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Related Experiment Video

Updated: Jun 20, 2025

Culture and Imaging of Ex Vivo Organotypic Pseudomyxoma Peritonei Tumor Slices from Resected Human Tumor Specimens

Culture and Imaging of Ex Vivo Organotypic Pseudomyxoma Peritonei Tumor Slices from Resected Human Tumor Specimens

Published on: December 9, 2022

Precision Oncology and Systemic Targeted Therapy in Pseudomyxoma Peritonei.

Jordi Martínez-Quintanilla¹, Débora Cabot¹, Doménico Sabia²

¹Translational Program, Stem Cells and Cancer Laboratory, Vall d'Hebron Institute of Oncology (VHIO), Vall d'Hebron Barcelona Hospital Campus, Barcelona, Spain.

Clinical Cancer Research : an Official Journal of the American Association for Cancer Research

|July 17, 2024

Summary

This summary is machine-generated.

New preclinical models of pseudomyxoma peritonei (PMP) identified BRAFV600E as a druggable target. Systemic BRAF inhibition with encorafenib effectively controlled PMP tumors in preclinical models, offering a new therapeutic strategy.

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Last Updated: Jun 20, 2025

Culture and Imaging of Ex Vivo Organotypic Pseudomyxoma Peritonei Tumor Slices from Resected Human Tumor Specimens

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Published on: December 9, 2022

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Implantation and Monitoring by PET/CT of an Orthotopic Model of Human Pleural Mesothelioma in Athymic Mice

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Published on: December 21, 2019

Area of Science:

Oncology
Gastroenterology
Translational Research

Background:

Pseudomyxoma peritonei (PMP) is a rare malignancy characterized by mucin accumulation.
Current treatments like cytoreductive surgery have high recurrence rates, necessitating novel therapeutic strategies.

Purpose of the Study:

To develop preclinical models for Pseudomyxoma peritonei (PMP).
To identify druggable targets and evaluate targeted therapies for PMP.

Main Methods:

Generated 50 patient-derived organoid (PDO) and xenograft (PDX) models from 120 PMP samples.
Performed whole exome sequencing, immunohistochemistry, and in vitro/in vivo drug efficacy studies.
Utilized droplet digital PCR to detect mutations in patient biopsies.

Main Results:

Identified BRAFV600E, KRASG12C, and KRASG12D as druggable targets in PMP models and patient biopsies.
BRAFV600E PMP-PDO models showed reduced viability with BRAFV600E inhibitor encorafenib.
Encorafenib treatment significantly reduced tumor growth and prolonged survival in BRAFV600E-PMP-PDX mouse models.

Conclusions:

Systemic targeted therapies can effectively control PMP tumors.
BRAF signaling pathway inhibition offers a new therapeutic avenue for BRAFV600E PMP patients.
Developed preclinical models provide a platform for evaluating other targeted therapies in PMP, advancing precision oncology.