Issue #115
July 2021
Every child diagnosed with cancer to have access to personalised medicine for the first time

Associate Professor Paul Ekert, a speaker at the RCPA’s annual conference, says the Zero Childhood Cancer National Personalised Medicine Program (ZERO) is currently delivering significant outcomes for children in Australia who are suffering from the most aggressive and high-risk cancers. Consequently, the Program has been expanded to include all children with cancer over the next four years.

A/Prof Ekert is Group Leader, Translational Tumour Biology and co-Theme head, Personalised Medicine at Children’s Cancer Institute. He explains that preliminary results from ZERO, which aims to find personalised treatments for children with cancer, have been very optimistic and that it is feasible to apply the techniques, potentially, to all children with cancer.

“What we found with phase one of the study is that our approach is feasible in high-risk cancer and that it offers the potential to find a significant number of possible treatment targets in these children with cancer. This next phase of the project will expand to not only include children with high-risk cancer, but all children diagnosed with cancer in Australia. By the end of 2023, we expect that all Australian children and young people with cancer will be able to benefit from ZERO, regardless of the cancer they have. It is our aim that every child diagnosed with cancer in Australia will have access to personalised medicine for the very first time,” said A/Prof Ekert.

Once a child is enrolled in the trial, a sample of their cancer is sent to the laboratory for assessment. ZERO combines whole genome and transcriptome sequencing and DNA methylation profiling for central nervous system (CNS) tumours and sarcomas. Where possible, drugs considered most likely to be effective against a child’s cancer will be tested on laboratory models of that disease to see how they perform. Once the cancer has been assessed, a potential treatment plan will be tailored to each individual child by an expert team of clinicians and scientists.

“Through ZERO, we identified the genetic basis of a child’s cancer in more than 90 per cent of cases and in 70 per cent, we identified at least one new potential treatment option based on their cancer’s genetic makeup. What we are now trying to understand is what kind of value this kind of testing has, and under what circumstances, for the rest of the childhood cancer population. From this year, all children in Australia will be eligible to be enrolled in this study, progressively over the next four years. If their treating physicians and their family see value in being enrolled in this study then it would be made available to them, funded through the Medical Research Future Fund (MRFF) and Minderoo Foundation,” said A/Prof Ekert.

Every year in Australia, more than 1,000 children and young adults are diagnosed with cancer. The survivors of childhood cancer often suffer serious side effects, sometimes for the rest of their lives. At least two-thirds of survivors are significantly impacted later in life by the effects of treatment, including organ dysfunction, impaired fertility, and secondary cancers.

“Through our approach, we are able to extract the medically relevant information from a particular cancer. This information then informs patient diagnosis, identifies new potential treatment options, and identifies families that may be at higher risk of developing additional childhood cancers. Beyond knowing whether this makes a clinical difference to children with high-risk cancers, there is more to come from this work. We might be able to find out which children could benefit from specific therapies like immunotherapies or we may be able to apply this to children with more curable or lower risk cancers.

“What we have also shown is that at a national scale we can bring the whole cancer community together and all participate in a really cutting-edge study and show that this is really feasible for children with cancers. By personalising treatment, we can make sure each child has the best chance of getting the best treatment – the one that’s going to be most effective against their cancer,” said A/Prof Ekert.

 

 

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