A New VMAT-2 Inhibitor NBI-641449 in the Treatment of Huntington Disease

CNS Neuroscience and Therapeutics

Volumn 21, Issue 8, 2015, Pages 662-671

  Chen, Sheng ^a,b   Zhang, Xiao Jie ^a   Xie, Wen Jie ^c   Qiu, Hong Yan ^a   Liu, Hui ^a   Le, Wei Dong ^a,d  

^a SHANGHAI JIAO TONG UNIVERSITY SCHOOL OF MEDICINE   (China)

^b BAYLOR COLLEGE OF MEDICINE   (United States)

^c Diana Helis Henry Medical Research Foundation   (United States)

^d DALIAN MEDICAL UNIVERSITY   (China)

Author keywords

Endoplasmic reticulum stress; Huntington disease; NBI 641449; Therapeutics effects; VMAT 2 inhibitor

Indexed keywords

3,4 DIHYDROXYPHENYLACETIC ACID; DOPAMINE; DOPAMINE RECEPTOR BLOCKING AGENT; HOMOVANILLIC ACID; HUNTINGTIN; NBI 641449; NEURON SPECIFIC NUCLEAR PROTEIN; PHOSPHOPROTEIN DARPP 32; PROTEIN INHIBITOR; UNCLASSIFIED DRUG; NERVE PROTEIN; NEUN PROTEIN, MOUSE; NEUROPROTECTIVE AGENT; NUCLEAR PROTEIN; PPP1R1B PROTEIN, MOUSE; SEROTONIN TRANSPORTER; SLC18A2 PROTEIN, MOUSE; SLC6A4 PROTEIN, MOUSE; TYROSINE 3 MONOOXYGENASE; VESICULAR MONOAMINE TRANSPORTER;

AMINO ACID BRAIN LEVEL; ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; CELL SURVIVAL; CONTROLLED STUDY; CORPUS STRIATUM; DOPAMINE BRAIN LEVEL; DOSE RESPONSE; DOWN REGULATION; DRUG DOSE COMPARISON; DRUG EFFECT; DRUG EFFICACY; DRUG MEGADOSE; ENDOPLASMIC RETICULUM STRESS; FEMALE; HUNTINGTON CHOREA; HYPERKINESIA; LOCOMOTION; LOW DRUG DOSE; MOUSE; NERVE CELL; NONHUMAN; OPEN FIELD TEST; PROTEIN AGGREGATION; PROTEIN EXPRESSION; ROTAROD TEST; SEDATION; ANIMAL; ANTAGONISTS AND INHIBITORS; DISEASE MODEL; DRUG EFFECTS; HUNTINGTON DISEASE; METABOLISM; MOTOR ACTIVITY; PATHOLOGY; PATHOPHYSIOLOGY; PHYSIOLOGY; TRANSGENIC MOUSE;

3,4-DIHYDROXYPHENYLACETIC ACID; ANIMALS; CELL SURVIVAL; CORPUS STRIATUM; DISEASE MODELS, ANIMAL; DOPAMINE; DOPAMINE AND CAMP-REGULATED PHOSPHOPROTEIN 32; DOSE-RESPONSE RELATIONSHIP, DRUG; FEMALE; HOMOVANILLIC ACID; HUNTINGTON DISEASE; MICE, TRANSGENIC; MOTOR ACTIVITY; NERVE TISSUE PROTEINS; NEURONS; NEUROPROTECTIVE AGENTS; NUCLEAR PROTEINS; SEROTONIN PLASMA MEMBRANE TRANSPORT PROTEINS; TYROSINE 3-MONOOXYGENASE; VESICULAR MONOAMINE TRANSPORT PROTEINS;

EID: 84937517138     PISSN: 17555930     EISSN: 17555949     Source Type: Journal    
DOI: 10.1111/cns.12425     Document Type: Article

References (45)

