Researchers have identified a new generation of anti-cancer drug that could be repurposed as an effective treatment for an aggressive childhood brain tumour called medulloblastoma.

The international collaboration, led by WEHI and researchers from The University of Queensland (UQ), could give parents hope in the fight against the most common and fatal brain cancer in children.

The team mapped the genetics of these aggressive brain tumours for five years to find new pathways that existing drugs could potentially target.

The study showed a drug called ixabepilone, typically used to treat breast cancer, blocked tumour growth and significantly extended the survival rate in preclinical models.

Associate Professor Melissa Davis said the chemotherapy drug had a ‘dramatic survival benefit’, with very little sign of any remaining tumour following treatment.

“This is the second, highly effective drug we have identified using the genetic map, and it is highly effective in treating our pre-clinical models of medulloblastoma,” she said.

“The impact of this drug in our model systems gives hope for children diagnosed with highly aggressive forms of medulloblastoma.

“But even more promising is the potential to use our genetic map to find other treatments for this disease.”

Mapping aggressive brain tumours

Associate Professor Davis likened their work to a street map.

“Like a map shows streets connecting places, the genetic map shows connections between different genes that contribute to more aggressive brain tumours,” she said.

Drugs that can block these connections are more likely to be effective in treating the cancer.

UQ researcher Dr Laura Genovesi said the research would lay the foundation for drugs that could potentially be used in the future to treat paediatric brain cancers.

“This gives us the best chance to identify drugs that will have the least impact on the normal developing brain,” she said.

The side-effects of current treatments can be difficult for families to live with.

“In the short-term, we are looking at existing drugs that can target certain overlapping areas on the genetic map. But in the long-term, we now have an entire list of proteins and pathways 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,” Dr Genovesi said.