Deborah Shelton

Title: Overview of appendix cancer PMP (ACPMP) Research foundation & epithelial appendiceal malignant neoplasms: A rare but challenging cancer with critical unmet medical need

Abstract:

Appendiceal cancer is a rare cancer for which there is critical unmet medical need. Although it is a rare cancer, findings from a 2020 study reflect that the overall incidence of malignant appendiceal tumors has significantly increased in the U.S. and Canada from 2000 to 2016.  Singh H, Koomson AS, Decker KM, Park J. Demers AA. Continued increasing incidence of malignant aopendiceal tumors in Canada and the United States a population based study. Cancer 2020;126(10):2206-2216.  One of the challenges of diagnosing appendiceal cancer is that it often presents with nonspecific symptoms and is often mistaken for colon cancer. By the time a proper diagnosis of appendiceal cancer is made, the disease is advanced. It has spread throughout the abdomen and, depending on histology, presents as pseudomyxoma peritonei (for low grade tumors) or peritoneal carcinomatosis (for high grade tumors). This is deemed Stage 4. For years, and continuing today, cytoreductive surgery and intraperitoneal heated chemotherapy with mitomycin C or another perfusion agent remains the standard of care. This procedure, known as CRS/HIPEC, however, is not an option for many patients due to a high-grade histology, tumor burden, or unresectable tumor due to location (e.g., small bowel). For these patients, quality of life is poor and the disease is fatal. In terms of non-surgical options, patients are typically offered systemic chemotherapy (5FU-based) that are typically used to treat colorectal cancer. However, as shown by a team at MD Anderson in a recently published paper, this approach is not effective, particularly in the case of low grade appendiceal histologies. Shen JP, Yourself A, Zeneddine F et al. Efficacy of Systemic Chemotherapy in Patients with Low-grade Mucinous Appendiceal Carcinoma: A randomized crossover trial. JAMA June 2023. (Dr. Shen is a guest presenter for ACPMP’s free June webinar on June 22 and will be discussing this study in greater detail.) The ACPMP Research Foundation is a small, all-volunteer led 501(c)3 foundation whose mission is to fund educational programs to increase awareness among clinicians researchers, patients/families about appendix cancer and to fund research to discover innovative treatments for appendiceal cancer with the  hope of one day finding a cure. This presentation will provide a brief overview of appendiceal cancer with a focus on the current challenges, limited treatments options, and critical need for innovative therapies. It will also include an overview of the ACPMP Research Foundation and its research grant program and some of the more recent research funded, including this year a study to explore a vaccine for a specific mutation often seen in appendiceal cancer that, to date has not been targetable.

Audience Take Away Notes:

• Increase awareness of the rare cancer of appendiceal cancer and the critical need for targeted therapies
• Identify ACPMP funding opportunities for tinterested researchers 
• Foster ongoing thinking and collaborations about how to leverage the promise of precision medicine for the purposes of earlier diagnosis, more efficacious treatments, and/or more reliable surveillance

Biography:

Deborah M. Shelton is grateful for the privilege that she has each and every day to contribute to the ACPMP Research Foundation her deep knowledge and extensive professional experience in drug development with a focus on rare cancers. Deborah has spent over 25 years as an FDA regulatory advisor to a diverse set of stakeholders, including patients and caregivers, researchers, drug and device developers, and legislative bodies. In her professional capacity, Deborah has helped this diverse group of stakeholders to navigate the complex considerations and regulations pertinent to clinical research, and the testing and commercialization of cutting-edge pharmaceuticals and medical devices directed at enhancing the timely diagnosis and effective treatments for cancers and other serious diseases. She is an honors graduate of the University of Maryland School of Law, a frequent presenter at FDA-related conferences and has authored numerous publications on various topics of interest in the regulatory sphere.As with all of her colleagues at ACPMP, Deborah’s personal journey with appendix cancer inspired her to dedicate her time to ACPMP. Deborah’s spouse is a survivor of appendix cancer. She was diagnosed in August 2018 and underwent CRS/HIPEC that October. For several years prior to her spouse being diagnosed, Deborah had been actively engaged in patient advocacy and issues involving the need to incentivize innovation for the development of therapies to treat cancers and other diseases where there is critical unmet medical need. Her spouse’s subsequent diagnosis with ACPMP and their collective personal experience with the numerous challenges presented following that diagnosis has further fueled Deborah’s zeal for that work, taking the fight now to both a professional and personal level. Deborah’s primary focus in her work at ACPMP is in further developing the ACPMPs research grant program, clinical trial network, and the organization’s scientific and educational initiatives. Deborah resides in Silver Spring, Maryland with her spouse (Kate), Deborah's 84-year “young” mother, and their two rescue dogs. In her spare time, she loves exploring salt marshes by kayak with Kate, exploring beaches, and reading Nordic detective novels with a good cup of strong coffee in hand.