Chimeric antigen receptors against CD33/CD123 antigens efficiently target primary acute myeloid leukemia cells in vivo

I Pizzitola, F Anjos-Afonso, K Rouault-Pierre… - Leukemia, 2014 - nature.com
I Pizzitola, F Anjos-Afonso, K Rouault-Pierre, F Lassailly, S Tettamanti, O Spinelli, A Biondi
Leukemia, 2014nature.com
As significant numbers of acute myeloid leukemia (AML) patients are still refractory to
conventional therapies or experience relapse, immunotherapy using T cells expressing
chimeric antigen receptors (CARs) might represent a valid treatment option. AML cells
frequently overexpress the myeloid antigens CD33 and CD123, for which specific CARs can
be generated. However, CD33 is also expressed on normal hematopoietic stem/progenitor
cells (HSPCs), and its targeting could potentially impair normal hematopoiesis. In contrast …
Abstract
As significant numbers of acute myeloid leukemia (AML) patients are still refractory to conventional therapies or experience relapse, immunotherapy using T cells expressing chimeric antigen receptors (CARs) might represent a valid treatment option. AML cells frequently overexpress the myeloid antigens CD33 and CD123, for which specific CARs can be generated. However, CD33 is also expressed on normal hematopoietic stem/progenitor cells (HSPCs), and its targeting could potentially impair normal hematopoiesis. In contrast, CD123 is widely expressed by AML, while low expression is detected on HSPCs, making it a much more attractive target. In this study we describe the in vivo efficacy and safety of using cytokine-induced killer (CIK) cells genetically modified to express anti-CD33 or anti-CD123 CAR to target AML. We show that both these modified T cells are very efficient in reducing leukemia burden in vivo, but only the anti-CD123 CAR has limited killing on normal HSPCs, thus making it a very attractive immunotherapeutic tool for AML treatment.
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