A world-first study involving more than 100 cancer researchers and clinicians across Australia – including from Telethon Kids Institute and Perth Children’s Hospital – has shown that precision medicine can significantly improve outcomes for children with high-risk cancer.
Precision medicine involves the delivery of treatment tailored to the individual child’s cancer, based on detailed genetic analysis of each tumour’s driver genes.
In a study that is rewriting the narrative for children with aggressive cancers, a staggering 55 per cent of children who received personalised treatment as part of the Australia-wide trial achieved complete or partial remission, or had their disease stabilised for at least six months.
The study, led by the Children’s Cancer Institute in Sydney and published overnight in the journal Nature Medicine, included 384 children with high-risk cancers which, in many cases, had failed to respond to standard therapy.
The children were recruited from every children’s hospital in Australia through the Zero Childhood Cancer Program (ZERO), Australia’s national precision medicine program for children with cancer. ZERO is jointly led by the Children’s Cancer Institute and Kids Cancer Centre at Sydney Children’s Hospital, Randwick and involves cancer research and treatment organisations across the country, including the Telethon Kids Cancer Centre and Perth Children’s Hospital.
The just-published study found precision medicine was superior to standard or non-guided therapy, in terms of both clinical response and survival.
Children involved in the study received tailored treatment recommended to their oncologists by ZERO’s experts following detailed genetic analysis of their individual tumour’s driver genes. This approach enabled drugs to be better matched to the child’s cancer driver genes, with the analysis often suggesting drugs not normally used to treat that cancer type.
Children who received the personalised medicine were observed for an average of three years following treatment, with the results showing an improvement in progression-free survival – the length of time a patient lives without their cancer getting any worse.
The results showed children who received a ZERO-recommended treatment did significantly better than those who did not. In fact, their two-year progression-free survival was more than double that of children who received standard therapy (26% vs 12%), and five times higher than that of children who received an unguided treatment (a novel agent not chosen on the basis of molecular findings).
Importantly, the study found that children who received their recommended therapy early on in their treatment pathway did significantly better than those who received it after their disease had progressed, suggesting that the sooner a personalised treatment strategy can be implemented, the better the chance of preventing relapse and death.
A team led by clinician-researchers Professor Rishi Kotecha and Professor Nick Gottardo, from Telethon Kids Cancer Centre and Perth Children’s Hospital, led the Perth arm of the trial and are also involved in the next phase, ZERO2, which has begun expanding personalised treatment to every child diagnosed with cancer in Australia, rather than just those with high-risk cancers.
Professor Kotecha, who is Co-Head of Leukaemia Translational Research at Telethon Kids, said the newly published results confirmed precision medicine as a valuable new tool for tackling childhood cancer.
“What precision medicine gives us is another avenue with which to tackle these tumours in children, in addition to existing therapies,” Associate Professor Kotecha said.
“It’s effectively a fifth arm that we can now think about using in conjunction with those other therapies to improve cure rates – a tool that gives us further options to guide therapy, and provides a lot more information about each individual tumour than we previously had access to.”
For more information on the trial and on ZERO, see here.