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Updated: Sep 12, 2025
Culture of Bladder Cancer Organoids as Precision Medicine Tools
Published on: December 28, 2021
Top advances of the year: Bladder cancer
Andrea B Apolo1, Saad Atiq1, Andre R Kydd1
1Genitourinary Malignancies Branch, Center for Cancer Research, National Cancer Institute, National Institutes of Health, Bethesda, Maryland, USA.
Abstract:
Looking back at 2024, the authors highlight the top five clinical advances in bladder cancer (urothelial carcinoma), from: (1) novel drug-delivery mechanisms in intravesical therapy for nonmuscle-invasive bladder cancer and muscle-invasive bladder cancer (MIBC); (2) immune checkpoint inhibition (ICI) as adjuvant and (3) perioperative therapy in MIBC; (4) circulating tumor DNA as a biomarker in MIBC; to (5) a new standard of care in first-line metastatic urothelial carcinoma. TAR-200 is a new intravesical drug-delivery system that enables controlled release of gemcitabine but may be used with other anticancer drugs to treat nonmuscle-invasive bladder cancer and MIBC. Two phase 3 studies of adjuvant ICI (nivolumab and pembrolizumab) have both reported a doubling of disease-free survival in patients with high-risk MIBC receiving therapy. Perioperative durvalumab, including neoadjuvant therapy plus gemcitabine and cisplatin before radical cystectomy followed by adjuvant durvalumab, demonstrated an improvement in event-free survival and overall survival for patients with MIBC. Circulating tumor DNA is a promising biomarker to select patients with MIBC for adjuvant ICI therapy. Finally, the combination of enfortumab vedotin, an antibody-drug conjugate, plus pembrolizumab doubled overall survival compared with standard gemcitabine plus platinum in patients with metastatic urothelial carcinoma and has been implemented in treatment guidelines in the United States, Europe, and Asia as the new standard of care in this setting, transforming the treatment landscape for bladder cancer.
Insights
In 2024, bladder cancer treatment advanced with new intravesical therapies, adjuvant and perioperative immune checkpoint inhibition (ICI), circulating tumor DNA biomarkers, and a new standard of care for metastatic urothelial carcinoma.
Area of Science:
- Oncology
- Urothelial Carcinoma Research
- Clinical Advances
Background:
- Bladder cancer treatment has seen significant progress in 2024.
- Key areas of advancement include intravesical therapy, immune checkpoint inhibition, and biomarkers.
- The landscape of metastatic urothelial carcinoma treatment has been transformed.
Purpose of the Study:
- To review the top five clinical advances in bladder cancer for 2024.
- To highlight novel therapeutic strategies and biomarkers.
- To summarize the impact of these advances on patient outcomes.
Main Methods:
- Review of novel intravesical drug-delivery systems (e.g., TAR-200).
- Analysis of Phase 3 studies on adjuvant and perioperative immune checkpoint inhibition (ICI) in muscle-invasive bladder cancer (MIBC).
- Evaluation of circulating tumor DNA (ctDNA) as a biomarker for MIBC.
- Assessment of new first-line treatments for metastatic urothelial carcinoma.
Main Results:
- Novel intravesical therapies show promise for nonmuscle-invasive and muscle-invasive bladder cancer (MIBC).
- Adjuvant ICI (nivolumab, pembrolizumab) doubled disease-free survival in high-risk MIBC.
- Perioperative durvalumab improved event-free and overall survival in MIBC.
- ctDNA is a promising biomarker for selecting patients for adjuvant ICI therapy.
- Enfortumab vedotin plus pembrolizumab doubled overall survival in metastatic urothelial carcinoma, becoming a new standard of care.
Conclusions:
- Significant clinical advances in bladder cancer treatment were achieved in 2024.
- New intravesical drug delivery, ICI strategies, and biomarkers are transforming care.
- The combination of enfortumab vedotin and pembrolizumab represents a major breakthrough for metastatic disease.
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