RT Journal Article
SR Electronic
T1 Significance and implications of FDA approval of pembrolizumab for biomarker-defined disease
JF Journal for ImmunoTherapy of Cancer
JO J Immunother Cancer
FD BMJ Publishing Group Ltd
SP 35
DO 10.1186/s40425-018-0342-x
VO 6
IS 1
A1 Michael M. Boyiadzis
A1 John M. Kirkwood
A1 John L. Marshall
A1 Colin C. Pritchard
A1 Nilofer S. Azad
A1 James L. Gulley
YR 2018
UL http://jitc.bmj.com/content/6/1/35.abstract
AB The U.S. Food and Drug Administration (FDA) recently approved pembrolizumab, an anti- programmed cell death protein 1 cancer immunotherapeutic, for use in advanced solid tumors in patients with the microsatellite-high/DNA mismatch repair-deficient biomarker. This is the first example of a tissue-agnostic FDA approval of a treatment based on a patient’s tumor biomarker status, rather than on tumor histology. Here we discuss key issues and implications arising from the biomarker-based disease classification implied by this historic approval.Abbreviations:ABLAbelsonALLAcute lymphoblastic leukemiaAMLAcute myeloid leukemiaASCOAmerican Society of Clinical OncologyBCRBreakpoint cluster regionCLIAClinical Laboratory Improvement AmendmentsCLLChronic lymphocytic leukemiaCMLChronic myeloid leukemiaCRCColorectal cancerdMMRDNA mismatch repair-deficientEORTCEuropean Organization for the Research and Treatment of CancerFDAU.S. Food and Drug AdministrationIFNInterferonIHCImmunohistochemistryMHCMajor histocompatibility complexMSIMicrosatellite instabilityMSI-HMicrosatellite instability-highNCCNNational Comprehensive Cancer NetworkNGSNext-generation sequencingNSCLCNon-Small cell lung cancerORRObjective response ratePCRPolymerase chain reactionPD-1Programmed cell death-proteinPD-L1Programmed death-ligand 1RCCRenal cell carcinoma