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Updated: Jun 12, 2026
Intra-Peritoneal Transplantation for Generating Acute Myeloid Leukemia in Mice
Published on: January 6, 2023
Kit inhibitor APcK110 extends survival in an AML xenograft mouse model
Stefan Faderl1, Carlos Bueso-Ramos, Zhiming Liu
1Department of Leukemia, Unit 428, MD Anderson Cancer Center, The University of Texas, 1515 Holcombe Blvd., Houston, TX 77030, USA. sfaderl@mdanderson.org
Background:
Constitutive activation of kit contributes to pathogenesis of acute myeloid leukemia (AML) and targeting Kit may be of therapeutic benefit. APcK110, a novel inhibitor of Kit, has potent proapoptotic and antiproliferative activity in AML cell lines and primary AML samples. Here we extend our studies to the activity of APcK110 in a xenograft mouse model.
Methods:
After sub-lethal whole body radiation, OCI/AML3 cells were injected intravenously in NOD-SCID mice. Ten days later, either APcK110 or phosphate buffered saline (PBS) was injected intraperitoneally every other day. Kaplan-Meier estimates were used to calculate survival.
Results:
We show that 1) all mice injected with OCI/AML3 cells developed a clinical and histological picture consistent with myelomonocytic AML; and 2) survival of APcK110-treated mice was significantly longer compared with mice injected with PBS (p = .02).
Conclusions:
APcK110 is a novel kit kinase inhibitor with anti-AML activity in vitro and in vivo. Further evaluation in toxicology and clinical studies is warranted.
Insights
APcK110, a novel Kit inhibitor, demonstrated significant anti-leukemia activity in a mouse model of acute myeloid leukemia (AML). This study shows APcK110 extends survival in vivo, supporting its therapeutic potential for AML patients.
Area of Science:
- Oncology
- Hematology
- Pharmacology
Background:
- Constitutive activation of the Kit kinase is implicated in acute myeloid leukemia (AML) pathogenesis.
- Targeting Kit presents a potential therapeutic strategy for AML.
- APcK110, a novel Kit inhibitor, exhibits proapoptotic and antiproliferative effects in AML models.
Purpose of the Study:
- To evaluate the in vivo efficacy of APcK110 in a xenograft mouse model of AML.
- To assess the impact of APcK110 on survival in a preclinical AML model.
Main Methods:
- NOD-SCID mice were engrafted with OCI/AML3 cells following sub-lethal irradiation.
- APcK110 or phosphate-buffered saline (PBS) was administered intraperitoneally every other day.
- Survival analysis was performed using Kaplan-Meier estimates.
Main Results:
- Mice developed myelomonocytic AML, confirmed by clinical and histological findings.
- APcK110 treatment significantly prolonged survival compared to PBS control (p = .02).
Conclusions:
- APcK110 demonstrates anti-AML activity in vivo, complementing its in vitro findings.
- Further investigation, including toxicology and clinical trials, is warranted for APcK110.
- APcK110 represents a promising novel Kit kinase inhibitor for AML therapy.