1. Imarisio S, Carmichael J, Korolchuk V, et al. Huntington's disease: From pathology and genetics to potential therapies. Biochem J 2008;412:191-209.
2. Ross CA, Tabrizi SJ. Huntington's disease: From molecular pathogenesis to clinical treatment. Lancet Neurol 2011;10:83-98.
3. Gusella JF, MacDonald ME. Huntington's disease: Seeing the pathogenic process through a genetic lens. Trends Biochem Sci 2006;31:533-540.
4. Li SH, Li XJ. Huntingtin-protein interactions and the pathogenesis of Huntington's disease. Trends Genet 2004;20:146-154.
5. Reiner A, Dragatsis I, Dietrich P. Genetics and neuropathology of Huntington's disease. Int Rev Neurobiol 2011;98:325-372.
6. Tang TS, Chen X, Liu J, Bezprozvanny I. Dopaminergic signaling and striatal neurodegeneration in Huntington's disease. J Neurosci 2007;27:7899-7910.
7. Walker FO. Huntington's disease. Lancet 2007;369:220.
8. Caudle WM, Richardson JR, Wang MZ, et al. Reduced vesicular storage of dopamine causes progressive nigrostriatal neurodegeneration. J Neurosci 2007;27:8138-8148.
9. Gros Y, Schuldiner S. Directed evolution reveals hidden properties of VMAT, a neurotransmitter transporter. J Biol Chem 2010;285:5076-5084.
10. Staal RG, Hogan KA, Liang CL, German DC, Sonsalla PK. In vitro studies of striatal vesicles containing of the vesicular monoamine transporter (VMAT2): Rat versus mouse differences in sequestration of 1-methyl-4-phenylpyridinium. J Pharmacol Exp Ther 2000;293:329-335.
11. Zheng G, Dwoskin LP, Crooks PA. Vesicular monoamine transporter 2: Role as a novel target for drug development. AAPS J 2006;8:E682-E692.
12. Fasano A, Cadeddu F, Guidubaldi A, et al. The long-term effect of tetrabenazine in the management of Huntington disease. Clin Neuropharmacol 2008;31:313-318.
13. Frank S. Tetrabenazine: The first approved drug for the treatment of chorea in US patients with Huntington disease. Neuropsychiatr Dis Treat 2010;6:657-665.
14. Huntington Study Group. Tetrabenazine as antichorea therapy in Huntington disease: A randomized controlled trial. Neurology 2006;66:366-372.
15. Kenney C, Jankovic J. Tetrabenazine in the treatment of hyperkinetic movement disorders. Expert Rev Neurother 2006;6:7-17.
16. Le W, Rowe D, Xie WJ, Ortiz I, He Y, Appel SH. Microglial activation and dopaminergic cell injury: An in vitro model relevant to Parkinson's disease. J Neurosci 2001;21:8447-8455.
17. Wang H, Chen X, Li Y, Tang TS, Bezprozvanny I. Tetrabenazine is neuroprotective in Huntington's disease mice. Mol Neurodegener 2010;5:18.
18. Fuentes P, Paris I, Nassif M, Caviedes P, Segura-Aguilar J. Inhibition of VMAT-2 and DT-diaphorase induce cell death in a substantia nigra-derived cell line-an experimental cell model for dopamine toxicity studies. Chem Res Toxicol 2007;20:776-783.
19. Slow EJ, van Raamsdonk J, Rogers D, et al. Selective striatal neuronal loss in a YAC128 mouse model of Huntington disease. Hum Mol Genet 2003;12:1555-1567.
20. Graham RK, Slow EJ, Deng Y, et al. Levels of mutant huntingtin influence the phenotypic severity of Huntington disease in YAC128 mouse models. Neurobiol Dis 2006;21:444-455.
21. Menalled L, El-Khodor BF, Patry M, et al. Systematic behavioral evaluation of Huntington's disease transgenic and knock-in mouse models. Neurobiol Dis 2009;35:319-336.
22. Dey ND, Bombard MC, Roland BP, et al. Genetically engineered mesenchymal stem cells reduce behavioral deficits in the YAC128 mouse model of Huntington's disease. Behav Brain Res 2010;214:193-200.
23. Vidal RL, Hetz C. Crosstalk between the UPR and autophagy pathway contributes to handling cellular stress in neurodegenerative disease. Autophagy 2012;8:970-972.
24. Pan T, Kondo S, Le W, Jankovic J. The role of autophagy-lysosome pathway in neurodegeneration associated with Parkinson's disease. Brain 2008;131:1969-1978.
