Venetoclax combination study

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

Dr. Jamie’s project is in line with Neuroblastoma Australia’s mission to develop better, safer and more effective treatments for children with neuroblastoma. The 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

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