General considerations regarding the in vitro and in vivo properties of block copolymer micelle products and their evaluation

Journal of Controlled Release

Volumn 210, Issue , 2015, Pages 76-83

  Sakai Kato, Kumiko ^a   Nishiyama, Nobuhiro ^b   Kozaki, Masato ^c   Nakanishi, Takeshi ^d   Matsuda, Yoshihiro ^e   Hirano, Mai ^e   Hanada, Hiroyuki ^f   Hisada, Shigeru ^g   Onodera, Hiroshi ^e   Harashima, Hideyoshi ^h   Matsumura, Yasuhiro ⁱ   Kataoka, Kazunori ^j,k   Goda, Yukihiro ^a   Okuda, Haruhiro ^a   Kawanishi, Toru ^a  

^a NATIONAL INSTITUTE OF HEALTH SCIENCES   (Japan)

^b TOKYO INSTITUTE OF TECHNOLOGY   (Japan)

^c KOWA COMPANY LTD   (Japan)

^d NIPPON KAYAKU CO LTD   (Japan)

^e PHARMACEUTICALS AND MEDICAL DEVICES AGENCY   (Japan)

^f NanoCarrier Co Ltd   (Japan)

^g Grelan Pharmaceutical Co Ltd   (Japan)

^h HOKKAIDO UNIVERSITY   (Japan)

ⁱ NATIONAL CANCER CENTER HOSPITAL EAST   (Japan)

^j UNIVERSITY OF TOKYO   (Japan)

^k UNIVERSITY OF TOKYO   (Japan)

more..

Author keywords

Block copolymer micelle; Drug development; Nanotechnology; Regulatory affairs

Indexed keywords

AMINO ACIDS; COPOLYMERS; DRUG PRODUCTS; MICELLES; NANOTECHNOLOGY; PHARMACOKINETICS; POLYETHYLENE GLYCOLS;

BLOCK CO-POLYMER MICELLES; CLINICAL INVESTIGATION; CLINICAL PHARMACOKINETICS; DISTRIBUTION PROFILES; DRUG DEVELOPMENT; HYDROPHILIC POLYMERS; PHYSICOCHEMICAL PROPERTY; REGULATORY AFFAIRS;

BLOCK COPOLYMERS;

BLOCK COPOLYMER MICELLE PRODUCT; COPOLYMER; NANOPARTICLE; UNCLASSIFIED DRUG; MICELLE; POLYMER;

ABSORPTION SPECTROPHOTOMETRY; CHEMICAL BINDING; CHEMICAL STRUCTURE; DRUG MANUFACTURE; ENCAPSULATION; EVALUATION STUDY; HUMAN; IN VITRO STUDY; IN VIVO STUDY; MASS SPECTROMETRY; MICELLE; MICELLIZATION; NANOTECHNOLOGY; NONHUMAN; PARTICLE SIZE; PHARMACOKINETICS; PHARMACOLOGY; PHYSICAL CHEMISTRY; PRIORITY JOURNAL; PROCESS CONTROL; QUALITY CONTROL; REVIEW; SOLUBILIZATION; SPECTROFLUOROMETRY; SURFACE PROPERTY; ANIMAL; CHEMISTRY; DRUG DELIVERY SYSTEM;

ANIMALS; DRUG DELIVERY SYSTEMS; HUMANS; MICELLES; POLYMERS;

EID: 84929591367     PISSN: 01683659     EISSN: 18734995     Source Type: Journal    
DOI: 10.1016/j.jconrel.2015.05.259     Document Type: Review

References (76)

