Alzheimer's disease drug development and the problem of the blood-brain barrier

Alzheimer's and Dementia

Volumn 5, Issue 5, 2009, Pages 427-432

  Pardridge, William M ^a  

^a UNIVERSITY OF CALIFORNIA   (United States)

Author keywords

Biopharmaceuticals; Blood brain barrier; Brain drug targeting; Endogenous transporters

Indexed keywords

ADJUVANT; AMYLOID BETA PROTEIN; AMYLOID BETA PROTEIN ANTIBODY; ANTIBODY; BETA SECRETASE INHIBITOR; BRAIN DERIVED NEUROTROPHIC FACTOR; CHOLINESTERASE INHIBITOR; DOPAMINE; FREUND ADJUVANT; GAMMA SECRETASE INHIBITOR; GLIAL CELL LINE DERIVED NEUROTROPHIC FACTOR; HYBRID PROTEIN; IMMUNOLOGICAL ADJUVANT; INSULIN RECEPTOR ANTIBODY; MANNAN ANTIBODY; MONOCLONAL ANTIBODY; UNCLASSIFIED DRUG;

ALZHEIMER DISEASE; ARTICLE; BLOOD BRAIN BARRIER; DRUG DELIVERY SYSTEM; DRUG DESIGN; DRUG PENETRATION; DRUG TARGETING; HUMAN; HYDROGEN BOND; MOLECULAR WEIGHT; NONHUMAN; PRIORITY JOURNAL; RESOURCE ALLOCATION;

EID: 69949149176     PISSN: 15525260     EISSN: None     Source Type: Journal    
DOI: 10.1016/j.jalz.2009.06.003     Document Type: Note

References (45)

