Related Experiment Video
Updated: Oct 19, 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
Molecular-driven treatment for biliary tract cancer: the promising turning point
Mara Persano1, Marco Puzzoni1, Pina Ziranu1
1Medical Oncology Unit, University Hospital and University of Cagliari, Cagliari, Italy.
Introduction:
In the past, targeted therapies have not shown positive results as they have been used without adequate molecular selection of patients with biliary tract cancer (BTC). This has led to an expansion of research on characteristics and molecular selection to identify new effective strategies in this setting. Improved knowledge of the molecular biology of these neoplasms has highlighted their extraordinary heterogeneity and has made it possible to identify targetable gene alterations, including fibroblast growth factor receptor (FGFR) 2 gene fusions, and isocitrate dehydrogenase (IDH) mutations. The FDA recently approved ivosidenib and pemigatinib for the treatment of BTCs.
Areas Covered:
We review data in the literature regarding targeted therapies for the treatment of BTCs, as well as on the prospects deriving from the extraordinary molecular heterogeneity of these neoplasms.
Expert Opinion:
At present, it is essential to evaluate the expression of the genetic alterations expressed by these neoplasms to offer patients an increasingly personalized therapeutic approach. Studies are needed to better define the limits and potentials of targeted therapies and their role in the therapeutic algorithm to improve the poor prognosis of these patients.
Insights
Targeted therapies for biliary tract cancer (BTC) are improving with molecular selection. Identifying gene fusions like FGFR2 and IDH mutations allows for personalized treatment strategies, enhancing patient outcomes.
Area of Science:
- Oncology
- Molecular Biology
- Genetics
Background:
- Biliary tract cancer (BTC) treatment historically lacked efficacy due to inadequate patient molecular selection for targeted therapies.
- Recent advancements in understanding BTC molecular biology reveal significant heterogeneity and identify targetable genetic alterations.
- FDA approvals of ivosidenib and pemigatinib mark progress in BTC targeted treatment.
Purpose of the Study:
- To review current literature on targeted therapies for BTC.
- To explore the potential of molecularly targeted treatments based on BTC's heterogeneity.
- To discuss the future role of targeted therapies in improving BTC patient prognosis.
Main Methods:
- Literature review of studies on targeted therapies in BTC.
- Analysis of research on molecular characteristics and heterogeneity of BTC.
- Examination of data on specific gene alterations (FGFR2 fusions, IDH mutations) and their targeted treatments.
Main Results:
- Targeted therapies show promise when guided by molecular selection, addressing BTC's heterogeneity.
- Identification of specific actionable mutations, such as FGFR2 gene fusions and IDH mutations, is crucial.
- New drugs like ivosidenib and pemigatinib are now available for specific BTC patient populations.
Conclusions:
- Personalized therapeutic approaches based on genetic alterations are essential for improving BTC outcomes.
- Further research is needed to define the optimal role and potential of targeted therapies in the BTC treatment algorithm.
- Evaluating genetic alterations is key to advancing personalized medicine and improving the prognosis for patients with biliary tract cancer.
Related Concept Videos
Targeted Cancer Therapies
There are several types of targeted therapies against...
Treatment Resistant Cancers
Combination 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...
Cancer 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...

