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A Human Peripheral Blood Mononuclear Cell (PBMC) Engrafted Humanized Xenograft Model for Translational Immuno-oncology (I-O) Research
Published on: August 15, 2019
Human AML cells in NOD/SCID mice: engraftment potential and gene expression
R Lumkul1, N-C Gorin, M T Malehorn
1Sidney Kimmel Comprehensive Cancer Center, Johns Hopkins University School of Medicine, Baltimore, MD 21231, USA.
Leukemia
|August 30, 2002
Summary
Human acute myeloid leukemia (AML) cells engraft in NOD/SCID mice, providing a renewable source for research. This model supports AML studies without selecting for increased virulence.
Area of Science:
- Hematology
- Immunology
- Cancer Biology
Background:
- Acute myeloid leukemia (AML) is a heterogeneous hematologic malignancy.
- Establishing reliable preclinical models is crucial for AML research.
- Non-obese diabetic/severe combined immunodeficient (NOD/SCID) mice are commonly used for xenograft studies.
Purpose of the Study:
- To evaluate the engraftment efficiency and characteristics of human acute myeloid leukemia (AML) cells in NOD/SCID mice.
- To determine if AML cells from relapsed patients engraft differently than those from initial diagnosis.
- To assess the potential of NOD/SCID mice as a source for renewable AML cells for experimental use.
Main Methods:
- Intravenous injection of varying numbers of human AML cells into irradiated NOD/SCID mice.
- Assessment of engraftment efficiency based on cell dose and patient clinical features.
- Serial transplantation experiments to evaluate virulence.
- cDNA microarray analysis to compare gene expression profiles of engrafted vs. patient-derived AML cells.
Main Results:
- Most human AML cases engrafted efficiently in NOD/SCID mice, even with low cell numbers (10^5).
- AML cases with poor prognostic markers, like FLT3 mutations, showed efficient engraftment.
- Engraftment efficiency did not increase for AML cells from patients at relapse compared to initial diagnosis.
- Serial transplantation did not select for increased AML cell virulence.
- Gene expression analysis revealed minimal changes (approx. 5%) in immunopurified AML cells post-engraftment.
Conclusions:
- NOD/SCID mice effectively support the engraftment of diverse human AML samples.
- Engraftment efficiency correlates with certain clinical features but not relapse status.
- Engrafted AML cells maintain their molecular profile and do not exhibit increased virulence.
- NOD/SCID mice serve as a valuable and renewable source of human AML cells for preclinical research.
