Published January 2022 | Version v1
Journal article

Discovery and mechanism of action of small molecule inhibitors of ceramidases

Description

Sphingolipid metabolism is tightly controlled by enzymes to regulate essential processes in human physiology. The central metabolite is ceramide, a pro-apoptotic lipid catabolized by ceramidase enzymes to produce pro-proliferative sphingosine-1phosphate. Alkaline ceramidases are transmembrane enzymes that recently attracted attention for drug development in fatty liver diseases. However, due to their hydrophobic nature, no specific small molecule inhibitors have been reported. Here, we present the discovery and mechanism of action of the first drug-like inhibitors of alkaline ceramidase 3 (ACER3). In particular, we chemically engineered novel fluorescent ceramide substrates enabling screening of large compound libraries and characterized enzyme:inhibitor interactions using mass spectrometry and MD simulations. In addition to reveal a new paradigm for inhibition of lipid metabolising enzymes with nonlipidic small molecules, our data lay the ground for targeting ACER3 in drug discovery efforts.

Abstract

International audience

Additional details

Identifiers

URL
https://hal.archives-ouvertes.fr/hal-03406152
URN
urn:oai:HAL:hal-03406152v1

Origin repository

Origin repository
UNICA