1. Y. Barenholz Doxil® - the first FDA-approved nano-drug: lessons learned J. Control. Release 160 2012 117 134
2. R. Duncan, and R. Gaspar Nanomedicine(s) under the microscope Mol. Pharm. 8 2011 2101 2141
3. Guidance for Industry Considering whether an FDA-regulated product involves the application of nanotechnology 2014 U.S. Department of Health and Human Services, Food and Drug Administration
4. M. Ferrari Cancer nanotechnology: opportunities and challenges Nat. Rev. Cancer 5 2005 161 171
5. L. Gao, G. Liu, J. Ma, X. Wang, L. Zhou, and X. Li Drug nanocrystals: in vivo performances J. Control. Release 160 2012 418 430
6. R. Shegokar, and R.H. Müller Nanocrystals: industrially feasible multifunctional formulation technology for poorly soluble actives Int. J. Pharm. 399 2010 129 139
7. I. Lynch, A. Salvati, and K.A. Dawson Protein-nanoparticle interactions: what does the cell see? Nat. Nanotechnol. 4 2009 546 547
8. M.P. Monopoli, C. Aberg, A. Salvati, and K.A. Dawson Biomolecular coronas provide the biological identity of nanosized materials Nat. Nanotechnol. 12 2012 779 786
9. H. Harashima, S. Iida, Y. Urakami, M. Tsuchihashi, and H. Kiwada Optimization of antitumor effect of liposomally encapsulated doxorubicin based on simulations by pharmacokinetic/pharmacodynamic modeling J. Control. Release 61 1999 93 106
10. Z. Cheng, A.A.L. Zaki, J.Z. Hui, V.R. Muzykantov, and A. Tsourkas Multifunctional nanoparticles: cost versus benefit of adding targeting and imaging capabilities Science 338 2012 903 910
11. F. Perche, and V.P. Torchilin Recent trends in multifunctional liposomal nanocarriers for enhanced tumor targeting J. Drug Deliv. 2013 705265
12. H. Cabral, and K. Kataoka Progress of drug-loaded polymeric micelles into clinical studies J. Control. Release 190 2014 465 476
13. Y. Matsumura The drug discovery by nanomedicine and its clinical experience Jpn. J. Clin. Oncol. 44 2014 515 525
14. V.P. Torchilin PEG-based micelles as carriers of contrast agents for different imaging modalities Adv. Drug Deliv. 54 2002 235 252
15. A. Kabanov, J. Zhu, and V. Alakhov Pluronic block copolymers for gene delivery Adv. Genet. 53 2005 231 261
16. N. Nishiyama, and K. Kataoka Nanostructured devices based on block copolymer assemblies for drug delivery: designing structures for enhanced drug function Adv. Polym. Sci. 193 2006 67 101
17. English version of the latest version of original policy is available as Japan's Science and Technology Basic Policy Report Available at: http://www8.cao.go.jp/cstp/english/basic/4th-BasicPolicy.pdf Council for Science and Technology Policy, December 24, 2010 2010 (Accessed March 22,2015)
18. Japan Revitalization Strategy Available at: http://www.kantei.go.jp/jp/singi/keizaisaisei/pdf/en-saikou-jpn-hon.pdf 2013 (Assessed March 22,2015)
19. The Healthcare Policy Available at: http://www.kantei.go.jp/jp/singi/kenkouiryou/en/pdf/policy.pdf (Assessed March 22,2015)
20. Joint MHLW/EMA reflection paper on the development of block copolymer micelle medicinal products January 10, 2014, PFSB/ELD Notification No. 0110-1PFSB/ELD Notification No.0110-4, January 10, 2014
