Summary and recommendations from the national cancer institute's clinical trials planning meeting on novel therapeutics for non-muscle invasive bladder cancer

Seth P. Lerner, Dean F. Bajorin, Colin P. Dinney, Jason A. Efstathiou, Susan Groshen, Noah M. Hahn, Donna Hansel, David Kwiatkowski, Michael O'Donnell, Jonathan Rosenberg, Robert Svatek, Jeffrey S. Abrams, Hikmat Al-Ahmadie, Andrea B. Apolo, Joaquim Bellmunt, Margaret Callahan, Eugene K. Cha, Charles Drake, Jonathan Jarow, Ashish KamatWilliam Kim, Margaret Knowles, Bhupinder Mann, Luigi Marchionni, David McConkey, Lisa McShane, Nilsa Ramirez, Andrew Sharabi, Arlene H. Sharpe, David Solit, Catherine M. Tangen, Abdul Tawab Amiri, Eliezer Van Allen, Pamela J. West, J. A. Witjes, Diane Zipursky Quale

Research output: Contribution to journalArticle

17 Citations (Scopus)

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 defi ning 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).

Original languageEnglish (US)
Pages (from-to)165-201
Number of pages37
JournalBladder Cancer
Volume2
Issue number2
DOIs
StatePublished - Jan 1 2016

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National Cancer Institute (U.S.)
Urinary Bladder Neoplasms
Clinical Trials
Mycobacterium bovis
Therapeutics
valrubicin
Neoplasms
Urinary Bladder
Urinary Bladder Diseases
Molecular Targeted Therapy
Urologic Neoplasms
Drug Industry
Proxy
Advisory Committees
Research
Immunotherapy
Radiotherapy
Education

Keywords

  • Immunotherapy
  • Non-muscle invasive bladder cancer
  • Radiation therapy
  • Targeted therapy
  • Trial design

ASJC Scopus subject areas

  • Oncology
  • Urology

Cite this

Summary and recommendations from the national cancer institute's clinical trials planning meeting on novel therapeutics for non-muscle invasive bladder cancer. / Lerner, Seth P.; Bajorin, Dean F.; Dinney, Colin P.; Efstathiou, Jason A.; Groshen, Susan; Hahn, Noah M.; Hansel, Donna; Kwiatkowski, David; O'Donnell, Michael; Rosenberg, Jonathan; Svatek, Robert; Abrams, Jeffrey S.; Al-Ahmadie, Hikmat; Apolo, Andrea B.; Bellmunt, Joaquim; Callahan, Margaret; Cha, Eugene K.; Drake, Charles; Jarow, Jonathan; Kamat, Ashish; Kim, William; Knowles, Margaret; Mann, Bhupinder; Marchionni, Luigi; McConkey, David; McShane, Lisa; Ramirez, Nilsa; Sharabi, Andrew; Sharpe, Arlene H.; Solit, David; Tangen, Catherine M.; Amiri, Abdul Tawab; Van Allen, Eliezer; West, Pamela J.; Witjes, J. A.; Quale, Diane Zipursky.

In: Bladder Cancer, Vol. 2, No. 2, 01.01.2016, p. 165-201.

Research output: Contribution to journalArticle

Lerner, SP, Bajorin, DF, Dinney, CP, Efstathiou, JA, Groshen, S, Hahn, NM, Hansel, D, Kwiatkowski, D, O'Donnell, M, Rosenberg, J, Svatek, R, Abrams, JS, Al-Ahmadie, H, Apolo, AB, Bellmunt, J, Callahan, M, Cha, EK, Drake, C, Jarow, J, Kamat, A, Kim, W, Knowles, M, Mann, B, Marchionni, L, McConkey, D, McShane, L, Ramirez, N, Sharabi, A, Sharpe, AH, Solit, D, Tangen, CM, Amiri, AT, Van Allen, E, West, PJ, Witjes, JA & Quale, DZ 2016, 'Summary and recommendations from the national cancer institute's clinical trials planning meeting on novel therapeutics for non-muscle invasive bladder cancer', Bladder Cancer, vol. 2, no. 2, pp. 165-201. https://doi.org/10.3233/BLC-160053
Lerner, Seth P. ; Bajorin, Dean F. ; Dinney, Colin P. ; Efstathiou, Jason A. ; Groshen, Susan ; Hahn, Noah M. ; Hansel, Donna ; Kwiatkowski, David ; O'Donnell, Michael ; Rosenberg, Jonathan ; Svatek, Robert ; Abrams, Jeffrey S. ; Al-Ahmadie, Hikmat ; Apolo, Andrea B. ; Bellmunt, Joaquim ; Callahan, Margaret ; Cha, Eugene K. ; Drake, Charles ; Jarow, Jonathan ; Kamat, Ashish ; Kim, William ; Knowles, Margaret ; Mann, Bhupinder ; Marchionni, Luigi ; McConkey, David ; McShane, Lisa ; Ramirez, Nilsa ; Sharabi, Andrew ; Sharpe, Arlene H. ; Solit, David ; Tangen, Catherine M. ; Amiri, Abdul Tawab ; Van Allen, Eliezer ; West, Pamela J. ; Witjes, J. A. ; Quale, Diane Zipursky. / Summary and recommendations from the national cancer institute's clinical trials planning meeting on novel therapeutics for non-muscle invasive bladder cancer. In: Bladder Cancer. 2016 ; Vol. 2, No. 2. pp. 165-201.
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