Central nervous system delivery of large molecules: challenges and new frontiers for intrathecally administered therapeutics

Bibliography

• Misra A, Ganesh S, Shahiwala A, Drug delivery to the central nervous system: a review. J Pharm Pharm Sci 2003;6(2):252-73
   PubMed Web of Science ®Google Scholar
• Martin-Villalba A, Okuducu AF, von Deimling A. The evolution of our understanding on glioma. Brain Pathol 2008;18(3):455-63
   PubMed Web of Science ®Google Scholar
• Prince DA, Parada I, Scalise K, Epilepsy following cortical injury: cellular and molecular mechanisms as targets for potential prophylaxis. Epilepsia 2009;50:30-40
   PubMed Web of Science ®Google Scholar
• Viswanathan A, Rocca WA, Tzourio C. Vascular risk factors and dementia how to move forward? Neurology 2009;72(4):368-74
   PubMed Web of Science ®Google Scholar
• Strecker K, Schwarz J. Parkinson's disease: emerging pharmacotherapy. Expert Opin Emerging Drugs 2008;13(4):573-91
   PubMed Web of Science ®Google Scholar
• Biran Y, Masters CL, Barnham KJ, Pharmacotherapeutic targets in Alzheimer's disease. J Cell Mol Med 2009;13(1):61-86
   PubMed Web of Science ®Google Scholar
• Vernino S, Geschwind M, Boeve B. Autoimmune encephalopathies. Neurologist 2007;13(3):140-7
   PubMed Web of Science ®Google Scholar
• Marchand F, Perretti M, McMahon SB. Role of the immune system in chronic pain. Nat Rev Neurosci 2005;6(7):521-32
   PubMed Web of Science ®Google Scholar
• Nagahara AH, Merrill DA, Coppola G, Neuroprotective effects of brain-derived neurotrophic factor in rodent and primate models of Alzheimer's disease. Nat Med 2009;15:331-7
   Google Scholar
• Dawbarn D, Allen SJ. Neurotrophins and neurodegeneration. Neuropathol Appl Neurobiol 2003;29(3):211-30
   PubMed Web of Science ®Google Scholar
• Opal SM, DePalo VA. Anti-inflammatory cytokines. Chest 2000;117(4):1162-72
   PubMed Web of Science ®Google Scholar
• Walker D, Lue LF. Anti-inflammatory and immune therapy for Alzheimer's disease: current status and future directions. Curr Neuropharmacol 2007;5(4):232-43
   PubMed Web of Science ®Google Scholar
• Pettingill LN, Minter RL, Shepherd RK. Schwann cells genetically modified to express neurotrophins promote spiral ganglion neuron survival in vitro. Neuroscience 2008;152(3):821-8
   PubMed Web of Science ®Google Scholar
• Blesch A, Tuszynski MH. Transient growth factor delivery sustains regenerated axons after spinal cord injury. J Neurosci 2007;27(39):10535-45
   PubMed Web of Science ®Google Scholar
• Yao MZ, Gu JF, Wang JH, Interleukin-2 gene therapy of chronic neuropathic pain. Neuroscience 2002;112(2):409-16
   PubMed Web of Science ®Google Scholar
• Sloane E, Ledeboer A, Seibert W, Anti-inflammatory cytokine gene therapy decreases sensory and motor dysfunction in experimental multiple sclerosis. Brain Behav Immun 2009;23(1):92-100
   PubMed Web of Science ®Google Scholar
• Patel MM, Goyal BR, Bhadada SV, Getting into the brain approaches to enhance brain drug delivery. CNS Drugs 2009;23(1):35-58
   PubMed Web of Science ®Google Scholar
• Tosi G, Costantino L, Ruozi B, Polymeric nanoparticles for the drug delivery to the central nervous system. Expert Opin Drug Deliv 2008;5(2):155-74
   PubMed Web of Science ®Google Scholar
• Lagarce F, Benoit JP. Sustained release formulations for spinal drug delivery. J Drug Deliv Sci Technol 2004;14(5):331-43
   Web of Science ®Google Scholar
• Smith HS, Deer TR, Staats PS, Intrathecal drug delivery. Pain Physician 2008; Opiod Special Issue(11):S89-S104
   Google Scholar
• Goss JR, Goins WF, Glorioso JC. Gene therapy applications for the treatment of neuropathic pain. Expert Rev Neurother 2007;7(5):487-506
   PubMedGoogle Scholar
• Gallagher RM. Intrathecal drug delivery for chronic back pain: better science for clinical innovation. Pain Med 2004;5(1):1-3
