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Bladder Cancer. 2016 Apr 27;2(2):165-202.

Summary and Recommendations from the National Cancer Institute's Clinical Trials Planning Meeting on Novel Therapeutics for Non-Muscle Invasive Bladder Cancer.

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Baylor College of Medicine , Houston, TX, USA.
Memorial Sloan Kettering Cancer Center, New York, NY, USA; Weill Medical College of Cornell University, New York, NY, USA.
The University of Texas MD Anderson Cancer Center , Houston, TX, USA.
Massachusetts General Hospital , Harvard Medical School, Boston, MA, USA.
USC Norris Comprehensive Cancer Center, University of Southern California , Los Angeles, CA, USA.
Johns Hopkins Sidney Kimmel Comprehensive Cancer Center , Baltimore, MD, USA.
University of California , La Jolla, San Diego, CA, USA.
Brigham and Women's Hospital , Harvard Medical School, Boston, MA, USA.
The University of Iowa , IA, USA.
UT Health Science Center San Antonio , San Antonio, TX, USA.
Cancer Therapy Evaluation Program, Division of Cancer Treatment and Diagnosis, National Cancer Institute, National Institutes of Health , Bethesda, MD, USA.
Memorial Sloan Kettering Cancer Center , New York, NY, USA.
Genitourinary Malignancies Branch, Center for Cancer Research, National Cancer Institute , National Institutes of Health, Bethesda, MD, USA.
Dana-Farber Cancer Institute and Harvard Medical School , Boston, MA, USA.
Office of Hematology and Oncology Products , U.S. Food and Drug Administration, Silver Spring, MD, USA.
University of North Carolina Lineberger Comprehensive Cancer Center , Chapel Hill, NC, USA.
Leeds Institute of Cancer and Pathology , University of Leeds, Leeds, UK.
Biometric Research Branch, Division of Cancer Treatment and Diagnosis, National Cancer Institute , Bethesda, MD, USA.
The Research Institute at Nationwide Children's Hospital , Columbus, OH, USA.
USC Norris Comprehensive Cancer Center, University of Southern California, Los Angeles, CA, USA; Johns Hopkins Sidney Kimmel Comprehensive Cancer Center, Baltimore, MD, USA.
SWOG Statistical Center , Fred Hutchinson Cancer Research Center, Seattle, WA, USA.
The Emmes Corporation , Rockville, MD, USA.
Department of Urology, Radboud UMC, Nijmegen, The Netherlands.
Bladder Cancer Advocacy Network , Bethesda, MD, USA.


The NCI Bladder Cancer Task Force convened a Clinical Trials Planning Meeting (CTPM) Workshop focused on Novel Therapeutics for Non-Muscle Invasive Bladder Cancer (NMIBC). Meeting attendees included a broad and multi-disciplinary group of clinical and research stakeholders and included leaders from NCI, FDA, National Clinical Trials Network (NCTN), advocacy and the pharmaceutical and biotech industry. The meeting goals and objectives were to: 1) create a collaborative environment in which the greater bladder research community can pursue future optimally designed novel clinical trials focused on the theme of molecular targeted and immune-based therapies in NMIBC; 2) frame the clinical and translational questions that are of highest priority; and 3) develop two clinical trial designs focusing on immunotherapy and molecular targeted therapy. Despite successful development and implementation of large Phase II and Phase III trials in bladder and upper urinary tract cancers, there are no active and accruing trials in the NMIBC space within the NCTN. Disappointingly, there has been only one new FDA approved drug (Valrubicin) in any bladder cancer disease state since 1998. Although genomic-based data for bladder cancer are increasingly available, translating these discoveries into practice changing treatment is still to come. Recently, major efforts in defining the genomic characteristics of NMIBC have been achieved. Aligned with these data is the growing number of targeted therapy agents approved and/or in development in other organ site cancers and the multiple similarities of bladder cancer with molecular subtypes in these other cancers. Additionally, although bladder cancer is one of the more immunogenic tumors, some tumors have the ability to attenuate or eliminate host immune responses. Two trial concepts emerged from the meeting including a window of opportunity trial (Phase 0) testing an FGFR3 inhibitor and a second multi-arm multi-stage trial testing combinations of BCG or radiotherapy and immunomodulatory agents in patients who recur after induction BCG (BCG failure).


Non-muscle invasive bladder cancer; immunotherapy; radiation therapy; targeted therapy; trial design

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