In a breakthrough for oncology, researchers from the Australian National University (ANU) have launched a pioneering clinical trial aimed at treating some of the most aggressive and previously untreatable forms of cancer.
The trial focuses on malignancies driven by the MYC protein, a gene involved in up to 70% of human cancers, according to a report by the Xinhua News Agency. The initiative has the potential to redefine cancer treatment strategies worldwide and offer new hope to patients with therapy-resistant tumours.
The experimental drug, PMR-116, will be tested on patients with prostate, breast, ovarian, and blood cancers, particularly those whose tumours are characterized by MYC overexpression.
“MYC is one of the most notorious cancer-causing genes, and tumours driven by MYC overexpression are often among the most aggressive and difficult to treat,” said Professor Mark Polizzotto, a haematologist at ANU and lead investigator of the trial. “MYC has long been considered ‘undruggable,’ but early results of PMR-116 show promise in changing that perception.”
Unlike traditional clinical trials, this study adopts a “basket trial” approach, grouping participants based on shared molecular biomarkers rather than the type or origin of their cancer. This method enables researchers to target MYC’s downstream effects, thereby disrupting the cancer-promoting processes it activates, rather than attempting to block the protein directly.
PMR-116, developed in partnership with biotech firm Pimera Therapeutics, inhibits ribosomal biogenesis, a cellular mechanism hijacked by MYC-driven tumours to fuel uncontrolled growth.
Professor Ross Hannan, co-developer of PMR-116, emphasized the significance of the new approach: “This represents a new direction in precision oncology, focusing on the molecular drivers of cancer rather than its location. It has the potential to accelerate treatment development for many patients globally.”
The trial is scheduled to commence in late 2025 at major hospitals in Canberra, Melbourne, and Sydney. The research team has confirmed that it will prioritize patients whose cancers are resistant to standard therapies.
If successful, the trial could serve as a blueprint for future therapies targeting other hard-to-treat cancers, marking a transformative step forward in cancer research and treatment.
–Input IANS
