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Target intelligence / Profile preview
Branched-chain-amino-acid transaminase 1 (BCAT1)
- Target
- BCAT1
- Molecular classification
- Enzyme
01
Overview
Branched-chain-amino-acid transaminase 1 (BCAT1) is a cytosolic enzyme that catalyzes the first step in the catabolism of branched-chain amino acids (leucine, isoleucine, valine) by reversible transamination, essential for cellular energy metabolism and growth. It regulates BCAA degradation and thereby controls metabolic flux through the TCA cycle and glutamate synthesis. BCAT1 is abundantly expressed in embryonic and select adult tissues (notably brain, ovary, placenta, macrophages) and has a distinct cytosolic distribution, while its mitochondrial isoform is BCAT2. Clinically, BCAT1 is overexpressed in several cancers, contributes to macrophage activation in inflammation, and is implicated in congenital amino acid metabolic disorders. It is a validated therapeutic target in metabolic and inflammatory diseases, and experimental agents such as the leucine analogue ERG240 have demonstrated preclinical efficacy by inhibiting BCAT1 function in immune and inflammatory models.
- Other names
- Branched-chain-amino-acid aminotransferase, cytosolicBCAT1BCT1ECA39BCAT(c)BCATcBCATCMECA39PNAS121PP18placental protein 18
02
Mechanism of action
Inhibition of BCAT1 aminotransferase activity; metabolic modulation (decreases BCAA catabolism, alters TCA cycle and glycolytic flux); reduces activation of IRG1/itaconate axis in macrophages
03
Biological functions
Branched-chain amino acid (BCAA) catabolismAmino acid transaminationMetabolic reprogrammingRegulation of cell growth
04
Disease associations
Cancer (glioblastoma, breast cancer, acute myeloid leukemia, gastric cancer, chronic myeloid leukemia)InflammationAutoimmune disease (rheumatoid arthritis, glomerulonephritis)Congenital metabolic disorders (hypervalinemia, hyperleucine-isoleucinemia)
05
Safety considerations
Potential risk of disrupting amino acid homeostasispossible effects on cell proliferation and immune functionrisk of metabolic imbalance in patients with pre-existing BCAA metabolism disorders
06
Interacting drugs
ERG240 (experimental, selective BCAT1 inhibitor)
07
Biomarkers
BCAT1 expression (in cancer, weight regulation, macrophage activation)IRG1/itaconate levels as downstream metabolic markers
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