The adult human brain in preclinical drug development (original) (raw)

• Zivin, J. A., Fisher, M., DeGirolami, U., Hemenway, C. C. & Stashak, J. A. Tissue plasminogen activator reduces neurological damage after cerebral embolism. Science 230, 1289–1292 (1985).
  Article CAS Google Scholar
• Shuaib, A. et al. NXY-059 for the treatment of acute ischemic stroke. N. Engl. J. Med. 357, 562–571 (2007).
  Article CAS Google Scholar
• Lees, K. R. et al. Tolerability of NXY-059 at higher target concentrations in patients with acute stroke. Stroke 34, 482–487 (2003).
  Article CAS Google Scholar
• Gordon, P. H. et al. Efficacy of minocycline in patients with amyotrophic lateral sclerosis: a phase III randomised trial. Lancet Neurol. 6, 1045–1053 (2007).
  Article CAS Google Scholar
• Fitzgerald, G. A. Opinion: anticipating change in drug development: the emerging era of translational medicine and therapeutics. Nature Rev. Drug Discov. 4, 815–818 (2005).
  Article CAS Google Scholar
• Anisimov, V. N., Ukraintseva, S. V. & Yashin, A. I. Cancer in rodents: does it tell us about cancer in humans? Nature Rev. Cancer 5, 807–819 (2005).
  Article CAS Google Scholar
• Kola, I. & Landis, J. Can the pharmaceutical industry reduce attrition rates? Nature Rev. Drug Discov. 3, 711–715 (2004).
  CAS Google Scholar
• Oberheim, N. A., Wang, X., Goldman, S. & Nedergaard, M. Astrocytic complexity distinguishes the human brain. Trends Neurosci. 29, 547–553 (2006).
  Article CAS Google Scholar
• Bachoo, R. M. et al. Molecular diversity of astrocytes with implications for neurological disorders. Proc. Natl Acad. Sci. USA 101, 8384–8389 (2004).
  Article CAS Google Scholar
• Block, M. L., Zecca, L. & Hong, J. S. Microglia-mediated neurotoxicity: uncovering the molecular mechanisms. Nature Rev. Neurosci. 8, 57–69 (2007).
  Article CAS Google Scholar
• Klegeris, A., McGeer, E. G. & McGeer, P. L. Therapeutic approaches to inflammation in neurodegenerative disease. Curr. Opin. Neurol. 20, 351–357 (2007).
  Article CAS Google Scholar
• Meda, L. et al. Activation of microglial cells by β-amyloid protein and interferon-γ. Nature 374, 647–650 (1995).
  Article CAS Google Scholar
• Xiao, Q. et al. Mutant SOD1(G93A) microglia are more neurotoxic relative to wild-type microglia. J. Neurochem. 102, 2008–19 (2007).
  Article CAS Google Scholar
• Zhao, M. L., Liu, J. S., He, D., Dickson, D. W. & Lee, S. C. Inducible nitric oxide synthase expression is selectively induced in astrocytes isolated from adult human brain. Brain Res. 813, 402–405 (1998).
  Article CAS Google Scholar
• Wallace, M. N., Geddes, J. G., Farquhar, D. A. & Masson, M. R. Nitric oxide synthase in reactive astrocytes adjacent to β-amyloid plaques. Exp. Neurol. 144, 266–272 (1997).
  Article CAS Google Scholar
• Giulian, D. et al. Specific domains of β-amyloid from Alzheimer plaque elicit neuron killing in human microglia. J. Neurosci. 16, 6021–6037 (1996).
  Article CAS Google Scholar
• Petrova, T. V., Akama, K. T. & Van Eldik, L. J. Cyclopentenone prostaglandins suppress activation of microglia: down-regulation of inducible nitric-oxide synthase by 15-deoxy-Δ12,14-prostaglandin J2 . Proc. Natl Acad. Sci. USA 96, 4668–4673 (1999).
  Article CAS Google Scholar
• Janabi, N. Selective inhibition of cyclooxygenase-2 expression by 15-deoxy-Δ12,14-prostaglandin J2 in activated human astrocytes, but not in human brain macrophages. J. Immunol. 168, 4747–4755 (2002).
  Article CAS Google Scholar
• Chan, G. C. et al. Epigenetic basis for the transcriptional hyporesponsiveness of the human inducible nitric oxide synthase gene in vascular endothelial cells. J. Immunol. 175, 3846–3861 (2005).
