Chemical optimization of new ligands of the low-density lipoprotein receptor as potential vectors for central nervous system targeting

Journal of Medicinal Chemistry

Volumn 55, Issue 5, 2012, Pages 2227-2241

  Malcor, Jean Daniel ^a   Payrot, Nadine ^a,b   David, Marion ^b   Faucon, Aude ^b   Abouzid, Karima ^b   Jacquot, Guillaume ^b   Floquet, Nicolas ^a   Debarbieux, Franck ^c   Rougon, Geneviève ^c   Martinez, Jean ^a   Khrestchatisky, Michel ^b,d   Vlieghe, Patrick ^b   Lisowski, Vincent ^a  

^a UNIVERSITÉ DE MONTPELLIER   (France)

^b AIX MARSEILLE UNIVERSITY   (France)

^c AIX MARSEILLE UNIVERSITÉ   (France)

^d Mediterranée University   (France)

Author keywords

[No Author keywords available]

Indexed keywords

CENTRAL NERVOUS SYSTEM AGENTS; LIGAND; LOW DENSITY LIPOPROTEIN RECEPTOR;

ANIMAL CELL; ANIMAL EXPERIMENT; ANIMAL MODEL; ARTICLE; BINDING AFFINITY; BIOPANNING; BLOOD BRAIN BARRIER; CENTRAL NERVOUS SYSTEM; CONTROLLED STUDY; DRUG DELIVERY SYSTEM; DRUG RECEPTOR BINDING; DRUG TARGETING; ENDOTHELIUM; MICROSCOPY; MOLECULAR DOCKING; MOUSE; NONHUMAN; NUCLEAR MAGNETIC RESONANCE; PHAGE DISPLAY; PROTEIN DOMAIN; TRANSCYTOSIS;

ANIMALS; BLOOD-BRAIN BARRIER; FLUORESCENT DYES; HUMANS; LIGANDS; MAGNETIC RESONANCE SPECTROSCOPY; MICE; MICE, INBRED C57BL; MODELS, MOLECULAR; OLIGOPEPTIDES; PEPTIDES, CYCLIC; RECEPTORS, LDL; SPINAL CORD; STRUCTURE-ACTIVITY RELATIONSHIP; TRANSCYTOSIS;

EID: 84858039289     PISSN: 00222623     EISSN: 15204804     Source Type: Journal    
DOI: 10.1021/jm2014919     Document Type: Article

References (61)

