Synthesis and biological evaluation of new creatine fatty esters revealed Dodecyl creatine ester as a promising drug candidate for the treatment of the creatine transporter deficiency

Journal of Medicinal Chemistry

Volumn 56, Issue 12, 2013, Pages 5173-5181

  Trotier Faurion, Alexandra ^a   Dézard, Sophie ^b   Taran, Frédéric ^b   Valayannopoulos, Vassili ^c   De Lonlay, Pascale ^c   Mabondzo, Aloïse ^a  

^a Institut National de la Recherche Agronomique   (France)

^b CEA   (France)

^c HÔPITAL NECKER ENFANTS MALADES   (France)

Author keywords

[No Author keywords available]

Indexed keywords

CREATINE FATTY ESTER; CREATININE; DODECYL CREATINE ESTER; ESTER DERIVATIVE; PRODRUG; UNCLASSIFIED DRUG;

ANIMAL CELL; ARTICLE; ASTROCYTE; BLOOD BRAIN BARRIER; CONTROLLED STUDY; CREATINE TRANSPORTER DEFICIENCY; DRUG SCREENING; DRUG SYNTHESIS; ENDOTHELIUM CELL; FIBROBLAST; HUMAN; HUMAN CELL; IN VITRO STUDY; NERVE CELL; NEUROLOGIC DISEASE; NONHUMAN; ORGANIC CHEMISTRY; PROTEIN FOLDING; STRUCTURE ACTIVITY RELATION;

ANIMALS; BLOOD-BRAIN BARRIER; CELL SURVIVAL; CHEMISTRY TECHNIQUES, SYNTHETIC; CREATINE; CREATININE; DRUG STABILITY; ESTERS; FEMALE; HUMANS; MEMBRANE TRANSPORT PROTEINS; PREGNANCY; RATS;

EID: 84879572441     PISSN: 00222623     EISSN: 15204804     Source Type: Journal    
DOI: 10.1021/jm400545n     Document Type: Article

References (25)

