What targeted therapies are available for Bladder Cancer

Targeted Therapies for Bladder Cancer

Targeted therapies represent an important treatment option for bladder cancer patients, particularly those with advanced disease. These drugs work differently than traditional chemotherapy—they target specific genetic mutations or protein markers found in cancer cells rather than attacking all rapidly dividing cells.

Key Targeted Therapy Options

FGFR3-Targeted Therapy: Erdafitinib

According to the NCCN Guidelines for Bladder Cancer, erdafitinib is an FDA-approved targeted therapy for patients with metastatic urothelial carcinoma (advanced bladder cancer) that has specific FGFR3 genetic alterations.

How it works: FGFR3 is a growth signal protein that's mutated in some bladder cancers. Erdafitinib blocks this signal, slowing or stopping cancer cell growth.

Important note: This therapy requires genetic testing to confirm FGFR3 mutations are present in your cancer cells.

HER2-Targeted Therapy: Fam-trastuzumab Deruxtecan

This antibody-drug conjugate (a newer class of targeted therapy) targets HER2 overexpression in bladder cancer cells. According to NCCN Guidelines, HER2 testing by immunohistochemistry (IHC) should be performed on advanced bladder cancers to determine eligibility for this treatment.

How it works: The drug delivers chemotherapy directly to cancer cells that have high levels of HER2 protein on their surface.

Emerging Treatment Approaches

The NCCN Guidelines note that immune checkpoint inhibitors, antibody-drug conjugates, and targeted therapies have emerged as new options for the treatment of metastatic bladder cancer, with experts predicting that treatment approaches will evolve rapidly over the coming years.

Molecular Testing: The Foundation for Targeted Therapy

According to NCCN Guidelines, molecular/genomic testing should be performed for stages IVA and IVB (metastatic) bladder cancer and may be considered for stage IIIB. This testing is critical because:

• It identifies which targeted therapies your specific cancer may respond to
• It should be done early at diagnosis to guide treatment decisions
• Testing must be performed in CLIA-certified laboratories (Clinical Laboratory Improvement Amendments)
• A comprehensive study found that 93% of advanced urothelial carcinoma cases had at least one clinically relevant genetic alteration, with an average of 2.6 alterations per case

Important Considerations

Targeted therapies are NOT one-size-fits-all. Your cancer's specific genetic profile determines which targeted therapies might work for you. This is why genetic testing is so important—it's the "map" that helps your oncologist select the most effective treatment.

Questions to Ask Your Oncology Team

1. Has my cancer been tested for FGFR3 mutations and HER2 overexpression? If not, should it be?
2. Based on my test results, am I eligible for any targeted therapies?
3. What are the expected benefits and side effects of the targeted therapy you're recommending?
4. Are there clinical trials available that might include targeted therapy options for my specific cancer profile?
5. How will we monitor whether the targeted therapy is working?

The Bigger Picture

While targeted therapies show promise, the NCCN Guidelines emphasize that treatment decisions for bladder cancer are individualized and depend on many factors including disease stage, your overall health, kidney function, and your preferences. Your oncologist will determine the most appropriate approach for your specific situation, which may involve targeted therapy alone, combined with other treatments, or as part of a clinical trial.

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This information is for educational purposes only. Always consult your healthcare team for personalized medical advice and decisions.