title: Presurgical window of opportunity trial design as a platform for testing anticancer drugs: Pros, cons and a focus on breast cancer.
abstract: The high attrition rate is a major issue in anticancer drug development. Among the alternative trial designs, presurgical window of opportunity trials envision a short course treatment in the time window between diagnostic biopsy and surgery in a moderately-sized patient population. This approach allows testing therapeutics when pre- and post-treatment tumor tissues are available for comprehensive molecular analyses. The emerging evidence may help define the ability of a given agent to modulate its target(s) and help obtain a broader picture of the molecular changes operated by the treatment. The resulting gain may outweigh the potential harms for patients in the early disease setting. Window of opportunity trials have been extensively applied to breast cancer. Overall, a wider use of these trial designs might lead to the identification of potential responders, ineffective drugs or combinations, and ultimately contribute to enhance the efficiency of the clinical developmental process.
authors.affiliation: Division of Medical Oncology 2, Regina Elena National Cancer Institute, Via Elio Chianesi 53, 00144 Rome, Italy; Scientific Direction, Regina Elena National Cancer Institute, Via Elio Chianesi 53, 00144 Rome, Italy. Electronic address: maugeri@ifo.it.
authors.forename: Marcello
authors.initials: M
authors.lastname: Maugeri-Saccà
authors.affiliation: Division of Medical Oncology 2, Regina Elena National Cancer Institute, Via Elio Chianesi 53, 00144 Rome, Italy; Scientific Direction, Regina Elena National Cancer Institute, Via Elio Chianesi 53, 00144 Rome, Italy. Electronic address: maddalena.barba@gmail.com.
authors.forename: Maddalena
authors.initials: M
authors.lastname: Barba
authors.affiliation: Division of Medical Oncology 2, Regina Elena National Cancer Institute, Via Elio Chianesi 53, 00144 Rome, Italy.
authors.forename: Patrizia
authors.initials: P
authors.lastname: Vici
authors.affiliation: Division of Medical Oncology 2, Regina Elena National Cancer Institute, Via Elio Chianesi 53, 00144 Rome, Italy.
authors.forename: Laura
authors.initials: L
authors.lastname: Pizzuti
authors.affiliation: Division of Medical Oncology 2, Regina Elena National Cancer Institute, Via Elio Chianesi 53, 00144 Rome, Italy.
authors.forename: Domenico
authors.initials: D
authors.lastname: Sergi
authors.affiliation: Division of Medical Oncology 2, Regina Elena National Cancer Institute, Via Elio Chianesi 53, 00144 Rome, Italy.
authors.forename: Teresa
authors.initials: T
authors.lastname: Catenaro
authors.affiliation: Division of Medical Oncology 2, Regina Elena National Cancer Institute, Via Elio Chianesi 53, 00144 Rome, Italy.
authors.forename: Luigi
authors.initials: L
authors.lastname: Di Lauro
authors.affiliation: Department of Pathology, Regina Elena National Cancer Institute, Via Elio Chianesi 53, 00144 Rome, Italy.
authors.forename: Marcella
authors.initials: M
authors.lastname: Mottolese
authors.affiliation: Department of Medical Oncology, Campus Bio-Medico, University of Rome, 00128 Rome, Italy.
authors.forename: Daniele
authors.initials: D
authors.lastname: Santini
authors.affiliation: Division of Medical Oncology A, Regina Elena National Cancer Institute, Via Elio Chianesi 53, 00144 Rome, Italy.
authors.forename: Michele
authors.initials: M
authors.lastname: Milella
authors.affiliation: Scientific Direction, Regina Elena National Cancer Institute, Via Elio Chianesi 53, 00144 Rome, Italy.
authors.forename: Ruggero
authors.initials: R
authors.lastname: De Maria
mesh_terms: D000970:Antineoplastic Agents / Q000627:therapeutic use*; D001943:Breast Neoplasms / Q000188:drug therapy* / Q000601:surgery; D002986:Clinical Trials as Topic / Q000379:methods*; D005260:Female; D006801:Humans; D057234:Preoperative Period
chemical_list: D000970:Antineoplastic Agents
keywords: Attrition rate; Breast cancer; Cancer therapeutics; Window of opportunity trials