1. Pardridge, W. M. Blood-brain barrier delivery Drug Discovery Today 2007, 12, 54-61
2. Pardridge, W. M. Why is the global CNS pharmaceutical market so under-penetrated? Drug Discovery Today 2002, 7, 5-7
3. Pardridge, W. M. Blood-brain barrier drug targeting: the future of brain drug development Mol. Interventions 2003, 3, 90-105
4. Nag, S. Morphology and Molecular Properties of Cellular Components of Normal Cerebral Vessels. In Blood-Brain Barrier; Humana Press: NJ, 2003; Vol. 89, pp 3-36.
5. de Boer, A. G.; Gaillard, P. J. Strategies to improve drug delivery across the blood-brain barrier Clin. Pharmacokinet. 2007, 46, 553-576
6. Grammas, P.; Martinez, J.; Miller, B. Cerebral microvascular endothelium and the pathogenesis of neurodegenerative diseases Expert Rev. Mol. Med. 2011, 13, e19
7. Lipinski, C. A.; Lombardo, F.; Dominy, B. W.; Feeney, P. J. Experimental and computational approaches to estimate solubility and permeability in drug discovery and development settings Adv. Drug Delivery Rev. 2001, 46, 3-26
8. Hitchcock, S. A.; Pennington, L. D. Structure-brain exposure relationships J. Med. Chem. 2006, 49, 7559-7583
9. Pardridge, W. M. Drug targeting to the brain Pharm. Res. 2007, 24, 1733-1744
10. Vlieghe, P.; Lisowski, V.; Martinez, J.; Khrestchatisky, M. Synthetic therapeutic peptides: science and market Drug Discovery Today 2010, 15, 40-56
11. Pardridge, W. M. The blood-brain barrier: bottleneck in brain drug development NeuroRx 2005, 2, 3-14
12. Pammolli, F.; Magazzini, L.; Riccaboni, M. The productivity crisis in pharmaceutical R&D Nat. Rev. Drug Discovery 2011, 10, 428-438
13. Paul, S. M.; Mytelka, D. S.; Dunwiddie, C. T.; Persinger, C. C.; Munos, B. H.; Lindborg, S. R.; Schacht, A. L. How to improve R&D productivity: the pharmaceutical industry's grand challenge Nat. Rev. Drug Discovery 2010, 9, 203-214
14. Patel, M. M.; Goyal, B. R.; Bhadada, S. V.; Bhatt, J. S.; Amin, A. F. Getting into the brain: approaches to enhance brain drug delivery CNS Drugs 2009, 23, 35-58
15. De Boer, A. G.; Van der Sandt, I. C.; Gaillard, P. J. The role of drug transporters at the blood-brain barrier Annu. Rev. Pharmacol. Toxicol. 2003, 43, 629-656
16. Jones, A. R.; Shusta, E. V. Blood-brain barrier transport of therapeutics via receptor-mediation Pharm. Res. 2007, 24, 1759-1771
17. Visser, C. C.; Stevanović, S.; Voorwinden, L. H.; van Bloois, L.; Gaillard, P. J.; Danhof, M.; Crommelin, D. J. A.; de Boer, A. G. Targeting liposomes with protein drugs to the blood-brain barrier in vitro Eur. J. Pharm. Sci. 2005, 25, 299-305
18. Wu, D.; Yang, J.; Pardridge, W. M. Drug targeting of a peptide radiopharmaceutical through the primate blood-brain barrier in vivo with a monoclonal antibody to the human insulin receptor J. Clin. Invest. 1997, 100, 1804-1812
19. Ulbrich, K.; Hekmatara, T.; Herbert, E.; Kreuter, J. Transferrin- and transferrin-receptor-antibody-modified nanoparticles enable drug delivery across the blood-brain barrier (BBB) Eur. J. Pharm. Biopharm. 2009, 71, 251-256
20. Olivier, J.-C. Drug transport to brain with targeted nanoparticles NeuroRx 2005, 2, 108-119
21. Demeule, M.; Régina, A.; Ché, C.; Poirier, J.; Nguyen, T.; Gabathuler, R.; Castaigne, J.-P.; Béliveau, R. Identification and design of peptides as a new drug delivery system for the brain J. Pharmacol. Exp. Ther. 2008, 324, 1064-1072
22. Demeule, M.; Currie, J.-C.; Bertrand, Y.; Ché, C.; Nguyen, T.; Régina, A.; Gabathuler, R.; Castaigne, J.-P.; Béliveau, R. Involvement of the low-density lipoprotein receptor-related protein in the transcytosis of the brain delivery vector angiopep-2 J. Neurochem. 2008, 106, 1534-1544
23. Gent, J.; Braakman, I. Low-density lipoprotein receptor structure and folding Cell. Mol. Life Sci. 2004, 61, 2461-2470
24. Chung, N. S.; Wasan, K. M. Potential role of the low-density lipoprotein receptor family as mediators of cellular drug uptake Adv. Drug Delivery Rev. 2004, 56, 1315-1334