   PubMed Web of Science ®Google Scholar
• Rosenblum LY, Johnson RC, Schmahai TJ. Preclinical safety evaluation of recombinant human interleukin-10. Regul Toxicol Pharmacol 2002;35(1):56-71
   PubMed Web of Science ®Google Scholar
• Lin JH. CSF as a surrogate for assessing CNS exposure: an industrial perspective. Curr Drug Metab 2008;9(1):46-59
   PubMed Web of Science ®Google Scholar
• Rambeck B, Jurgens UH, May TW, Comparison of brain extracellular fluid, brain tissue, cerebrospinal fluid, and serum concentrations of antiepileptic drugs measured intraoperatively in patients with intractable epilepsy. Epilepsia 2006;47(4):681-94
   PubMed Web of Science ®Google Scholar
• Sharma HS, Johanson CE. Blood-cerebrospinal fluid barrier in hyperthermia. Neurobiology of hyperthermia. Elsevier Science Bv, Amsterdam; 2007. p. 459-78
   Google Scholar
• Krewson CE, Klarman ML, Saltzman WM. Distribution of nerve growth-factor following direct delivery to brain interstitium. Brain Res 1995;680(1-2):196-206
   PubMed Web of Science ®Google Scholar
• Shen DD, Artru AA, Adkison KK. Principles and applicability of CSF sampling for the assessment of CNS drug delivery and pharmacodynamics. Adv Drug Deliv Rev 2004;56(12):1825-57
   PubMed Web of Science ®Google Scholar
• Newcomb R, Abbruscato TJ, Singh T, Bioavailability of Ziconotide in brain: influx from blood, stability, and diffusion. Peptides 2000;21(4):491-501
   PubMed Web of Science ®Google Scholar
• Kandel ER, Schwartz JH, Jessell TM. Principles of neural science. 4th edition. McGraw-Hill, New York; 2000
   Google Scholar
• Abbott NJ. Evidence for bulk flow of brain interstitial fluid: significance for physiology and pathology. Neurochem Int 2004;45(4):545-52
   PubMed Web of Science ®Google Scholar
• Greitz D, Hannerz J. A proposed model of cerebrospinal fluid circulation: observations with radionuclide cisternography. Am J Neuroradiol 1996;17(3):431-8
   PubMed Web of Science ®Google Scholar
• Whittlesey KJ, Shea LD. Delivery systems for small molecule drugs, proteins, and DNA: the neuroscience/biomaterial interface. Exp Neurol 2004;190(1):1-16
   PubMed Web of Science ®Google Scholar
• Wieseler-Frank J, Jekich BM, Mahoney JH, A novel immune-to-CNS communication pathway: Cells of the meninges surrounding the spinal cord CSF space produce proinflammatory cytokines in response to an inflammatory stimulus. Brain Behav Immun 2007;21(5):711-8
   PubMed Web of Science ®Google Scholar
• Mercier F, Hatton GI. Immunocytochemical basis for a meningeo-glial network. J Comp Neurol 2000;420(4):445-65
   PubMed Web of Science ®Google Scholar
• Tsutsami Y, Tsunoda S-I, Kamada H, PEGylation of Interleukin-6 effectively increases its thrombopoietic potency. Thromb Haemost 1997;77(1):168-73
   PubMed Web of Science ®Google Scholar
• Basu A, Yang K, Wang ML, Structure-function engineering of interferon-beta-1b for improving stability, solubility, potency, immunogenicity, and pharmacokinetic properties by site-selective mono-PEGylation. Bioconjug Chem 2006;17(3):618-30
   PubMed Web of Science ®Google Scholar
• Ramon J, Saez V, Baez R, PEGylated interferon-alpha 2b: a branched 40K polyethylene glycol derivative. Pharm Res 2005;22(8):1374-86
   PubMed Web of Science ®Google Scholar
• Bailon P, Berthold W. Polyethylene glycol-conjugated pharmaceutical proteins. Pharm Sci Technol Today 1998;1(8):352-6
   Google Scholar
• Roberts MJ, Bentley MD, Harris JM. Chemistry for peptide and protein PEGylation. Adv Drug Deliv Rev 2002;54(4):459-76
   PubMed Web of Science ®Google Scholar
• Veronese FM, Pasut G. PEGylation, successful approach to drug delivery. Drug Discov Today 2005;10(21-24):1451-8
   PubMed Web of Science ®Google Scholar
