Assessment of partially deoxygenated deoxynojirimycin derivatives as glucosylceramide synthase inhibitors

ACS Medicinal Chemistry Letters

Volumn 2, Issue 7, 2011, Pages 519-522

  Van Den Berg, Richard J B H N ^a   Wennekes, Tom ^a   Ghisaidoobe, Amar ^a   Donker Koopman, Wilma E ^b   Strijland, Anneke ^b   Boot, Rolf G ^b   Van Der Marel, Gijsbert A ^a   Aerts, Johannes M F G ^b   Overkleeft, Herman S ^a  

^a LEIDEN UNIVERSITY   (Netherlands)

^b ACADEMIC MEDICAL CENTER   (Netherlands)

Author keywords

ceramide; deoxynojirimycin; Gaucher disease; Glucosylceramide synthase; iminosugar

Indexed keywords

CERAMIDE; CERAMIDE GLUCOSYLTRANSFERASE; MIGLUSTAT;

ARTICLE; BINDING AFFINITY; CONTROLLED STUDY; DEOXYGENATION; ENZYME INHIBITION; NONHUMAN; PRIORITY JOURNAL;

EID: 79960427628     PISSN: None     EISSN: 19485875     Source Type: Journal    
DOI: 10.1021/ml200050s     Document Type: Article

References (34)

