Arylsulfatase a bound to poly(butyl cyanoacrylate) nanoparticles for enzyme replacement therapy - Physicochemical evaluation

Pharmazie

Volumn 69, Issue 7, 2014, Pages 518-524

  Muhlstein A ^a   Gelperina, S ^b   Shipulo, E ^b   Maksimenko, O ^b   Kreuter, Jörg ^a  

^a GOETHE UNIVERSITY   (Germany)

^b Nanosystem LTD   (Russian Federation)

Author keywords

[No Author keywords available]

Indexed keywords

CEREBROSIDE SULFATASE; NANOPARTICLE; POLY(BUTYL CYANOACRYLATE); POLYSORBATE 80; SUCROSE; UNCLASSIFIED DRUG; 4-NITROCATECHOL SULFATE; CATECHOL DERIVATIVE; DRUG CARRIER; DYES, REAGENTS, INDICATORS, MARKERS AND BUFFERS; ENBUCRILATE; EXCIPIENT; PROTEIN BINDING; TISSUE ADHESIVE;

ADSORPTION; ARTICLE; DISPERSION; DRUG RELEASE; ENZYME ACTIVITY; ENZYME REPLACEMENT; FREEZE DRYING; PARTICLE SIZE; PHYSICAL CHEMISTRY; STATIC ELECTRICITY; STEREOSPECIFICITY; ZETA POTENTIAL; CHEMISTRY; DRUG FORMULATION; ELECTROCHEMISTRY; GAS CHROMATOGRAPHY; GEL CHROMATOGRAPHY; KINETICS; PROCEDURES; SOLUBILITY; SURFACE PROPERTY;

CATECHOLS; CEREBROSIDE-SULFATASE; CHROMATOGRAPHY, GAS; CHROMATOGRAPHY, GEL; DRUG CARRIERS; DRUG COMPOUNDING; ELECTROCHEMISTRY; ENBUCRILATE; ENZYME REPLACEMENT THERAPY; EXCIPIENTS; FREEZE DRYING; INDICATORS AND REAGENTS; KINETICS; NANOPARTICLES; PROTEIN BINDING; SOLUBILITY; SURFACE PROPERTIES; TISSUE ADHESIVES;

EID: 84904576442     PISSN: 00317144     EISSN: None     Source Type: Journal    
DOI: 10.1691/ph.2014.3250     Document Type: Article

References (46)