• Harris MJ, Martin NE, Modi M. Pegylation: a novel process for modifying pharmacokinetics. Clin Pharmacokinet 2001;40(7):539-51
   PubMed Web of Science ®Google Scholar
• Koumenis IL, Shahrokh Z, Leong S, Modulating pharmacokinetics of an anti-interleukin-8 F(ab')(2) by amine-specific PEGylation with preserved bioactivity. Int J Pharm 2000;198(1):83-95
   PubMedGoogle Scholar
• Clark R, Olson K, Fuh G, Long-acting growth hormones produced by conjugation with polyethylene glycol. J Biol Chem 1996;271(36):21969-77
   PubMed Web of Science ®Google Scholar
• Francis GE, Fisher D, Delgado C, PEGylation of cytokines and other therapeutic proteins and peptides: the importance of biological optimisation of coupling techniques. Int J Hematol 1998;68:1-18
   PubMed Web of Science ®Google Scholar
• Molineux G. Pegylation: engineering improved biopharmaceuticals for oncology. Pharmacotherapy 2003;23(8 Pt 2):3S-8S
   PubMed Web of Science ®Google Scholar
• Dhalluin C, Ross A, Leuthold LA, Structural and biophysical characterization of the 40 kDa PEG-interferon-alpha(2a) and its individual positional isomers. Bioconjug Chem 2005;16(3):504-17
   PubMed Web of Science ®Google Scholar
• Stroh M, Zipfel WR, Williams RM, Multiphoton microscopy guides neurotrophin modification with poly(ethylene glycol) to enhance interstitial diffusion. Nat Mater 2004;3(7):489-94
   PubMed Web of Science ®Google Scholar
• Dang W, Colvin MO, Brem H, Covalent coupling of methotrexate to dextran enhances the penetration of cytotoxicity into a tissue-like matrix. Cancer Res 1994;54:1729-35
   PubMed Web of Science ®Google Scholar
• Ankeny DP, McTigue DM, Guan Z, Pegylated brain-derived neurotrophic factor shows improved distribution into the spinal cord and stimulates locomotor activity and morphological changes after injury. Exp Neurol 2001;170(1):85-100
   PubMed Web of Science ®Google Scholar
• Greenwald RB, Yang K, Zhao H, Controlled release of proteins from their poly(ethylene glycol) conjugates: drug delivery systems employing 1,6-elimination. Bioconjug Chem 2003;14(2):395-403
   PubMed Web of Science ®Google Scholar
• Scarisbrick IA, Towner MD, Isackson PJ. Nervous system-specific expression of a novel serine protease: regulation in the adult rat spinal cord by excitotoxic injury. J Neurosci 1997;17(21):8156-68
   PubMed Web of Science ®Google Scholar
• Polfliet MMJ, Goede PH, van Kesteren-Hendrikx EML, A method for the selective depletion of perivascular and meningeal macrophages in the central nervous system. J Neuroimmunol 2001;116(2):188-95
   PubMed Web of Science ®Google Scholar
• McMenamin PG, Wealthall RJ, Deverall M, Macrophages and dendritic cells in the rat meninges and choroid plexus: three-dimensional localisation by environmental scanning electron microscopy and confocal microscopy. Cell Tissue Res 2003;313(3):259-69
   PubMed Web of Science ®Google Scholar
• Soderquist RG, Milligan ED, Sloane EM, PEGylation of brain-derived neurotrophic factor for preserved biological activity and enhanced spinal cord distribution. J Biomed Mater Res A 2009;91(3):719-29
   PubMedGoogle Scholar
• Soderquist RG, Milligan ED, Harrison JA, PEGylation of interleukin-10 for the mitigation of enhanced pain states. J Biomed Mater Res A 2009. Published online 18 Sep 2009. Available at http://dx.doi.org/10.1002/jbm.a.32611
   Google Scholar
• Radler JO, Koltover I, Salditt T, Structure of DNA-cationic liposome complexes: DNA intercalation in multilamellar membranes in distinct interhelical packing regimes. Science 1997;275(5301):810-4
   PubMed Web of Science ®Google Scholar
• Park JH, Lee S, Kim JH, Polymeric nanomedicine for cancer therapy. Prog Polym Sci 2008;33(1):113-37
   Web of Science ®Google Scholar
