Appendix cancer is a rare tumor, but it is unfortunately becoming more common, especially in young adults. Nearly half (40%) of patients afflicted with appendix cancer are under the age of 50, and it is no longer uncommon to see patients in their 30s diagnosed with this lethal and currently incurable disease. Because appendix is rare very few clinical trials have ever been performed in appendix cancer. Unlike most cancers there are no drugs that are approved by the FDA to treat appendix cancer. Traditionally, appendix cancer has been treated with chemotherapy as if it were the same as colon cancer and in 2023 this is still accepted practice by many. However, new data in the last five years gen...

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Appendix cancer is a rare tumor, but it is unfortunately becoming more common, especially in young adults. Nearly half (40%) of patients afflicted with appendix cancer are under the age of 50, and it is no longer uncommon to see patients in their 30s diagnosed with this lethal and currently incurable disease. Because appendix is rare very few clinical trials have ever been performed in appendix cancer. Unlike most cancers there are no drugs that are approved by the FDA to treat appendix cancer. Traditionally, appendix cancer has been treated with chemotherapy as if it were the same as colon cancer and in 2023 this is still accepted practice by many. However, new data in the last five years generated by our lab and others has shown convincingly that appendix tumors are very different than colon tumors. Given that appendix tumors are so different than colon tumors, we believe that practice of giving colon cancer chemotherapy to appendix cancer patients is outdated and that we can find better treatments. A major reason that no appendix cancer chemotherapy has been developed previously is that there are very few mouse and cell models of appendix cancer to allow scientists to test potential treatments in the lab. Our lab now has considerable experience making these mouse and cell models of appendix cancer which we will use to test potential new drugs. We will also use next-generation sequencing technology to make detailed measurements of appendix tumors. Having these detailed molecular measurements will allow us to understand the differences in each patient's tumor and knowing what differences are important will allow us to pick the best chemotherapy plan to fit each patient. Taken a whole, we are confident that the proposed research will lead to the development of one or more drugs that are effective for appendix cancer. Although these ideas will need further testing in the future, we believe this work will lead to multiple new clinical trials for appendix cancer patients.

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