25. Herz, J. The LDL receptor gene family: (un)expected signal transducers in the brain Neuron 2001, 29, 571-581
26. Brown, M. S.; Goldstein, J. L. Receptor-mediated endocytosis: insights from the lipoprotein receptor system Proc. Natl. Acad. Sci. U.S.A. 1979, 76, 3330-3337
27. Dehouck, B.; Fenart, L.; Dehouck, M. P.; Pierce, A.; Torpier, G.; Cecchelli, R. A new function for the LDL receptor: transcytosis of LDL across the blood-brain barrier J. Cell Biol. 1997, 138, 877-889
28. Ché, C.; Yang, G.; Thiot, C.; Lacoste, M.-C.; Currie, J.-C.; Demeule, M.; Régina, A.; Béliveau, R.; Castaigne, J.-P. New angiopep-modified doxorubicin (ANG1007) and etoposide (ANG1009) chemotherapeutics with increased brain penetration J. Med. Chem. 2010, 53, 2814-2824
29. Khrestchatisky, M.; David, M.; Molino, Y.; Vlieghe, P. Peptides Derivatives and Use Therefore as Carriers for Molecules in the Form of Conjugates. WO2010046588A1, 2010.
30. Armishaw, C. J.; Daly, N. L.; Nevin, S. T.; Adams, D. J.; Craik, D. J.; Alewood, P. F. Alpha-selenoconotoxins, a new class of potent α7 neuronal nicotinic receptor antagonists J. Biol. Chem. 2006, 281, 14136-14143
31. Cerovsky, V.; Wunsch, E.; Brass, J. Enzymatic semisynthesis of dicarba analogs of calcitonin Eur. J. Biochem. 1997, 247, 231-237
32. Hase, S.; Morikawa, T.; Sakakibara, S. Synthesis of a biologically active analog of deamino-8-arginine-vasopressin which does not contain a disulphide bond Experientia 1969, 25, 1239-1240
33. Kambayashi, Y.; Nakajima, S.; Ueda, M.; Inouye, K. A dicarba analog of beta-atrial natriuretic peptide (beta-ANP) inhibits guanosine 3′,5′-cyclic monophosphate production induced by alpha-ANP in cultured rat vascular smooth muscle cells FEBS Lett. 1989, 248, 28-34
34. Stymiest, J. L.; Mitchell, B. F.; Wong, S.; Vederas, J. C. Synthesis of biologically active dicarba analogues of the peptide hormone oxytocin using ring-closing metathesis Org. Lett. 2003, 5, 47-49
35. Nestor, J. J., Jr. The medicinal chemistry of peptides Curr. Med. Chem. 2009, 16, 4399-4418
36. Galande, A. K.; Trent, J. O.; Spatola, A. F. Understanding base-assisted desulfurization using a variety of disulfide-bridged peptides Pept. Sci. 2003, 71, 534-551
37. Robinson, A. J.; Elaridi, J.; Van Lierop, B. J.; Mujcinovic, S.; Jackson, W. R. Microwave-assisted RCM for the synthesis of carbocyclic peptides J. Pept. Sci. 2007, 13, 280-285
38. Jacobsen, O.; Klaveness, J.; Petter Ottersen, O.; Amiry-Moghaddam, M. R.; Rongved, P. Synthesis of cyclic peptide analogues of the 3(10) helical Pro138-Gly144 segment of human aquaporin-4 by olefin metathesis Org. Biomol. Chem. 2009, 7, 1599-1611
39. Hossain, M. A.; Rosengren, K. J.; Zhang, S.; Bathgate, R. A.; Tregear, G. W.; van Lierop, B. J.; Robinson, A. J.; Wade, J. D. Solid phase synthesis and structural analysis of novel A-chain dicarba analogs of human relaxin-3 (INSL7) that exhibit full biological activity Org. Biomol. Chem. 2009, 7, 1547-1553
40. Illesinghe, J.; Guo, C. X.; Garland, R.; Ahmed, A.; van Lierop, B.; Elaridi, J.; Jackson, W. R.; Robinson, A. J. Metathesis assisted synthesis of cyclic peptides Chem. Commun. 2009, 3, 295-297
41. Alexandra Le Chevalier, I.; Anna Maria, P.; Michael, C.; Paolo, R. Side chain-to-side chain cyclization by click reaction J. Pept. Sci. 2009, 15, 451-454
42. Jagasia, R.; Holub, J. M.; Bollinger, M.; Kirshenbaum, K.; Finn, M. G. Peptide cyclization and cyclodimerization by CuI-mediated azide-alkyne cycloaddition J. Org. Chem. 2009, 74, 2964-2974
43. Himo, F.; Lovell, T.; Hilgraf, R.; Rostovtsev, V. V.; Noodleman, L.; Sharpless, K. B.; Fokin, V. V. Copper(I)-catalyzed synthesis of azoles. DFT study predicts unprecedented reactivity and intermediates J. Am. Chem. Soc. 2004, 127, 210-216
