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Induction of Invasive Transitional Cell Bladder Carcinoma in Immune Intact Human MUC1 Transgenic Mice: A Model for Immunotherapy Development
Published on: October 30, 2013
Emerging therapeutic targets in bladder cancer
Benedito A Carneiro1, Joshua J Meeks2, Timothy M Kuzel3
1Northwestern Medicine Developmental Therapeutics Institute, Feinberg School of Medicine, Northwestern University, United States; Division of Hematology and Oncology, Feinberg School of Medicine, Northwestern University, United States; Robert H. Lurie Comprehensive Cancer Center of Northwestern University, United States.
Abstract:
Treatment of muscle invasive urothelial bladder carcinoma (BCa) remains a major challenge. Comprehensive genomic profiling of tumors and identification of driver mutations may reveal new therapeutic targets. This manuscript discusses relevant molecular drivers of the malignant phenotype and agents with therapeutic potential in BCa. Small molecule pan-FGFR inhibitors have shown encouraging efficacy and safety results especially among patients with activating FGFR mutations or translocations. mTOR inhibitors for patients with TSC1 mutations and concomitant targeting of PI3K and MEK represent strategies to block PI3K/AKT/mTOR pathway. Encouraging preclinical results with ado-trastuzumab emtansine (T-DM1) exemplifies a new potential treatment for HER2-positive BCa along with innovative bispecific antibodies. Inhibitors of cell cycle regulators (aurora kinase, polo-like kinase 1, and cyclin-dependent kinase 4) are being investigated in combination with chemotherapy. Early results of clinical studies with anti-CTLA4 and anti-PDL1 are propelling immune modulating drugs to the forefront of emerging treatments for BCa. Collectively, these novel therapeutic targets and treatment strategies hold promise to improve the outcome of patients afflicted with this malignancy.
Insights
Muscle invasive urothelial bladder cancer (BCa) treatment is challenging. Genomic profiling identifies new targets like FGFR, HER2, and immune checkpoints, offering promising novel therapies for BCa patients.
Area of Science:
- Oncology
- Genomics
- Pharmacology
Background:
- Muscle invasive urothelial bladder carcinoma (BCa) presents significant treatment challenges.
- Comprehensive genomic profiling of tumors is crucial for identifying actionable molecular drivers.
- Understanding these drivers can reveal novel therapeutic targets for BCa.
Purpose of the Study:
- To review molecular drivers of the malignant phenotype in BCa.
- To discuss emerging therapeutic agents targeting these drivers.
- To highlight novel treatment strategies for BCa.
Main Methods:
- Review of current literature on molecular drivers in BCa.
- Analysis of preclinical and clinical data for targeted therapies.
- Discussion of emerging treatment modalities including small molecule inhibitors and immunotherapies.
Main Results:
- Activating FGFR mutations/translocations show efficacy with pan-FGFR inhibitors.
- mTOR inhibitors and PI3K/MEK targeting are strategies for TSC1 mutations.
- Ado-trastuzumab emtansine (T-DM1) shows promise for HER2-positive BCa.
- Investigational inhibitors of cell cycle regulators (aurora kinase, PLK1, CDK4) are studied with chemotherapy.
- Anti-CTLA4 and anti-PDL1 therapies are emerging as potent immunomodulating treatments for BCa.
Conclusions:
- Novel therapeutic targets and agents, including FGFR inhibitors, T-DM1, and immunotherapies, hold significant promise.
- Targeted therapies and combination strategies are advancing BCa treatment.
- These advancements offer hope for improved outcomes in patients with BCa.
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