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The Zero Childhood Cancer Program

Zero Childhood Cancer is the most exciting childhood cancer research initiative ever undertaken in Australia. This world-class program brings together all major Australian clinical and research groups working in childhood cancer to offer Australia’s first ever personalised medicine program for children with high-risk or relapsed cancer.

Led by Children’s Cancer Institute and The Kids Cancer Centre at Sydney Children’s Hospital Randwick, part of The Sydney Children’s Hospitals Network, Zero Childhood Cancer is an unprecedented collaboration that stands to revolutionise the way childhood cancer is treated, and represents an integral step towards curing every child.

What has happened so far?

The first stage of the Zero Childhood Cancer program began in 2016 with a pilot study for children with high-risk cancer being treated in New South Wales. During this pilot, the platform required to create a personalised medicine pipeline was established and tested. This involved multiple steps, including developing a reception process for cancer samples being sent from all over Australia to a centralised tumour bank, and establishing the best possible methods for molecular profiling, drug screening and drug efficacy testing.

In 2017, following successful completion of the pilot study, a national clinical trial was launched. At 31 October 2018, 127 children with high-risk cancers from all over Australia have been enrolled on the state-of-the-art trial, which will run for three years. The trial is free to all children who meet enrolment criteria and is being sponsored by the Australian and New Zealand Haematology/Oncology group (ANZCHOG).

How does it work?

Each year in Australia, about 200 children and adolescents are diagnosed with a type of cancer that is very difficult to treat and has a less than 30% survival rate, either on first diagnosis or after relapse. It is these children – including those suffering the most aggressive forms of brain tumours, sarcomas, infant leukaemias and neuroblastomas – who Zero Childhood Cancer is for.

As soon as a child is diagnosed with high-risk or relapsed cancer and is enrolled in the Zero Childhood Cancer clinical trial, a sample is taken of their cancer and sent to Children’s Cancer Institute. Here, researchers leverage the most innovative technology available to analyse the genetic and molecular make-up of those cells, and use robotic technologies to test hundreds of medicinal drugs against the cells to identify which drugs are most likely to be effective. Once a promising drug is identified, it is tested in living laboratory models to see how it performs. The results of all the laboratory analysis and testing are then made available to an expert team of clinicians and scientists (the Multi-Disciplinary Tumour Board) who use the data to develop a precise personalised treatment plan – a plan that is specifically tailored to suit that individual child.

This personalised approach is what sets Zero Childhood Cancer apart and makes it so exciting. The challenge in curing every child is that every child’s cancer is unique. A treatment that works for one child may not work for another. Anti-cancer treatments have harsh effects on children’s developing bodies, so nobody wants to subject a child to a toxic treatment that is not going to cure their cancer. What’s more, almost half of all children treated for cancer go on to suffer late effects of that treatment as adults. By analysing each child’s unique cancer cells and finding therapies that specifically target these cells (‘targeted therapy’), Zero Childhood Cancer gives each child the best chance of survival while at the same time minimizing their risk of debilitating side effects. This approach holds the promise of one day allowing us to cure every child of cancer and give them the best possible quality of life.

What results are available?

The Zero Childhood Cancer clinical trial is showing some very promising results. During the first 12 months of the trial, a personalised treatment plan was able to be recommended for almost 70% of participating children, within an average turnaround time of only 9 weeks from the time their tumour sample was received. And for some, the trial has proven to be a life-saver.

One such child is Ellie, who at 11 months of age was diagnosed with a rare and aggressive tumour called infantile fibrosarcoma. At the time of diagnosis, Ellie’s tumour had grown so large it had forced her heart over to one side of her chest and her lungs could no longer work properly. Ellie was on life support when the Zero Childhood Cancer team received her cancer sample. Extensive genetic analysis revealed that Ellie’s tumour had a rare translocation (a type of chromosome mutation) likely to be driving the growth of the cancer. A new drug targeting this exact translocation was identified and Ellie was put on the drug straight away. Within six weeks of treatment, Ellie was able to go home.

Ellie’s case shows the power of Zero Childhood Cancer as a personalised medicine program. By recognising that each child’s cancer is unique, and tailoring treatment on the basis of the scientific analysis of that specific cancer, it is possible to change outcomes.

Where to from here?

The Zero Childhood Cancer clinical trial will remain open until 2020 and will be recruiting new children with aggressive cancer each year until then. Due to the complex nature of childhood cancer, the extraordinary outcome that Ellie experienced will not be possible for every child. However, the more that is learned through the trial, the more remarkable success stories like Ellie’s are likely in the future.

Through the trial, Zero Childhood Cancer is generating a wealth of new research data, including valuable molecular and genetic information about childhood cancers. This data is adding enormously to our knowledge and understanding of childhood cancer and will be shared nationally and internationally to contribute to future research discoveries. In this way, Zero Childhood Cancer is not only benefiting children today, but will continue to benefit all children with cancer in the future.