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 Development of an HLA-matched humanized immune system mouse model with primary AML patient samples for immunotherapy testing  

Acute Myeloid Leukemia (AML) remains one of the most lethal hematologic malignancies, with limited treatment options and a high relapse rate. Traditional preclinical models often lack the genetic complexity and immune microenvironment reflective of real-world disease, hindering the development of effective immunotherapies.

Champions Oncology, in collaboration with Taconic Biosciences, developed a novel in vivo humanized AML model by co-engrafting primary, never-passaged AML patient-derived xenografts (PDXs) and HLA-matched CD34+ hematopoietic stem cells (HSCs) into NOG-EXL mice, which support multilineage immune system reconstitution.

• This platform preserves AML’s genetic and clonal heterogeneity, leveraging a library of over 50 deeply characterized patient-derived models. AML progression and immune cell dynamics were longitudinally tracked using custom flow cytometry panels and multi-omic characterization.

• Flow cytometry confirmed successful engraftment of both human immune cells (T cells, B cells, NK cells, M2 macrophages) and AML subpopulations (progenitors, blasts, monoblasts), reflecting the immunosuppressive bone marrow microenvironment seen in patients.

• The humanized AML model enables robust testing of immuno-oncology therapies—including checkpoint inhibitors and CAR-T cells—in a clinically relevant context, setting a new standard for evaluating immune-based treatments in AML.