Australian researchers have embarked on a groundbreaking clinical trial aiming to combat aggressive cancers previously considered untreatable.
The trial targets malignancies driven by the elusive MYC protein, which is implicated in 70 percent of human cancers. The study will test an experimental drug called PMR-116, designed to disrupt cancer growth mechanisms in patients with prostate, breast, ovarian, and blood cancers, as well as other MYC-driven tumors.
Developed by scientists at the Australian National University (ANU) and biotech company Pimera Therapeutics, PMR-116 works by inhibiting ribosomal biogenesis—a cellular process exploited by MYC-driven cancers. Instead of attempting to block the MYC protein directly, which has been deemed “undruggable,” the drug blocks the processes that MYC triggers within cells.
“MYC has long been a challenging target in cancer therapy, but early results of PMR-116 show promise in changing that perception,” said Professor Mark Polizzotto, a hematologist at ANU who will lead the clinical trial.
The trial, backed by a federal grant, adopts an innovative “basket” approach. This means participants are grouped based on molecular biomarkers rather than the type of cancer they have. Such an approach streamlines research and could accelerate the development of effective treatments.
“This represents a new direction in precision oncology,” said Professor Ross Hannan, co-developer of PMR-116. “By focusing on the molecular drivers of cancer instead of its location in the body, we can potentially speed up treatments for many patients worldwide.”
The trial is set to enroll patients at major hospitals in Canberra, Melbourne, and Sydney starting in late 2025. It will prioritize individuals whose cancers have resisted standard therapies, offering new hope to those with limited treatment options.
