The discovery draws on screening capabilities at the National Drug Discovery Centre, a facility supported by Therapeutic innovation Australia (TIA) through the National Collaborative Research Infrastructure Strategy (NCRIS). Using a library of over 270,000 compounds, the team identified several molecules that could inhibit this process.

Minor splicing, while affecting only a small subset of genes, is essential for cancer cell growth. The study showed that blocking it causes DNA damage in cancer cells and activates the p53 tumour suppressor pathway, slowing tumour growth in liver, lung and stomach cancers while leaving healthy cells largely unharmed.

The study, led by Professor Joan Heath and first author Dr Karen Doggett, demonstrated that reducing the activity of a key minor splicing protein significantly slowed tumour growth across a range of models, including zebrafish, mice and human cancer cells.

As explained by Joan Heath, this strategy takes a different approach to a long-standing challenge:

“Instead of trying to target specific mutations that may only apply to a subset of patients, we’re disrupting a fundamental process that these fast-growing cancers rely on.”

The findings provide a strong foundation for future drug development, with early screening efforts at the NDDC identifying several compounds that may be able to inhibit this process.

This next phase, translating a biological insight into a viable therapeutic, relies on access to specialised infrastructure and expertise that can support large-scale screening and optimisation.

The NDDC provides researchers with access to industrial-scale screening technologies that are critical for identifying and developing new drug candidates. It forms part of the national TIA network designed to connect researchers with the capabilities needed to progress from early discovery through to therapeutic development.

In this case, the ability to screen a large and diverse library of drug-like molecules has enabled researchers to move beyond identifying a target, toward exploring how it might be translated into a treatment.

While further work is needed to develop safe and effective inhibitors of minor splicing, the study provides a clear direction for future research.

It also illustrates how access to national research infrastructure can support the progression of complex biological discoveries toward new therapeutic opportunities, particularly in areas where conventional approaches have struggled to deliver results.