Gallbladder Cancer Medication

Updated: Dec 03, 2021
  • Author: Eric Fox, DO, MA; Chief Editor: N Joseph Espat, MD, MS, FACS  more...
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Medication Summary

Historically, chemotherapy has not shown significant activity in gallbladder carcinoma. Typically, 5-fluorouracil (5-FU) has been used with response rates of 10-24% in advanced disease. Often 5-FU is administered either as a bolus or as a prolonged infusion regimen with radiation. Capecitabine is a currently available oral alternative to a prolonged 5-FU infusion.

More recently, gemcitabine has shown activity in gallbladder cancer. Early-phase studies show an increased response rate with gemcitabine combination therapy over historical treatment response rates with 5-FU alone. Gemcitabine has been studied in combination with cis-platinum and capecitabine.

The programmed death ligand (PD-L) inhibitor pembrolizumab has demonstrated benefit in tumors with high microsatellite instability/deficient mismatch repair (MSI-H/dMMR). [38] The tyrosine kinase inhibitor entrectinib may be useful in NTRK gene fusion–positive tumors. [30]

Currently, no clearly defined standard exists for chemotherapy in gallbladder cancer. Patients should be encouraged to participate in clinical trials.


Antineoplastic agents

Class Summary

These agents inhibit cell growth and proliferation.

Gemcitabine (Gemzar)

Cytidine analog, after intracellular metabolism to active nucleotide, inhibits ribonucleotide reductase and competes with deoxycytidine triphosphate for incorporation into DNA. Cell cycle-specific for S phase.

This drug has been shown to have activity in a phase-2 trial against relapsed germ cell tumors.

Cisplatin (Platinol)

Platinum-containing compound that exerts antineoplastic effect by covalently binding to DNA with preferential binding to N-7 position of guanine and adenosine. Can react with 2 different sites on DNA to cause cross-links. Platinum complex also can bind to nucleus and cytoplasmic protein. A bifunctional alkylating agent, once activated to aquated form in cell, binds to DNA, resulting in interstrand and intrastrand cross-linking and denaturation of double helix.

Modify dose on basis of CrCl. Avoid use if CrCl < 60 mL/min.

Capecitabine (Xeloda)

Prodrug of fluorouracil that undergoes hydrolysis in liver and tissues to form the active moiety (fluorouracil), inhibiting thymidylate synthetase, which in turn blocks methylation of deoxyuridylic acid to thymidylic acid. This step interferes with DNA, and to a lesser degree with RNA synthesis.


PD-1/PD-L1 Inhibitors

Pembrolizumab (Keytruda)

Anti PD-1 humanized monoclonal antibody which inhibits programmed cell death 1 activity by binding the PD-1 receptor on T cells to block PD-1 ligands (PDL-1 and PDL-2) from binding


Antineoplastics, Tyrosine Kinase Inhibitor

Entrectinib (Rozlytrek)

Inhibitor of tropomyosin tyrosine receptor kinases. Entrectinib also inhibits proto-oncogenic tyrosine-protein kinase (ROS1) and anaplastic lymphoma kinase (ALK). Entrectinib targets these oncogenic drivers to disrupt this unchecked cell proliferation