A study into precision medicine for high-risk childhood cancer has revealed encouraging results.
Australian researchers and clinicians have shown that precision medicine - personalised treatment tailored to an individual child's cancer - leads to significantly improved outcomes in children with high-risk cancer.
In a world-first study published this week in Nature Medicine, the researchers showed that precision medicine was superior to standard therapy, and to therapy that wasn't guided by molecular findings - both in terms of clinical response and survival.
The researchers followed the children in the study cohort for an average of three years after receiving their personalised treatment, and gave treatment recommendations to their oncologists after detailed genetic analysis of the driver genes in the tumour. This strategy matched the drug better to each child's cancer driver genes, often suggesting drugs that are not normally used to treat that cancer type.
The results show, for the first time, that this led to an improvement in progression-free survival: 55 per cent of children who received their personalised treatment achieved complete or partial remission, or had their disease stabilised for at least six months. Given that that these children had highly aggressive cancers which, in many cases, had already failed to respond to standard therapy, the researchers say these are remarkable results.
"These are very exciting results which we believe have important implications for the treatment of children with cancer," said UNSW Conjoint Professor David Ziegler, Chair of Clinical Trials for the Zero Childhood Cancer Program and senior author on the paper.
"We've already shown that precision medicine can help identify new treatment options for many high-risk patients. Now we've shown that it not only can shrink their tumours, but also lead to a significant improvement in long term survival for those patients."
Over a minimum follow-up period of 18 months, the study included 384 children with high-risk cancers (with a very low chance of cure) enrolled on the Zero Childhood Cancer Program (ZERO), Australia's national precision medicine program for children with cancer. ZERO is jointly led by Children's Cancer Institute and Kids Cancer Centre at Sydney Children's Hospital, Randwick and involves nine child cancer treatment centres in the country. This study involved more than 100 scientists and clinicians working together across these treatment centres.
