Antihuman CD117 chimeric antigen receptor (CAR) T cells may be effective in treating CD117-positive acute myeloid leukemia (AML), according to study results published in Leukemia.

AML is derived from hematopoietic stem and progenitor cells (HSPCs), which can be targeted using anti­-pan-B-cell, -pan-plasma-cell, or -pan-T-cell antigen therapies; however, targeted therapies are associated with evolved resistance. Therefore, new therapies are needed to improve survival rates in this patient setting.

CAR T cells have shown promise in a number of hematologic settings, most notably in B-cell and plasma cell malignancies. CD117, the cognate receptor for stem cell factor expressed on most primary human AML cells and HSPCs, may be an effective target for CAR-T therapy. Researchers evaluated the potential efficacy of anti-CD117 CAR-T cell therapy for AML treatment in vitro and in vivo.

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The researchers designed anti-CD117 CAR T cells using samples from healthy donors and patients with AML. In the in vitro setting, the engineered CAR T cells effectively targeted CD117-expressing healthy and AML cells. In mice xenografted with CD117-positive AML, the CAR T cells successfully eliminated the disease entirely.

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“In summary, we here established anti-human CD117 CAR T-cells as highly effective immunotherapeutic modality for AML and envision that this approach can increase the curative potential of preconditioning regimens prior to allogeneic HSCT,” the authors concluded.


Myburgh R, Kiefer JD, Russkamp NF, et al. Anti-human CD117 CAR T-cells efficiently eliminate healthy and malignant CD117-expressing hematopoietic cells [published online May 1, 2020]. Leukemia. doi: 10.1038/s41375-020-0818-9