1. Abdelwahed W, Degobert G, Stainmesse S, Fessi H (2006) Freeze-drying of nanoparticles: formulation, process and storage considerations. Adv Drug Deliv Rev 58: 1688-1713. (Pubitemid 44827432)
2. Agogbua SI, Wynn CH (1976) Purification and properties of arylsulphatase B of human liver. Biochem J 153: 415-421.
3. Alyautdin RN, Petrov VE, Langer K, Berthold A, Kharkevich DA, Kreuter J (1997) Delivery of loperamide across the blood-brain barrier with polysorbate 80-coated polybutylcyanoacrylate nanoparticles. Pharm Res 14: 325-328. (Pubitemid 27160944)
4. Alyautdin RN, Tezikov EB, Ramge P, Kharkevich DA, Begley DJ, Kreuter J (1998) Significant entry of tubocurarine into the brain of rats by adsorption to polysorbate 80-coated polybutylcyanoacrylate nanoparticles: an in situ brain perfusion study. J Microencapsul 15: 67-74. (Pubitemid 28031791)
5. Anhorn MG, Mahler HC, Langer K (2008) Freeze-drying of human serum albumin (HSA) nanoparticles with different excipients. Int J Pharm 363: 162-169.
6. Baum H, Dodgson KS, Spencer B (1959) The assay of arylsulphatases A and B in human urine. Clin Chim Acta 4: 453-455.
7. Beck M (2007) New therapeutic options for lysosomal storage disorders: enzyme replacement, small molecules and gene therapy. Hum Genet 121: 1-22.
8. Beck M (2010) Therapy for lysosomal storage disorders. IUBMB Life 62: 33-40.
9. Bond CS, Clements PR, Ashby SJ, Collyer CA, Harrop SJ, Hopwood JJ, Guss JM (1997) Structure of a human lysosomal sulfatase. Structure 5: 277-289. (Pubitemid 27158733)
10. Carpenter JF, Pikal MJ, Chang BS, Randolph TW (1997) Rational design of stable lyophilized protein formulations: some practical advice. Pharm Res 14: 969-975. (Pubitemid 27371393)
11. Cha P, Krishnan A, Fiore VF, Vogler EA (2008) Interfacial energetics of protein adsorption from aqueous buffer to surfaces with varying hydrophilicity. Langmuir 24: 2553-2563. (Pubitemid 351542656)
12. Couvreur P, Kante B, Roland M, Guiot P, Bauduin P, Speiser P (1979) Polycyanoacrylate nanocapsules as potential lysosomotropic carriers: preparation, morphological and sorptive properties. J Pharm Pharmacol 31: 331-332. (Pubitemid 9207599)
13. Dali, C. and Lund, A.M. (2009) Intravenous enzyme replacement therapy for metachromatic leukodystrophy (MLD). Abstract from the Annual Clinical Genetics (ACMG) Meeting on 25 March 2009 in Tampa, FL. Available at: http:// mldfoundation.org/pdfs/Shire-ERT-P12-2009-02.pdf.
14. Fischer S, Hoernschemeyer J, Mahler HC (2008) Glycation during storage and administration of monoclonal antibody formulations. Eur J Pharm Biopharm 70: 42-50.
15. Ghosh D (2007) Human sulfatases: a structural perspective to catalysis. Cell Mol Life Sci 64: 2013-2022.
16. Gibson GJ, Saccone GT, Brooks DA, Clements PR, Hopwood JJ (1987) Human N-acetylgalactosamine-4-sulphate sulphatase. Purification, monoclonal antibody production and native and subunit Mr values. Biochem J 248: 755-764. (Pubitemid 18012725)
17. Gieselmann V (1995) Lysosomal storage diseases. Biochim Biophys Acta 1270: 103-136.
18. Gieselmann V, Krageloh-Mann I (2010) Metachromatic leukodystrophy - an update. Neuropediatrics 41: 1-6.
19. Heim P, Claussen M, Hoffmann B, Conzelmann E, Gartner J, Harzer K, Hunneman DH, Kohler W, Kurlemann G, Kohlschutter A (1997) Leukodystrophy incidence in Germany. Am J Med Genet 71: 475-478. (Pubitemid 27371851)
20. Jiang S, Nail SL (1998) Effect of process conditions on recovery of protein activity after freezing and freeze-drying. Eur J Pharm Biopharm 45: 249-257. (Pubitemid 28270813)
21. Koutsopoulos S, Patzsch K, Bosker WT, Norde W (2007) Adsorption of trypsin on hydrophilic and hydrophobic surfaces. Langmuir 23: 2000-2006. (Pubitemid 46406154)
22. Kolodny EH. 1989. Metachromatic leukodystrophy and multiple sulfatase deficiency: Sulfatide lipidosis. In: Scriver CR, Beaudet AL, Sly WS, Valle DE, eds. The metabolic basis of inherited disease. New York: p.1721-50.
23. Kreuter J, Petrov VE, Kharkevich DA, Alyautdin RN (1997) Influence of the type of surfactant on the analgesic effects induced by the peptide dalargin after its delivery across the blood-brain barrier using surfactant-coated nanoparticles. J Control Release 49: 81-87. (Pubitemid 27417903)
