The Victorian Paediatric Oncology Network for Drug Safety

The challenge

Cure rates for childhood cancers are now approaching 85%. However, many children undergoing cancer treatment, radiation, and bone marrow transplants (BMT) experience serious side effects. These reactions can be mild, including hair loss, nausea, and vomiting, or more serious, such as problems with blood cell production, liver damage, or trouble breathing. Sometimes these side effects are so severe that they lead to hospital stays, impact the effectiveness of the treatment, or, in rare cases, may even be life-threatening.  

In addition, the effectiveness of chemotherapy and supportive care medications such as antibiotics, antifungals or antiemetics, may be reduced due to how their developing bodies process these drugs.

Even after finishing cancer treatment, many children experience long-term health issues caused by the treatments that saved their lives. These can include serious or chronic health conditions, from needing joint replacements and hearing loss, to developing other cancers - secondary malignant neoplasms - throughout their life.

One promising way to reduce these side effects is through pharmacogenomics (PGx) - a field of medicine that uses genetic information to predict how individuals will respond to different drugs. By understanding each child’s genetic makeup, doctors could choose medications and doses that are safer and work better for each patient.

Although guidelines exist for adopting pharmacogenomics in clinical care, they are not widely used in everyday medical practice. Most guidelines are based on research in adults, which makes it harder to apply the information to younger patients whose bodies are still developing. Research specifically focused on children is needed, and many healthcare professionals would benefit from additional education and training to feel confident using these tools.

The research

To better understand the challenges of this unmet need, 30 people with a lived experience of childhood cancer were interviewed. As a result, five research priorities were identified:

1. Better tracking of side effects in real time: By closely monitoring and reporting any unwanted reactions children have to their treatment as they happen, researchers can quickly learn more about which treatments cause which problems.

2. Understanding how children’s bodies handle different treatments: This research aims to discover how children of different ages process modern, targeted treatments.  This is the first step towards being able to check and adjust dosages as needed for each child.  

3. Learning how a child’s genes affect their response to targeted treatment: By collecting blood samples from children, adolescents and young adults that are being treated with a type of targeted therapy, tyrosine kinase inhibitors or high-cost cancer therapeutics used in BMT, their samples can be later analysed to determine whether their genetics can be used to predict their response to treatment.  

4. Making bone marrow transplants safer: By improving the monitoring of busulfan, which is used in BMTs, serious side effects or treatment failures can be reduced, and Victorian BMT care will be lifted to meet the best international standards.  

5. Improving education for healthcare providers: An education program will be developed to close the knowledge gap by giving doctors and nurses better training in using genetic information when prescribing medicines, making it easier to use these new approaches in everyday care

Collectively, these research projects have the potential to transform the way cancer treatments are tailored for children, making them safer and more effective. As a collaborative research network involving collaborators and hospitals across Victoria, research findings can be quickly implemented into clinical care, improving outcomes for children and their families.

The impact

This project unites innovative approaches to improve the safety and effectiveness of treatments for childhood cancer. The team will gather and combine detailed reports from both patients and pharmacists on medication side effects, using the latest technology to analyse and report this information. They will also lead Australia’s first study to better understand how children process complex cancer medicines, combining drug level monitoring, genetic testing, and side effect tracking for drugs that currently have limited information in young people.

Importantly, the project includes a world-first investigation into cases where a child’s reaction to a medicine doesn’t match what their genes would predict, offering new insights that could make treatments much more personalised.

The team will also evaluate whether performing critical drug level tests locally within Victoria, rather than interstate, is feasible, which has the potential to improve accuracy and speed in patient care.

Finally, the team is committed to building expertise among healthcare professionals, laying the groundwork for more advanced research in the future and ultimately aiming to improve results for children and young people with cancer.

Children's Cancer CoLab Funding Information

Grant Awarded: $1.76 million

Project Timeline: August 2025 – July 2027

Impact Programs: Safer Therapies

Scientific Review: Children’s Cancer CoLab Scientific Advisory Committee

Lead Institution: Murdoch Children’s Research Institute

Collaborating Institutions: Peter MacCallum Cancer Centre, Monash Children’s Hospital, Princess Máxima Center for Pediatric Oncology and Royal Melbourne Hospital

Lead Researcher: A/Prof Rachel Conyers

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