Carcinoma of the Ampulla of Vater (CAV) is an exceedingly uncommon and complex cancer that develops at the ampulla of Vater, the anatomical syphon where the common bile duct and pancreatic duct join and open into the duodenum. At Protheragen, we offer comprehensive services tailored to accelerate the development of innovative diagnostics and therapeutics for Carcinoma of the Ampulla of Vater.
Overview of Carcinoma of the Ampulla of Vater (CAV)
Carcinoma of the Ampulla of Vater (CAV) is an uncommon cancer that affects the ampullary area of the common bile duct within 2 cm of its distal region, where the duodenum and ampullary papilla are located. This distinct anatomy renders CAV as a complex, multifaceted form of cancer that is difficult to diagnose and manage. CAV usually manifests with signs of obstruction in the biliary or pancreatic duct, which include jaundice, weight loss, and abdominal pain. Regardless of its infrequency, CAV has received more focus because of the possible curative surgical therapy and the considerable efforts needed toward diagnostic and therapeutic methodologies.

Fig.1 Immunohistochemical findings of invasive micropapillary carcinoma in the ampulla of Vater. (Taniguchi S. H.,
et al., 2025)
Pathogenesis of Carcinoma of the Ampulla of Vater (CAV)
The origin of CAV is multi-causative, including changes in heredity, long-standing inflammation, and characteristics of the surroundings. Genetic changes such as KRAS, APC, TP53, and ELF3 have been described in CAV, as in other neoplasms of the gastrointestinal tract. Persistent inflammation of the ampullary region from recurrent biliary or pancreatic infections may increase the risk of CAV. Other risks include obesity, smoking, and other environmental factors. Furthermore, a previous cholecystectomy can increase the risk of developing CAV owing to changes in the dynamics of bile flow.
Diagnostics Development for Carcinoma of the Ampulla of Vater (CAV)
- Tumor Markers: Elevated levels of tumor markers such as CA19-9 and CEA can be indicative of CAV. These markers are useful for monitoring therapeutic response and detecting recurrence.
- Genetic Profiling: Next-generation sequencing (NGS) can identify specific genetic mutations and alterations in CAV tumors. This information is crucial for developing personalized therapeutic plans and selecting appropriate targeted therapies.
- Endoscopic Ultrasonography (EUS): EUS provides detailed images of the ampullary region and helps in assessing tumor invasion and lymph node involvement. It is a crucial tool for accurate staging and planning surgical interventions.
- Computed Tomography (CT): CT scans are used to evaluate the extent of tumor invasion into surrounding tissues and to detect distant metastases. They provide comprehensive information about the tumor's size, location, and potential spread.
Therapeutics of Carcinoma of the Ampulla of Vater (CAV)
Therapeutics |
Drug Name |
Target |
Description |
Stage |
Chemotherapy |
5-fluorouracil |
DNA and RNA synthesis |
An antimetabolite that inhibits DNA and RNA synthesis, primarily used as adjuvant chemotherapy to reduce recurrence risk after surgery. |
Approved |
Chemotherapy |
Folinic acid |
None (potentiates 5-fluorouracil) |
A leucovorin derivative that enhances the cytotoxic effects of 5-fluorouracil by stabilizing its binding to thymidylate synthase. |
Approved |
Chemotherapy |
Gemcitabine |
DNA synthesis |
A nucleoside analog that inhibits DNA synthesis and repair. Used in adjuvant settings for periampullary adenocarcinomas, including CAV. |
Approved |
Radiotherapy |
N/A |
Cancer cells (via radiation) |
High-energy radiation used to kill cancer cells or shrink tumors. Mentioned as adjuvant therapy, sometimes combined with 5-fluorouracil. |
Approved |
Immunotherapy |
N/A |
Immune checkpoint inhibition |
Immune checkpoint inhibitors, which harness the body's immune system to fight cancer, are also being investigated. While data for CAV specifically are limited compared to other gastrointestinal cancers, ongoing research explores the potential of agents targeting PD-1/PD-L1 pathways, especially in patients with high microsatellite instability (MSI-H) or a high tumor mutational burden (TMB). |
Preclinical |
Disclaimer: Protheragen focuses on providing preclinical research services. This table is for information exchange purposes only. This table is not a treatment plan recommendation. For guidance on treatment options, please visit a regular hospital.
Our Services
Protheragen offers comprehensive diagnostics and therapeutics development services for Carcinoma of the Ampulla of Vater (CAV). Our services are designed to support the entire spectrum of research and development, from early-stage discovery to preclinical studies. We leverage cutting-edge technologies and expertise to provide tailored solutions for our clients, ensuring that each project is optimized for success.
Diagnostics Development
- Karyotype Analysis Service
- Omics Analysis Service
- Biomarker Development Service
- Artificial Intelligence Service
Therapeutic Development
- Anticancer Peptide
- Gene Therapy
- Immunotherapy
- Monoclonal Antibody
- Phytotherapy
- Small Molecule Drug
- Therapeutic Cancer Vaccine
Disease Models
- Human CAV Cell Lines
- Immortalized Epithelial Cell Lines
- CAV Organoids
- QBC939 Cell Line Xenograft Models
- Genetically Engineered Mouse Models (GEMMs)
At Protheragen, we recognize the uniqueness of every research project. Our tailored services are crafted to address the particular requirements of our clients, offering adaptable and expandable options for the development of diagnostics and therapeutics. If you are interested in our services, please feel free to contact us.
References
- Noguchi, Hirotsugu, et al. "Rare histological subtype of invasive micropapillary carcinoma in the ampulla of Vater: A case report." World Journal of Clinical Cases 9.11 (2021): 2671.
- Rostain, Florian, et al. "Trends in incidence and management of cancer of the ampulla of Vater." World Journal of Gastroenterology: WJG 20.29 (2014): 10144.
All of our services and products are intended for preclinical research use only and cannot be used to diagnose, treat or manage patients.