1. Jeckel, D.; Karrenbauer, A.; Burger, K. N.; van Meer, G.; Wieland, F. Glucosylceramide is synthesized at the cytosolic surface of various Golgi subfractions J. Cell. Biol. 1992, 117, 259-267
2. Sakabe, K.; Hart, G. W. O-GlcNAc transferase regulates mitotic chromatin dynamics J. Biol. Chem. 2010, 285, 34460-34468
3. Platt, F. M.; Neises, G. R.; Dwek, R. A.; Butters, T. D. N-Butyldeoxynojirimycin is a novel inhibitor of glycolipid biosynthesis J. Biol. Chem. 1994, 269, 8362-8365
4. Platt, F. M.; Jeyakumar, M.; Andersson, U.; Priestman, D. A.; Dwek, R. A.; Butters, T. D.; Cox, T. M.; Lachmann, R. H.; Hollak, C.; Aerts, J. M.; van Weely, S.; Hrebícek, M.; Moyses, C.; Gow, I.; Elstein, D.; Zimran, A. Inhibition of substrate synthesis as a strategy for glycolipid lysosomal storage disease therapy J. Inherited Metab. Dis. 2001, 24, 275-290 (Pubitemid 32479421)
5. Aerts, J. M.; Hollak, C. E.; Boot, R. G.; Groener, J. E.; Maas, M. Substrate reduction therapy of glycosphingolipid storage disorders J. Inherited Metab. Dis. 2006, 29, 449-456 (Pubitemid 43880650)
6. Cox, T.; Lachmann, R.; Hollak, C.; Aerts, J.; van Weely, S.; Hrebícek, M.; Platt, F.; Butters, T.; Dwek, R.; Moyses, C.; Gow, I.; Elstein, D.; Zimran, A. Novel oral treatment of Gaucher's disease with N-butyldeoxynojirimycin (OGT 918) to decrease substrate biosynthesis Lancet 2000, 355, 1481-1485 (Pubitemid 30237353)
7. Overkleeft, H. S.; Renkema, G. H.; Neele, J.; Vianello, P.; Hung, I. O.; Strijland, A.; van der Burg, A. M.; Koomen, G. J.; Pandit, U. K.; Aerts, J. M. Generation of specific deoxynojirimycin-type inhibitors of the non-lysosomal glucosylceramidase J. Biol. Chem. 1998, 273, 26522-26527 (Pubitemid 28471661)
8. Wennekes, T.; van den Berg, R. J. B. H. N.; Donker, W.; van der Marel, G. A.; Strijland, A.; Aerts, J. M. F. G.; Overkleeft, H. S. Development of adamantan-1-yl-methoxy-functionalized 1-deoxynojirimycin derivatives as selective inhibitors of glucosylceramide metabolism in man J. Org. Chem. 2007, 72, 1088-1097
9. Aerts, J. M.; Ottenhoff, R.; Powlson, A. S.; Grefhorst, A.; van Eijk, M.; Dubbelhuis, P. F.; Aten, J.; Kuipers, F.; Serlie, M. J.; Wennekes, T.; Sethi, J. K.; O'Rahilly, S.; Overkleeft, H. S. Pharmacological inhibition of glucosylceramide synthase enhances insulin sensitivity Diabetes 2007, 56, 1341-1349 (Pubitemid 46715620)
10. Bijl, N.; Sokolović, M.; Vrins, C.; Langeveld, M.; Moerland, P. D.; Ottenhoff, R.; van Roomen, C. P.; Claessen, N.; Boot, R. G.; Aten, J.; Groen, A. K.; Aerts, J. M.; van Eijk, M. Modulation of glycosphingolipid metabolism significantly improves hepatic insulin sensitivity and reverses hepatic steatosis in mice Hepatology 2009, 50, 1431-1441
11. Bietrix, F.; Lombardo, E.; van Roomen, C. P.; Ottenhoff, R.; Vos, M.; Rensen, P. C.; Verhoeven, A. J.; Aerts, J. M.; Groen, A. K. Inhibition of glycosphingolipid synthesis induces a profound reduction of plasma cholesterol and inhibits atherosclerosis development in APOE*3 Leiden and low-density lipoprotein receptor-/- mice Arterioscler. Thromb. Vasc. Biol. 2010, 30, 931-937
12. Shen, C.; Bullens, D.; Kasran, A.; Maerten, P.; Boon, L.; Aerts, J. M.; van Assche, G.; Geboes, K.; Rutgeerts, P.; Ceuppens, J. L. Inhibition of glycolipid biosynthesis by N-(5-adamantane-1-yl-methoxy-pentyl)-deoxynojirimycin protects against the inflammatory response in hapten-induced colitis Int. Immunopharmacol. 2004, 4, 939-951 (Pubitemid 38739099)
13. van Eijk, M.; Aten, J.; Bijl, N.; Ottenhoff, R.; van Roomen, C. P.; Dubbelhuis, P. F.; Seeman, I.; Ghauharali-van der Vlugt, K.; Overkleeft, H. S.; Arbeeny, C.; Groen, A. K.; Aerts, J. M. Reducing glycosphingolipid content in adipose tissue of obese mice restores insulin sensitivity, adipogenesis and reduces inflammation PLoS One 2009, 4, e4723
14. Natoli, T. A.; Smith, L. A.; Rogers, K. A.; Wang, B.; Komarnitsky, S.; Budman, Y.; Belenky, A.; Bukanov, N. O.; Dackowski, W. R.; Husson, H.; Russo, R. J.; Shayman, J. A.; Ledbetter, S. R.; Leonard, J. P.; Ibraghimov-Beskrovnaya, O. Inhibition of glucosylceramide accumulation results in effective blockade of polycystic kidney disease in mouse models Nat. Med. 2010, 16, 788-792
15. Kato, A.; Kato, N.; Kano, E.; Adachi, I.; Ikeda, K.; Yu, L.; Okamoto, T.; Banba, Y.; Ouchi, H.; Takahata, H.; Asano, N. Biological properties of d-and l -1-deoxyazasugars J. Med. Chem. 2005, 48, 2036-2044
16. Wennekes, T.; van den Berg, R. J. B. H. N.; Boot, R. G.; van der Marel, G. A.; Overkleeft, H. S.; Aerts, J. M. F. G. Glycosphingolipids-nature, function, and pharmacological modulation Angew. Chem., Int. Ed. 2009, 48, 8848-8869