21. European Medicines Agency Reflection paper on the pharmaceutical development of intravenous medicinal products containing active substances solubilised in micellar systems EMA/CHMP/QWP/799402/2011micellar systems, EMA/CHMP/QWP/799402/2011, March 5, 2012
22. N. Nishiyama, and K. Kataoka Current state, achievements, and future prospects of polymeric micelles as nanocarriers for drug and gene delivery Pharmacol. Ther. 112 2006 630 648
23. G.S. Kwon, M. Naito, M. Yokoyama, T. Okano, Y. Sakurai, and K. Kataoka Physical entrapment of adriamycin in AB block copolymer micelles Pharm. Res. 12 1995 192 195
24. T. Hamaguchi, Y. Matsumura, M. Suzuki, K. Shimizu, R. Goda, I. Nakamura, I. Nakatomi, M. Yokoyama, K. Kataoka, and T. Kakizoe NK105, a paclitaxel-incorporating micellar nanoparticle formulation, can extend in vivo antitumour activity and reduce the neurotoxicity of paclitaxel Br. J. Cancer 92 2005 1240 1246
25. S. Danson, D. Ferry, V. Alakhov, J. Margison, D. Kerr, D. Jowle, M. Brampton, G. Halbert, and M. Ranson Phase I dose escalation and pharmacokinetic study of pluronic polymer-bound doxorubicin (SP1049C) in patients with advanced cancer Br. J. Cancer 90 2004 2085 2091
26. T.Y. Kim, D.W. Kim, J.Y. Chung, S.G. Shin, S.C. Kim, D.S. Heo, N.K. Kim, and Y.J. Bang Phase I and pharmacokinetic study of Genexol-PM, a cremophor-free, polymeric micelle-formulated paclitaxel, in patients with advanced malignancies Clin. Cancer Res. 10 2004 3708 3716
27. Y. Matsumura Preclinical and clinical studies of NK012, an SN-38-incorporating polymeric micelles, which is designed based on EPR effect Adv. Drug Deliv. Rev. 63 2011 184 192
28. N. Nishiyama, S. Okazaki, H. Cabral, M. Miyamoto, Y. Kato, Y. Sugiyama, K. Nishio, Y. Matsumura, and K. Kataoka Novel cisplatin-incorporated polymeric micelles can eradicate solid tumors in mice Cancer Res. 63 2003 8977 8983
29. M. Murakami, H. Cabral, Y. Matsumoto, S. Wu, M.R. Kano, T. Yamori, N. Nishiyama, and K. Kataoka Improving drug potency and efficacy by nanocarrier-mediated subcellular targeting Sci. Transl. Med. 3 2011 64ra2
30. R.J. Christie, Y. Matsumoto, K. Miyata, T. Nomoto, S. Fukushima, K. Osada, J. Halnaut, F. Pittella, H.J. Kim, N. Nishiyama, and K. Kataoka Targeted polymeric micelles for siRNA treatment of experimental cancer by intravenous injection ACS Nano 6 2012 5174 5189
31. M. Harada, M. Ohuchi, T. Hayashi, and Y. Kato Prolonged circulation and in vivo efficacy of recombinant human granulocyte colony-stimulating factor encapsulated in polymeric micelles J. Control. Release 156 2011 101 108
32. A.M. Jhaveri, and V.P. Torchilin Multifunctional polymeric micelles for delivery of drugs and siRNA Front. Pharmacol. 5 2014 77
33. M.J. Ernsting, M. Murakami, A. Roy, and S.D. Li Factors controlling the pharmacokinetics, biodistribution and intratumoral penetration of nanoparticles J. Control. Release 172 2013 782 794
34. M. Yokoyama, M. Miyauchi, N. Yamada, T. Okano, Y. Sakurai, K. Kataoka, and S. Inoue Characterization and anticancer activity of the micelle-forming polymeric anticancer drug adriamycin-conjugated poly(ethylene glycol)-poly(aspartic acid) block copolymer Cancer Res. 50 1990 1693 1700
35. J.W. Valle, A. Armstrong, C. Newman, V. Alakhov, G. Pietrzynski, J. Brewer, S. Campbell, P. Corrie, E.K. Rowinsky, and M. Ranson A phase 2 study of SP1049C, doxorubicin in P-glycoprotein-targeting pluronics, in patients with advanced adenocarcinoma of the esophagus and gastroesophageal junction Invest. New Drugs 29 2011 1029 1037
