title: Recommendations from the iSBTc-SITC/FDA/NCI Workshop on Immunotherapy Biomarkers.
abstract: OBJECTIVE
  To facilitate development of innovative immunotherapy approaches, especially for treatment concepts exploiting the potential benefits of personalized therapy, there is a need to develop and validate tools to identify patients who can benefit from immunotherapy. Despite substantial effort, we do not yet know which parameters of antitumor immunity to measure and which assays are optimal for those measurements.
  
  
  METHODS
  The iSBTc-SITC (International Society for Biological Therapy of Cancer-Society for Immunotherapy of Cancer), FDA (Food and Drug Administration), and NCI (National Cancer Institute) partnered to address these issues for immunotherapy of cancer. Here, we review the major challenges, give examples of approaches and solutions, and present our recommendations.
  
  
  CONCLUSIONS
  Although specific immune parameters and assays are not yet validated, we recommend following standardized (accurate, precise, and reproducible) protocols and use of functional assays for the primary immunologic readouts of a trial; consideration of central laboratories for immune monitoring of large, multi-institutional trials; and standardized testing of several phenotypic and functional potential potency assays specific to any cellular product. When reporting results, the full QA (quality assessment)/QC (quality control) should be conducted and selected examples of truly representative raw data and assay performance characteristics should be included. Finally, to promote broader analysis of multiple aspects of immunity, and gather data on variability, we recommend that in addition to cells and serum, RNA and DNA samples be banked (under standardized conditions) for later testing. We also recommend that sufficient blood be drawn to allow for planned testing of the primary hypothesis being addressed in the trial, and that additional baseline and posttreatment blood is banked for testing novel hypotheses (or generating new hypotheses) that arise in the field.
authors.affiliation: Department of Medicine, University of Pittsburgh, Graduate School of Public Health, University of Pittsburgh, Pittsburgh, Pennsylvania 15213, USA.
authors.forename: Lisa H
authors.initials: LH
authors.lastname: Butterfield
authors.forename: A Karolina
authors.initials: AK
authors.lastname: Palucka
authors.forename: Cedrik M
authors.initials: CM
authors.lastname: Britten
authors.forename: Madhav V
authors.initials: MV
authors.lastname: Dhodapkar
authors.forename: Leif
authors.initials: L
authors.lastname: Håkansson
authors.forename: Sylvia
authors.initials: S
authors.lastname: Janetzki
authors.forename: Yutaka
authors.initials: Y
authors.lastname: Kawakami
authors.forename: Thomas-Oliver
authors.initials: TO
authors.lastname: Kleen
authors.forename: Peter P
authors.initials: PP
authors.lastname: Lee
authors.forename: Cristina
authors.initials: C
authors.lastname: Maccalli
authors.forename: Holden T
authors.initials: HT
authors.lastname: Maecker
authors.forename: Vernon C
authors.initials: VC
authors.lastname: Maino
authors.forename: Michele
authors.initials: M
authors.lastname: Maio
authors.forename: Anatoli
authors.initials: A
authors.lastname: Malyguine
authors.forename: Giuseppe
authors.initials: G
authors.lastname: Masucci
authors.forename: Graham
authors.initials: G
authors.lastname: Pawelec
authors.forename: Douglas M
authors.initials: DM
authors.lastname: Potter
authors.forename: Licia
authors.initials: L
authors.lastname: Rivoltini
authors.forename: Lupe G
authors.initials: LG
authors.lastname: Salazar
authors.forename: Dolores J
authors.initials: DJ
authors.lastname: Schendel
authors.forename: Craig L
authors.initials: CL
authors.lastname: Slingluff
authors.forename: Wenru
authors.initials: W
authors.lastname: Song
authors.forename: David F
authors.initials: DF
authors.lastname: Stroncek
authors.forename: Hideaki
authors.initials: H
authors.lastname: Tahara
authors.forename: Magdalena
authors.initials: M
authors.lastname: Thurin
authors.forename: Giorgio
authors.initials: G
authors.lastname: Trinchieri
authors.forename: Sjoerd H
authors.initials: SH
authors.lastname: van Der Burg
authors.forename: Theresa L
authors.initials: TL
authors.lastname: Whiteside
authors.forename: Jon M
authors.initials: JM
authors.lastname: Wigginton
authors.forename: Francesco
authors.initials: F
authors.lastname: Marincola
authors.forename: Samir
authors.initials: S
authors.lastname: Khleif
authors.forename: Bernard A
authors.initials: BA
authors.lastname: Fox
authors.forename: Mary L
authors.initials: ML
authors.lastname: Disis
mesh_terms: D014408:Biomarkers, Tumor / Q000032:analysis*; D003245:Consensus Statements as Topic; D006287:Health Planning Guidelines; D006801:Humans; D007167:Immunotherapy / Q000331:legislation & jurisprudence / Q000379:methods*; D007390:International Agencies / Q000331:legislation & jurisprudence; D008495:Medical Oncology / Q000331:legislation & jurisprudence / Q000379:methods / Q000458:organization & administration; D054547:National Cancer Institute (U.S.) / Q000331:legislation & jurisprudence; D009369:Neoplasms / Q000175:diagnosis* / Q000628:therapy*; D017410:Practice Guidelines as Topic; D012955:Societies, Medical / Q000331:legislation & jurisprudence / Q000458:organization & administration; D014481:United States; D014486:United States Food and Drug Administration / Q000331:legislation & jurisprudence
chemical_list: D014408:Biomarkers, Tumor