17. Stütz, A. E. Iminosugars as Glycosidase Inhibitors: Nojirimycin and Beyond; Wiley-VCH: Weinheim, 1999.
18. Martin, O. R.; Compain, P. Iminosugars: From synthesis to therapeutic applications; Wiley-VHC: Weinheim, 2007.
19. Compain, P.; Martin, O. R. Carbohydrate mimetics-based glycosyltransferase inhibitors Bioorg. Med. Chem. 2001, 9, 3077-3092 (Pubitemid 33065608)
20. Compain, P.; Martin, O. R. Design, synthesis and biological evaluation of iminosugar-based glycosyltransferase inhibitors Curr. Top. Med. Chem. 2003, 3, 541-560
21. Platt, F. M.; Neises, G. R.; Karlsson, G. B.; Dwek, R. A.; Butters, T. D. N-butyldeoxygalactonojirimycin inhibits glycolipid biosynthesis but does not effect N-linked oligosaccharide processing J. Biol. Chem. 1994, 269, 27108-27114
22. Butters, T. D.; van den Broek, L. A. G. M.; Fleet, G. W. J.; Krulle, T. M.; Wormald, M. R.; Dwek, R. A.; Platt, F. M. Molecular requirements of imino sugars for the selective control of N-linked glycosylation and glycosphingolipid biosynthesis Tetrahedron: Asymmetry 2000, 11, 113-124 (Pubitemid 30116779)
23. Butters, T. D. Iminosugar inhibitors for substrate reduction therapy for the lysosomal glycosphingolipidoses. In Iminosugars: From synthesis to therapeutic applications; T. D. Martin, O. R.; Compain, P., Eds.; Wiley-VHC: Weinheim, 2007; pp 249-268.
24. Wennekes, T.; Meijer, A. J.; Groen, A. K.; Boot, R. G.; Groener, J. E.; van Eijk, M.; Ottenhoff, R.; Bijl, N.; Ghauharali, K.; Song, H.; O'Shea, T. J.; Liu, H.; Yew, N.; Copeland, D.; van den Berg, R. J.; van der Marel, G. A.; Overkleeft, H. S.; Aerts, J. M. Dual-action lipophilic iminosugar improves glycemic control in obese rodents by reduction of visceral glycosphingolipids and buffering of carbohydrate assimilation J. Med. Chem. 2010, 53, 689-698
25. Butters, T. D.; Mellor, H. R.; Narita, K.; Dwek, R. A.; Platt, F. M. Small-molecule therapeutics for the treatment of glycolipid lysosomal storage disorders Philos. Trans. R. Soc. London, B: Biol. Sci. 2003, 358, 927-945 (Pubitemid 36639718)
26. Takahata, H.; Banba, Y.; Ouchi, H.; Nemoto, H.; Kato, A.; Adachi, I. Aymmetric synthesis of the four possible fagomine isomers J. Org. Chem. 2003, 68, 3603-3607 (Pubitemid 36520385)
27. Takahata, H.; Banba, Y.; Ouchi, H.; Nemoto, H. Concise and highly stereocontrolled synthesis of 1-deoxygalactonojirimycin and its congeners using dioxanylpiperidene, a promising chiral building block Org. Lett. 2003, 5, 2527-2529 (Pubitemid 37140901)
28. Takahata, H.; Banba, Y.; Sasatani, M.; Nemoto, H.; Kato, A.; Adachi, I. Asymmetric synthesis of 1-deoxynojirimycin and its congeners from a common chiral building block Tetrahedron 2004, 60, 8199-8205 (Pubitemid 39094335)
29. van den Broek, L. A. G. M.; Vermaas, D. J.; Heskamp, B. M.; van Boeckel, C. A. A.; Tan, M. C. A. A.; Bolscher, J. G. M.; Ploegh, H. L.; van Kemenade, F. J.; de Goede, R. E. Y.; Miedema, F. Chemical modification of azasugars, inhibition of N-glycoprotein-processing glycosidases and of HIV-I infection Recl. Trav. Chim. Pays-Bas 1993, 112, 82-94
30. van den Broek, L. A. G. M.; Vermaas, D. J.; van Kemenade, F. J.; Tan, M. C. C. A.; Rotteveel, F. T. M.; Zandberg, P.; Butters, T. D.; Miedema, F.; Ploegh, H. L.; van Boeckel, C. A. A. Synthesis of oxygen-substituted N-alkyl 1-deoxynojirimycin derivatives: aza sugar α-glucosidase inhibitors showing antiviral (HIV-1) and immunosuppressive activity Recl. Trav. Chim. Pays-Bas 1994, 113, 507-516
31. van Weely, S.; van Leeuwen, M. B.; Jansen, I. D. C.; de Bruijn, M. A. C.; Brouwer-Kelder, E. M.; Schram, A. W.; Sa Miranda, M. C.; Barranger, J. A.; Petersen, E. M.; Goldblatt, J.; Stotz, H.; Schwarzmann, G.; Sandhoff, K.; Svennerholm, L.; Erikson, A.; Tager, J. M.; Aerts, J. M. F. G. Clinical phenotype of Gaucher disease in relation to properties of mutant glucocerebrosidase in cultured fibroblasts Biochim. Biophys. Acta 1991, 1096, 301-311
32. Aerts, J. M.; Donker-Koopman, W. E.; van der Vliet, M. K.; Jonsson, L. M.; Ginns, E. I.; Murray, G. J.; Barranger, J. A.; Tager, J. M.; Schram, A. W. The occurrence of two immunologically distinguishable beta-glucocerebrosidases in human spleen Eur. J. Biochem. 1985, 150, 565-574 (Pubitemid 15024833)
33. van Weely, S.; Brandsma, M.; Strijland, A.; Tager, J. M.; Aerts, J. M. Demonstration of the existence of a second, non-lysosomal glucocerebrosidase that is not deficient in Gaucher disease Biochim. Biophys. Acta 1993, 1181, 55-62 (Pubitemid 23087522)
34. Boot, R. G.; Verhoek, M.; Donker-Koopman, W.; Strijland, A.; van Marle, J.; Overkleeft, H. S.; Wennekes, T.; Aerts, J. M. Identification of the non-lysosomal glucosylceramidase as beta-glucosidase 2 J. Biol. Chem. 2007, 282, 1305-1312