Current advances in BCG-unresponsive non-muscle invasive bladder cancer

Jennifer Tse, Nirmish Singla, Rashed Ghandour, Yair Lotan, Vitaly Margulis

Research output: Contribution to journalReview articlepeer-review

17 Scopus citations


Introduction: The current first line therapy for high grade (HG) non-muscle invasive bladder cancer (NMIBC) is intravesical Bacillus Calmette–Guerin (BCG). Patients who recur or progress despite BCG are recommended to undergo radical cystectomy or participate in clinical trials. There is an urgent need for alternative therapies in the BCG-unresponsive NMIBC realm. Areas covered: We queried and for current and recently completed early clinical trials pertaining to investigational agents used for the treatment of BCG-unresponsive NMIBC. These included intravesical chemotherapy, immunotherapy, vaccines, gene therapy, viruses, and agents used with novel drug delivery methods. In this article, we discuss the treatment guidelines for non-muscle invasive bladder cancer and therapeutic approaches under investigation in clinical trials. Expert opinion: The FDA is currently allowing single-arm studies as a pathway for approval in BCG-refractory patients with CIS. Although many agents are currently undergoing testing, none have been approved since Valrubicin. Hopefully, we will identify therapies sufficiently effective and durable to achieve FDA approval. Other considerations in this realm include the use of biomarkers in NMIBC to identify patients who will most likely respond to specific interventions. In addition, as systemic agents such as checkpoint inhibitors, are studied further, a multidisciplinary approach may be needed to treat this subset of patients.

Original languageEnglish (US)
Pages (from-to)757-770
Number of pages14
JournalExpert Opinion on Investigational Drugs
Issue number9
StatePublished - Sep 2 2019


  • BCG-refractory
  • BCG-unresponsive
  • clinical trials
  • non-muscle invasive bladder cancer

ASJC Scopus subject areas

  • Pharmacology
  • Pharmacology (medical)


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