ERK1/2 Signaling in Intrahepatic Cholangiocarcinoma: From Preclinical Advances to Therapeutic Strategies

Extracellular signal-regulated kinase 1/2 (ERK1/2) is involved in the regulation of the key cellular processes that are essential for the proper functioning of the cell under physiological conditions. Notably, the hyperactivation of ERK1/2 is implicated in oncogenesis and metastatic dissemination ac...

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Main Authors: Veronica Porreca, Luca Sallustio, Ludovica Giancola, Pietro Angelone, Giuseppina Mignogna, Bruno Maras, Carmine Mancone
Format: Article
Language:English
Published: MDPI AG 2025-06-01
Series:Biology
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Online Access:https://www.mdpi.com/2079-7737/14/7/776
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author Veronica Porreca
Luca Sallustio
Ludovica Giancola
Pietro Angelone
Giuseppina Mignogna
Bruno Maras
Carmine Mancone
author_facet Veronica Porreca
Luca Sallustio
Ludovica Giancola
Pietro Angelone
Giuseppina Mignogna
Bruno Maras
Carmine Mancone
author_sort Veronica Porreca
collection DOAJ
description Extracellular signal-regulated kinase 1/2 (ERK1/2) is involved in the regulation of the key cellular processes that are essential for the proper functioning of the cell under physiological conditions. Notably, the hyperactivation of ERK1/2 is implicated in oncogenesis and metastatic dissemination across various tumor types, making it an attractive candidate for targeted therapy (TT) through functional inhibition. In intrahepatic cholangiocarcinoma (iCCA), sustained ERK1/2 activation represents one of the major events within the complex signaling network that drives tumor development and progression. In this review, we dissect the biological role of ERK1/2 signaling in iCCA and highlight recent preclinical advances involving selective small-molecule ERK1/2 inhibitors. In vitro and in vivo studies have demonstrated how these inhibitors present effective anti-tumorigenic properties. In particular, PD901 and U0126 effectively reduce iCCA cell proliferation and invasion. Furthermore, Ulixertinib has shown a favorable therapeutic index and encouraging activity in clinical trials involving advanced solid tumors, including iCCA, paving the way for a new therapeutic approach targeting ERK1/2. Nevertheless, the heterogeneous and dynamic molecular landscape of iCCA, often accompanied by drug resistance, presents significant therapeutic challenges. We underscore how targeting the ERK1/2 pathway could represent a cornerstone within a multifaceted therapeutic strategy, fostering the development of personalized treatment approaches and improving clinical outcomes in iCCA patients.
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spelling doaj-art-2a8adbd7b17f4ea9a1d0db43adb4f0a02025-08-20T03:32:31ZengMDPI AGBiology2079-77372025-06-0114777610.3390/biology14070776ERK1/2 Signaling in Intrahepatic Cholangiocarcinoma: From Preclinical Advances to Therapeutic StrategiesVeronica Porreca0Luca Sallustio1Ludovica Giancola2Pietro Angelone3Giuseppina Mignogna4Bruno Maras5Carmine Mancone6Department of Molecular Medicine, Sapienza University of Rome, 00161 Rome, ItalyDepartment of Molecular Medicine, Sapienza University of Rome, 00161 Rome, ItalyDepartment of Molecular Medicine, Sapienza University of Rome, 00161 Rome, ItalyDepartment of Molecular Medicine, Sapienza University of Rome, 00161 Rome, ItalyDepartment of Biochemical Science, Sapienza University of Rome, Piazzale Aldo Moro 5, 00185 Rome, ItalyDepartment of Biochemical Science, Sapienza University of Rome, Piazzale Aldo Moro 5, 00185 Rome, ItalyDepartment of Molecular Medicine, Sapienza University of Rome, 00161 Rome, ItalyExtracellular signal-regulated kinase 1/2 (ERK1/2) is involved in the regulation of the key cellular processes that are essential for the proper functioning of the cell under physiological conditions. Notably, the hyperactivation of ERK1/2 is implicated in oncogenesis and metastatic dissemination across various tumor types, making it an attractive candidate for targeted therapy (TT) through functional inhibition. In intrahepatic cholangiocarcinoma (iCCA), sustained ERK1/2 activation represents one of the major events within the complex signaling network that drives tumor development and progression. In this review, we dissect the biological role of ERK1/2 signaling in iCCA and highlight recent preclinical advances involving selective small-molecule ERK1/2 inhibitors. In vitro and in vivo studies have demonstrated how these inhibitors present effective anti-tumorigenic properties. In particular, PD901 and U0126 effectively reduce iCCA cell proliferation and invasion. Furthermore, Ulixertinib has shown a favorable therapeutic index and encouraging activity in clinical trials involving advanced solid tumors, including iCCA, paving the way for a new therapeutic approach targeting ERK1/2. Nevertheless, the heterogeneous and dynamic molecular landscape of iCCA, often accompanied by drug resistance, presents significant therapeutic challenges. We underscore how targeting the ERK1/2 pathway could represent a cornerstone within a multifaceted therapeutic strategy, fostering the development of personalized treatment approaches and improving clinical outcomes in iCCA patients.https://www.mdpi.com/2079-7737/14/7/776ERK1/2intrahepatic cholangiocarcinomatarget therapypersonalized medicinetumor microenvironment
spellingShingle Veronica Porreca
Luca Sallustio
Ludovica Giancola
Pietro Angelone
Giuseppina Mignogna
Bruno Maras
Carmine Mancone
ERK1/2 Signaling in Intrahepatic Cholangiocarcinoma: From Preclinical Advances to Therapeutic Strategies
Biology
ERK1/2
intrahepatic cholangiocarcinoma
target therapy
personalized medicine
tumor microenvironment
title ERK1/2 Signaling in Intrahepatic Cholangiocarcinoma: From Preclinical Advances to Therapeutic Strategies
title_full ERK1/2 Signaling in Intrahepatic Cholangiocarcinoma: From Preclinical Advances to Therapeutic Strategies
title_fullStr ERK1/2 Signaling in Intrahepatic Cholangiocarcinoma: From Preclinical Advances to Therapeutic Strategies
title_full_unstemmed ERK1/2 Signaling in Intrahepatic Cholangiocarcinoma: From Preclinical Advances to Therapeutic Strategies
title_short ERK1/2 Signaling in Intrahepatic Cholangiocarcinoma: From Preclinical Advances to Therapeutic Strategies
title_sort erk1 2 signaling in intrahepatic cholangiocarcinoma from preclinical advances to therapeutic strategies
topic ERK1/2
intrahepatic cholangiocarcinoma
target therapy
personalized medicine
tumor microenvironment
url https://www.mdpi.com/2079-7737/14/7/776
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