• Boussif O, Lezoualch F, Zanta MA, A versatile vector for gene and oligonucleotide transfer into cells in culture and in vivo–polyethylenimine. Proc Natl Acad Sci USA 1995;92(16):7297-301
   PubMed Web of Science ®Google Scholar
• Shi L, Tang GP, Gao SJ, Repeated intrathecal administration of plasmid DNA complexed with polyethylene glycol-grafted polyethylenimine led to prolonged transgene expression in the spinal cord. Gene Ther 2003;10(14):1179-88
   PubMed Web of Science ®Google Scholar
• Meuli-Simmen C, Liu Y, Yeo TT, Gene expression along the cerebral-spinal axis after regional gene delivery. Hum Gene Ther 1999;10(16):2689-700
   PubMed Web of Science ®Google Scholar
• Milligan ED, Soderquist RG, Malone SM, Intrathecal polymer-based interleukin-10 gene delivery for neuropathic pain. Neuron Glia Biol 2006;2(4):293-308
   PubMedGoogle Scholar
• Tang GP, Zeng JM, Gao SJ, Polyethylene glycol modified polyethylenimine for improved CNS gene transfer: effects of PEGylation extent. Biomaterials 2003;24(13):2351-62
   PubMed Web of Science ®Google Scholar
• Wang X, Wang CY, Zeng JM, Gene transfer to dorsal root ganglia by intrathecal injection: effects on regeneration of peripheral nerves. Mol Ther 2005;12(2):314-20
   PubMed Web of Science ®Google Scholar
• Godbey WT, Wu KK, Mikos AG. Poly(ethylenimine)-mediated gene delivery affects endothelial cell function and viability. Biomaterials 2001;22(5):471-80
   PubMed Web of Science ®Google Scholar
• Fischer D, Li YX, Ahlemeyer B, In vitro cytotoxicity testing of polycations: influence of polymer structure on cell viability and hemolysis. Biomaterials 2003;24(7):1121-31
   PubMed Web of Science ®Google Scholar
• Tiera MJ, Winnik FM, Fernandes JC. Synthetic and natural polycations for gene therapy: state of the art and new perspectives. Curr Gene Ther 2006;6(1):59-71
   PubMed Web of Science ®Google Scholar
• Tang GP, Guo HY, Alexis F, Low molecular weight polyethylenimines linked by beta-cyclodextrin for gene transfer into the nervous system. J Gene Med 2006;8(6):736-44
   PubMed Web of Science ®Google Scholar
• Arote R, Kim TH, Kim YK, A biodegradable poly(ester amine) based on polycaprolactone and polyethylenimine as a gene carrier. Biomaterials 2007;28(4):735-44
   PubMed Web of Science ®Google Scholar
• Edlund U, Albertsson AC. Degradable polymer microspheres for controlled drug delivery. Degradable Aliphatic Polyesters 2002;157:67-112
   Google Scholar
• Birnbaum DT, Brannon-Peppas L. Microparticle drug delivery systems. In: Brown DM, editor, Drug delivery systems in cancer therapy. Humana Press Inc., Totowa, NJ; 2003. p. 117-35
   Google Scholar
• Kumar M, Bakowsky U, Lehr CM. Preparation and characterization of cationic PLGA nanospheres as DNA carriers. Biomaterials 2004;25(10):1771-7
   PubMed Web of Science ®Google Scholar
• Barman SP, Lunsford L, Chambers P, Two methods for quantifying DNA extracted from poly(lactide-co-glycolide) microspheres. J Control Release 2000;69(3):337-44
   PubMed Web of Science ®Google Scholar
• Tinsley-Bown AM, Fretwell R, Dowsett AB, Formulation of poly(D,L-lactic-co-glycolic acid) microparticles for rapid plasmid DNA delivery. J Control Release 2000;66(2-3):229-41
   PubMed Web of Science ®Google Scholar
• Cohen S, Yoshioka T, Lucarelli M, Controlled delivery systems for proteins based on poly(lactic/glycolic acid) microspheres. Pharm Res 1991;8(6):713-20
   PubMed Web of Science ®Google Scholar
• Liu Y, Deng XM. Influences of preparation conditions on particle size and DNA-loading efficiency for poly(DL-lactic acid-polyethylene glycol) microspheres entrapping free DNA. J Control Release 2002;83(1):147-55
   PubMed Web of Science ®Google Scholar
• Sendil D, Bonney IM, Carr DB, Antinociceptive effects of hydromorphone, bupivacaine and biphalin released from PLGA polymer after intrathecal implantation in rats. Biomaterials 2003;24(11):1969-76