  Article CAS Google Scholar
• Lanosa, X. A. & Colombo, J. A. Astroglial injury in an ex vivo model: contributions to its analysis in enriched cell cultures. In Vitro Cell Dev. Biol. Anim. 43, 186–195 (2007).
  Article Google Scholar
• Flanary, B. E. & Streit, W. J. Alpha-tocopherol (vitamin E) induces rapid, nonsustained proliferation in cultured rat microglia. Glia 53, 669–674 (2006).
  Article Google Scholar
• Gibbons, H. M. et al. Cellular composition of human glial cultures from adult biopsy brain tissue. J. Neurosci. Methods 166, 89–98 (2007).
  Article CAS Google Scholar
• Silver, J. & Miller, J. H. Regeneration beyond the glial scar. Nature Rev. Neurosci. 5, 146–156 (2004).
  Article CAS Google Scholar
• Lim, J. H. et al. Extracellular signal-regulated kinase involvement in human astrocyte migration. Brain Res. 1164, 1–13 (2007).
  Article CAS Google Scholar
• Sandvig, A., Berry, M., Barrett, L. B., Butt, A. & Logan, A. Myelin-, reactive glia-, and scar-derived CNS axon growth inhibitors: expression, receptor signaling, and correlation with axon regeneration. Glia 46, 225–251 (2004).
  Article Google Scholar
• Simard, A. R., Soulet, D., Gowing, G., Julien, J. P. & Rivest, S. Bone marrow-derived microglia play a critical role in restricting senile plaque formation in Alzheimer's disease. Neuron 49, 489–502 (2006).
  Article CAS Google Scholar
• Fiala, M. et al. Innate immunity and transcription of MGAT-III and Toll-like receptors in Alzheimer's disease patients are improved by bisdemethoxycurcumin. Proc. Natl Acad. Sci. USA 104, 12849–12854 (2007).
  Article CAS Google Scholar
• Nicoll, J. A. et al. Neuropathology of human Alzheimer disease after immunization with amyloid-β peptide: a case report. Nature Med. 9, 448–452 (2003).
  Article CAS Google Scholar
• Floden, A. M. & Combs, C. K. β-Amyloid stimulates murine postnatal and adult microglia cultures in a unique manner. J. Neurosci. 26, 4644–4648 (2006).
  Article CAS Google Scholar
• Pouton, C. W. & Haynes, J. M. Embryonic stem cells as a source of models for drug discovery. Nature Rev. Drug Discov. 6, 605–616 (2007).
  Article CAS Google Scholar
• Sanchez-Gonzalez, M. A., Garcia-Cabezas, M. A., Rico, B. & Cavada, C. The primate thalamus is a key target for brain dopamine. J. Neurosci. 25, 6076–6083 (2005).
  Article CAS Google Scholar
• Dorus, S. et al. Accelerated evolution of nervous system genes in the origin of Homo sapiens. Cell 119, 1027–1040 (2004).
  Article CAS Google Scholar
• Tippett, L. J. et al. Striosomes and mood dysfunction in Huntington's disease. Brain 130, 206–221 (2007).
  Article Google Scholar
• Watanabe, Y., Himeda, T. & Araki, T. Mechanisms of MPTP toxicity and their implications for therapy of Parkinson's disease. Med. Sci. Monit. 11, RA17–RA23 (2005).
  CAS PubMed Google Scholar
• Hsiao, K. et al. Correlative memory deficits, Aβ elevation, and amyloid plaques in transgenic mice. Science 274, 99–102 (1996).
  Article CAS Google Scholar
• Dragunow, M. et al. In situ evidence for DNA fragmentation in Huntington's disease striatum and Alzheimer's disease temporal lobes. Neuroreport 6, 1053–1057 (1995).
  Article CAS Google Scholar
• Oddo, S. et al. Triple-transgenic model of Alzheimer's disease with plaques and tangles: intracellular Aβ and synaptic dysfunction. Neuron 39, 409–421 (2003).
  Article CAS Google Scholar
• Miller, G. Neuroscience. The dark side of glia. Science 308, 778–781 (2005).
  Article CAS Google Scholar
• Nagai, M. et al. Astrocytes expressing ALS-linked mutated SOD1 release factors selectively toxic to motor neurons. Nature Neurosci. 10, 615–622 (2007).
  Article CAS Google Scholar
• Connor, B. et al. Trk receptor alterations in Alzheimer's disease. Brain Res. Mol. Brain Res. 42, 1–17 (1996).
