Pioneering Project To Provide Safer Childhood Cancer Treatments

Murdoch Children's Research Institute

The safety and effectiveness of childhood cancer treatments will be transformed under a pioneering genetics project.

The project, led by Murdoch Children's Research Institute (MCRI), has received $1.76 million from the Children's Cancer CoLab to explore how a person's genes are impacted by cancer treatments, with the aim of reducing side effects, improving quality of life and maintaining survival outcomes for young patients.

The announcement was made at MCRI by Medical Research Minister Danny Pearson, MCRI Prof Kathryn North AC, MCRI Professor David Eisenstat and Children's Cancer CoLab CEO Dr Udani Reets.

Image: Professor David Eisenstat, Associate Professor Michelle Yong, Karen and Marco, Minister Danny Pearson, Dr Udani Reets, Professor Kathryn North and Professor Mark Dawson

Image: Professor David Eisenstat, Associate Professor Michelle Yong, Karen and Marco, Minister Danny Pearson, Dr Udani Reets, Professor Kathryn North and Professor Mark Dawson

Despite childhood cancer cure rates having reached 85 per cent, most are treated with drugs originally developed for adults, causing uncertainty about how each child's body will respond to toxicities of treatment.

Project lead MCRI Associate Professor Rachel Conyers said pharmacogenomics, which uses genetic information to predict drug responses, could help reduce side effects caused by cancer treatments.

"Pharmacogenomics is like having a personalised instruction manual for each child's body, telling doctors which medications will work best, and which doses will be safest," she said. This information is crucial because children process medications differently from adults, yet most treatment guidelines are based on adult research."

Children's cancer expert Rachel Conyers

Image: Associate Professor Rachel Conyers

Professor Conyers' team interviewed 30 people with a lived experience of childhood cancer to help guide the program's direction. These conversations lead to the creation of five interconnected initiatives that will use pharmacogenomics to improve outcomes for childhood cancer patients.

1. Better real-time tracking of side effects

Monitoring reactions as they happen and analysing each child's genetic makeup to identify which treatments cause specific side effects to provide safer care.

2. Understanding how children's bodies handle different treatments

Conducting Australia's first clinical study into how children process targeted cancer medicines, combining drug level monitoring, genetic testing and detailed side effect tracking. The results could help doctors predict which children need lower doses or different medications, preventing serious complications before they occur.

3. Learning how genetics affect responses to targeted therapies

A world-first clinical study examining cases where children's reactions to medications don't match what their genetics would predict, unlocking new insights into personalised medicine.

4. Building Victorian capacity for safer bone marrow transplants

Exploring whether Victoria could create its own program to monitor levels of Busulfan, a key chemotherapy drug used in the bone marrow transplants, rather than sending blood samples interstate, allowing doctors to track drug levels and adjust doses in real-time to reduce treatment complications, cut costs and eliminate delays.

5. Improving healthcare education

An extensive education program will train doctors and nurses to confidently use genetic information when prescribing medicines, closing the knowledge gap that prevents widespread adoption of personalised treatment approaches.

Children's Cancer CoLab CEO Dr Reets said for families facing childhood cancer the project represented hope for a future where treatments were safer and more effective.

"Due to each child's genetic makeup, some treatments simply do not work as intended or are metabolised differently, which can lead to devastating outcomes - a fact we are determined to change," she said. This project will put world-class Victorian research at the forefront of global efforts to make childhood cancer treatments more personalised and less toxic on developing bodies."

The project, which is part of the Children's Cancer CoLab's Safer Therapies Impact Program, is supported by the Children's Cancer Foundation and the State Government.

Children's Cancer Foundation CEO Margaret Fitzherbert said, "Children with cancer currently receive the same aggressive treatments as adults. As survival rates climb, the next frontier is ensuring children with cancer can undergo treatments free from devastating side effects."

Karen's son Marco, 18, was diagnosed with leukemia two years ago.

Karen and her son Marco, who was diagnosed with Leukemia as a child

Image: Marco, 18, has recovered from cancer.

After undergoing pharmacogenomic testing, his doctors were able to better tailor his care and medicine dosage levels.

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