Drug intelligence / Profile preview

toosendanin

Development stage
Preclinical
Lead developer
Nanjing KMK Pharmaceutical
Modality
Metabolically Activated Prodrugs → Prodrugs/Conditional Activator Small Molecules → Small Molecules, Classical Binding Small Molecules → Small Molecules
Administration
Intraperitoneal (experimental, Animal Studies)
01

Overview

**Toosendanin** is a naturally occurring tetracyclic triterpenoid compound extracted from the bark and fruits of *Melia toosendan* (Sieb et Zucc), a medicinal plant used in traditional Chinese medicine[1][2][5]. It is primarily recognized for its use as an ascaricide (anti-parasitic agent)[3][5], agricultural insecticide[1][2][5], and has demonstrated analgesic, anti-inflammatory, antibotulinum, and anticancer activities in preclinical settings[1][3][4][5][6]. Toosendanin acts as a selective presynaptic blocker, interfering with neurotransmitter release by initially facilitating and then depressing synaptic transmission, ultimately blocking neuro-muscular and central synapse activity[1][5]. It can also inhibit the growth and induce apoptosis of various tumor cell lines—including glioma, osteosarcoma, colorectal, liver, breast, and ovarian cancers—via multiple mechanisms involving suppression of PI3K/Akt/mTOR and STAT3 signaling, mitochondrial apoptotic pathways, and V-ATPase inhibition[3][4][6][7]. It has significant antibotulismic effects by preventing botulinum neurotoxin from accessing its SNARE protein substrate[1][5]. Its anti-inflammatory effects are related to modulation of M1 macrophage polarization, NLRP3 inflammasome activity, and Nrf2/HO-1 signaling[2]. Research also points to its effect on potassium channels, facilitation of calcium influx via L-type calcium channels, and intracellular calcium overload[1][5]. No approved pharmaceutical use in humans is reported; use is limited to agricultural and experimental research settings.

Other names
ToosendaniChuanliansu28-Deacetylsendanin12-AcetoxyaMoorastatin
02

Targets

STAT3 (Signal Transducer and Activator of Transcription 3)DCK (Deoxycytidine kinase)HCK (Haematopoietic Cell Kinase)

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