  Article CAS Google Scholar
• Sauter, G., Simon, R. & Hillan, K. Tissue microarrays in drug discovery. Nature Rev. Drug Discov. 2, 962–972 (2003).
  Article CAS Google Scholar
• LeBaron, M. J. et al. Ultrahigh density microarrays of solid samples. Nature Methods 2, 511–513 (2005).
  Article CAS Google Scholar
• Sjobeck, M., Haglund, M., Persson, A., Sturesson, K. & Englund, E. Brain tissue microarrays in dementia research: white matter microvascular pathology in Alzheimer's disease. Neuropathology 23, 290–295 (2003).
  Article Google Scholar
• Curtis, M. A. et al. Human neuroblasts migrate to the olfactory bulb via a lateral ventricular extension. Science 315, 1243–1249 (2007).
  Article CAS Google Scholar
• Narayan, P. J., Gibbons, H. M., Mee, E. W., Faull, R. L. & Dragunow, M. High throughput quantification of cells with complex morphology in mixed cultures. J. Neurosci. Methods 164, 339–349 (2007).
  Article Google Scholar
• Scotter, E. L., Narayan, P., Glass, M. & Dragunow, M. High throughput quantification of mutant huntingtin aggregates. J. Neurosci. Methods 171, 174–179 (2008).
  Article CAS Google Scholar
• Ciammola, A. et al. Increased apoptosis, Huntingtin inclusions and altered differentiation in muscle cell cultures from Huntington's disease subjects. Cell Death Differ. 13, 2068–2078 (2006).
  Article CAS Google Scholar
• Khan, T. K. & Alkon, D. L. An internally controlled peripheral biomarker for Alzheimer's disease: Erk1 and Erk2 responses to the inflammatory signal bradykinin. Proc. Natl Acad. Sci. USA 103, 13203–13207 (2006).
  Article CAS Google Scholar
• Klegeris, A. & McGeer, P. L. Interaction of various intracellular signaling mechanisms involved in mononuclear phagocyte toxicity toward neuronal cells. J. Leukoc. Biol. 67, 127–133 (2000).
  Article CAS Google Scholar
• Klegeris, A., Bissonnette, C. J. & McGeer, P. L. Modulation of human microglia and THP-1 cell toxicity by cytokines endogenous to the nervous system. Neurobiol. Aging 26, 673–682 (2005).
  Article CAS Google Scholar
• Klegeris, A. et al. Prolyl endopeptidase is revealed following SILAC analysis to be a novel mediator of human microglial and THP-1 cell neurotoxicity. Glia 56, 675–685 (2008).
  Article Google Scholar
• Walsh, K., Megyesi, J. & Hammond, R. Human central nervous system tissue culture: a historical review and examination of recent advances. Neurobiol. Dis. 18, 2–18 (2005).
  Article CAS Google Scholar
• Konishi, Y., Lindholm, K., Yang, L. B., Li, R. & Shen, Y. Isolation of living neurons from human elderly brains using the immunomagnetic sorting DNA-linker system. Am. J. Pathol. 161, 1567–1576 (2002).
  Article CAS Google Scholar
• Brewer, G. J. et al. Culture and regeneration of human neurons after brain surgery. J. Neurosci. Methods 107, 15–23 (2001).
  Article CAS Google Scholar
• Verwer, R. W. et al. Cells in human postmortem brain tissue slices remain alive for several weeks in culture. Faseb J. 16, 54–60 (2002).
  Article CAS Google Scholar
• Brewer, G. J. & Torricelli, J. R. Isolation and culture of adult neurons and neurospheres. Nature Protoc. 2, 1490–1498 (2007).
  Article CAS Google Scholar
• Ayuso-Sacido, A., Roy, N. S., Schwartz, T. H., Greenfield, J. P. & Boockvar, J. A. Long-term expansion of adult human brain subventricular zone precursors. Neurosurgery 62, 223–229; discussion 229–231 (2008).
  Article Google Scholar
• Walton, N. M. et al. Derivation and large-scale expansion of multipotent astroglial neural progenitors from adult human brain. Development 133, 3671–3681 (2006).
  Article CAS Google Scholar
• Moe, M. C. et al. Multipotent progenitor cells from the adult human brain: neurophysiological differentiation to mature neurons. Brain 128, 2189–2199 (2005).
  Article Google Scholar
• Richardson, R. M., Holloway, K. L., Bullock, M. R., Broaddus, W. C. & Fillmore, H. L. Isolation of neuronal progenitor cells from the adult human neocortex. Acta Neurochir. (Wien) 148, 773–777 (2006).
