Australian researchers have initiated a groundbreaking clinical trial aimed at addressing aggressive cancers that had previously been considered untreatable. This effort focuses specifically on malignancies driven by the often elusive MYC protein.

If successful, this trial has the potential to redefine global treatment paradigms for aggressive cancers, potentially providing a framework to address other molecularly complex diseases, as emphasized by the Australian National University (ANU) in a recent release.

The clinical study will investigate the efficacy of an experimental drug known as PMR-116, which is designed to interrupt cancer growth mechanisms in patients with MYC-driven tumors, including prostate, breast, ovarian, and blood cancers.

The trial is being led by the ANU in collaboration with Canberra Health Services and is supported by a federal grant. Notably, the trial employs an innovative 'basket' approach, grouping participants based on their molecular biomarkers instead of the traditional classification by cancer type.

This method streamlines the research process by targeting the MYC protein, which is implicated in around 70 percent of human cancers. The approach involves blocking downstream effects triggered by MYC rather than attempting to inhibit MYC itself.

PMR-116, which has been developed by researchers at ANU in partnership with Pimera Therapeutics, functions by inhibiting ribosomal biogenesis, a crucial cellular process that is often exploited by MYC-driven tumors.

"MYC has long been labeled as 'undruggable,' but early findings related to PMR-116 suggest that this perception may be changing," stated hematologist and ANU professor Mark Polizzotto, who is overseeing the clinical trial.

The trial plans to enroll patients from major hospitals located in Canberra, Melbourne, and Sydney, with participant recruitment set to commence in late 2025. The research team aims to prioritize patients whose cancers demonstrate resistance to conventional therapies.

ANU professor Ross Hannan, who co-developed PMR-116, pointed out that this trial marks a significant shift in the field of precision oncology. The focus is now shifting toward the molecular drivers of cancer rather than its anatomical location, potentially expediting treatment options for numerous patients around the globe.