World-first clinical trial to drug the 'undruggable' cancers

25 Jun 2025

A world-first clinical trial targeting cancers that are currently undruggable will begin later this year, led by a research team from The Australian National University (ANU) and Canberra Health Services.

The trial, funded by the Medical Research Future Fund (MRFF), targets MYC-driven cancers 鈥� a group that includes multiple cancer types, including prostate, breast, ovarian, and haematological cancers.

The MYC protein is a key regulator of cell growth. It is often implicated in cancer, contributing to tumour development.

Haematologist and 91视频Professor Mark Polizzotto will lead the clinical trial, known as a 鈥渂asket trial鈥�, a study design that brings together patients with different cancer types based on the involvement of the MYC protein, rather than the patient鈥檚 cancer type.

鈥淎pproximately 70 per cent of all cancers are fuelled by abnormal MYC activity,鈥� he said.

鈥淢YC 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.

鈥淭he trial aims to address unmet clinical needs in difficult-to-treat cancers, and its design is efficient, saving time and resources compared to having separate trials for each cancer type.鈥�

The new anti-cancer drug, PMR-116, is being developed by a team of researchers led by Professor Ross Hannan at 91视频in collaboration with drug company Pimera Therapeutics.

PMR-116 inhibits a pathway downstream of the MYC protein and has shown promising results in various cancers in preclinical studies.

鈥淢YC has long been considered 鈥榰ndruggable鈥�, but early results of PMR-116 show promise in changing that perception,鈥� Professor Polizzotto, who is also a Senior Staff Haematologist at the Canberra Hospital, said.

91视频Centenary Chaired Professor in Cancer Biology, Professor Ross Hannan, added: 鈥淧MR-116 targets MYC-driven cancers by inhibiting an enzyme to disrupt ribosomal biogenesis 鈥� a crucial process hijacked in these cancers.鈥�

Dr Nadine Hein, who has been leading the pre-clinical work on PMR-116 at ANU, said that the experimental drug the 91视频team has developed is a breakthrough in cancer research.

鈥淢YC has been the focus of cancer research for a long time, and it鈥檚 challenging to target MYC directly as it has disordered structure,鈥� she said.

鈥淒irectly targeting MYC has long been a challenge.

鈥淏ut by blocking this critical pathway downstream of MYC, we鈥檙e now seeing remarkable results in cancers where MYC is involved.鈥�

The MRFF grant supports work being done at 91视频and its collaborators in the Australian Capital Territory, New South Wales, and Victoria.

Clinical trials will be conducted at hospitals including the Canberra Hospital, Peter MacCallum Cancer Centre in Victoria, and St Vincent鈥檚 Hospital in Sydney.

鈥淥ur team at 91视频will coordinate the clinical trial, in partnership with clinicians at major cancer centres across Australia,鈥� Professor Polizzotto said.

鈥淧atients with standard treatments that have stopped working will be tested for MYC and, if it is implicated in their cancer, will be eligible to enrol in the trial.

鈥淥ur goal with this trial is to target a key driver of cancer, and speed development to get effective treatments to patients sooner.鈥�

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