Anti-cancer drug shows promise

A genetic map of an aggressive childhood brain tumour called medulloblastoma has helped researchers identify a new generation anti-cancer drug that can be repurposed as an effective treatment for the disease.

This international collaboration, led by researchers from The University of Queensland’s Diamantina Institute and WEHI in Melbourne, could give parents hope in the fight against the most common and fatal brain cancer in children.

UQ lead researcher Dr Laura Genovesi said the team had mapped the genetics of these aggressive brain tumours for five years to find new pathways that existing drugs could potentially target.

"In this study, we predicted a drug called Ixabepilone, typically used to treat breast cancer, would block tumour growth and significantly extend the survival rate in preclinical models,” says Dr Genovesi.

Dr Genovesi says drugs that could block these connections were more likely to be effective in treating the cancer, giving researchers a head start on the best possible treatment options.

“We’re really using biology to define the next round of drugs that will hopefully have a fantastic benefit for children with this condition,” Dr Genovesi says.

“This gives us the best chance to identify drugs that will have the least impact on the normal developing brain, an important consideration for paediatric brain cancers."

“At the moment, the side-effects of treatment can be almost impossible for families to live with. Short-term, we are looking at existing drugs that can target certain overlapping areas on the genetic map."

"But long-term, we now have an entire list of proteins and pathways that new therapeutics could target that we know would kill cancer cells, and we want to work with drug companies to try and develop these life-saving medications.”

This work is a culmination of a five-year collaborative study, which first began at UQ’s Institute for Molecular Bioscience, and is led by UQ Diamantina Institute's Professor Brandon Wainwright and Associate Professor Melissa Davis, a computational biologist from WEHI, along with a team of national and international collaborators.

Read more: New therapies to treat incurable paediatric brain tumours

The research was funded by the US National Institutes of Health, the Seattle Children’s Brain Tumour Endowment, The Kids' Cancer Project, Brainchild, the Children’s Hospital Foundation, The Pirate Ship Foundation, Cure Brain Cancer, the National Breast Cancer Foundation, The Betty Smyth Centenary Fellowship and the Victorian Government.

This research is published in Genome Medicine (DOI: 10.1186/s13073-021-00920-z).

Image caption: Associate Professor Melissa Davis, courtesy WEHI and Dr Laura Genovesi courtesy UQ.