Genetic map reveals potential new treatment for aggressive childhood brain tumor

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

This international collaboration, led by researchers from the Diamantina Institute at the University of Queensland (UQ) and WEHI in Melbourne, could give parents hope in the fight against the most common and deadly childhood brain cancer.

UQ Principal Investigator Dr. Laura Genovesi said the team spent five years mapping the genetics of these aggressive brain tumors to find new avenues for existing drugs to potentially target.

These are drugs that have already been approved for other diseases or cancers, but have never been tested for brain tumors in children. In this study, we predicted that a drug called Ixabepilone, typically used to treat breast cancer, would block tumor growth and significantly prolong survival in preclinical models.”

dr. Laura Genovesi, principal investigator of UQ

Associate professor Melissa Davis, co-senior author of WEHI, said the chemotherapy drug had a “dramatic survival benefit,” with very little evidence of residual tumor after treatment.

“This is the second drug we’ve identified using the genetic map that is highly effective in treating this type of childhood brain cancer,” said Associate Professor Davis.

“The impact of this drug in our model systems provides 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.”

Associate professor Davis compared their work to a map.

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

dr. Genovesi said drugs that can block these compounds are more likely to be effective in treating cancer, giving researchers an edge 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,” said Dr. Genovese.

“This gives us the best chance of identifying drugs that have the least impact on the normally developing brain, an important consideration for childhood brain cancer.

“Right now, the side effects of treatment can be nearly impossible for families to live with.

“In the short term, we’re looking at existing drugs that can target certain overlapping regions of the genetic map.

“But in the long run, we now have a whole list of proteins and pathways that new therapies can target that we know would kill cancer cells, and we want to work with pharmaceutical companies to try to develop these life-saving drugs.”

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

The research was funded by the US National Institutes of Health, the Seattle Children’s Brain Tumor 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.


Walter and Eliza Hall Institute

Reference magazine:

Genovesi, LA, et al. (2021) Systemic pharmacogenomics identifies novel targets and clinically useful therapies for medulloblastoma. Genome Medicine.

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