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Trackable Intratumor Microdosing and Spatial Profiling Provide Early Insights into Activity of Investigational Agents in the Intact Tumor Microenvironment.
Derry, Jonathan M J; Burns, Connor; Frazier, Jason P; Beirne, Emily; Grenley, Marc; DuFort, Christopher C; Killingbeck, Emily; Leon, Michael; Williams, Claire; Gregory, Mark; Houlton, Jeffrey; Clayburgh, Daniel; Swiecicki, Paul; Huszar, Dennis; Berger, Allison; Klinghoffer, Richard A.
Afiliación
  • Derry JMJ; Presage Biosciences, Seattle, Washington.
  • Burns C; Presage Biosciences, Seattle, Washington.
  • Frazier JP; Presage Biosciences, Seattle, Washington.
  • Beirne E; Presage Biosciences, Seattle, Washington.
  • Grenley M; Presage Biosciences, Seattle, Washington.
  • DuFort CC; Presage Biosciences, Seattle, Washington.
  • Killingbeck E; NanoString Technologies, Seattle, Washington.
  • Leon M; NanoString Technologies, Seattle, Washington.
  • Williams C; NanoString Technologies, Seattle, Washington.
  • Gregory M; NanoString Technologies, Seattle, Washington.
  • Houlton J; University of Washington, Seattle, Washington.
  • Clayburgh D; Oregon Health Sciences University, Portland, Oregon.
  • Swiecicki P; University of Michigan, Ann Arbor, Michigan.
  • Huszar D; Takeda Development Center Americas, Inc., Boston, Massachusetts.
  • Berger A; Takeda Development Center Americas, Inc., Boston, Massachusetts.
  • Klinghoffer RA; Presage Biosciences, Seattle, Washington.
Clin Cancer Res ; 29(18): 3813-3825, 2023 09 15.
Article en En | MEDLINE | ID: mdl-37389981
PURPOSE: Cancer drug development is currently limited by a paradigm of preclinical evaluation that does not adequately recapitulate the complexity of the intact human tumor microenvironment (TME). To overcome this, we combined trackable intratumor microdosing (CIVO) with spatial biology readouts to directly assess drug effects in patient tumors in situ. EXPERIMENTAL DESIGN: In a first-of-its-kind phase 0 clinical trial, we explored the effects of an investigational stage SUMOylation-activating enzyme (SAE) inhibitor, subasumstat (TAK-981) in 12 patients with head and neck carcinoma (HNC). Patients scheduled for tumor resection received percutaneous intratumor injections of subasumstat and vehicle control 1 to 4 days before surgery, resulting in spatially localized and graded regions of drug exposure (∼1,000-2,000 µm in diameter). Drug-exposed (n = 214) and unexposed regions (n = 140) were compared by GeoMx Digital Spatial Profiler, with evaluation at single-cell resolution in a subset of these by CosMx Spatial Molecular Imager. RESULTS: Localized regions of subasumstat exposure revealed SUMO pathway inhibition, elevation of type I IFN response, and inhibition of cell cycle across all tumor samples. Single-cell analysis by CosMx demonstrated cell-cycle inhibition specific to the tumor epithelium, and IFN pathway induction commensurate with a TME shift from immune-suppressive to immune-permissive. CONCLUSIONS: Pairing CIVO with spatial profiling enabled detailed investigation of response to subasumstat across a diverse sampling of native and intact TME. We demonstrate that drug mechanism of action can be directly evaluated in a spatially precise manner in the most translationally relevant setting: an in situ human tumor.
Asunto(s)

Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Neoplasias de Cabeza y Cuello / Antineoplásicos Tipo de estudio: Clinical_trials Límite: Humans Idioma: En Revista: Clin Cancer Res Asunto de la revista: NEOPLASIAS Año: 2023 Tipo del documento: Article Pais de publicación: Estados Unidos

Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Neoplasias de Cabeza y Cuello / Antineoplásicos Tipo de estudio: Clinical_trials Límite: Humans Idioma: En Revista: Clin Cancer Res Asunto de la revista: NEOPLASIAS Año: 2023 Tipo del documento: Article Pais de publicación: Estados Unidos