Our world class Scientific Advisory Board strongly recommended we fund this project due to the quality of research and potential to make a difference to treatments.

A promising new drug combination for aggressive neuroblastoma

Many of our supporters will be familiar with Dr Jamie Fletcher from Children's Cancer Institute and Dr Toby Trahair from Sydney Children’s Hospital, and the incredible research they lead on high-risk neuroblastoma.

In the following video, the team outlines their proposal to identify new treatment combinations for high-risk neuroblastoma based on the drug venetoclax.

At present nearly half of all children diagnosed with high-risk neuroblastoma either do not respond to therapy or initially respond but subsequently relapse with incurable disease. This is an unacceptably low success rate.

Venetoclax is a relatively new drug that is proving effective against a range of cancers. Jamie and Toby’s initial studies suggest that venetoclax may also be effective against high-risk neuroblastoma, however the experience with adult cancers suggests that it is likely to be most effective in combination with other drugs. At present, the optimal approach to venetoclax combination therapy for high-risk neuroblastoma is completely unknown.

This study aims to identify effective combinations with venetoclax using the state-of-the-art robotic drug screening facility at Children’s Cancer Institute. Promising combinations will be assessed for effectiveness in representative panels of laboratory models of high-risk neuroblastoma developed directly from children’s tumours. The models were previously developed with the support of Neuroblastoma Australia.

The study is designed to generate the evidence required to support a clinical trial for the most effective combination. There are also opportunities to identify features of an individual child’s tumour that predict the likely effectiveness of the combination. In the longer term, this study has the potential to directly impact on survival rates and quality of life for children with high-risk neuroblastoma by identifying a more effective and safer therapy.

Achieving our mission

Jamie and research fellow discussing project

Jamie's project is in line with Neuroblastoma Australia’s mission to develop better, safer and more effective treatments for children with neuroblastoma. Jamie’s research is driven by his desire to improve the lives of children diagnosed with cancer and he’s proud to be working with Neuroblastoma Australia to achieve this aim.My research is all about improving outcomes for children with high-risk neuroblastoma … making existing therapies work better, identifying new therapeutic approaches, and understanding disease progression for each patient. Dr Jamie Fletcher

What makes this and our other research projects so compelling for us can be found in our family pages, the #44KMfor44kids, our Run2Cure Superheroes and all the stories of the families affected by this heartbreaking disease.

We need the funding for research because the only way children are treated now is they’re hit hard and fast with as much as they can because if it comes back they can’t treat it. They’re given absolutely everything and it’s awful for the kids. Funding and research is needed to reach the point that these precious children are not taken from us or made to suffer so intensely. Max's mum Sharvita


About Dr Jamie Fletcher

Jamie conducts research into the high-risk neuroblastoma at Children’s Cancer Institute. His work spans understanding disease progression and drug resistance, developing new approaches to disease monitoring and developing new treatment approaches. He also contributes to laboratory testing for high-risk neuroblastoma patients as part of the Zero Childhood Cancer personalised medicine program.

In collaboration with Toby, and with the support of Neuroblastoma Australia, Jamie has developed a panel of highly specialised pre-clinical models (mouse models) developed direct from children with high-risk neuroblastoma. These models are critical to testing the effectiveness of different drugs and drug combinations and underpin Jamie’s research aimed at identifying and prioritising new agents to go into clinical trials in children with high-risk neuroblastoma. The models are also utilised by other scientists at the Institute, and elsewhere in the country.

Jamie is extremely grateful for the support he has received from Neuroblastoma Australia and would like to continue this partnership to progress his research further to one day put an end to neuroblastoma.

More information