36. E.V. Batrakova, S. Li, V.Y. Alakhov, D.W. Miller, and A.V. Kabanov Optimal structure requirements for pluronic block copolymers in modifying P-glycoprotein drug efflux transporter activity in bovine brain microvessel endothelial cells J. Pharmacol. Exp. Ther. 304 2003 845 854
37. ICH Harmonised Tripartite Guideline Specifications: test procedures and acceptance criteria for biotechnological/biological products Q6B PMSB/ELD Notification No. 571, May 1, 2001
38. ICH Harmonised Tripartite Guideline Pharmaceutical development Q8 (R2) PFSB/ELD Notification No. 0628-1, June 28, 2010
39. ICH Harmonised Tripartite Guideline Development and manufacture of drug substances (chemical entities and biotechnological/biological entities) Q11 PFSB/ELD Notification No. 0710-9, July 10, 2014
40. ICH Harmonised Tripartite Guideline Note for guidance on comparability of biotechnological/biological products subject to changes in their manufacturing process Q5E PFSB/ELD Notification No. 0426001, April 26, 2005
41. F. Ehmann, K. Sakai-Kato, R. Duncan, D. Pérez de la Ossa, R. Pita Hernán, J.M. Vidal, A. Kohli, L. Tothfalusi, A. Sanh, S. Tinton, J.L. Robert, L.B. Silva, and M.P. Amati Next-generation nanomedicines and nanosimilars: EU regulators' initiatives relating to the development and evaluation of nanomedicines Nanomedicine (Lond.) 8 2013 849 856
42. L.W. Seymour, R. Duncan, J. Strohalm, and J. Kopecek Effect of molecular weight (Mw) of N-(2-hydroxypropyl) methacrylamide copolymers on body distribution and rate of excretion after subcutaneous, intraperitoneal, and intravenous administration to rats J. Biomed. Mater. Res. 21 1987 1341 1358
43. S. Stolnik, L. Illum, and S.S. Davis Long circulating microparticulate drug carriers Adv. Drug Deliv. Rev. 16 1995 195 214
44. V.C. Mosqueira, P. Legrand, A. Gulik, O. Bourdon, R. Gref, D. Labarre, and G. Barratt Relationship between complement activation, cellular uptake and surface physicochemical aspects of novel PEG-modified nanocapsules Biomaterials 22 2001 2967 2979
45. Y. Matsumura, and H. Maeda A new concept for macromolecular therapeutics in cancer chemotherapy: mechanism of tumoritropic accumulation of proteins and the antitumor agent smancs Cancer Res. 46 1986 6387 6392
46. H. Nakamura, F. Jun, and H. Maeda Development of next-generation macromolecular drugs based on the EPR effect: challenges and pitfalls Expert Opin. Drug Deliv. 12 2015 53 64
47. S. Kim, Y. Shi, J.Y. Kim, K. Park, and J.X. Cheng Overcoming the barriers in micellar drug delivery: loading efficiency, in vivo stability, and micelle-cell interaction Expert Opin. Drug Deliv. 7 2010 49 62
48. T. Hamaguchi, K. Kato, H. Yasui, C. Morizane, M. Ikeda, H. Ueno, K. Muro, Y. Yamada, T. Okusaka, K. Shirao, Y. Shimada, H. Nakahama, and Y. Matsumura A phase I and pharmacokinetic study of NK105, a paclitaxel-incorporating micellar nanoparticle formulation Br. J. Cancer 97 2007 170 176
49. European Medicines Agency Reflection paper on the data requirements for intravenous liposomal products developed with reference to an innovator liposomal product EMA/CHMP/806058/2009/Rev. 02 February 21, 2013
