2ⁿᵈ Edition of the Cancer R&D World Conference 2026

Abstract

Immune cells within the tumor microenvironment are increasingly recognized as key targets for cancer therapy. However, findings from conventional animal models often fail to translate to humans due to fundamental interspecies differences. This underscores the growing need for humanized mouse models that can more accurately support the preclinical development of immunotherapeutic strategies.

To recreate the human immune system in immunodeficient mice, researchers typically use human peripheral blood mononuclear cells (PBMCs) or hematopoietic stem cells (HSCs). These “humanized” mouse models have been developed to investigate the complex interactions of the human immune system in disease contexts where syngeneic mouse models are insufficient. Humanized immunodeficient mice, engrafted with both human immune cells and human tumor cells, offer a relevant platform for evaluating a wide range of oncology therapies, including adoptive cell transfer, immune checkpoint inhibitors, and oncolytic viruses.

Tumor growth dynamics in humanized mice will be presented using both subcutaneous models (including cell lines and patient-derived xenografts) and disseminated intravenous models. Randomization criteria were defined to account for both tumor and immune cell variables. Key readouts for assessing immune cell modulation and associated antitumor efficacy will be detailed.

The poster will feature illustrative data and case studies that showcase the platform’s capabilities and its application in preclinical evaluation of immune-oncology drug candidates.

PROPOSITION CORE

Immune cells within the tumor microenvironment are increasingly recognized as critical targets for cancer therapy. However, findings from conventional animal models often fail to translate effectively to humans due to fundamental interspecies differences. This limitation has driven the development of humanized mouse models capable of more accurately recapitulating human immune biology and supporting the preclinical evaluation of emerging immunotherapeutic strategies.

To recreate a functional human immune system in immunodeficient mice, researchers commonly engraft either human peripheral blood mononuclear cells (PBMCs) or hematopoietic stem cells (HSCs). These humanized mouse models enable the study of human immune cell development, activation, and tumor–immune interactions in vivo, addressing key gaps that traditional syngeneic models cannot. When coupled with the implantation of human tumor cell lines or patient-derived xenografts, humanized mice provide a uniquely relevant platform for assessing a broad spectrum of oncological therapeutic strategies, including adoptive immune cell therapies, immune checkpoint inhibitors, and oncolytic viruses.

In this work, we present tumor growth dynamics and immune profiling outcomes across both subcutaneous and disseminated intravenous humanized tumor models. Randomization strategies incorporating tumor burden and immune cell engraftment are outlined to enhance experimental robustness. Key readouts capturing immune modulation and antitumor efficacy are detailed.

Illustrative datasets and case studies demonstrate the versatility and translational value of these platforms for preclinical assessment of immune-oncology drug candidates. Overall, the findings highlight humanized mice as a powerful bridge between discovery research and clinical development.