Researchers launch pioneering trial targeting untreatable cancers
New Delhi: Researchers have launched a pioneering clinical trial that aims to combat aggressive cancers previously deemed untreatable.
The trial led by a team from the Australian National University (ANU) focussed on malignancies driven by the elusive MYC protein -- a protein implicated in 70 per cent of human cancers --, Xinhua news agency reported.
The success of the trial could redefine treatment paradigms for aggressive malignancies globally, offering a template for tackling other molecularly complex diseases, said the team. The study will test an experimental drug, PMR-116, designed to disrupt cancer growth mechanisms in patients with prostate, breast, ovarian, and blood cancers, or MYC-driven tumours.
The MYC protein is a key regulator of cell growth. It is often implicated in cancer, contributing to tumour development.
Led by the ANU and Canberra Health Services the trial adopts an innovative 'basket' approach, grouping participants based on molecular biomarkers rather than cancer type.
This method streamlines research by targeting MYC through its downstream effects, meaning it blocks the processes MYC triggers in the cell rather than trying to block MYC itself, according to the study.
PMR-116, developed by ANU researchers and biotech firm Pimera Therapeutics, inhibits ribosomal biogenesis, a cellular process exploited by MYC-driven tumours.
'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 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,' he added.
The trial will enroll patients at major hospitals in Canberra, Melbourne, and Sydney starting in late 2025, prioritising those whose cancers resist standard therapies, said the research team.
ANU professor Ross Hannan, co-developer of PMR-116, highlighted that this approach represents a new direction in precision oncology, focusing on cancer's molecular drivers instead of its location, potentially speeding up treatments for many patients worldwide.
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