title: Investigational therapies for non-muscle invasive bladder cancer.
abstract: BACKGROUND
  Bacillus Calmette-Guérin (BCG) is currently the most effective adjuvant intravesical agent at preventing disease recurrence and the only therapy shown to inhibit disease progression in non-muscle invasive bladder cancer (NMIBC). However, recurrence rates as high as 30% and significant local/systemic toxicity have resulted in an increased interest in the use of alternative intravesical agents.
  
  
  METHODS
  Our aim is to discuss recent clinical trial evidence utilizing novel intravesical agents for treatment of NMIBC. A systematic literature review was performed via the National Center for Biotechnology Information databases to identify pertinent studies from 2000-2009.
  
  
  RESULTS
  A durable response has been demonstrated with alternative agents in patients refractory to or intolerant of BCG. This review compares the merits and shortcomings of these emerging agents, focusing on clinical trial safety and efficacy results.
  
  
  CONCLUSIONS
  Despite recent enthusiasm for novel agents, radical cystectomy remains the treatment of choice for patients with NMIBC who have failed intravesical therapy. However, evidence is accumulating that novel agents provide an efficacious alternative in patients refractory or intolerable to BCG or unfit for cystectomy. Further randomized prospective data are required to demonstrate a recurrence- and progression-free benefit compared with BCG.
authors.affiliation: University of Pittsburgh Medical Center, Department of Urology, Kaufmann Building, 3471 5th Avenue, Pittsburgh, PA 15213, USA. smaldonemc@upmc.edu
authors.forename: Marc C
authors.initials: MC
authors.lastname: Smaldone
authors.forename: Daniel P
authors.initials: DP
authors.lastname: Casella
authors.forename: Daniel R
authors.initials: DR
authors.lastname: Welchons
authors.forename: Jeffrey R
authors.initials: JR
authors.lastname: Gingrich
mesh_terms: D000276:Adjuvants, Immunologic / Q000009:adverse effects / Q000627:therapeutic use; D000818:Animals; D000970:Antineoplastic Agents / Q000009:adverse effects / Q000627:therapeutic use*; D001500:BCG Vaccine / Q000009:adverse effects / Q000627:therapeutic use; D002986:Clinical Trials as Topic; D015653:Cystectomy / Q000379:methods; D018450:Disease Progression; D006801:Humans; D007155:Immunologic Factors / Q000009:adverse effects / Q000627:therapeutic use*; D009364:Neoplasm Recurrence, Local; D001749:Urinary Bladder Neoplasms / Q000473:pathology / Q000628:therapy*
chemical_list: D000276:Adjuvants, Immunologic; D000970:Antineoplastic Agents; D001500:BCG Vaccine; D007155:Immunologic Factors