1. Stockler, S.; Schutz, P. W.; Salomons, G. S. Cerebral creatine deficiency syndromes: clinical aspects, treatment and pathophysiology Subcell. Biochem. 2007, 46, 149-166
2. Schulze, A. Creatine deficiency syndromes Mol. Cell. Biochem. 2003, 244, 143-150
3. Wyss, M.; Kaddurah-Daouk, R. Creatine and creatinine metabolism Physiol. Rev. 2000, 80, 1107-1213
4. Stockler, S.; Hanefeld, F.; Frahm, J. Creatine replacement therapy in guanidinoacetate methyltransferase deficiency, a novel inborn error of metabolism Lancet 1996, 348, 789-790
5. Poo-Arguelles, P.; Arias, A.; Vilaseca, M. A.; Ribes, A.; Artuch, R.; Sans-Fito, A.; Moreno, A.; Jakobs, C.; Salomons, G. X-Linked creatine transporter deficiency in two patients with severe mental retardation and autism J. Inherit. Metab. Dis. 2006, 29, 220-223
6. van de Kamp, J. M.; Pouwels, P. J.; Aarsen, F. K.; ten Hoopen, L. W.; Knol, D. L.; de Klerk, J. B.; de Coo, I. F.; Huijmans, J. G.; Jakobs, C.; van der Knaap, M. S.; Salomons, G. S.; Mancini, G. M. Long-term follow-up and treatment in nine boys with X-linked creatine transporter defect J. Inherit. Metab. Dis. 2012, 35, 141-149
7. Cecil, K. M.; Salomons, G. S.; Ball, W. S., Jr.; Wong, B.; Chuck, G.; Verhoeven, N. M.; Jakobs, C.; DeGrauw, T. J. Irreversible brain creatine deficiency with elevated serum and urine creatine: a creatine transporter defect? Ann. Neurol. 2001, 49, 401-404
8. Ohtsuki, S.; Tachikawa, M.; Takanaga, H.; Shimizu, H.; Watanabe, M.; Hosoya, K.; Terasaki, T. The blood-brain barrier creatine transporter is a major pathway for supplying creatine to the brain J. Cereb. Blood Flow. Metab. 2002, 22, 1327-1335
9. deGrauw, T. J.; Cecil, K. M.; Byars, A. W.; Salomons, G. S.; Ball, W. S.; Jakobs, C. The clinical syndrome of creatine transporter deficiency Mol. Cell. Biochem. 2003, 244, 45-48
10. Perasso, L.; Lunardi, G. L.; Risso, F.; Pohvozcheva, A. V.; Leko, M. V.; Gandolfo, C.; Florio, T.; Cupello, A.; Burov, S. V.; Balestrino, M. Protective effects of some creatine derivatives in brain tissue anoxia Neurochem. Res. 2008, 33, 765-775
11. Perasso, L.; Adriano, E.; Ruggeri, P.; Burov, S. V.; Gandolfo, C.; Balestrino, M. In vivo neuroprotection by a creatine-derived compound: phosphocreatine-Mg-complex acetate Brain Res. 2009, 1285, 158-163
12. Burov, S.; Leko, M.; Dorosh, M.; Dobrodumov, A.; Veselkina, O. Creatinyl amino acids: new hybrid compounds with neuroprotective activity J. Pept. Sci. 2011, 17, 620-626
13. Skelton, M. R.; Schaefer, T. L.; Graham, D. L.; Degrauw, T. J.; Clark, J. F.; Williams, M. T.; Vorhees, C. V. Creatine transporter (CrT; Slc6a8) knockout mice as a model of human CrT deficiency PLoS One 2011, 6, e16187
14. Kurosawa, Y.; Degrauw, T. J.; Lindquist, D. M.; Blanco, V. M.; Pyne-Geithman, G. J.; Daikoku, T.; Chambers, J. B.; Benoit, S. C.; Clark, J. F. Cyclocreatine treatment improves cognition in mice with creatine transporter deficiency J. Clin. Invest. 2012, 122, 2837-2846
15. Lunardi, G.; Parodi, A.; Perasso, L.; Pohvozcheva, A. V.; Scarrone, S.; Adriano, E.; Florio, T.; Gandolfo, C.; Cupello, A.; Burov, S. V.; Balestrino, M. The creatine transporter mediates the uptake of creatine by brain tissue, but not the uptake of two creatine-derived compounds Neuroscience 2006, 142, 991-997
16. Adriano, E.; Garbati, P.; Damonte, G.; Salis, A.; Armirotti, A.; Balestrino, M. Searching for a therapy of creatine transporter deficiency: some effects of creatine ethyl ester in brain slices in vitro Neuroscience 2011, 199, 386-393
17. Fons, C.; Arias, A.; Sempere, A.; Poo, P.; Pineda, M.; Mas, A.; Lopez-Sala, A.; Garcia-Villoria, J.; Vilaseca, M. A.; Ozaez, L.; Lluch, M.; Artuch, R.; Campistol, J.; Ribes, A. Response to creatine analogs in fibroblasts and patients with creatine transporter deficiency Mol. Genet. Metab. 2010, 99, 296-299
18. Katseres, N. S.; Reading, D. W.; Shayya, L.; DiCesare, J. C.; Purser, G. H. Non-enzymatic hydrolysis of creatine ethyl ester Biochem. Biophys. Res. Commun. 2009, 386, 363-367
19. Katseres, N. S.; Reading, D. W.; Shayya, L.; DiCesare, J.; Purser, G. H. Hydrolysis of creatine ethyl ester. 237th National Meeting of the American Chemical Society, Salt Lake City, Utah, March 22-26, 2009, 350.
20. Giese, M. W.; Lecher, C. S. Non-enzymatic cyclization of creatine ethyl ester to creatinine Biochem. Biophys. Res. Commun. 2009, 388, 252-255
21. Lacombe, O.; Videau, O.; Chevillon, D.; Guyot, A. C.; Contreras, C.; Blondel, S.; Nicolas, L.; Ghettas, A.; Benech, H.; Thevenot, E.; Pruvost, A.; Bolze, S.; Krzaczkowski, L.; Prevost, C.; Mabondzo, A. In vitro primary human and animal cell-based blood-brain barrier models as a screening tool in drug discovery Mol. Pharmaceutics 2011, 8, 651-663
22. Salomons, G. S.; van Dooren, S. J.; Verhoeven, N. M.; Cecil, K. M.; Ball, W. S.; Degrauw, T. J.; Jakobs, C. X-linked creatine-transporter gene (SLC6A8) defect: a new creatine-deficiency syndrome Am. J. Hum. Genet. 2001, 68, 1497-500
23. Brun, E.; Carriere, M.; Mabondzo, A. In vitro evidence of dysregulation of blood-brain barrier function after acute and repeated/long-term exposure to TiO(2) nanoparticles Biomaterials 2012, 33, 886-896
24. Valayannopoulos, V.; Bakouh, N.; Mazzuca, M.; Nonnenmacher, L.; Hubert, L.; Makaci, F. L.; Chabli, A.; Salomons, G. S.; Mellot-Draznieks, C.; Brule, E.; de Lonlay, P.; Toulhoat, H.; Munnich, A.; Planelles, G.; de Keyzer, Y. Functional and electrophysiological characterization of four non-truncating mutations responsible for creatine transporter (SLC6A8) deficiency syndrome J. Inherit. Metab. Dis. 2012, 36, 103-112
25. Valayannopoulos, V.; Boddaert, N.; Chabli, A.; Barbier, V.; Desguerre, I.; Philippe, A.; Afenjar, A.; Mazzuca, M.; Cheillan, D.; Munnich, A.; de Keyzer, Y.; Jakobs, C.; Salomons, G. S.; de Lonlay, P. Treatment by oral creatine, l -arginine and l -glycine in six severely affected patients with creatine transporter defect J. Inherit. Metab. Dis. 2012, 35, 151-157