Appendiceal cancers are extremely rare, affecting only 2-4 people per million each year.  Because of their rarity, these cancers are significantly understudied, and treatment options have remained unchanged for over 25 years.  For most patients, the only chance of extended survival is major surgery to remove affected areas, followed by heated chemotherapy.  Unfortunately, even with this aggressive approach, many patients still face relapse due to the return of metastatic disease.

With a St Vincent’s Clinic Research Foundation grant, Dr Sharissa Latham and her team undertook a research project focused on developing new treatments to stop cancer from spreading and to improve survival for people diagnosed with metastatic appendiceal mucinous neoplasms (AMNs).

The team has been investigating two promising new drugs, K11 and K12, originally developed to block a cancer-driving pathway called cytoplasmic JNK signalling.  These drugs had previously shown success in slowing the spread of breast cancer in the lab, and Dr Latham’s research is now exploring their potential in appendiceal cancers.  Early results are encouraging: the JNK pathway appears to be highly active in all tumour samples studied so far, suggesting it may be a common target across different cases.  In lab tests, both K11 and K12 have effectively slowed the growth of appendiceal cancer cells.

But testing treatments for rare cancers like this is challenging.  While lab-based studies using cancer cells are well established, there are very few suitable animal models available to study how appendiceal tumours behave and respond to treatment in the body.  That’s why another primary focus of the project has been creating patient-derived tumour models in mice.  These models closely mimic the behaviour of cancer in patients, making them a powerful tool for testing new therapies.  Despite early setbacks, the team has now successfully developed two promising models that are being refined and expanded.

“This grant has been instrumental in getting this research off the ground,” says Dr Latham.  “We’ve generated critical early data that confirms this pathway is active in appendiceal cancer, and that our inhibitors are having an effect.  We’ve also laid the groundwork for experimental models that will support future studies and clinical trial development.”

This project has also sparked new collaborations between Dr Latham’s lab and leading oncologists, including A/Prof Kate Mahon at Chris O’Brien Lifehouse, Dr Madeline Strach, and Dr Dannel Yeo.  It is now being co-funded by Cancer Council NSW as part of a larger research initiative focused on rare and hard-to-treat cancers.

While the research is still in early stages, the progress so far marks an important step toward new and much-needed treatment options for people diagnosed with metastatic appendiceal cancer.