AMB4269951

AMB4269951 is a plant-derived small molecule isoquinoline derivative that acts as a potent inhibitor of choline transporter-like protein 1 (CTL1). Developed by researchers at Tokyo Medical University, AMB4269951 blocks CTL1-mediated choline uptake, which is a rate-limiting step in phospholipid synthesis and cell membrane formation in cancer cells. This inhibition leads to intracellular choline deficiency, triggering ceramide production, downregulating the anti-apoptotic protein survivin, and activating caspase-3/7 to induce apoptotic cell death. AMB4269951 has demonstrated significant antitumor efficacy in preclinical models of glioma, pancreatic cancer, hepatocellular carcinoma, and small cell lung cancer (SCLC) without causing significant systemic toxicity or weight loss.

Other names

4-methoxy-6,6-dimethyl-5-(2-oxo-2-(4-pentylphenyl)ethyl)-5,6,7,8-tetrahydro-[1,3]dioxolo[4,5-g]isoquinolin-6-ium iodide