New Delhi: A team of Australian researchers has initiated a clinical trial aimed at treating aggressive cancers previously considered untreatable, by targeting the MYC protein - a major contributor to cancer development.
Led by the Australian National University (ANU) and Canberra Health Services, the trial focuses on testing a new experimental drug, PMR-116, designed to disrupt the cancer-promoting effects of the MYC protein. This protein is involved in approximately 70 per cent of all human cancers, including prostate, breast, ovarian, and blood cancers.
The study employs a "basket" trial design, where participants are grouped based on shared molecular features, rather than the type of cancer they have. This innovative approach enables researchers to target the disease at its molecular source. In this case, the drug blocks the downstream effects of the MYC protein instead of attempting to directly inhibit the protein itself.
“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 and ANU researcher who is leading the clinical trial. “MYC has long been considered ‘undruggable,’ but early results of PMR-116 show promise in changing that perception.”
PMR-116, developed by researchers at ANU in partnership with biotech company Pimera Therapeutics, works by inhibiting ribosomal biogenesis—a key process that MYC-driven tumours rely on for growth.
According to the research team, the clinical trial is expected to begin in late 2025 and will enroll patients from major hospitals in Canberra, Melbourne, and Sydney. The trial will prioritise individuals with cancers that have not responded to standard treatments.
Professor Ross Hannan, co-developer of the drug, described the effort as a step forward in precision oncology. “This approach represents a new direction in cancer treatment, targeting cancer’s molecular drivers rather than where it appears in the body,” he said. “It could potentially accelerate access to effective therapies for patients worldwide.”
The trial’s outcome may help reshape global cancer treatment strategies and offer hope to those with previously limited treatment options.
