Novel molecular targets for the therapy of urothelial carcinoma

Bagi R.P. Jana, Matthew D. Galsky, Noah M. Hahn, Matthew I. Milowsky, Guru Sonpavde

Research output: Contribution to journalReview articlepeer-review

2 Scopus citations


Introduction: First-line platinum-based combinations are active in locally advanced and metastatic urothelial carcinoma; however, long-term outcomes including disease-specific and overall survival remain suboptimal. In addition, approximately 40 50% of patients with advanced urothelial carcinoma have coexisting medical issues that preclude the use of cisplatin-based therapy. Improvements in our understanding of the molecular mechanisms of urothelial tumorigenesis have led to first-generation clinical trials evaluating novel agents targeting molecular pathways. These are particularly relevant in regard to subpopulations. Novel trial designs warrant consideration to accelerate accrual. Areas covered: In this review, novel molecular targets for the therapy of urothelial carcinoma, as well as recently completed and ongoing clinical trials utilizing novel targeted agents, are discussed. A Medline search with key words, abstracts reported at national conferences on urothelial carcinoma and NCI clinical trial identifiers was used for this review. Expert opinion: Improved understanding of molecular biology has identified a number of new molecular targets, but there is a seeming absence of one dominant molecular driver for urothelial cancer. An adaptive and biomarker-derived strategy may be warranted. Clinical trials utilizing targeted agents are ongoing and results are awaited.

Original languageEnglish (US)
Pages (from-to)499-513
Number of pages15
JournalExpert opinion on therapeutic targets
Issue number5
StatePublished - May 2012
Externally publishedYes


  • Novel molecular targets
  • Targeted therapy
  • Transitional cell carcinoma
  • Urothelial carcinoma

ASJC Scopus subject areas

  • Molecular Medicine
  • Pharmacology
  • Drug Discovery
  • Clinical Biochemistry


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