Lucerastat | CAS#141206-42-0 | a-D-galactosidase inhibitor MedKoo

MedKoo Cat#: 319731 | Name: Lucerastat

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Description:

WARNING: This product is for research use only, not for human or veterinary use.

Lucerastat is an extremely potent and selective α-gal A (a-D-galactosidase) inhibitor for the treatment of lipid storage disorders and Fabry's disease.

Chemical Structure

Lucerastat

CAS#141206-42-0

Theoretical Analysis

MedKoo Cat#: 319731

Name: Lucerastat

CAS#: 141206-42-0

Chemical Formula: C10H21NO4

Exact Mass: 219.1471

Molecular Weight: 219.28

Elemental Analysis: C, 54.77; H, 9.65; N, 6.39; O, 29.18

Price and Availability

Size	Price	Availability	Quantity
1mg	USD 450.00	2 weeks

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Synonym

NBDGJ; N-(n-Butyl)deoxygalactonojirimycin; Lucerastat

IUPAC/Chemical Name

(2R,3S,4R,5S)-1-butyl-2-(hydroxymethyl)piperidine-3,4,5-triol

InChi Key

UQRORFVVSGFNRO-XFWSIPNHSA-N

InChi Code

InChI=1S/C10H21NO4/c1-2-3-4-11-5-8(13)10(15)9(14)7(11)6-12/h7-10,12-15H,2-6H2,1H3/t7-,8+,9+,10-/m1/s1

SMILES Code

O[C@H]1[C@@H](CO)N(CCCC)C[C@H](O)[C@H]1O

Appearance

Solid powder

Purity

>95% (or refer to the Certificate of Analysis)

Shipping Condition

Shipped under ambient temperature as non-hazardous chemical. This product is stable enough for a few weeks during ordinary shipping and time spent in Customs.

Storage Condition

Dry, dark and at 0 - 4 C for short term (days to weeks) or -20 C for long term (months to years).

Solubility

Soluble in DMSO, not in water

Shelf Life

>2 years if stored properly

Drug Formulation

This drug may be formulated in DMSO

Stock Solution Storage

0 - 4 C for short term (days to weeks), or -20 C for long term (months).

HS Tariff Code

2934.99.9001

More Info

Product Data

Product Data

Instruction

View handling instruction

Biological target:

Lucerastat, the galactose form of Miglustat, is an orally-available inhibitor of glucosylceramide synthase (GCS).

In vitro activity:

Lucerastat dose dependently reduced Gb3 in all cell lines. For 13 cell lines the Gb3 data could be fit to an IC50 curve, giving a median IC50 [interquartile range (IQR)] = 11 μM (8.2-18); the median percent reduction (IQR) in Gb3 was 77% (70-83). Lucerastat treatment also dose dependently reduced LysoTracker Red staining of acidic compartments. Lucerastat's effects in the cell lines were compared to those with current treatments-agalsidase alfa and migalastat. Reference: Hum Mol Genet. 2018 Oct 1;27(19):3392-3403. https://pubmed.ncbi.nlm.nih.gov/29982630/

In vivo activity:

TBD

	Solvent	mg/mL	mM
Solubility
	DMF	20.0	91.21
	DMSO	26.0	118.57
	Ethanol	5.0	22.80
	PBS (pH 7.2)	10.0	45.60
	Water	24.0	109.45

Note: There can be variations in solubility for the same chemical from different vendors or different batches from the same vendor. The following factors can affect the solubility of the same chemical: solvent used for crystallization, residual solvent content, polymorphism, salt versus free form, degree of hydration, solvent temperature. Please use the solubility data as a reference only. Warming and sonication will facilitate dissolving. Still have questions? Please contact our Technical Support scientists.

Preparing Stock Solutions

The following data is based on the product molecular weight 219.28 Batch specific molecular weights may vary from batch to batch due to the degree of hydration, which will affect the solvent volumes required to prepare stock solutions.

