Australian researchers launch pioneering trial targeting untreatable cancers

Australian researchers have launched a pioneering clinical trial that aims to combat aggressive cancers previously deemed untreatable, focusing on malignancies driven by the elusive MYC protein.

Success could redefine treatment paradigms for aggressive malignancies globally, offering a template for tackling other molecularly complex diseases, the Australian National University (ANU) said in a release on Friday.

The study will test an experimental drug, PMR-116, designed to disrupt cancer growth mechanisms in patients with prostate, breast, ovarian, and blood cancers, or MYC-driven tumors, according to the ANU.

Led by the ANU and Canberra Health Services and backed by a federal grant, the trial adopts an innovative "basket" approach, grouping participants based on molecular biomarkers rather than cancer type, the release said.

This method streamlines research by targeting MYC – a protein implicated in 70 percent of human cancers – through its downstream effects, meaning it blocks the processes MYC triggers in the cell rather than trying to block MYC itself, according to the study.

PMR-116, developed by ANU researchers and biotech firm Pimera Therapeutics, inhibits ribosomal biogenesis, a cellular process exploited by MYC-driven tumors, it said.

"MYC has long been considered 'undruggable,' but early results of PMR-116 show promise in changing that perception," said hematologist and ANU professor Mark Polizzotto, who will lead the clinical trial.

The trial will enroll patients at major hospitals in Canberra, Melbourne and Sydney starting in late 2025, prioritizing those whose cancers resist standard therapies, said the research team.

ANU professor Ross Hannan, co-developer of PMR-116, highlighted that this approach represents a new direction in precision oncology, focusing on cancer's molecular drivers instead of its location, potentially speeding up treatments for many patients worldwide.