Top advances of the year: Bladder cancer

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.