Drug intelligence / Profile preview

l-cycloserine

Development stage
Preclinical
Lead developer
Pfizer
Modality
Small Molecules
Administration
Oral, Intraperitoneal
01

Overview

L-cycloserine is the L-enantiomer of the antibiotic cycloserine. It is a potent inhibitor of several pyridoxal 5'-phosphate (PLP)-dependent enzymes, most notably serine palmitoyltransferase (SPT), which catalyzes the rate-limiting step in the de novo biosynthesis of ceramides. By reducing ceramide levels, L-cycloserine is being investigated as a substrate reduction therapy (SRT) for sphingolipidoses such as Krabbe disease and as a protective agent in retinal degenerative diseases and certain cancers, including MYC-driven liver cancer and chemoresistant renal cancer. It also inhibits other PLP-dependent enzymes such as glutamic-pyruvic transaminase 2 (GPT2), alanine:glyoxylate aminotransferase (AGT), and various bacterial aminotransferases and desulfurases. Unlike its D-enantiomer, which is an FDA-approved treatment for tuberculosis, L-cycloserine is currently an experimental compound and is not approved for clinical use in humans.

Other names
(S)-4-amino-1,2-oxazolidin-3-oneLevocycloserine
02

Targets

SPT (Serine palmitoyltransferase)ALT (Alanine aminotransferase)Alr (Alanine racemase)NFS1 (NFS1 cysteine desulfurase)BCAT (Branched-chain-amino-acid transaminase 1)DdlA (D-alanyl-D-alanine Synthetase)

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