44. Tornøe, C. W.; Christensen, C.; Meldal, M. Peptidotriazoles on solid phase: [1,2,3]-triazoles by regiospecific copper(I)-catalyzed 1,3-dipolar cycloadditions of terminal alkynes to azides J. Org. Chem. 2002, 67, 3057-3064
45. Touati-Jallabe, Y.; Chiche, L.; Hamze, A.; Aumelas, A.; Lisowski, V.; Berthomieu, D.; Martinez, J.; Hernandez, J. F. Cyclic peptides with a diversely substituted guanidine bridge: solid-phase synthesis and structural analysis Chem.-Eur. J. 2011, 17, 2566-2570
46. Breznik, M.; Grdadolnik, S. G.; Giester, G.; Leban, I.; Kikelj, D. Influence of chirality of the preceding acyl moiety on the cis/trans ratio of the proline peptide bond J. Org. Chem. 2001, 66, 7044-7050
47. Jacobson, K. A.; Marr-Leisy, D.; Rosenkranz, R. P.; Verlander, M. S.; Melmon, K. L.; Goodman, M. Conjugates of catecholamines. 1. N -Alkyl-functionalized carboxylic acid congeners and amides related to isoproterenol J. Med. Chem. 1983, 26, 492-499
48. Martinez, J.; Bali, J. P.; Rodriguez, M.; Castro, B.; Magous, R.; Laur, J.; Lignon, M. F. Synthesis and biological activities of some pseudo-peptide analogues of tetragastrin: the importance of the peptide backbone J. Med. Chem. 1985, 28, 1874-1879
49. Doulut, S.; Rodriguez, M.; Lugrin, D.; Vecchini, F.; Kitabgi, P.; Aumelas, A.; Martinez, J. Reduced peptide bond pseudopeptide analogues of neurotensin Pept. Res. 1992, 5, 30-38
50. Lugrin, D.; Vecchini, F.; Doulut, S.; Rodriguez, M.; Martinez, J.; Kitabgi, P. Reduced peptide bond pseudopeptide analogues of neurotensin: binding and biological activities, and in vitro metabolic stability Eur. J. Pharmacol. 1991, 205, 191-198
51. 2NH]Gly- d -Phe-Pro] Int. J. Pept. Protein Res. 1993, 41, 204-206
52. Fehrentz, J. A.; Heitz, A.; Castro, B. Synthesis of aldehydic peptides inhibiting renin Int. J. Pept. Protein Res. 1985, 26, 236-241
53. Ganneau, C.; Moulin, A.; Demange, L.; Martinez, J.; Fehrentz, J. A. The epimerization of peptide aldehydes: a systematic study J. Pept. Sci. 2006, 12, 497-501
54. Lefevre, C.; Kang, H. C.; Haugland, R. P.; Malekzadeh, N.; Arttamangkul, S.; Haugland, R. P. Texas Res-X and Rhodamine Red-X, new derivatives of sulforhodamine 101 and Lissamine Rhodamine B with improved labeling and fluorescence properties Bioconjugate Chem. 1996, 7, 482-489
55. Brooks, B. R.; Brooks, C. L.; Mackerell, A. D.; Nilsson, L.; Petrella, R. J.; Roux, B.; Won, Y.; Archontis, G.; Bartels, C.; Boresch, S.; Caflisch, A.; Caves, L.; Cui, Q.; Dinner, A. R.; Feig, M.; Fischer, S.; Gao, J.; Hodoscek, M.; Im, W.; Kuczera, K.; Lazaridis, T.; Ma, J.; Ovchinnikov, V.; Paci, E.; Pastor, R. W.; Post, C. B.; Pu, J. Z.; Schaefer, M.; Tidor, B.; Venable, R. M.; Woodcock, H. L.; Wu, X.; Yang, W.; York, D. M.; Karplus, M. CHARMM: the biomolecular simulation program J. Comput. Chem. 2009, 30, 1545-1614
56. Floquet, N.; Héry-Huynh, S.; Dauchez, M.; Derreumaux, P.; Tamburro, A. M.; Alix, A. J. P. Structural characterization of VGVAPG, an elastin-derived peptide Biopolymers 2004, 76, 266-280
57. Humphrey, W.; Dalke, A.; Schulten, K. VMD: visual molecular dynamics J. Mol. Graphics 1996, 14 (33-38) 27-28
58. Case, D. A.; Cheatham, T. E.; Darden, T.; Gohlke, H.; Luo, R.; Merz, K. M.; Onufriev, A.; Simmerling, C.; Wang, B.; Woods, R. J. The Amber biomolecular simulation programs J. Comput. Chem. 2005, 26, 1668-1688
59. Trott, O.; Olson, A. J. AutoDock Vina: improving the speed and accuracy of docking with a new scoring function, efficient optimization, and multithreading J. Comput. Chem. 2010, 31, 455-461
60. Kwon, H. J.; Lagace, T. A.; McNutt, M. C.; Horton, J. D.; Deisenhofer, J. Molecular basis for LDL receptor recognition by PCSK9 Proc. Natl. Acad. Sci. U.S.A. 2008, 105, 1820-1825
61. Dray, C.; Rougon, G.; Debarbieux, F. Quantitative analysis by in vivo imaging of the dynamics of vascular and axonal networks in injured mouse spinal cord Proc. Natl. Acad. Sci. U.S.A. 2009, 106, 9459-9464