24. Kreuter J (2001) Nanoparticulate systems for brain delivery of drugs. Adv Drug Deliv Rev 47: 65-81.
25. Krishnan A, Liu YH, Cha P, Allara D, Vogler EA (2006) Interfacial energetics of globular-blood protein adsorption to a hydrophobic interface from aqueous-buffer solution. J R Soc Interface 3: 283-301.
26. Kufleitner J,Wagner S,Worek F, von Briesen H, Kreuter J (2010) Adsorption of obidoxime onto human serum albumin nanoparticles: drug loading, particle size and drug release. J Microencapsul 27: 506-513.
27. Kurakhmaeva KB, Djindjikhashvili IA, Petrov VE, Balabanyan VU, Voronina TA, Trofimov SS, Kreuter J, Gelperina S, Begley D, Alyautdin RN (2009) Brain targeting of nerve growth factor using poly(butyl cyanoacrylate) nanoparticles. J Drug Target 17: 564-574.
28. Langer K, Seegmüller E, Zimmer A, Kreuter J (1994) Characterisation of polybutylcyanoarylate nanoparticles: I. Quantification of PCBA polymer and dextrans. Int J Pharm110: 21-27.
29. Lukatela G, Krauss N, Theis K, Selmer T, Gieselmann V, von Figura K, Saenger W (1998) Crystal structure of human arylsulfatase A: the aldehyde function and the metal ion at the active site suggest a novel mechanism for sulfate ester hydrolysis. Biochemistry 37: 3654-3664. (Pubitemid 28162920)
30. Lundqvist M, Stigler J, Cedervall T, Berggard T, Flanagan MB, Lynch I, Elia G, Dawson K (2011) The evolution of the protein corona around nanoparticles: a test study. ACS Nano 5: 7503-7509.
31. McGonigle P (2012) Peptide therapeutics for CNS indications. Biochem Pharmacol 83: 559-566.
32. Moghimi SM, Hunter AC, Murray JC (2001) Long-circulating and target-specific nanoparticles: theory to practice. Pharmacol Rev 53: 283-318. (Pubitemid 32476118)
33. Mollmann SH, Jorgensen L, Bukrinsky JT, Elofsson U, Norde W, Frokjaer S (2006) Interfacial adsorption of insulin conformational changes and reversibility of adsorption. Eur J Pharm Sci 27: 194-204. (Pubitemid 41831254)
34. Mühlstein A, Gelperina S, Kreuter J (2013) Development of nanoparticle-bound arylsulfatase B for enzyme replacement therapy of mucopolysaccharidosis VI. Pharmazie, 68: 549-554.
35. Pardridge WM (2005) Molecular biology of the blood-brain barrier. Mol Biotechnol 30: 57-70.
36. Pardridge WM (2007a) Blood-brain barrier delivery. Drug Discov Today 12: 54-61.
37. Pardridge WM (2007b) Blood-brain barrier delivery of protein and non-viral gene therapeutics with molecular Trojan horses. J Control Release 122: 345-348.
38. Poorthuis BJ, Wevers RA, Kleijer WJ, Groener JE, de Jong JG, van Weely S, Niezen-Koning KE, van Diggelen OP (1999) The frequency of lysosomal storage diseases in The Netherlands. Hum Genet 105: 151-156. (Pubitemid 29396979)
39. Rubin LL, Staddon JM (1999) The cell biology of the blood-brain barrier. Annu Rev Neurosci 22: 11-28. (Pubitemid 29144879)
40. Scheld WM (1989) Drug delivery to the central nervous system: general principles and relevance to therapy for infections of the central nervous system. Rev Infect Dis 11 Suppl 7: S1669-1690.
41. Sommerlade HJ, Selmer T, Ingendoh A, Gieselmann V, von Figura K, Neifer K, Schmidt B (1994) Glycosylation and phosphorylation of arylsulfatase A. J Biol Chem 269: 20977-20981. (Pubitemid 24266463)
42. Soppimath KS, Aminabhavi TM, Kulkarni AR, Rudzinski WE (2001) Biodegradable polymeric nanoparticles as drug delivery devices. J Control Release 70: 1-20. (Pubitemid 32144788)
43. von Figura K, Gieselmann V, Jaeken J. 2001. Metachromatic Leukodystrophy. In: Scriver C, Beaudet A, Sly W, Valle D, Childs B, Kinzler K, Vogelstein B, eds. The Metabolic and Molecular Bases of Inherited Disease. New York: p. 3695-3724.
44. Wohlfart S, Gelperina S, Kreuter J (2012) Transport of drugs across the blood-brain barrier by nanoparticles. J Control Release 161: 264-273.
45. Yaghootfam A, Schestag F, Dierks T, Gieselmann V (2003) Recognition of arylsulfatase A and B by the UDP-N-acetylglucosamine: lysosomal enzyme N-acetylglucosamine-phosphotransferase. J Biol Chem 278: 32653-32661. (Pubitemid 37055704)
46. Zensi A, Begley D, Pontikis C, Legros C, Mihoreanu L, Wagner S, Buchel C, von Briesen H, Kreuter J (2009) Albumin nanoparticles targeted with Apo E enter the CNS by transcytosis and are delivered to neurones. J Control Release137: 78-86.