Concentration / Solvent Volume / Mass	1 mg	5 mg	10 mg
1 mM	1.15 mL	5.76 mL	11.51 mL
5 mM	0.23 mL	1.15 mL	2.3 mL
10 mM	0.12 mL	0.58 mL	1.15 mL
50 mM	0.02 mL	0.12 mL	0.23 mL

Formulation protocol:

1. Welford RWD, Mühlemann A, Garzotti M, Rickert V, Groenen PMA, Morand O, Üçeyler N, Probst MR. Glucosylceramide synthase inhibition with lucerastat lowers globotriaosylceramide and lysosome staining in cultured fibroblasts from Fabry patients with different mutation types. Hum Mol Genet. 2018 Oct 1;27(19):3392-3403. doi: 10.1093/hmg/ddy248. PMID: 29982630; PMCID: PMC6140777.

In vitro protocol:

In vivo protocol:

TBD

1: Takai T, Higaki K, Aguilar-Moncayo M, Mena-Barragán T, Hirano Y, Yura K, Yu L, Ninomiya H, García-Moreno MI, Sakakibara Y, Ohno K, Nanba E, Ortiz Mellet C, García Fernández JM, Suzuki Y. A bicyclic 1-deoxygalactonojirimycin derivative as a novel pharmacological chaperone for GM1 gangliosidosis. Mol Ther. 2013 Mar;21(3):526-32. doi: 10.1038/mt.2012.263. Epub 2013 Jan 22. PubMed PMID: 23337983; PubMed Central PMCID: PMC3589148. 2: Kato T, Muraoka M, Hatanaka K. Novel method for chase analysis of oligosaccharide metabolic error caused by xenobiotics. Anal Biochem. 2010 Oct 1;405(1):103-8. doi: 10.1016/j.ab.2010.06.002. Epub 2010 Jun 4. PubMed PMID: 20570645. 3: Kato A, Yamashita Y, Nakagawa S, Koike Y, Adachi I, Hollinshead J, Nash RJ, Ikeda K, Asano N. 2,5-Dideoxy-2,5-imino-d-altritol as a new class of pharmacological chaperone for Fabry disease. Bioorg Med Chem. 2010 Jun 1;18(11):3790-4. doi: 10.1016/j.bmc.2010.04.048. Epub 2010 Apr 21. PubMed PMID: 20457528. 4: Gerrard G, Butters TD, Ganeshaguru K, Mehta AB. Glucosylceramide synthase inhibitors sensitise CLL cells to cytotoxic agents without reversing P-gp functional activity. Eur J Pharmacol. 2009 May 1;609(1-3):34-9. doi: 10.1016/j.ejphar.2009.03.018. Epub 2009 Mar 12. PubMed PMID: 19285492. 5: Voss AA, Díez-Sampedro A, Hirayama BA, Loo DD, Wright EM. Imino sugars are potent agonists of the human glucose sensor SGLT3. Mol Pharmacol. 2007 Feb;71(2):628-34. Epub 2006 Nov 16. PubMed PMID: 17110502. 6: Norris-Cervetto E, Callaghan R, Platt FM, Dwek RA, Butters TD. Inhibition of glucosylceramide synthase does not reverse drug resistance in cancer cells. J Biol Chem. 2004 Sep 24;279(39):40412-8. Epub 2004 Jul 19. PubMed PMID: 15263008. 7: Mellor HR, Platt FM, Dwek RA, Butters TD. Membrane disruption and cytotoxicity of hydrophobic N-alkylated imino sugars is independent of the inhibition of protein and lipid glycosylation. Biochem J. 2003 Sep 1;374(Pt 2):307-14. PubMed PMID: 12769816; PubMed Central PMCID: PMC1223602. 8: Day AJ, Gee JM, DuPont MS, Johnson IT, Williamson G. Absorption of quercetin-3-glucoside and quercetin-4'-glucoside in the rat small intestine: the role of lactase phlorizin hydrolase and the sodium-dependent glucose transporter. Biochem Pharmacol. 2003 Apr 1;65(7):1199-206. PubMed PMID: 12663055. 9: Durantel D, Branza-Nichita N, Carrouée-Durantel S, Butters TD, Dwek RA, Zitzmann N. Study of the mechanism of antiviral action of iminosugar derivatives against bovine viral diarrhea virus. J Virol. 2001 Oct;75(19):8987-98. PubMed PMID: 11533162; PubMed Central PMCID: PMC114467.