A team of Peter Mac researchers, led by A/Prof Paul Ekert, have been awarded a Cancer Council Victoria Venture Grant to look into a potential new approach to treating childhood cancers.

The ambitious project will test an innovative new way to shut down cancers cells, focused on eliminating abnormal or over-abundant RNA molecules.

If successful, this research could lead to a brand new personalised approach to treating cancer, in particular paediatric cancers.

Led by A/Prof Paul Ekert, Clinical Associate and Research Group Leader in Peter Mac’s Centre for Cancer Immunotherapy, the project will bring together a multidisciplinary team including Peter Mac’s Dr Mohamed Fareh and Prof Joe Trapani, and is linked to the national Zero Childhood Cancer program - the largest and most ambitious program dedicated to delivering personalised medicine to children with high-risk cancers.

The team will use cancer genetics to guide their personalised approach to cancer treatment.

Genes are the blueprints for making proteins, and mutant genes lead to the production of abnormal proteins, which can cause cells to divide and grow in an uncontrolled way, driving cancer growth.

Most targeted anti-cancer therapies focus on blocking the action of these abnormal proteins. However, this approach only works in some cases.

The new approach proposed by A/Prof Ekert and colleagues is completely unique as it focuses on targeting RNA – molecules that are produced in the interim between genes and proteins.

The basis for this approach is therefore not to shut down the action of cancer-causing proteins, but rather to stop their formation completely by eliminating mutant RNA.

To do this, the researchers will exploit a powerful ‘weapon’ discovered in bacteria called CRISPR-CAS13, developed over millions of years of evolution to help bacteria fight off viral infections by destroying viral RNA.

For the first time, the research team will redesigned these bacterial CRISPR-CAS13 weapons as new personalised ‘drugs’ targeting tumour RNAs, and in doing so can turn off the messages from mutant cancer genes.

In principle, this approach would mean that individual cancer RNAs could be eliminated specifically and many mutant cancer RNAs could be targeted at the same time. In addition, RNA-targeting CRISPR-CAS13 could be designed and deployed rapidly, and the specificity would mean improved survival chances and diminished treatment side effects.

While this technology is very much in the early phases of development, CRISPR-CAS13 has immense potential as a new approach to personalized cancer therapy.

Supported by the CCV, this groundbreaking research will allow A/Prof Ekert and the research team to test whether this approach works in principle in laboratory and preclinical models, which could lead to developing this into a new type of anti-cancer therapy in the future.