Personalised Medicine Will Be Game-changer

Paediatric cancer is now understood to be a disease driven by genetic mutations and children respond differently to standard treatments based on the biological subtype of their cancer.

This revolution in molecular and genomic techniques has allowed researchers to develop targeted therapies for children with particular genetic changes, resulting in a dramatic improvement in survival rates for patients with specific genetic markers. This approach is often referred to as ‘personalised medicine’.

The Children’s Cancer Foundation has prioritised clinical research, building on its long legacy of both constructing the research laboratory at The Royal Children's Hospital in 2004 and funding research initiatives since the 1990s.

The appointment of Associate Professor Paul Ekert, Head of Molecular Diagnostics at the Children’s Cancer Centre at The Royal Children's Hospital and Head of Cancer Research at Murdoch Childrens Research Institute in 2014, funded in partnership with the Steven Walter Children’s Cancer Foundation, is key to our commitment to molecular diagnostics and the translation of genetic research into clinical applications.

Over the past 18 months, Ekert has built a team of researchers to work on a suite of research projects. Most importantly he and his team are changing the treatment pathway for children by developing the diagnostic platforms that can support clinical decision-making.

By identifying the genetic changes, for example in high risk Acute Lymphoblastic Leukaemia, some children have received a different treatment protocol that targets the genetic mutations that cause high risk disease.

“The incredible advances in genomics have fundamentally changed how we think about childhood cancer and how we will approach treatment in the future. In the coming years, the clinical trials that test the great promise of personalised medicine will be game-changers. It is exciting and a privilege to be part of this work,” Ekert said.

Ekert’s research team is also working on a gene panel for solid tumours that can be used to enhance clinical decision-making. The selection and assessment of genetic abnormalities to build an evidence base is critical for improving treatment decisions. Ekert and his team are defining the clinical significance and the clinical actions that can be linked to abnormalities in a panel of 300 genes to produce a diagnostic tool that will be available for use within the next 2 years.

The Foundation has committed more than $1 million from 2014 to 2018 in salary and research funding to advance the development of molecular diagnostics through Ekert and his team, to better customise therapies to the unique needs of each patient. This innovative research is working towards lifting survival rates and reducing the debilitating side effects of treatment, including infertility and brain damage.

AProf Paul Ekert