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

被引：44

作者：

Lerner, Seth P. ^{[1
]}

Bajorin, Dean F. ^{[2
,3
]}

Dinney, Colin P. ^{[4
]}

Efstathiou, Jason A. ^{[5
]}

Groshen, Susan ^{[6
]}

Hahn, Noah M. ^{[7
]}

Hansel, Donna ^{[8
]}

Kwiatkowski, David ^{[9
]}

O'Donnell, Michael ^{[10
]}

Rosenberg, Jonathan ^{[2
,3
]}

Svatek, Robert ^{[11
]}

Abrams, Jeffrey S. ^{[12
]}

Al-Ahmadie, Hikmat ^{[2
]}

Apolo, Andrea B. ^{[13
]}

Bellmunt, Joaquim ^{[14
,15
]}

Callahan, Margaret ^{[2
,3
]}

Cha, Eugene K. ^{[2
]}

Drake, Charles ^{[7
]}

Jarow, Jonathan ^{[16
]}

Kamat, Ashish ^{[4
]}

Kim, William ^{[17
]}

Knowles, Margaret ^{[18
]}

Mann, Bhupinder ^{[12
]}

Marchionni, Luigi ^{[7
]}

McConkey, David ^{[4
]}

McShane, Lisa ^{[19
]}

Ramirez, Nilsa ^{[20
]}

Sharabi, Andrew ^{[6
,7
]}

Sharpe, Arlene H. ^{[14
,15
]}

Solit, David ^{[2
,3
]}

Tangen, Catherine M. ^{[21
]}

Amiri, Abdul Tawab ^{[22
]}

Van Allen, Eliezer ^{[14
,15
]}

West, Pamela J. ^{[23
]}

Witjes, J. A. ^{[24
]}

Quale, Diane Zipursky ^{[25
]}

机构：

[1] Baylor Coll Med, Houston, TX 77030 USA

[2] Mem Sloan Kettering Canc Ctr, 1275 York Ave, New York, NY 10021 USA

[3] Cornell Univ, Weill Med Coll, New York, NY 10021 USA

[4] Univ Texas MD Anderson Canc Ctr, Houston, TX 77030 USA

[5] Harvard Med Sch, Massachusetts Gen Hosp, Boston, MA USA

[6] Univ Southern Calif, USC Norris Comprehens Canc Ctr, Los Angeles, CA USA

[7] Johns Hopkins Sidney Kimmel Comprehens Canc Ctr, Baltimore, MD USA

[8] Univ Calif San Diego, La Jolla, CA 92093 USA

[9] Harvard Med Sch, Brigham & Womens Hosp, Boston, MA USA

[10] Univ Iowa, Iowa City, IA 52242 USA

[11] UT Hlth Sci Ctr San Antonio, San Antonio, TX USA

[12] NCI, Canc Therapy Evaluat Program, Div Canc Treatment & Diag, NIH, Bethesda, MD USA

[13] NCI, Genitourinary Malignancies Branch, Ctr Canc Res, NIH, Bethesda, MD 20892 USA

[14] Dana Farber Canc Inst, Boston, MA 02115 USA

[15] Harvard Med Sch, Boston, MA 02115 USA

[16] US FDA, Off Hematol & Oncol Prod, Silver Spring, MD USA

[17] Univ N Carolina, Lineberger Comprehens Canc Ctr, Chapel Hill, NC 27515 USA

[18] Univ Leeds, Leeds Inst Canc & Pathol, Leeds, W Yorkshire, England

[19] NCI, Biometr Res Branch, Div Canc Treatment & Diag, Bethesda, MD 20892 USA

[20] Nationwide Childrens Hosp, Res Inst, Columbus, OH USA

[21] Fred Hutchinson Canc Res Ctr, SWOG Stat Ctr, 1124 Columbia St, Seattle, WA 98104 USA

[22] NCI, Coordinating Ctr Clin Trials, Bethesda, MD 20892 USA

[23] Emmes Corp, Rockville, MD USA

[24] Radboud UMC, Dept Urol, Nijmegen, Netherlands

[25] Bladder Canc Advocacy Network, Bethesda, MD USA

来源：

BLADDER CANCER | 2016年 / 2卷 / 02期

关键词：

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

D O I：

10.3233/BLC-160053

中图分类号：

R73 [肿瘤学];

学科分类号：

100214 ;

摘要：

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 inununomodulatory agents in patients who recur after induction BCG (BCG failure).

引用

页码：165 / 201

页数：37

共 126 条

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