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Australian Researchers Launch Groundbreaking Trial to Target Untreatable Cancers

Australian researchers have initiated a pioneering clinical trial targeting aggressive and previously untreatable cancers.

Australian researchers have initiated a pioneering clinical trial targeting aggressive and previously untreatable cancers. Spearheaded by the Australian National University (ANU), the trial focuses on malignancies driven by the elusive MYC protein — a protein implicated in nearly 70% of human cancers, according to Xinhua news agency.

New Drug PMR-116 Brings Fresh Hope

The experimental drug, PMR-116, has been developed to interrupt the growth mechanisms of MYC-driven cancers such as prostate, breast, ovarian, and blood cancers. The MYC protein plays a crucial role in regulating cell growth and is often a key factor in tumor development.

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Innovative Basket Trial Approach

The ANU-led trial uses an innovative “basket” trial approach, grouping participants based on molecular biomarkers rather than cancer type. This method allows researchers to target MYC through its downstream effects, disrupting the processes MYC triggers in the cells rather than targeting the protein directly.

Collaboration with Pimera Therapeutics

PMR-116, developed collaboratively by ANU researchers and biotech firm Pimera Therapeutics, works by inhibiting ribosomal biogenesis—a cellular process exploited by MYC-driven tumors. This novel approach offers a fresh direction in the treatment of cancers that have long resisted conventional therapies.

Early Results Show Encouraging Signs

“MYC is one of the most notorious cancer-causing genes, and tumors driven by MYC overexpression are among the most aggressive and difficult to treat,” said hematologist and ANU professor Mark Polizzotto, who will lead the clinical trial. “MYC has long been considered ‘undruggable,’ but early results of PMR-116 show promise in changing that perception.”

Clinical Trials to Begin in Late 2025

The trial is set to begin enrolling patients in late 2025 at major hospitals in Canberra, Melbourne, and Sydney. Priority will be given to patients whose cancers are resistant to standard treatments, the research team confirmed.

A New Era in Precision Oncology

ANU professor Ross Hannan, co-developer of PMR-116, emphasized that this trial signifies a new direction in precision oncology. By focusing on molecular drivers instead of cancer locations, this approach could accelerate treatments for countless patients worldwide.

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