The scientific foundation needed to work on curing a class of cancer-causing mutations is here, in the form of a tool from St. Jude Children’s Research Hospital. Computational biologists at St. Jude comprehensively categorized and identified the mechanism underlying oncogenic fusions in pediatric cancer cells. Oncogenic fusions are mutations that drive cancer. The researchers showed that targeting them with genome editing tools such as CRISPR has the potential to cure certain tumors. The findings were published in Nature Communications.
The new St. Jude tool lays a foundation for using genome editing to cure cancer. The mutations that cause fusion genes are only present in cancer cells. That means a highly specific genetic engineering tool, such as the CRISPR-Cas9 system, could selectively cut out the fusion gene in cancer cells – removing their ability to make the hybrid protein, leading to a cure.
Even with the challenges facing its use in therapy, the computational tool already predicts some clinical outcomes. The St. Jude authors were able to explain why a small group of pediatric patients with relapsed acute myeloid leukemia (AML) had poor outcomes. They found subtle differences in the oncogenic fusion mutations, which explained survival outcomes better than any existing clinical diagnostics. The result demonstrated that the tool can be used for clinical predictions, which will help physicians choose more personalized and effective treatments for patients in the future.