50. H. Uchino, Y. Matsumura, T. Negishi, F. Koizumi, T. Hayashi, T. Honda, N. Nishiyama, K. Kataoka, S. Naito, and T. Kakizoe Cisplatin-incorporating polymeric micelles (NC-6004) can reduce nephrotoxicity and neurotoxicity of cisplatin in rats Br. J. Cancer 93 2005 678 687
51. Y. Mochida, H. Cabral, Y. Miura, F. Albertini, S. Fukushima, K. Osada, N. Nishiyama, and K. Kataoka Bundled assembly of helical nanostructures in polymeric micelles loaded with platinum drugs enhancing therapeutic efficiency against pancreatic tumor ACS Nano 8 2014 6724 6738
52. K. Kataoka, M. Yokoyama, T. Okano, and Y. Sakurai Block copolymers micelles as vehicles for drug delivery J. Control. Release 24 1993 119 132
53. H. Cabral, M. Murakami, H. Hojo, Y. Terada, M.R. Kano, U.I. Chung, N. Nishiyama, and K. Kataoka Targeted therapy of spontaneous murine pancreatic tumors by polymeric micelles prolongs survival and prevents peritoneal metastasis Proc. Natl. Acad. Sci. U. S. A. 110 2013 11397 11402
54. A.S. Mikhail, S. Eetezadi, and C. Allen Multicellular tumor spheroids for evaluation of cytotoxicity and tumor growth inhibitory effects of nanomedicines in vitro: a comparison of docetaxel-loaded block copolymer micelles and taxotere (R) PLoS One 8 2013 e62630
55. H. Cabral, Y. Matsumoto, K. Mizuno, Q. Chen, M. Murakami, M. Kimura, Y. Terada, M.R. Kano, K. Miyazono, M. Uesaka, N. Nishiyama, and K. Kataoka Accumulation of sub-100 nm polymeric micelles in poorly permeable tumours depends on size Nat. Nanotechnol. 6 2011 815 823
56. M. Yasunaga, M. Furuta, K. Ogata, Y. Koga, Y. Yamamoto, M. Takigahira, and Y. Matsumura The significance of microscopic mass spectrometry with high resolution in the visualisation of drug distribution Sci. Rep. 3 2013 3050
57. ICH Harmonised Tripartite Guideline, Nonclinical Evaluation for Anticancer Pharmaceuticals S9. PFSB/ELD Notification No. 0604-1, June 4, 2010
58. I.C.H. Harmonised Tripartite Guideline Preclinical Safety Evaluation of Biotechnology-Derived Pharmaceuticals S6(R1) PFSB/ELD Notification No. 0323-1 March 23, 2012
59. J. Szebeni Hemocompatibility testing for nanomedicines and biologicals: predictive assays for complement mediated infusion reactions Eur. J. Nanomed. 4 2012 33 53
60. R. Plummer, R.H. Wilson, H. Calvert, A.V. Boddy, M. Griffin, J. Sludden, M.J. Tilby, M. Eatock, D.G. Pearson, C.J. Ottley, Y. Matsumura, K. Kataoka, and T. Nishiya A Phase I clinical study of cisplatin-incorporated polymeric micelles (NC-6004) in patients with solid tumours Br. J. Cancer 104 2011 593 598
61. H. Hussain, K.Y. Mya, and C. He Self-assembly of brush-like poly[poly(ethylene glycol) methyl ether methacrylate] synthesized via aqueous atom transfer radical polymerization Langmuir 24 2008 13279 13286
62. J. Cao, A. Lu, C. Li, M. Cai, Y. Chen, S. Li, and X. Luo Effect of architecture on the micellar properties of poly(epsilon-caprolactone) containing sulfobetaines Colloids Surf. B Biointerfaces 112 2013 35 41
63. Y. Sanada, I. Akiba, K. Sakurai, K. Shiraishi, M. Yokoyama, E. Mylonas, N. Ohta, N. Yagi, Y. Shinohara, and Y. Amemiya Hydrophobic molecules infiltrating into the poly(ethylene glycol) domain of the core/shell interface of a polymeric micelle: evidence obtained with anomalous small-angle X-ray scattering J. Am. Chem. Soc. 135 2013 2574 2582
