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December 8, 2025Blood

Targeting CD117 with engineered invariant natural killer T cells (iNKT Cells) as a novel approach for acute myeloid leukemia treatment

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Authors

HYHao YanQBQuenton R. BubbRNRobert S. Negrin

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Overview

Adoptive cell therapy with CD117-CAR-iNKT cells enhances survival in AML models, suggesting clinical potential.

Key Points

  • To evaluate the therapeutic efficacy of CD117-CAR-iNKT cells for treating acute myeloid leukemia (AML).
  • Isolated peripheral blood mononuclear cells (PBMCs) and enriched iNKT cells using anti-iNKT microbeads.
  • Stimulated iNKT cells with cytokines IL2, IL15, and IL21 to promote expansion.
  • Transduced iNKT cells with a gamma-retroviral vector expressing a CD117-targeting CAR.
  • Assessed in vitro cytotoxicity against CD117 leukemia cell lines and evaluated in vivo efficacy in NSG mice.
  • CD117-CAR-iNKT cells showed dose-dependent cytotoxic effects against AML cell lines.
  • Significant survival improvement in NSG mice treated with CD117-CAR-iNKT cells compared to controls.
  • 40% of CAR-iNKT treated mice survived 151 days, with no GVHD observed.

Cite This Study

Yan et al. (2025) studied this question.

synapsesocial.com/papers/69362f444fa91c937236d648https://doi.org/10.1182/blood-2025-7662
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Also Consider

Synapse has enriched 5 closely related papers on similar clinical questions. Consider them for comparative context:

  1. 1Abstract 137: Harnessing allogeneic CAR-iNKT cells for next-generation treatments in oncology and autoimmunity.2026
  2. 2Off-the-shelf dual CAR-iNKT cell immunotherapy eradicates medullary and leptomeningeal high-risk KMT2A-rearranged leukemia2025 · 9 citations
  3. 3Novel allogeneic BCMA CAR-expressing iNKT cells with PD1 deletion demonstrate superior tumor control in a multiple myeloma preclinical model2025
  4. 4Abstract 1332: Allogeneic CD19-directed CAR-iNKT cells and their phenotypic subsets for the treatment of CD19+ hematological malignancies2024
  5. 5Effiefficacy and safety of allogeneic CD123 CAR-NK cell therapy in Relapsed/Refractory Acute Myeloid Leukemia2025 · 3 citations