A phase II clinical trial led by the Olivia Newton-John Cancer Research Institute (ONJCRI), is showing exciting results for patients with rareupper gastrointestinal, neuroendocrine and gynaecological cancers.

The results of the rare upper gastrointestinal cancer cohort, that predominately enrolled patients who have biliary tract cancers like Anna pictured (and story below), have been published in JAMA Oncology. While results for trial participants with advanced neuroendocrine tumours were recently published in Clinical Cancer Research and the results for trial participants withadvanced gynaecological tumours were presented at the American Society of Clinical Oncology Annual Meeting – ASCO20 Virtual Scientific Program in May 2020.

The findings from this trial, which was generously supported by a grant from Bristol-Myers Squibb Ltd and Australian Government Medical Research Futures Fund, and championed by project partners Rare Cancers Australia, will have significant global implications for the treatment of patients with rare upper gastrointestinal, neuroendocrine and gynaecological cancers.

The current data demonstrates that trial participants who were treated with a combination of immune-stimulating anti-cancer drugs (Ipilimumab and Nivolumab)showed significant clinical benefit and life changing results.

Dr Oliver Klein, clinical trial co-lead, ONJCRI Clinician Fellow and Oncologist at Austin Health said, “These are promising results, in particular given that patients with rare cancers have limited treatment options compared to more common cancers”.

“Responses to immunotherapy were, as already seen in other malignancies, durable which contrasts with the generally short-lived responses obtained with chemotherapy”.

In this trial, ONJCRI, as the La Trobe University School of Cancer Medicine, investigated the use of the drugs Ipilimumab and Nivolumab as a combination immunotherapy which has so far not been trialled in patients with rare cancers. This combination treatment has already been shown to be an effective treatment for patients with metastatic melanoma, renal cell carcinoma, microsatellite unstable colorectal cancer and non-small cell lung cancer.

Globally, 20% of all cancer diagnoses, are for rare cancers. These patients currently have very limited access to treatment options and, as a result, have significantly reduced overall survival rate compared to patients with more common cancers.

Prof Jonathan Cebon, trial co-lead, Head, ONJCRI Cancer Immunobiology Cancer Program and Oncologist at Austin Health said, “This is an exciting day for people with rareupper gastrointestinal, neuroendocrine and gynaecological cancers”.

“As key players in the Australian cancer research community, we are very proud to be able to make this contribution in providing hope to patients with otherwise untreatable cancers.”

“This important trial is supported by our trial partners and generous Australians who donate to our research at ONJCRI,” said Prof Cebon.

The trial enrolment closed early in 2020 with trial participants currently in follow up. Ongoing translational research on tumour tissue and blood samples is aimed at identifying biomarkers to predict treatment response.

ONJCRI and the trial partners are also actively seeking additional opportunities and funding to expand the trial to more patients with rare cancers in rural and regional Australia.



Anna Anderson is grateful to be alive. When she was diagnosed with stage four gallbladder cancer her surgeon told her she had just months to live.

That was in October 2017.

The cancer was rare and aggressive and had metastasised to her liver.  With the outlook bleak, the mother-of-three was encouraged to get her affairs in order.

In January 2018 she joined the immunotherapy trial led by the Olivia Newton-John Cancer Research Institute, almost immediately things started to improve. Following less than a year on the trial, scans revealed Anna’s tumours had reduced to five per cent of their original size.

“The doctors are calling it a miracle,” she said. “I wouldn’t be here if not for the clinical trial”.

“I am incredibly grateful to the ONJCRI Team for the life changing work that they do. I hope that trial’s such as this will make it possible for others to benefit from the most up to date treatments modern medicine has to offer.”


Publication details

JAMA Oncology: https://jamanetwork.com/journals/jamaoncology/article-abstract/2768631

Clinical Cancer Research: https://clincancerres.aacrjournals.org/content/early/2020/06/12/1078-0432.CCR-20-0621

American Society of Clinical Oncology Annual Meeting – ASCO20 Virtual Scientific Program: https://meetinglibrary.asco.org/record/187511/abstract