Australian researchers have initiated a groundbreaking clinical trial targeting aggressive cancers driven by the MYC protein – a molecular culprit in 70% of human malignancies. The study focuses on PMR-116, an experimental drug developed through collaboration between Australian National University (ANU) and Pimera Therapeutics, designed to disrupt cancer growth mechanisms in prostate, breast, ovarian, and blood cancers.
The MYC Challenge
Dubbed 'undruggable' for decades due to its complex role in cellular processes, the MYC protein has evaded traditional treatment approaches. PMR-116 employs an innovative strategy by blocking ribosomal biogenesis – a cellular process hijacked by MYC-driven tumors – rather than targeting the protein directly.
Basket Trial Innovation
The trial adopts a 'basket' methodology, grouping participants by molecular biomarkers instead of cancer type. This precision oncology approach could accelerate treatment development for patients worldwide whose cancers resist standard therapies, particularly those in advanced stages.
Global Implications
"This isn't just about one cancer type – it's about rewriting the rulebook for molecularly complex diseases," said ANU Professor Ross Hannan, PMR-116 co-developer. Hematologist Mark Polizzotto added that early results suggest MYC's 'undruggable' status might soon be reconsidered.
The phase I trial, supported by federal funding, will enroll patients at major hospitals in Canberra, Melbourne, and Sydney starting late 2025. Success could establish new global standards for treating MYC-driven cancers while providing a template for addressing other molecularly complex diseases.
