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Discovering new approaches to treating incurable childhood cancers

Thursday 10 February, 2022

In Australia, three children and adolescents die  of childhood cancer every week. 

Cancer is caused by changes, or mutations, that occur in the genes of previously normal cells. Genome sequencing – the technical ability to read the mutations in cancer cells – has revealed the broad range of mutations that drive paediatric cancers. 

Research conducted by Associate Professor Paul Ekert and his team at the University of Melbourne has the potential to make a major contribution to improving survival and giving children with cancer the best possible quality of life. This project aims to reprogram a new bacterial protein called CRISPR-Cas13 to eliminate the root of cancer cells – RNAs.

In the first year of this project, the research team developed original screening approaches that revealed hidden mechanisms that are crucial for the silencing of any RNA of interest including tumour RNAs.

“Our work has shown how to design Cas13 guides which can be used to target any particular gene with very high efficiency”, said A/Prof Ekert.

“We have also shown that we can shut down the mutated genes that drive many cancers, including those genes which cannot be targeted by specific drugs. We are developing the capacity to do this in ‘undruggable’ childhood cancers, to identify new ways to treat these diseases.”

In response to the outbreak of COVID-19, A/Prof Ekert and his team used their findings to reprogram Cas13 to suppress SARS-CoV-2 (COVID-19 virus). They were able to demonstrate that reprogramming Cas13 can act as an anti-viral drug and suppress viral replication thanks to a new mechanism they discovered called ‘single-mismatch tolerance’. This research received significant public interest which reflects the potential of this method to not only to silence SARS-CoV-2, but also tumour RNAs as a personalized therapy.

“True innovation in cancer therapy is a high-risk endeavor. Without it, there is little realistic prospect that substantial advances in the understanding of cancer biology, which underpin all major advances in cancer treatment, can be made.”

This project is part of Cancer Council Victoria’s Venture Grants research program. Entirely funded by generous Victorians, this research program is designed to support innovative researchers with courageous ideas who would not be able to attract conventional funding. 

Learn more about the Venture Grants research program.

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