An autologous humanized patient-derived-xenograft platform to evaluate immunotherapy in ovarian cancer

Gynecol Oncol. 2020 Jan;156(1):222-232. doi: 10.1016/j.ygyno.2019.10.011. Epub 2019 Dec 7.

Abstract

Objective: The aim of this study was to "humanize" ovarian cancer patient-derived xenograft (PDX) models by autologous transfer of patient-matched tumor infiltrating lymphocytes (TILs) to evaluate immunotherapies.

Methods: Orthotopic high-grade serous ovarian cancer (HGSOC) PDX models were established from three patient donors. Models were molecularly and histologically validated by immunohistochemistry. TILs were expanded from donor tumors using a rapid expansion protocol. Ex vivo TIL and tumor co-cultures were performed to validate TIL reactivity against patient-matched autologous tumor cells. Expression of TIL activation markers and cytokine secretion was quantitated by flow cytometry and ELISA. As proof of concept, the efficacy of anti-PD-1 monotherapy was tested in autologous TIL/tumor HGSOC PDX models.

Results: Evaluation of T-cell activation in autologous TIL/tumor co-cultures resulted in an increase in HLA-dependent IFNγ production and T-cell activation. In response to increased IFNγ production, tumor cell expression of PD-L1 was increased. Addition of anti-PD-1 antibody to TIL/tumor co-cultures increased autologous tumor lysis in a CCNE1 amplified model. Orthotopic HGSOC PDX models from parallel patient-matched tumors maintained their original morphology and molecular marker profile. Autologous tumor-reactive TIL administration in patient-matched PDX models resulted in reduced tumor burden and increased survival, in groups that also received anti-PD-1 therapy.

Conclusions: This study validates a novel, clinically relevant model system for in vivo testing of immunomodulating therapeutic strategies for ovarian cancer, and provides a unique platform for assessing patient-specific T-cell response to immunotherapy.

Keywords: Anti-PD-1; Ovarian cancer; Patient derived xenograft; Tumor infiltrating lymphocytes.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Animals
  • Female
  • Humans
  • Immunotherapy / methods*
  • Lymphocyte Activation
  • Lymphocytes, Tumor-Infiltrating / immunology*
  • Lymphocytes, Tumor-Infiltrating / transplantation
  • Mice
  • Mice, Inbred NOD
  • Mice, SCID
  • Neoplasm Transplantation / methods
  • Ovarian Neoplasms / immunology*
  • Ovarian Neoplasms / pathology
  • Ovarian Neoplasms / therapy*
  • Programmed Cell Death 1 Receptor / antagonists & inhibitors
  • Programmed Cell Death 1 Receptor / immunology
  • T-Lymphocytes / immunology
  • Xenograft Model Antitumor Assays / methods*

Substances

  • PDCD1 protein, human
  • Programmed Cell Death 1 Receptor