Research Pipeline

From the lab bench towards the clinic, an outline of our research strategy

Our research strategy is centred around our biology and drug discovery pipeline: leveraging the existing expertise; recruiting and training new expertise; facilitating innovative and iterative research; and engaging international collaborators through our board members, the International Children’s Brain Tumour Board.

Our pipeline consists of the following four stages:

Stage One: Paediatric Brain Tumour Biology and Models

Developing accurate models to advance our understanding of the basic biology of paediatric brain tumours is at the heart of our work at the centre.  Work includes producing viable cell lines,  genetically engineered mouse models (GEMMs) and patient-derived orthotopic xenografts (PDOXs).These models are not only used to understand the basic biology of brain tumour development, but are also needed to accurately assess therapies pre-clinically to enable a better understanding of how potential therapies might work in patients.

Research highlights from the Centre 

One of the main centre projects is developing ZFTA-ependymoma models. These tumours  driven by the ZFTA-RELA fusion, account for >70% of all supratentorial ependymomas and are associated with a worse prognosis. Work in the Gilbertson Lab describes the first spontaneous GEMM of ZFTA-RELA fusion-driven ependymoma. Nestin-Flx-STOP-Flx-ZFTA-RELA (Fusion 1) or E1alpha-Flx-STOP-Flx-ZFTA-RELA open reading frames were targeted together with luciferase to the Rosa-26 locus. Breeding these mice with Nestin-CreERT2 or Blbp-Cre lines that drive recombination in neural progenitor cells resulted in forebrain tumours that could be tracked with bioluminescence imaging from P20. These tumours  have been characterised histologically and transcriptomically relative to the human disease and are being used to understand the lineage origins of ependymoma and to assess conventional and novel treatments.

Read more about stage one of our research pipeline here.

Stage two: Drug Target Identification

Work in the Centre is ongoing to discover tumour cell proteins and pathways (targets) critical for maintaining the malignant phenotype that may serve as therapeutic targets. This research is iterative and allows insights into tumour biology generated in stage one activities and collaborative input from the International Children’s Brain Tumour Board.  Both  in vitro and in vivo target validation is being carried out at the Centre, including identification of the targets of novel drugs and chemical probes discovered in Stage Three of the Centre pipeline.


Read more about this here.

Stage three: Drug Design and Discovery

Read more abut this here

Stage four: Preclinical Testing and Trial Design


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