A rare and often misunderstood form of cancer, appendiceal tumors present unique challenges in diagnosis and treatment. Without standardized screening protocols or clear risk factors, this disease frequently leads to delayed detection and poor patient outcomes. Recent efforts by a team of experts have identified six critical research areas aimed at enhancing our understanding of these tumors and improving patient care. This initiative, driven by the Appendix Cancer Pseudomyxoma Peritonei (ACPMP) Research Foundation, seeks to transform the landscape of appendiceal cancer research and provide hope for the approximately 3,000 new cases diagnosed annually.

The rarity of appendiceal cancer has historically hindered progress in its study. Up to half of all patients are diagnosed with advanced metastatic disease, leading to five-year survival rates that range from 10% to 63%. To address these issues, the ACPMP Research Foundation convened a group of leading experts at Vanderbilt-Ingram Cancer Center in December 2023. The meeting resulted in a groundbreaking set of recommendations published in Nature Reviews Cancer. These priorities aim to fill knowledge gaps and drive collaborative research that can ultimately improve early detection, diagnosis, and treatment strategies.

One of the key areas highlighted is the need for refined histopathological classification. Due to the variability in terminology and the rarity of the disease, there is an urgent requirement for expert pathology reviews and consistent application of tumor grading. Digitizing histology and leveraging computational approaches could significantly enhance diagnostic accuracy. Another focus is on molecular characterization, which involves preserving and profiling tissue removed during appendectomy to gain a comprehensive multiomics view of the tumors.

Understanding the tumor microenvironment is also crucial. By studying the ecosystem surrounding tumor cells, researchers can uncover new insights into disease progression and develop targeted therapies. Additionally, the creation of disease-specific models, such as patient-derived organoids, will support preclinical testing of new drugs. Collaborative clinical studies, like the Genetics of Appendix Cancer (GAP) Study, will yield evidence-based advancements, while population-level research will help identify potential risk factors and improve public health policies.

The publication of these research priorities marks a pivotal moment for appendiceal cancer. For too long, this disease has been underfunded and underresearched, leaving patients with limited options. The ACPMP Research Foundation is committed to ensuring the necessary funding and resources to propel these efforts forward. By addressing these critical areas, the foundation aims to revolutionize the field of appendiceal cancer research and bring about meaningful improvements in patient outcomes.