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 - 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 ANU Professor Mark Polizzotto will lead the clinical trial, known as a "basket trial", a study design that brings together patients with different cancer types based on the involvement of the MYC protein, rather than the patient's cancer type.
"Approximately 70 per cent of all cancers are fuelled by abnormal MYC activity," he said.
"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.
"The 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."
The new anti-cancer drug, PMR-116, is being developed by a team of researchers led by Professor Ross Hannan at ANU 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.
"MYC has long been considered 'undruggable', but early results of PMR-116 show promise in changing that perception," Professor Polizzotto, who is also a Senior Staff Haematologist at the Canberra Hospital, said.
ANU Centenary Chaired Professor in Cancer Biology, Professor Ross Hannan, added: "PMR-116 targets MYC-driven cancers by inhibiting an enzyme to disrupt ribosomal biogenesis - a crucial process hijacked in these cancers."
Dr Nadine Hein, who has been leading the pre-clinical work on PMR-116 at ANU, said that the experimental drug the ANU team has developed is a breakthrough in cancer research.
"MYC has been the focus of cancer research for a long time, and it's challenging to target MYC directly as it has disordered structure," she said.
"Directly targeting MYC has long been a challenge.
"But by blocking this critical pathway downstream of MYC, we're now seeing remarkable results in cancers where MYC is involved."
The MRFF grant supports work being done at ANU 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's Hospital in Sydney.
"Our team at ANU will coordinate the clinical trial, in partnership with clinicians at major cancer centres across Australia," Professor Polizzotto said.
"Patients 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.
"Our goal with this trial is to target a key driver of cancer, and speed development to get effective treatments to patients sooner."