  Article CAS Google Scholar
• Curtis, M. A. et al. Increased cell proliferation and neurogenesis in the adult human Huntington's disease brain. Proc. Natl Acad. Sci. USA 100, 9023–9027 (2003).
  Article CAS Google Scholar
• Curtis, M. A., Faull, R. L. & Eriksson, P. S. The effect of neurodegenerative diseases on the subventricular zone. Nature Rev. Neurosci. 8, 712–723 (2007).
  Article CAS Google Scholar
• Pillai, R. et al. Human astrocytes can be induced to differentiate into cells with neuronal phenotype. Exp. Cell Res. 312, 2336–2346 (2006).
  Article CAS Google Scholar
• Sharif, A. et al. Transforming growth factor alpha promotes sequential conversion of mature astrocytes into neural progenitors and stem cells. Oncogene 26, 2695–2706 (2007).
  Article CAS Google Scholar
• Takahashi, K. et al. Induction of pluripotent stem cells from adult human fibroblasts by defined factors. Cell 131, 861–872 (2007).
  Article CAS Google Scholar
• Chen, F. G. et al. Clonal analysis of nestin(–) vimentin(+) multipotent fibroblasts isolated from human dermis. J. Cell Sci. 120, 2875–2883 (2007).
  Article CAS Google Scholar
• Gingras, M., Champigny, M. F. & Berthod, F. Differentiation of human adult skin-derived neuronal precursors into mature neurons. J. Cell Physiol. 210, 498–506 (2007).
  Article CAS Google Scholar
• Kruse, C. et al. Towards the development of a pragmatic technique for isolating and differentiating nestin-positive cells from human scalp skin into neuronal and glial cell populations: generating neurons from human skin? Exp. Dermatol. 15, 794–800 (2006).
  Article Google Scholar
• Dhar, S., Yoon, E. S., Kachgal, S. & Evans, G. R. Long-term maintenance of neuronally differentiated human adipose tissue-derived stem cells. Tissue Eng. 13, 2625–2632 (2007).
  Article CAS Google Scholar
• Discher, D. E., Janmey, P. & Wang, Y. L. Tissue cells feel and respond to the stiffness of their substrate. Science 310, 1139–1143 (2005).
  Article CAS Google Scholar
• Edelman, D. B. & Keefer, E. W. A cultural renaissance: in vitro cell biology embraces three-dimensional context. Exp. Neurol. 192, 1–6 (2005).
  Article Google Scholar
• Roy, N. S., Chandler-Militello, D., Lu, G., Wang, S. & Goldman, S. A. Retrovirally mediated telomerase immortalization of neural progenitor cells. Nature Protoc. 2, 2815–2825 (2007).
  Article CAS Google Scholar
• Ponomarev, E. D., Novikova, M., Maresz, K., Shriver, L. P. & Dittel, B. N. Development of a culture system that supports adult microglial cell proliferation and maintenance in the resting state. J. Immunol. Methods 300, 32–46 (2005).
  Article CAS Google Scholar
• Pleasure, S. J., Page, C. & Lee, V. M. Pure, postmitotic, polarized human neurons derived from NTera 2 cells provide a system for expressing exogenous proteins in terminally differentiated neurons. J. Neurosci. 12, 1802–1815 (1992).
  Article CAS Google Scholar
• Gross, P. G., Kartalov, E. P., Scherer, A. & Weiner, L. P. Applications of microfluidics for neuronal studies. J. Neurol. Sci. 252, 135–143 (2007).
  Article Google Scholar
• Wang, Z., Kim, M. C., Marquez, M. & Thorsen, T. High-density microfluidic arrays for cell cytotoxicity analysis. Lab. Chip 7, 740–745 (2007).
  Article CAS Google Scholar
• Raiteri, M. Functional pharmacology in human brain. Pharmacol. Rev. 58, 162–193 (2006).
  Article CAS Google Scholar
• Whitebread, S., Hamon, J., Bojanic, D. & Urban, L. Keynote review: in vitro safety pharmacology profiling: an essential tool for successful drug development. Drug Discov. Today 10, 1421–1433 (2005).
  Article CAS Google Scholar
• Lee, S. M. et al. Minocycline inhibits apoptotic cell death via attenuation of TNF-α expression following iNOS/NO induction by lipopolysaccharide in neuron/glia co-cultures. J. Neurochem. 91, 568–578 (2004).
  Article CAS Google Scholar