1. Pardridge W.M. The blood-brain barrier: bottleneck in brain drug development. NeuroRx 2 (2005) 3-14
2. Blasberg R.G., Patlak C., and Fenstermacher J.D. Intrathecal chemotherapy: brain tissue profiles after ventriculocisternal perfusion. J Pharmacol Exp Ther 195 (1975) 73-83
3. Pardridge W.M. Brain drug targeting: the future of brain drug development (2001), Cambridge University Press, Cambridge 1-370
4. Fishman R.A., and Christy N.P. Fate of adrenal cortical steroids following intrathecal injections. Neurology 15 (1965) 1-6
5. Dykstra K.H., Arya A., Arriola D.M., Bungay P.M., Morrison P.F., and Dedrick R.L. Microdialysis study of zidovudine (AZT) transport in rat brain. J Pharmacol Exp Ther 267 (1993) 1227-1236
6. Fung L.K., Shin M., Tyler B., Brem H., and Saltzman W.M. Chemotherapeutic drugs released from polymers: distribution of 1,3-bis(2-chloroethyl)-1-nitrosourea in the rat brain. Pharm Res 13 (1996) 671-682
7. Lang A.E., Gill S., Patel N.K., Lozano A., Nutt J.G., Penn R., et al. Randomized controlled trial of intraputamenal glial cell line-derived neurotrophic factor infusion in Parkinson disease. Ann Neurol 59 (2006) 459-466
8. Salvatore M.F., Ai Y., Fischer B., Zhang A.M., Grondin R.C., Zhang Z., et al. Point source concentration of GDNF may explain failure of phase II clinical trial. Exp Neurol 202 (2006) 497-505
9. Lipinski C.A., Lombardo F., Dominy B.W., and Feeney P.J. Experimental and computational approaches to estimate solubility and permeability in drug discovery and development settings. Adv Drug Deliv Rev 46 (2001) 3-26
10. Cohen B.E., and Bangham A.D. Diffusion of small non-electrolytes across liposome membranes. Nature 236 (1972) 173-174
11. Fischer H., Gottschlich R., and Seelig A. Blood-brain barrier permeation: molecular parameters governing passive diffusion. J Membr Biol 165 (1998) 201-211
12. Lieb W.R., and Stein W.D. Non-Stokesian nature of transverse diffusion within human red cell membranes. J Membr Biol 92 (1986) 111-119
13. Träuble H. The movement of molecules across lipid membranes: a molecular theory. J Membr Biol 4 (1971) 193-208
14. Rishton G.M., LaBonte K., Williams A.J., Kassam K., and Kolovanov E. Computational approaches to the prediction of blood-brain barrier permeability: a comparative analysis of central nervous system drugs versus secretase inhibitors for Alzheimer's disease. Curr Opin Drug Discov Dev 9 (2006) 303-313
15. Ghose A.K., Viswanadhan V.N., and Wendoloski J.J. A knowledge-based approach in designing combinatorial or medicinal chemistry libraries for drug discovery. 1. A qualitative and quantitative characterization of known drug databases. J Comb Chem 1 (1999) 55-68
16. Lipinski C.A. Drug-like properties and the causes of poor solubility and poor permeability. J Pharmacol Toxicol Methods 44 (2000) 235-249
17. Boado R.J., Li J.Y., Nagaya M., Zhang C., and Pardridge W.M. Selective expression of the large neutral amino acid transporter at the blood-brain barrier. Proc Natl Acad Sci USA 96 (1999) 12079-12084
18. 1-40/vector complex. Proc Natl Acad Sci USA 92 (1995) 10227-10231
19. Ito S., Ohtsuki S., Kamiie J., Nezu Y., and Terasaki T. Cerebral clearance of human amyloid-β peptide (1-40) across the blood-brain barrier is reduced by self-aggregation and formation of low-density lipoprotein receptor-related protein-1 ligand complexes. J Neurochem 103 (2007) 2482-2490
20. Schenk D., Barbour R., Dunn W., Gordon G., Grajeda H., Guido T., et al. Immunization with amyloid-beta attenuates Alzheimer-disease-like pathology in the PDAPP mouse. Nature 400 (1999) 173-177
21. Solomon B., Koppel R., Frankel D., and Hanan-Aharon E. Disaggregation of Alzheimer beta-amyloid by site-directed mAb. Proc Natl Acad Sci USA 94 (1997) 4109-4112
22. Lombardo J.A., Stern E.A., McLellan M.E., Kajdasz S.T., Hickey G.A., Bacskai B.J., et al. Amyloid-beta antibody treatment leads to rapid normalization of plaque-induced neuritic alterations. J Neurosci 23 (2003) 10879-10883
23. Rabchevsky A.G., Degos J.D., and Dreyfus P.A. Peripheral injections of Freund's adjuvant in mice provoke leakage of serum proteins through the blood-brain barrier without inducing reactive gliosis. Brain Res 832 (1999) 84-96
24. Namer I.J., and Steibel J. Antibody directed against mannan of the Mycobacterium tuberculosis cell envelope provokes blood-brain barrier breakdown. J Neuroimmunol 103 (2000) 63-68