25. Zhang X, Chen S, Song L, et al. MTOR-independent, autophagic enhancer trehalose prolongs motor neuron survival and ameliorates the autophagic flux defect in a mouse model of amyotrophic lateral sclerosis. Autophagy 2014;10:588-602.
26. Jin ML, Park SY, Kim YH, Oh JI, Lee SJ, Park G. The neuroprotective effects of cordycepin inhibit glutamate-induced oxidative and ER stress-associated apoptosis in hippocampal HT22 cells. Neurotoxicology 2014;41:102-111.
27. Liu D, Zhang M, Yin H. Signaling pathways involved in endoplasmic reticulum stress-induced neuronal apoptosis. Int J Neurosci 2013;123:155-162.
28. Li X, Yang D, Li L, Peng C, Chen S, Le W. Proteasome inhibitor lactacystin disturbs the intracellular calcium homeostasis of dopamine neurons in ventral mesencephalic cultures. Neurochem Int 2007;50:959-965.
29. Du Y, Li X, Yang D, et al. Multiple molecular pathways are involved in the neuroprotection of GDNF against proteasome inhibitor induced dopamine neuron degeneration in vivo. Exp Biol Med (Maywood) 2008;233:881-890.
30. Ouimet CC, Langley-Guillon KC, Greengard P. Quantitative immunocytochemistry of DARPP-32-expressing neurons in the rat caudatoputamen. Brain Res 1998;808:8-12.
31. Gutekunst CA, Li SH, Yi H, et al. Nuclear and neuropil aggregates in Huntington's disease: Relationship to neuropathology. J Neurosci 1999;19:2522-2534.
32. Hayden MR, Leavitt BR, Yasothan U, Kirkpatrick P. Tetrabenazine. Nat Rev Drug Discov 2009;8:17-18.
33. Vonsattel JP, Myers RH, Stevens TJ, Ferrante RJ, Bird ED, Richardson EP. Neuropathological classification of Huntington's disease. J Neuropathol Exp Neurol 1985;44:559-577.
34. Montserrat A, Steven F. Protein aggregates in Huntington's disease. Exp Neurol 2012;238:1-11.
35. Davies SW, Scherzinger E. Nuclear inclusions in Huntington's disease. Trends Cell Biol 1997;7:422.
36. Becher MW, Kotzuk JA, Sharp AH, et al. Intranuclear neuronal inclusions in Huntington's disease and dentatorubral and pallidoluysian atrophy: Correlation between the density of inclusions and IT15 CAG triplet repeat length. Neurobiol Dis 1998;4:387-397.
37. Sapp EG, Aizawa H, Bird E, et al. Evidence for a preferential loss of enkephalin immunoreactivity in the external globus pallidus in low grade Huntington's disease using high resolution image analysis. Neuroscience 1995;64:397-404.
38. Zhang X, Smith DL, Meriin AB, et al. A potent small molecule inhibits polyglutamine aggregation in Huntington's disease neurons and suppresses neurodegeneration in vivo. Proc Natl Acad Sci U. S. A 2005;102:892-897.
39. Matus S, Glimcher LH, Hetz C. Protein folding stress in neurodegenerative diseases: A glimpse into the ER. Curr Opin Cell Biol 2011;23:239-252.
40. Reijonen S, Putkonen N, Norremolle A, Lindholm D, Korhonen L. Inhibition of endoplasmic reticulum stress counteracts neuronal cell death and protein aggregation caused by N-terminal mutant huntingtin proteins. Exp Cell Res 2008;314:950-960.
41. Chen S, Zhang X, Yang D, et al. D2/D3 receptor agonist ropinirole protects dopaminergic cell line against rotenone-induced apoptosis through inhibition of caspase- and JNK-dependent pathways. FEBS Lett 2008;582:603-610.
42. Zhang K, Kaufman RJ. The unfolded protein response: A stress signaling pathway critical for health and disease. Neurology 2006;66:S102-S109.
43. Aleksandar V. Treatment of Huntington disease. Curr Treat Options Neurol 2013;15:424-438.
44. Bonelli RM, Mahnert FA, Niederwieser G. Olanzapine for Huntington's disease: An open label study. Clin Neuropharmacol 2002;25:263-265.
45. Waters S, Ponten H, Klamer D, Waters N. Co-administration of the dopaminergic stabilizer pridopidine and tetrabenazine in rats. J Huntingtons Dis 2014;3:285-298.