# Humanised mice with an immune system matched to the tumour donor

Source: https://onco.cc/ideas/idea-bio1-immune-matched-humanised-mice/  
OnCo record `idea-bio1-immune-matched-humanised-mice` (Idea). Data CC BY-NC 4.0, attribute "Data from OnCo (onco.cc)"; commercial use needs a licence.

## TL;DR

Most cancer drugs are tested in mice with no immune system, then given to people who have one. Mice carrying the same patient's immune cells and tumour would be a fairer test.

## Summary

Standard PDX models use immunodeficient mice, which cannot evaluate immunotherapy or immune-mediated toxicity. Humanised immune system mice reconstituted with HLA-matched or autologous haematopoietic cells, ideally with human cytokine knock-in backgrounds to support myeloid development, allow tumour and immune system from the same donor. Cost and reproducibility have kept them niche.

## Fields

- Kind: Idea
- Last checked: 2026-09-08
- Hypothesis: Autologous or HLA-matched humanised models reproduce the individual patient's response and immune-related toxicity to checkpoint blockade better than chance, measured against the patient's actual outcome.
- Rationale: Immune context determines the outcome of most modern oncology drugs; syngeneic mouse tumours are immunologically unlike human cancers and have repeatedly over-predicted benefit.
- Proposed test: Build 40 paired autologous models from patients starting checkpoint therapy and score concordance between model response and patient outcome, pre-registering the concordance threshold.
- Maturity: preclinical-evidence
- Actor: research

## Sources

- Bottleneck evidence (Lab models that fail to predict what happens in patients): Wong, Siah & Lo, Estimation of clinical trial success rates (Biostatistics 2019): https://doi.org/10.1093/biostatistics/kxx069

## Connected records

- technologies: [Immune checkpoint inhibitors](https://onco.cc/technologies/checkpoint-inhibitor/), [Patient-derived xenografts](https://onco.cc/technologies/pdx-models/)
- companies: [Champions Oncology](https://onco.cc/companies/champions-oncology/)
- bottlenecks: [Lab models that fail to predict what happens in patients](https://onco.cc/bottlenecks/b-preclinical-models/), [No one can predict who responds to immunotherapy](https://onco.cc/bottlenecks/b-immunotherapy-response/)
- key papers: [Estimation of clinical trial success rates and related parameters](https://onco.cc/key-papers/paper-wong-biostatistics/)

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