The Hudson Monash Paediatric Precision Medicine (HMPPM) Program hopes to significantly improve treatment for childhood cancer patients with the greatest unmet clinical need – those diagnosed with brain cancers and solid tumours. The program was established in 2017 with investment from the Children’s Cancer Foundation.
In a collaborative program, scientists at Hudson Institute of Medical Research have established a living biobank of paediatric brain tumours and solid cancers, to trial and develop targeted treatments and improve clinical outcomes, survival rates and quality of life (limiting side-effects) for childhood cancer patients.
Innovation in the making
The HMPPM Program marks a significant investment in future clinical management and novel research discovery in childhood cancer. The program includes:
The development of a living tumour biobank for paediatric solid tumours
At present, very few reliable patient-derived preclinical models are available to researchers. To bridge this gap, our program establishes and banks cell lines, 2D and 3D xenograft models directly from childhood tumour tissue. The establishment of a living biobank for paediatric solid tumours provides a critical renewable resource for local, national and international researchers.
The establishment of a functional genomics pipeline
We capitalise on the living biobank tumour samples to integrate genomic data (next generation sequencing) with functional data obtained from high-throughput genetic screening (Cas9/CRISPR) and results from global pharmacological drug screens.
Multi-dimensional analyses of paediatric cancers
We will comprehensively profile paediatric cancers (both tissue and matched model) in 4 dimensions (genomic, epigenomic, transcriptomic, and proteomic) to provide both gene-specific and pathway level analyses of paediatric tumours. This work enables both a richer biological understanding of paediatric cancers and facilitates the utilisation of diverse molecular variables in cancer precision medicine.
Translation of genomic data into targeted therapy
The comprehensive molecular analysis of individual patient tumours will help identify both new and existing therapies that can be rapidly implemented in the clinic. This approach will facilitate clinical implications of data from the functional genomics pipeline for individual paediatric patients.
Molecular Tumour Board (MTB): integrating findings into clinical management
Working in collaboration with the Zero Childhood Cancer (ZCC) and PRISM program (zerochildhoodcancer.org.au), the paediatric cancer molecular MTB will integrate molecular and functional diagnostic assays into clinical management of childhood cancer patients, by providing expertise and recommendations on the clinical implications of findings that arise from molecular analyses.
Unique national and global collaborations
The establishment of a living biobank and functional genomic testing for paediatric solid tumours provides a critical resource for local, national and international researchers. Thus, a key element of the program includes national and international stakeholders’ involvement to build expertise, share resources and disseminate results that will advance the field of precision medicine for paediatric cancer patients. For more information, see the Affiliations and Partners section below.
Progress to Date
To date, we have collected tissue samples from 223 patients (including collaborator sites) and successfully established 3-dimensional cell cultures (‘organoids’) of paediatric solid tumours as well as 2-dimensional cell cultures. We have conducted over 200 high-throughput drug screens and identified a number of promising therapeutic targets for paediatric brain cancers and sarcomas.
Our scientific team has streamlined the process of drug screening (within a few weeks post sample collection), data generation and integrative analysis to efficiently identify cancer dependencies for individual patients; and have devised a functional genomics pipeline that generates CRISPR loss of function sensitivity data for patient-derived cell lines.
Other research directed at molecular and cell pathways, with investigation into pharmacogenomics, diversity of cancer genetics, pluripotent stem cells is underway, and offers excellent opportunities for young researchers.
A/Prof Ron Firestein | Head of Research and Chief Investigator
Dr Jason Cain | Chief Investigator
Dr Daniel Gough | Chief Investigator
Prof Annie Huang | Chief Investigator
Dr Peter Downie | Head of Clinical Enterprise
Naama Neeman | Children’s Cancer Foundation Administrative Director
Prof Bryan Williams | Strategic Advisor
Dr Paul Daniel | Children’s Cancer Foundation Postdoctoral Research Fellow
Caroline Drinkwater | Australian Lions Childhood Cancer Research Foundation Senior Biobank Specialist
Dr Claire Sun | Children’s Cancer Foundation Bioinformatician
Dr Nataliya Zhukova | My Room Clinical Research Fellow
Dr Paul Wood | Paediatric Oncologist
Dr Gabrielle Bradshaw | Alfie Chivers Research Fellow – funded by Robert Connor Dawes Foundation
Melissa Loi | Children’s Cancer Foundation Technical Sequencing Specialist
Hui (Claire) Shi | Children’s Cancer Foundation PhD Student
Sarah Parackal | Children’s Cancer Foundation PhD Student
Dasun Fernando | PhD Student
Motahhareh (Flora) Tourchi | PhD Student
Jay Yang | Masters Student
Mia Pörksen | Internship
Prof Roger Daly | Molecular analysis group (proteomics)
Prof Roger Reddel | Molecular analysis group (proteomics)
Affiliations and partners
Zero Childhood Cancer (Children’s Cancer Institute)
Australian & New Zealand Children’s Haematology/Oncology Group (ANZCHOG)
Monash Children’s Hospital
The Royal Children’s Hospital
Murdoch Children’s Research Institute
Queensland Institute of Medical Research Berghofer
Children’s Brain Tumor Tissue Consortium (CBTTC)
Hospital for Sick Children (SickKids)
Children’s Hospital of Philadelphia
University of Santa Cruz
KK Women’s and Children’s Hospital, VIVA Foundation for Children with Cancer
German Cancer Research Centre (DKFZ)
Beijing Genomic Institute (BGI)
The Hudson Monash Paediatric Precision Medicine Program is made possible thanks to generous and exclusive funding from the Children’s Cancer Foundation.
We are also thankful for the support of the Australian Lions Childhood Cancer Research Foundation (ALCCRF) and the Robert Connor Dawes (RCD) Foundation.
Children’s Cancer Foundation Administrative Director
Hudson Monash Paediatric Precision Medicine Program