Related Experiment Video
Updated: Aug 27, 2025

Induction of Invasive Transitional Cell Bladder Carcinoma in Immune Intact Human MUC1 Transgenic Mice: A Model for Immunotherapy Development
Published on: October 30, 2013
Immunotherapy With Checkpoint Inhibitors in FGFR-Altered Urothelial Carcinoma
David J Benjamin1, Nataliya Mar2, Arash Rezazadeh Kalebasty2
1Medical Oncology, Hoag Family Cancer Institute, Newport Beach, CA, USA.
Abstract:
The treatment landscape of metastatic urothelial cancer (mUC) remained unchanged for over 30 years until the approval of immune checkpoint inhibitors (ICIs) in 2016. Since then, several ICIs have been approved for the treatment of mUC. In addition, recent molecular characterization of bladder cancer has revealed several subtypes, including those harboring fibroblast growth factor receptor (FGFR) mutations and fusion proteins. Erdafitinib, a pan-FGFR inhibitor, was approved for the treatment of metastatic/advanced UC in 2019. Some available evidence suggests ICI may have inferior response in advanced FGFR+ UC for unclear reasons, but may possibly be related to the tumor microenvironment. Several ongoing trials are evaluating erdafitinib in metastatic/advanced UC including the ongoing phase IB/II NORSE trial combining erdafitinib plus ICI, which may prove to offer a more robust and durable response in patients with FGFR+ metastatic/advanced UC.
Insights
Immune checkpoint inhibitors (ICIs) revolutionized metastatic urothelial cancer (mUC) treatment. Combining erdafitinib with ICIs may improve outcomes for patients with FGFR+ mUC, addressing potential ICI resistance.
Area of Science:
- Oncology
- Genitourinary Cancer
- Cancer Therapeutics
Background:
- Metastatic urothelial cancer (mUC) treatment options were limited until immune checkpoint inhibitors (ICIs) emerged in 2016.
- Molecular profiling identified subtypes of bladder cancer, including those with fibroblast growth factor receptor (FGFR) alterations.
- Erdafitinib, a pan-FGFR inhibitor, gained approval in 2019 for advanced UC.
Purpose of the Study:
- To investigate the potential for improved treatment outcomes in FGFR-altered metastatic urothelial cancer.
- To explore the combination of erdafitinib and ICIs in mUC treatment.
- To address the observed inferior response to ICIs in advanced FGFR+ UC.
Main Methods:
- Review of current treatment paradigms for mUC.
- Analysis of molecular subtypes, focusing on FGFR alterations.
- Examination of ongoing clinical trials, including Phase IB/II studies combining erdafitinib with ICIs.
Main Results:
- Evidence suggests potential for reduced efficacy of ICIs in FGFR-altered mUC.
- The tumor microenvironment may play a role in ICI response in FGFR+ mUC.
- Ongoing trials are evaluating combination therapies for enhanced efficacy.
Conclusions:
- The combination of erdafitinib and ICIs shows promise for more robust and durable responses in FGFR+ mUC.
- Targeting FGFR alterations alongside immune modulation represents a potential advancement in mUC therapy.
- Further research and clinical trials are crucial to validate these findings.
More Related Videos
Related Concept Videos
Tumor Immunotherapy
Targeted Cancer Therapies
There are several types of targeted therapies against...
Mitogens and the Cell Cycle

