70%. This approach has now become standard of care for Australian children with ALL. This laid the foundation for establishment of the national ZERO Childhood Cancer Personalised Medicine Program. She has successfully led the development and roll-out of  ZERO, to every Australian paediatric oncology facility, currently available to children with the most aggressive malignancies, and to be made available to every child and young adult with cancer in Australia, by 2023. ZERO has improved outcomes for children with high-risk cancers and has transformed the approach of managing high-risk childhood cancers nationally. 

 Michelle is currently continuing work on identifying and validating the clinical role of molecular targets responsible for treatment failure in aggressive childhood cancers, with the overall goal of improving clinical outcomes for children with cancer.