For research use only. Not for therapeutic Use.
Sinbaglustat (OGT2378) is a dual inhibitor of glucosylceramide synthase (GCS) and non-lysosomal glucosyl ceramidase (GBA2). Sinbaglustat is an orally available N-alkyl iminosugar that crosses the blood-brain barrier. Sinbaglustat can be used for the research of central neurodegenerative diseases associated with lysosomal dysfunctions[1][2].
Sinbaglustat (OGT2378; 20 μM) reduces the synthesis of glucosylceramide and ganglioside by 93% and >95% in MEB4 melanoma cells compared with untreated MEB4 cells, respectively, without either cytotoxic or antiproliferative effects[1].
GBA2 is an enzyme involved in the catabolism of glycosphingolipids (GSLs). Sinbaglustat is 50-fold more potent in inhibiting GBA2 than GCS[2].
Sinbaglustat (OGT2378; administered p.o., in the powdered chow, at a dose of 2500 mg/kg/day, corresponding to 35-40 mg of Sinbaglustat per mouse per day) is highly effective in impeding melanoma tumor growth in vivo. The effectiveness of p.o. Sinbaglustat in this murine model suggests that inhibition of glycosphingolipid synthesis is a promising approach to inhibit tumor progression[1].
| Catalog Number | I008403 |
| CAS Number | 441061-33-2 |
| Synonyms | (2S,3R,4R,5S)-2-(hydroxymethyl)-1-pentylpiperidine-3,4,5-triol |
| Molecular Formula | C11H23NO4 |
| Purity | ≥95% |
| InChI | InChI=1S/C11H23NO4/c1-2-3-4-5-12-6-9(14)11(16)10(15)8(12)7-13/h8-11,13-16H,2-7H2,1H3/t8-,9-,10+,11+/m0/s1 |
| InChIKey | HCZQIIVHWYFIPW-UKKRHICBSA-N |
| SMILES | CCCCCN1CC(C(C(C1CO)O)O)O |
| Reference | [1]. Michael Weiss, et al. Inhibition of melanoma tumor growth by a novel inhibitor of glucosylceramide synthase. Cancer Res. 2003 Jul 1;63(13):3654-8. [2]. Martine Gehin, et al. Assessment of Target Engagement in a First-in-Human Trial with Sinbaglustat, an Iminosugar to Treat Lysosomal Storage Disorders. Clin Transl Sci. 2021 Mar;14(2):558-567. |
