@article{f4552e58cd344a72a00a23f8fd8de585,
title = "Significance and implications of FDA approval of pembrolizumab for biomarker-defined disease",
abstract = "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.",
keywords = "Biomarker, DNA mismatch repair, Immunotherapy, Microsatellite instability, Pembrolizumab",
author = "Boyiadzis, {Michael M.} and Kirkwood, {John M.} and Marshall, {John L.} and Pritchard, {Colin C.} and Azad, {Nilofer S.} and Gulley, {James L.}",
note = "Funding Information: ABL: Abelson; ALL: Acute lymphoblastic leukemia; AML: Acute myeloid leukemia; ASCO: American Society of Clinical Oncology; BCR: Breakpoint cluster region; CLIA: Clinical Laboratory Improvement Amendments; CLL: Chronic lymphocytic leukemia; CML: Chronic myeloid leukemia; CRC: Colorectal cancer; dMMR: DNA mismatch repair-deficient; EORTC: European Organization for the Research and Treatment of Cancer; FDA: U.S. Food and Drug Administration; IFN: Interferon; IHC: Immunohistochemistry; MHC: Major histocompatibility complex; MSI: Microsatellite instability; MSI-H: Microsatellite instability-high; NCCN: National Comprehensive Cancer Network; NGS: Next-generation sequencing; NSCLC: Non-Small cell lung cancer; ORR: Objective response rate; PCR: Polymerase chain reaction; PD-1: Programmed cell death-protein; PD-L1: Programmed death-ligand 1; RCC: Renal cell carcinoma Funding Information: 1University of Pittsburgh, Pittsburgh, PA, USA. 2University of Pittsburgh Hillman Cancer Center, Pittsburgh, PA, USA. 3Lombardi Cancer Research Center, Washington, D. C., USA. 4University of Washington, Seattle, WA, USA. 5Johns Hopkins University, Baltimore, MD, USA. 6Genitourinary Malignancies Branch, Center for Cancer Research, National Cancer Institute, National Institutes of Health, Bethesda, MD, USA. Publisher Copyright: {\textcopyright} 2018 The Author(s).",
year = "2018",
month = may,
day = "14",
doi = "10.1186/s40425-018-0342-x",
language = "English (US)",
volume = "6",
journal = "Journal for immunotherapy of cancer",
issn = "2051-1426",
publisher = "BioMed Central",
number = "1",
}