64. S.K. Filippov, J.M. Franklin, P.V. Konarev, P. Chytil, T. Etrych, A. Bogomolova, M. Dyakonova, C.M. Papadakis, A. Radulescu, K. Ulbrich, P. Stepanek, and D.I. Svergun Hydrolytically degradable polymer micelles for drug delivery: a SAXS/SANS kinetic study Biomacromolecules 14 2013 4061 4070
65. P.D. Petrov, M. Drechsler, and A.H.E. Müller Self-assembly of asymmetric poly(ethylene oxide)-block-poly(n-butyl acrylate) diblock copolymers in aqueous media to unexpected morphologies J. Phys. Chem. B 113 2009 4218 4225
66. Y. Tian, W.C. Wu, C.Y. Chen, T. Strovas, Y. Li, Y. Jin, F. Su, D.R. Meldrum, and A.K.Y. Jen 2,1,3-Benzothiadiazole (BTD)-moiety-containing red emitter conjugated amphiphilic poly(ethylene glycol)-block-poly(3-caprolactone) copolymers for bioimaging J. Mater. Chem. 20 2010 1728 1736
67. I. Akiba, N. Terada, S. Hashida, and K. Sakurai Encapsulation of a hydrophobic drug into a polymer-micelle core explored with synchrotron SAXS Langmuir 26 2010 7544 7551
68. A. Ghosh, S. Yusa, H. Matsuoka, and Y. Saruwatari Non-surface activity and micellization behavior of cationic amphiphilic block copolymer synthesized by reversible addition-fragmentation chain transfer process Langmuir 27 2011 9237 9244
69. M. Ashjari, S. Khoee, A.R. Mahdavian, and R. Rahmatolahzadeh Self-assembled nanomicelles using PLGA-PEG amphiphilic block copolymer for insulin delivery: a physicochemical investigation and determination of CMC values J. Mater. Sci. Mater. Med. 23 2012 943 953
70. P. Kaewsaiha, K. Matsumoto, and H. Matsuoka Non-surface activity and micellization of ionic amphiphilic diblock copolymers in water. Hydrophobic chain length dependence and salt effect on surface activity and the critical micelle concentration Langmuir 21 2005 9938 9945
71. M.S. Bakshi, R. Kaur, I. Kaur, R.K. Mahajan, P. Sehgal, and H. Doe Unlike surfactant-polymer interactions of sodium dodecyl sulfate and sodium dodecylbenzene sulfonate with water-soluble polymers Colloid Polym. Sci. 281 2003 716 726
72. N. Rehman, A. Khan, I. Bibi, C.I.D. Bica, and M. Siddiq Intermolecular interactions of polymer/surfactants mixture in aqueous solution investigated by various techniques J. Dispers. Sci. Technol. 34 2013 1202 1210
73. N. Li, D. Ziegemeiera, L. Bass, and W. Wang Quantitation of free polyethylene glycol in PEGylated protein conjugate by size exclusion HPLC with refractive index (RI) detection J. Pharm. Biomed. Anal. 48 2008 1332 1338
74. J.L. Haynes 3rd, E.J. Billiot, H.H. Yarabe, I.M. Warner, and S.A. Shamsi Chiral separation with dipeptide-terminated polymeric surfactants: the effect of an extra heteroatom on the polar head group Electrophoresis 21 2000 1597 1605
75. V. Zabaleta, M.A. Campanero, and J.M. Irache An HPLC with evaporative light scattering detection method for the quantification of PEGs and Gantrez in PEGylated nanoparticles J. Pharm. Biomed. Anal. 44 2007 1072 1078
76. R. Díaz-López, D. Libong, N. Tsapis, E. Fattal, and P. Chaminade Quantification of pegylated phospholipids decorating polymeric microcapsules of perfluorooctyl bromide by reverse phase HPLC with a charged aerosol detector J. Pharm. Biomed. Anal. 48 2008 702 707