25. Nadal A., Fuentes E., Pastor J., and McNaughton P.A. Plasma albumin is a potent trigger of calcium signals and DNA synthesis in astrocytes. Proc Natl Acad Sci USA 92 (1995) 1426-1430
26. Salahuddin T.S., Johansson B.B., Kalimo H., and Olsson Y. Structural changes in the rat brain after carotid infusions of hyperosmolar solutions. An electron microscopic study. Acta Neuropathol (Berl) 77 (1988) 5-13
27. Nicoll J.A., Wilkinson D., Holmes C., Steart P., Markham H., and Weller R.O. Neuropathology of human Alzheimer disease after immunization with amyloid-beta peptide: a case report. Nat Med 9 (2003) 448-452
28. Bard F., Cannon C., Barbour R., Burke R.L., Games D., Grajeda H., et al. Peripherally administered antibodies against amyloid beta-peptide enter the central nervous system and reduce pathology in a mouse model of Alzheimer disease. Nat Med 6 (2000) 916-919
29. DeMattos R.B., Bales K.R., Cummins D.J., Dodart J.C., Paul S.M., and Holtzman D.M. Peripheral anti-A beta antibody alters CNS and plasma A beta clearance and decreases brain A beta burden in a mouse model of Alzheimer's disease. Proc Natl Acad Sci USA 98 (2001) 8850-8855
30. Pfeifer M., Boncristiano S., Bondolfi L., Stalder A., Deller T., Staufenbiel M., et al. Cerebral hemorrhage after passive anti-Abeta immunotherapy. Science 298 (2002) 1379
31. Wilcock D.M., Rojiani A., Rosenthal A., Subbarao S., Freeman M.J., Gordon M.N., et al. Passive immunotherapy against Abeta in aged APP-transgenic mice reverses cognitive deficits and depletes parenchymal amyloid deposits in spite of increased vascular amyloid and microhemorrhage. J Neuroinflamm 1 (2004) 24
32. Wilcock D.M., Alamed J., Gottschall P.E., Grimm J., Rosenthal A., Pons J., et al. Deglycosylated anti-amyloid-beta antibodies eliminate cognitive deficits and reduce parenchymal amyloid with minimal vascular consequences in aged amyloid precursor protein transgenic mice. J Neurosci 26 (2006) 5340-5346
33. Su G.C., Arendash G.W., Kalaria R.N., Bjugstad K.B., and Mullan M. Intravascular infusions of soluble beta-amyloid compromise the blood-brain barrier, activate CNS glial cells and induce peripheral hemorrhage. Brain Res 818 (1999) 105-117
34. Jancso G., Domoki F., Santha P., Varga J., Fischer J., Orosz K., et al. Beta-amyloid (1-42) peptide impairs blood-brain barrier function after intracarotid infusion in rats. Neurosci Lett 253 (1998) 139-141
35. Pardridge W.M. Re-engineering biopharmaceuticals for delivery to brain with molecular Trojan horses. Bioconjug Chem 19 (2008) 1327-1338
36. Boado R.J., Zhang Y., Xia C.F., and Pardridge W.M. Fusion antibody for Alzheimer's disease with bidirectional transport across the blood-brain barrier and Abeta fibril disaggregation. Bioconjug Chem 18 (2007) 447-455
37. Boado R.J., Zhang Y., and Pardridge W.M. Humanization of anti-human insulin receptor antibody for drug targeting across the human blood-brain barrier. Biotechnol Bioeng 96 (2007) 381-391
38. Boado R.J., Zhang Y., and Pardridge W.M. Genetic engineering, expression, and activity of a fusion protein of a human neurotrophin and a molecular Trojan horse for delivery across the human blood-brain barrier. Biotechnol Bioeng 97 (2007) 1376-1386
39. Boado R.J., Zhang Y., Wang Y., and Pardridge W.M. GDNF fusion protein for targeted-drug delivery across the human blood-brain barrier. Biotechnol Bioeng 100 (2008) 387-396
40. Boado R.J., Zhang Y., Xia C.F., Wang Y., and Pardridge W.M. Genetic engineering of a lysosomal enzyme fusion protein for targeted delivery across the human blood-brain barrier. Biotechnol Bioeng 99 (2008) 475-484
41. Boado R.J., Zhang Y., Wang Y., and Pardridge W.M. IgG-paraoxonase-1 fusion protein for targeted drug delivery across the human blood-brain barrier. Mol Pharm 5 (2008) 1037-1043
42. Nagahara A.H., Merrill D.A., Coppola G., Tsukada S., Schroeder B.E., Shaked G.M., et al. Neuroprotective effects of brain-derived neurotrophic factor in rodent and primate models of Alzheimer's disease. Nat Med 15 (2009) 331-337
43. Sakane T., and Pardridge W.M. Carboxyl-directed pegylation of brain-derived neurotrophic factor markedly reduces systemic clearance with minimal loss of biologic activity. Pharm Res 14 (1997) 1085-1091
44. BDNF Study Group. A controlled trial of recombinant methionyl human BDNF in ALS: the BDNF Study Group (phase III). Neurology 52 (1999) 1427-1433
45. Wilkie N., Wingrove P.B., Bilsland J.G., Young L., Harper S.J., Hefti F., et al. The non-peptidyl fungal metabolite L-783,281 activates TRK neurotrophin receptors. J Neurochem 78 (2001) 1135-1145