Related Experiment Video
Updated: May 2, 2026

Author Spotlight: Finding New Therapeutic Targets for Malignant Peripheral Nerve Sheath Tumor Through Genome-Scale shRNA Screens
Published on: August 25, 2023
Peripheral medulloepithelioma: a rare tumor with a potential target therapy
Maria Debora De Pasquale1, Maria Antonietta De Ioris, Angela Gallo
1Department of Pediatric Hematology/Oncology and Stem Cell Transplantation, Bambino Gesù Children's Hospital, Piazza Sant'Onofrio, 4 - 00165 Rome, Italy. mdebora.depasquale@opbg.net.
Background:
Medulloepithelioma (ME) is a rare embryonal tumor predominantly located in the eye or in the central nervous system without an established treatment.
Case Presentation:
We report of a case of a localized peripheral ME treated with conventional and high dose chemotherapy, surgery and local radiotherapy. At relapse, the tumor tissue revealed a different molecular signature compared to the initial tumor mass. This molecular signature revealed a high expression of platelet derived growth factor receptor (PDGFR). Sorafenib plus irinotecan and temozolomide was started with a 5 month progression free survival.
Conclusion:
Our experience suggests a possible role of sorafenib or different PDGFR inhibitors in ME. Targeting treatment could represent an adjuvant and/or alternative therapy for ME and other rare tumors.
Related Concept Videos
Targeted Cancer Therapies
There are several types of targeted therapies against...
Mitogens and the Cell Cycle
Tumor Immunotherapy
The Tumor Microenvironment

