Target intelligence / Profile preview

Fucosyl-monosialoganglioside-1 (FucGM1)

Target
FucGM1
Molecular classification
Ganglioside, Glycosphingolipid, Tumor-associated carbohydrate antigen, Lipid membrane microdomain component
01

Overview

Fucosyl-monosialoganglioside-1 (Fucosyl-GM1, FucGM1) is a ganglioside consisting of a GM1 backbone with an α1,2-linked fucose moiety. It is selectively and highly expressed on the surface of small cell lung cancer cells and has sparse expression in most normal tissues, with the exception of some neurons. This molecular pattern makes FucGM1 a promising target for antibody-based immunotherapy, such as the monoclonal antibody BMS-986012, which acts by inducing immune-mediated cell death of FucGM1-positive tumor cells through mechanisms like ADCC and CDC. FucGM1 is also implicated as a biologically active membrane component distinct from the prototypical GM1 ganglioside, participating in neural differentiation and tumor cell biology via unique membrane signaling platforms. Its limited presence in healthy adult tissues, apart from some neural cells, underpins ongoing investigations for its use as both a therapeutic target and a cancer-selective biomarker.

Other names
Fucosyl-GM1FucGM1α1,2-fucosyl GM1Fuc-GM1Fuc(Gal)-GM1
02

Mechanism of action

Antibody-dependent cellular cytotoxicity (ADCC), Complement-dependent cytotoxicity (CDC), Antibody-dependent cellular phagocytosis (ADCP)

03

Biological functions

Signal transduction platformCell differentiation (neuritogenesis)Immune evasion in tumorsCell proliferation (cancer)
04

Disease associations

Cancer (especially small cell lung cancer)Potential function in neural diseases (limited evidence)
05

Safety considerations

Off-target effects on peripheral sensory neurons and dorsal root ganglia (limited expression in normal tissues, but not completely absent)Potential for immune-related adverse events due to antibody therapy
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Interacting drugs

BMS-986012 (anti-Fucosyl-GM1 monoclonal antibody)
07

Biomarkers

Fucosyl-GM1 surface expression (for patient selection in SCLC antibody trials)

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