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19
Mar
2018

Australian Paediatric Brain Cancer Trial using Organoids for a Personalised Medicine Program

An Australian first pilot study, undertaken by scientists at the Hudson Institute of Medical Research in Victoria, is using personalised medicine to target brain cancer and solid tumours in children, with a specific focus on Diffuse Intrinsic Pontine Glioma (DIPG).

This revolutionary trial is testing thousands of drug combinations to identify the treatment with the highest chance of success for each individual patient in the study. These drug combinations are being tested on organoids, clusters of cells that resemble a miniature version of the tumour, grown in a lab from the patient’s own tumour cells. These organoids are a three-dimensional culture that mimics the tumour’s unique genetic profile, making them ideal for applications in personalised medicine. 

Organoids are tested for genetic mutations using specialised genetic tests, called CRISPR screens. This aims to identify key genetic mutations in the tumour that can then be targeted with specific drugs. The drug combinations are then trialled on the organoid, in a process that takes approximately 3 weeks.

“Every child’s tumour is genetically unique and responds to cancer treatment in different ways. Knowledge of the genetic variability of paediatric tumours is building at a fast pace, and this program is aimed at translating this information into treatment”, remarked Associate Professor Ron Firestein, lead of Hudson’s Centre for Cancer Research.

The pilot study in Victoria is well underway, with 10 organoids already grown from paediatric tumours. Combining information about the tumour’s genetic mutations, drug sensitivity and drug resistance, allows clinicians to make treatment decisions specific to each child approximately 4 to 5 weeks after diagnosis, rather than months. 

An additional benefit of the study is the establishment of a biobank, a living collection of brain tumours and solid cancers. This developing biobank is part of a collaborative project with US researchers to conduct DNA sequencing on the tumour samples. This aims to build a library of data that correlates genetic mutations with appropriate responses to treatment, to provide more accurate and timely advice to doctors about the best medication for each patient.

This personalised approach has informed doctors to use a specific drug originally approved for melanoma, in a three-year old brain tumour patient. This follows genetic screening procedures in the pilot study that discovered the drug could be more effective than standard chemotherapy to treat the young girl’s tumour. The tumour has since halved in size. 

Head of Monash Children’s Hospital Cancer Centre, Dr Peter Downie remarked, “My hope would be that within another 15-20 years, chemotherapy will become almost redundant and that a lot of these newer, more specific agents will become drugs of choice.”

All children in Victoria with solid tumours are invited to join the clinical trial, with the aim to personalise cancer treatment and offer treatment options relevant to patients now, rather than waiting many years for laboratory research and future drug development. Firestein stated that this is purely about “giving the child the best treatment option we have right now.”