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Intraperitoneal Paclitaxel Is a Safe and Effective Therapeutic Strategy for Treating Mucinous Appendiceal
Ichiaki Ito1, Abdelrahman M G Yousef1, Saikat Chowdhury1
1Department of Gastrointestinal Medical Oncology, The University of Texas MD Anderson Cancer Center, Houston, Texas.
Abstract:
Appendiceal adenocarcinomas (AA) are a rare and heterogeneous mix of tumors for which few preclinical models exist. The rarity of AA has made performing prospective clinical trials difficult, which has partly contributed to AA remaining an orphan disease with no chemotherapeutic agents approved by the FDA for its treatment. AA has a unique biology in which it frequently forms diffuse peritoneal metastases but almost never spreads via a hematogenous route and rarely spreads to lymphatics. Given the localization of AA to the peritoneal space, intraperitoneal delivery of chemotherapy could be an effective treatment strategy. Here, we tested the efficacy of paclitaxel given by intraperitoneal administration using three orthotopic patient-derived xenograft (PDX) models of AA established in immunodeficient NSG mice. Weekly intraperitoneal paclitaxel treatment dramatically reduced AA tumor growth in all three PDX models. Comparing the safety and efficacy of intravenous with intraperitoneal administration, intraperitoneal delivery of paclitaxel was more effective, with reduced systemic side effects in mice. Given the established safety record of intraperitoneal paclitaxel in gastric and ovarian cancers, and lack of effective chemotherapeutics for AA, these data showing the activity of intraperitoneal paclitaxel in orthotopic PDX models of mucinous AA support the evaluation of intraperitoneal paclitaxel in a prospective clinical trial.
Significance:
The activity and safety of intraperitoneal paclitaxel in orthotopic PDX models of mucinous appendiceal adenocarcinoma supports the evaluation of intraperitoneal paclitaxel in a prospective clinical trial of this rare tumor type.
Insights
Intraperitoneal paclitaxel effectively reduced appendiceal adenocarcinoma tumor growth in preclinical models. This targeted chemotherapy approach offers a promising, safer treatment strategy for this rare cancer.
Area of Science:
- Oncology
- Gastroenterology
- Cancer Research
Background:
- Appendiceal adenocarcinomas (AA) are rare, heterogeneous tumors with limited preclinical models.
- AA's rarity and unique peritoneal spread pattern hinder clinical trials and treatment development, leaving it an orphan disease.
- Current treatment options for AA are limited, with no FDA-approved chemotherapeutic agents.
Purpose of the Study:
- To evaluate the efficacy and safety of intraperitoneal paclitaxel in orthotopic patient-derived xenograft (PDX) models of appendiceal adenocarcinoma.
- To compare the effectiveness and side effects of intraperitoneal versus intravenous paclitaxel administration for AA.
Main Methods:
- Three orthotopic patient-derived xenograft (PDX) models of appendiceal adenocarcinoma were established in immunodeficient NSG mice.
- Mice received weekly intraperitoneal paclitaxel administration.
- Tumor growth, safety, and systemic side effects were assessed and compared between intraperitoneal and intravenous administration routes.
Main Results:
- Weekly intraperitoneal paclitaxel significantly reduced tumor growth in all three AA PDX models.
- Intraperitoneal paclitaxel demonstrated superior efficacy compared to intravenous administration.
- Intraperitoneal delivery resulted in fewer systemic side effects in the treated mice.
Conclusions:
- Intraperitoneal paclitaxel exhibits significant activity and a favorable safety profile in preclinical models of appendiceal adenocarcinoma.
- These findings support the clinical investigation of intraperitoneal paclitaxel as a therapeutic strategy for appendiceal adenocarcinoma.
- Targeted intraperitoneal chemotherapy represents a promising approach for managing this rare malignancy.
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