Humanized mice for immune checkpoint blockade in human solid tumors

Henry Yip1, Carl Haupt1, Grace Maresh1

  • 1UQ-Ochsner Clinical School, Institute for Translational Research, Ochsner Clinic Foundation New Orleans, LA, USA.

Immunotherapy, specifically research involving immune checkpoint blockers (ICBs), has become a popular trend in anticancer research over the last three years. Due to the difficulties and often poor translation of results from in-vitro models, in-vivo models have become more relevant than ever. With the discovery of NOD, Prkdc , and Il2rγ mutations, patient-derived xenograft (PDX) mouse models were developed, providing an ideal environment for ICBs testing. By implanting a PDX with either CD34+ or peripheral blood mononuclear cells, we can create a human immune system capable of mounting a response against tumor burden. These animal models are currently being used to study molecular mechanisms, test drug efficacy, and trial drug combinations. Others have found use for these humanized mouse models as surrogates to represent otherwise uncommon diseases. Limitations remain with regards to what the models are capable of, but in the short amount of time between the development of these models and heightened interest in ICBs, these mice have already shown utility for future developments in the field of immunotherapy.