Drug intelligence / Profile preview

gliotoxin

Development stage
Preclinical
Modality
Small Molecules
Administration
Oral, Dermal, Inhalation, Subcutaneous, Intranasal, Intravenous
01

Overview

Gliotoxin is a sulfur-containing mycotoxin belonging to the epipolythiodioxopiperazine (ETP) class of natural products, characterized by a diketopiperazine core with a disulfide bridge. It is naturally produced by various fungi, most notably *Aspergillus fumigatus*, *Trichoderma virens*, and certain species of *Penicillium* and *Gliocladium*. Gliotoxin is primarily utilized as a research compound rather than a marketed pharmaceutical due to its significant systemic toxicity to mammalian cells. Its biological activity is pleiotropic, involving the inhibition of several key targets including the NF-κB signaling pathway, farnesyltransferase, geranylgeranyltransferase I, and NADPH oxidase. Research has explored its potential therapeutic applications as an antitumor agent (targeting breast, ovarian, and lung cancers), an antibacterial agent (active against MRSA), and an anti-fibrotic agent for liver fibrosis. Its mechanism of action often involves the modification of thiol groups in proteins and the generation of reactive oxygen species (ROS), leading to the induction of apoptosis in target cells.

Other names
Aspergillin
02

Targets

FTase (Protein Farnesyltransferase)GGTase I (Geranylgeranyl transferase type I)LUBAC (Linear Ubiquitin Chain Assembly Complex)

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