   PubMed Web of Science ®Google Scholar
• Lagarce F, Renaud P, Faisant N, Baclofen-loaded microspheres: preparation and efficacy testing in a new rabbit model. Eur J Pharm Biopharm 2005;59(3):449-59
   PubMed Web of Science ®Google Scholar
• Lagarce F, Faisant N, Desfontis JC, Baclofen-loaded microspheres in gel suspensions for intrathecal drug delivery: in vitro and in vivo evaluation. Eur J Pharm Biopharm 2005;61(3):171-80
   PubMed Web of Science ®Google Scholar
• Milligan ED, Langer SJ, Sloane EM, Controlling pathological pain by adenovirally driven spinal production of the anti-inflammatory cytokine, interleukin-10. Eur J Neurosci 2005;21(8):2136-48
   PubMed Web of Science ®Google Scholar
• Berg SL, Balis FM, McCully CL, Pharmacokinetics of peg-l-asparaginase and plasma and cerebrospinal-fluid l-asparagine concentrations in the rhesus-monkey. Cancer Chemother Pharmacol 1993;32(4):310-4
   PubMed Web of Science ®Google Scholar
• Hinds KD, Campbell KM, Holland KM, PEGylated insulin in PLGA microparticles. In vivo and in vitro analysis. J Control Release 2005;104(3):447-60
   PubMed Web of Science ®Google Scholar
• Gander B, Meinel L, Walter E, Polymers as a platform for drug delivery: reviewing our current portfolio on poly(lactide-co-glycolide) (PLGA) microspheres. Chimia 2001;55(3):212-7
   Web of Science ®Google Scholar
• Wang CY, Wang S. Adeno-associated virus inverted terminal repeats improve neuronal transgene expression mediated by baculoviral vectors in rat brain. Hum Gene Ther 2005;16(10):1219-26
   PubMed Web of Science ®Google Scholar
• Kaneda Y, Tabata Y. Non-viral vectors for cancer therapy. Cancer Sci 2006;97(5):348-54
   PubMed Web of Science ®Google Scholar
• Anderson WF. Human gene therapy. Nature 1998;392(6679):25-30
   PubMed Web of Science ®Google Scholar
• Milligan ED, Sloane EM, Langer SJ, Repeated intrathecal injections of plasmid DNA encoding interleukin-10 produce prolonged reversal of neuropathic pain. Pain 2006;126(1-3):294-308
   PubMed Web of Science ®Google Scholar
• Singh M, Briones M, Ott G, Cationic microparticles: a potent delivery system for DNA vaccines. Proc Natl Acad Sci USA 2000;97(2):811-6
   PubMed Web of Science ®Google Scholar
• Wang DQ, Robinson DR, Kwon GS, Encapsulation of plasmid DNA in biodegradable poly(D,L-lactic-co-glycolic acid) microspheres as a novel approach for immunogene delivery. J Control Release 1999;57(1):9-18
   PubMed Web of Science ®Google Scholar
• Li Y, Wang J, Lee CGL, CNS gene transfer mediated by a novel controlled release system based on DNA complexes of degradable polycation PPE-EA: a comparison with polyethylenimine/DNA complexes. Gene Ther 2004;11(1):109-14
   PubMed Web of Science ®Google Scholar
• Luo D, Woodrow-Mumford K, Belcheva N, Controlled DNA delivery systems. Pharm Res 1999;16(8):1300-8
   PubMed Web of Science ®Google Scholar
• Lunsford L, McKeever U, Eckstein V, Tissue distribution and persistence in mice of plasmid DNA encapsulated in a PLGA-based microsphere delivery vehicle. J Drug Target 2000;8(1):39-50
   PubMed Web of Science ®Google Scholar
• Ward AB, Kadies M. The management of pain in spasticity. Disabil Rehabil 2002;24(8):443-53
   PubMed Web of Science ®Google Scholar
• Plassat R, Verbe BP, Menei P, Treatment of spasticity with intrathecal baclofen administration: long-term follow-up, review of 40 patients. Spinal Cord 2004;42(12):686-93
   PubMed Web of Science ®Google Scholar
• Delhaas EM, Beersen N, Redekop WK, Long-term outcomes of continuous intrathecal baclofen infusion for treatment of spasticity: a prospective multicenter follow-up study. Neuromodulation 2008;11(3):227-36
   PubMed Web of Science ®Google Scholar