Options in Targeted Therapy for Advanced Cholangiocarcinoma: A 2024 Update
Related Experiment Video
Updated: Jun 24, 2025
An Oncogenic Hepatocyte-Induced Orthotopic Mouse Model of Hepatocellular Cancer Arising in the Setting of Hepatic Inflammation and Fibrosis
Published on: September 12, 2019
Anca Monica Oprescu Macovei1, Dana Paula Venter2, Gratiella Georgiana Makkai3
1Gastroenterology, Agrippa Ionescu Emergency Clinical Hospital, Bucharest, ROU.
Abstract:
Bile duct carcinomas have a different prognosis and genetic profile depending on their location; intrahepatic/extrahepatic or at the level of the gallbladder. Although in recent years there have been important advances in first-line therapy, second-line therapy in cholangiocarcinoma does not currently have a standard. Therefore at this level, there is an acute need for personalized treatment. The present article is a narrative review that aims to list the newest targeted therapeutic options for this type of cancer, based on identified genetic alterations. The literature selected for analysis includes phase 2 or 3 studies with targeted therapy in this disease and original articles no older than three years that describe the prevalence of the most common gene alterations in this type of cancer. PubMed/Medline, Scopus, and Clarivate-Web of Science databases were searched and keywords such as "cholangiocarcinoma," "biliary cancer," "targeted therapy," "gene amplifications," and "mutations" were used. This narrative review was designed taking into account the SANRA (Scale for the Assessment of Narrative Review Articles) criteria. The conclusions lead to the fact that next-generation sequencing testing is of particular usefulness in cholangiocarcinoma. Bile duct cancers are rich in targetable genetic alterations, and their treatment is in constant change, although much of the current data comes from phase II studies. There is a great need for the current options to be analyzed in phase III studies. Hence, the need of the oncological community to stay informed about targeted treatment options for cholangiocarcinoma is supported by the present article.
Insights
Targeted therapies offer new hope for cholangiocarcinoma (bile duct cancer) patients by targeting specific genetic alterations. Next-generation sequencing is crucial for identifying these alterations and guiding personalized treatment strategies.
Area of Science:
- Oncology
- Genetics
- Gastroenterology
Background:
- Cholangiocarcinoma (bile duct cancer) exhibits varied prognosis and genetic profiles based on tumor location (intrahepatic/extrahepatic, gallbladder).
- While first-line therapies have advanced, second-line treatments for cholangiocarcinoma lack standardization, highlighting a need for personalized approaches.
- Genetic alterations are increasingly recognized as key drivers in bile duct cancer development and progression.
Purpose of the Study:
- To review the latest targeted therapeutic options for cholangiocarcinoma, focusing on treatments linked to specific genetic alterations.
- To emphasize the importance of next-generation sequencing in identifying actionable mutations and amplifications.
- To provide an updated overview for the oncological community on emerging targeted treatments for this disease.
Main Methods:
- A narrative review of phase 2 or 3 clinical trials involving targeted therapy in cholangiocarcinoma.
- Inclusion of original articles (≤3 years old) detailing the prevalence of common gene alterations in bile duct cancer.
- Literature search conducted across PubMed/Medline, Scopus, and Clarivate-Web of Science databases using keywords like 'cholangiocarcinoma,' 'biliary cancer,' 'targeted therapy,' 'gene amplifications,' and 'mutations.'
Main Results:
- Bile duct cancers frequently harbor targetable genetic alterations, making them amenable to precision medicine.
- Next-generation sequencing (NGS) is highly valuable for identifying these genetic alterations in cholangiocarcinoma patients.
- Current targeted therapy data largely stems from phase II studies, indicating a need for further validation in phase III trials.
Conclusions:
- Next-generation sequencing is essential for personalized treatment selection in cholangiocarcinoma.
- The landscape of targeted therapy for bile duct cancer is rapidly evolving, driven by genetic discoveries.
- Further phase III studies are required to solidify the role of emerging targeted therapies in cholangiocarcinoma treatment regimens.
Related Concept Videos
02:57Targeted Cancer Therapies
There are several types of targeted therapies against...
02:50Combination Therapies and Personalized Medicine
The combination of the drug acetazolamide and sulforaphane is a good example of combination therapy to treat cancer. The cells in the interior of a large tumor often die due to the hypoxic and...
01:27Tumor Immunotherapy
02:49Cancer Therapies
However, cancer treatments can pose several challenges, as therapies used to kill cancer cells are generally also toxic to normal cells. Moreover, cancer cells mutate rapidly and can develop resistance to chemical agents or radiation therapy. Besides, all types of cancer cells may not respond to the same therapy. Some cancer cells respond to one...