title: Summary and Recommendations from the National Cancer Institute's Clinical Trials Planning Meeting on Novel Therapeutics for Non-Muscle Invasive Bladder Cancer.
abstract: 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).
authors.affiliation: Baylor College of Medicine , Houston, TX, USA.
authors.forename: Seth P
authors.initials: SP
authors.lastname: Lerner
authors.affiliation: Memorial Sloan Kettering Cancer Center, New York, NY, USA; Weill Medical College of Cornell University, New York, NY, USA.
authors.forename: Dean F
authors.initials: DF
authors.lastname: Bajorin
authors.affiliation: The University of Texas MD Anderson Cancer Center , Houston, TX, USA.
authors.forename: Colin P
authors.initials: CP
authors.lastname: Dinney
authors.affiliation: Massachusetts General Hospital , Harvard Medical School, Boston, MA, USA.
authors.forename: Jason A
authors.initials: JA
authors.lastname: Efstathiou
authors.affiliation: USC Norris Comprehensive Cancer Center, University of Southern California , Los Angeles, CA, USA.
authors.forename: Susan
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authors.lastname: Groshen
authors.affiliation: Johns Hopkins Sidney Kimmel Comprehensive Cancer Center , Baltimore, MD, USA.
authors.forename: Noah M
authors.initials: NM
authors.lastname: Hahn
authors.affiliation: University of California , La Jolla, San Diego, CA, USA.
authors.forename: Donna
authors.initials: D
authors.lastname: Hansel
authors.affiliation: Brigham and Women's Hospital , Harvard Medical School, Boston, MA, USA.
authors.forename: David
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authors.lastname: Kwiatkowski
authors.affiliation: The University of Iowa , IA, USA.
authors.forename: Michael
authors.initials: M
authors.lastname: O'Donnell
authors.affiliation: Memorial Sloan Kettering Cancer Center, New York, NY, USA; Weill Medical College of Cornell University, New York, NY, USA.
authors.forename: Jonathan
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authors.lastname: Rosenberg
authors.affiliation: UT Health Science Center San Antonio , San Antonio, TX, USA.
authors.forename: Robert
authors.initials: R
authors.lastname: Svatek
authors.affiliation: Cancer Therapy Evaluation Program, Division of Cancer Treatment and Diagnosis, National Cancer Institute, National Institutes of Health , Bethesda, MD, USA.
authors.forename: Jeffrey S
authors.initials: JS
authors.lastname: Abrams
authors.affiliation: Memorial Sloan Kettering Cancer Center , New York, NY, USA.
authors.forename: Hikmat
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authors.lastname: Al-Ahmadie
authors.affiliation: Genitourinary Malignancies Branch, Center for Cancer Research, National Cancer Institute , National Institutes of Health, Bethesda, MD, USA.
authors.forename: Andrea B
authors.initials: AB
authors.lastname: Apolo
authors.affiliation: Dana-Farber Cancer Institute and Harvard Medical School , Boston, MA, USA.
authors.forename: Joaquim
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authors.lastname: Bellmunt
authors.affiliation: Memorial Sloan Kettering Cancer Center, New York, NY, USA; Weill Medical College of Cornell University, New York, NY, USA.
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authors.lastname: Callahan
authors.affiliation: Memorial Sloan Kettering Cancer Center , New York, NY, USA.
authors.forename: Eugene K
authors.initials: EK
authors.lastname: Cha
authors.affiliation: Johns Hopkins Sidney Kimmel Comprehensive Cancer Center , Baltimore, MD, USA.
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authors.affiliation: Office of Hematology and Oncology Products , U.S. Food and Drug Administration, Silver Spring, MD, USA.
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authors.lastname: Jarow
authors.affiliation: The University of Texas MD Anderson Cancer Center , Houston, TX, USA.
authors.forename: Ashish
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authors.lastname: Kamat
authors.affiliation: University of North Carolina Lineberger Comprehensive Cancer Center , Chapel Hill, NC, USA.
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authors.lastname: Kim
authors.affiliation: Leeds Institute of Cancer and Pathology , University of Leeds, Leeds, UK.
authors.forename: Margaret
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authors.lastname: Knowles
authors.affiliation: Cancer Therapy Evaluation Program, Division of Cancer Treatment and Diagnosis, National Cancer Institute, National Institutes of Health , Bethesda, MD, USA.
authors.forename: Bhupinder
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authors.lastname: Mann
authors.affiliation: Johns Hopkins Sidney Kimmel Comprehensive Cancer Center , Baltimore, MD, USA.
authors.forename: Luigi
authors.initials: L
authors.lastname: Marchionni
authors.affiliation: The University of Texas MD Anderson Cancer Center , Houston, TX, USA.
authors.forename: David
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authors.lastname: McConkey
authors.affiliation: Biometric Research Branch, Division of Cancer Treatment and Diagnosis, National Cancer Institute , Bethesda, MD, USA.
authors.forename: Lisa
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authors.lastname: McShane
authors.affiliation: The Research Institute at Nationwide Children's Hospital , Columbus, OH, USA.
authors.forename: Nilsa
authors.initials: N
authors.lastname: Ramirez
authors.affiliation: USC Norris Comprehensive Cancer Center, University of Southern California, Los Angeles, CA, USA; Johns Hopkins Sidney Kimmel Comprehensive Cancer Center, Baltimore, MD, USA.
authors.forename: Andrew
authors.initials: A
authors.lastname: Sharabi
authors.affiliation: Dana-Farber Cancer Institute and Harvard Medical School , Boston, MA, USA.
authors.forename: Arlene H
authors.initials: AH
authors.lastname: Sharpe
authors.affiliation: Memorial Sloan Kettering Cancer Center, New York, NY, USA; Weill Medical College of Cornell University, New York, NY, USA.
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authors.lastname: Solit
authors.affiliation: SWOG Statistical Center , Fred Hutchinson Cancer Research Center, Seattle, WA, USA.
authors.forename: Catherine M
authors.initials: CM
authors.lastname: Tangen
authors.affiliation: Baylor College of Medicine , Houston, TX, USA.
authors.forename: Abdul Tawab
authors.initials: AT
authors.lastname: Amiri
authors.affiliation: Dana-Farber Cancer Institute and Harvard Medical School , Boston, MA, USA.
authors.forename: Eliezer
authors.initials: E
authors.lastname: Van Allen
authors.affiliation: The Emmes Corporation , Rockville, MD, USA.
authors.forename: Pamela J
authors.initials: PJ
authors.lastname: West
authors.affiliation: Department of Urology, Radboud UMC, Nijmegen, The Netherlands.
authors.forename: J A
authors.initials: JA
authors.lastname: Witjes
authors.affiliation: Bladder Cancer Advocacy Network , Bethesda, MD, USA.
authors.forename: Diane Zipursky
authors.initials: DZ
authors.lastname: Quale
keywords: Non-muscle invasive bladder cancer; immunotherapy; radiation therapy; targeted therapy; trial design