The first multitumor “exceptional responder” program in Australia is under way, adding to the growing effort around the world to understand and learn from these patients so drug developers can better tailor treatments. Currently, the United States, Canada, the United Kingdom, and France all have at least 1 similar type of program. Now, Australia has 2 programs, the other being the Australian Ovarian Cancer Study, which, as the name implies, solely focuses on patients with ovarian cancer.

The multitumor Australian program is being carried out by the Garvan Institute of Medical Research and The Kinghorn Cancer Centre in Darlinghurst, and the program progress to date was recently reported at the European Society for Medical Oncology (ESMO) Congress 2019.1

So far, the trial has recruited 76 patients who have had exceptional responses to therapy across several tumor types. The most common disease types for which patients had exceptional responses have been in malignant pleural mesothelioma (14 patients), colorectal cancer (10 patients), non-small cell lung cancer (10 patients), and glioblastoma multiforme (8 patients). The goal is to fully analyze up to 200 patients.

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During an interview with Cancer Therapy Advisor, lead author Megan Barnet, PhD, of The Kinghorn Cancer Centre, noted that the patients are spread across “a lot” of different clinical contexts and she and her colleagues are now trying to grow numbers in specific clinical contexts — that is, for patients with a specific type of cancer with a specific histology that has responded to a specific treatment. 

For example, for the enrolled patients who have pancreatic adenocarcinoma (6 individuals), some have indolent disease, some have responded to immunotherapy, and some have responded to chemotherapy and have gone on to live at least 5 years. “They’re all really interesting, but they’re all smaller subgroups that we all need to grow individually in order to get any power there,” Dr Barnet said.

Because treatments have been largely unsuccessful in this particular disease subgroup, pancreatic adenocarcinoma particularly stands to benefit from a program such as this.

“We can learn a lot from these types of analyses,” said Vinod Balachandran, MD, a member of the David M. Rubenstein Center for Pancreatic Cancer Research at Memorial Sloan Kettering Cancer Center in New York City, during an interview with Cancer Therapy Advisor.

In the past, Dr Balachandran explained, studies like this were hard to do for various logistical reasons, but now researchers have the technology, infrastructure, and ability to identify these patients and collect their tumor samples. “It gives us a really unprecedented look into what could be some of the reasons why these patients are so different.”