Target intelligence / Profile preview

Tumor-associated antigens on acute myeloid leukemia cells (AML TAAs)

Target
AML TAAs
Molecular classification
Receptor, Enzyme, Transcription factor, Other
01

Overview

Tumor-associated antigens (TAAs) on acute myeloid leukemia (AML) cells are a heterogeneous group of proteins that are either uniquely expressed or significantly overexpressed on leukemic blasts and stem cells (ASH Publications, 2006). These antigens serve as critical focal points for the development of targeted therapies, including monoclonal antibodies, antibody-drug conjugates (ADCs), and adoptive cellular therapies like CAR-T cells (NIH, 2021). Prominent examples include CD33, CD123, and FLT3, which are involved in various cellular processes such as signal transduction, proliferation, and survival (AACR, 2008). While targeting these antigens offers a pathway to more precise leukemia treatment, a significant challenge remains the 'on-target, off-tumor' toxicity, as many of these antigens are also present on healthy hematopoietic stem and progenitor cells (NIH, 2025). This can lead to severe clinical complications such as prolonged myelosuppression and bone marrow ablation (NIH, 2021). Current research is directed toward identifying novel, more specific antigens and developing multi-antigen targeting strategies to enhance efficacy while minimizing damage to normal tissues (AACR, 2021).

Other names
Leukemia-associated antigensLAAsAML-associated antigensAcute myeloblastic leukemia surface antigensAML-specific antigens
02

Mechanism of action

Drugs targeting these antigens utilize various mechanisms including direct inhibition of signaling (small molecules), antibody-dependent cellular cytotoxicity (ADCC), delivery of cytotoxic payloads (ADCs), and redirected T-cell killing via CAR-T cells or bispecific antibodies (NIH, 2021; AACR, 2021).

03

Biological functions

Signal transductionCell proliferationApoptosisImmune responseCell cycle
04

Disease associations

Cancer
05

Safety considerations

On-target off-tumor toxicity (NIH, 2025)Myelosuppression (NIH, 2021)Cytopenia (NIH, 2025)Cytokine release syndrome (CRS) (NIH, 2021)Veno-occlusive disease (VOD) (FDA, 2017)
06

Interacting drugs

Gemtuzumab ozogamicin

7 more in the full profile.

07

Biomarkers

CD33 expressionCD123 expressionFLT3-ITD mutationWT1 expression levelsCLL-1 expression

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