title: Biomarker-based adaptive trials for patients with glioblastoma--lessons from I-SPY 2.
abstract: The traditional clinical trials infrastructure may not be ideally suited to evaluate the numerous therapeutic hypotheses that result from the increasing number of available targeted agents combined with the various methodologies to molecularly subclassify patients with glioblastoma. Additionally, results from smaller screening studies are rarely translated to successful larger confirmatory studies, potentially related to a lack of efficient control arms or the use of unvalidated surrogate endpoints. Streamlining clinical trials and providing a flexible infrastructure for biomarker development is clearly needed for patients with glioblastoma. The experience developing and implementing the I-SPY studies in breast cancer may serve as a guide to developing such trials in neuro-oncology.
authors.affiliation: Department of Radiation Oncology, Dana-Farber/Brigham and Women’s Cancer Center, Harvard Medical School, Boston, MA, USA. bmalexander@lroc.harvard.edu
authors.forename: Brian M
authors.initials: BM
authors.lastname: Alexander
authors.forename: Patrick Y
authors.initials: PY
authors.lastname: Wen
authors.forename: Lorenzo
authors.initials: L
authors.lastname: Trippa
authors.forename: David A
authors.initials: DA
authors.lastname: Reardon
authors.forename: Wai-Kwan Alfred
authors.initials: WK
authors.lastname: Yung
authors.forename: Giovanni
authors.initials: G
authors.lastname: Parmigiani
authors.forename: Donald A
authors.initials: DA
authors.lastname: Berry
mesh_terms: D015415:Biomarkers / Q000032:analysis*; D001932:Brain Neoplasms / Q000175:diagnosis / Q000628:therapy*; D002986:Clinical Trials as Topic; D003952:Diagnostic Imaging; D005909:Glioblastoma / Q000175:diagnosis / Q000628:therapy*; D006801:Humans; D012107:Research Design
chemical_list: D015415:Biomarkers
keywords: Bayesian; adaptive; biomarker; clinical trials; glioblastoma