Targets in ALS: designing multidrug therapies

Trends in Pharmacological Sciences

Volumn 27, Issue 5, 2006, Pages 267-273

  Carrì, Maria Teresa ^a,b   Grignaschi, Giuliano ^c   Bendotti, Caterina ^c  

^a UNIVERSITY OF ROME TOR VERGATA   (Italy)

^b EUROPEAN BRAIN RESEARCH INSTITUTE   (Italy)

^c MARIO NEGRI INSTITUTE FOR PHARMACOLOGICAL RESEARCH   (Italy)

Author keywords

[No Author keywords available]

Indexed keywords

2,3 DIMETHYL 6 PHENYL 12H [1,3]DIOXOLO[4,5 H]IMIDAZOL[1,2 C][2,3]BENZODIAZEPINE; 5,5 DIMETHYL 1 PYRROLINE 1 OXIDE; AEOL 10150; AMPA RECEPTOR ANTAGONIST; ANTIINFLAMMATORY AGENT; ARIMOCLOMOL; BENZYLOXYCARBONYLVALYLALANYLASPARTYLFLUOROMETHYL KETONE; CEFTRIAXONE; CELECOXIB; COPPER ZINC SUPEROXIDE DISMUTASE; CREATINE; DYNACTIN; DYNEIN ADENOSINE TRIPHOSPHATASE; GABAPENTIN; GLATIRAMER; GLIAL CELL LINE DERIVED NEUROTROPHIC FACTOR; GLUTAMATE RECEPTOR ANTAGONIST; GLUTAMIC ACID; MANGANASE TETRAKIS(N,N' DIETHYLIMIDAZOLIUM 2 YL)PORPHYRIN; MINOCYCLINE; NIMODIPINE; ORPHAN DRUG; RECOMBINANT SOMATOMEDIN C; RILUZOLE; ROFECOXIB; SODIUM PHENYLBUTYRATE; SOMATOMEDIN C; TALAMPANEL; TOPIRAMATE; UBIDECARENONE; UNCLASSIFIED DRUG; VASCULOTROPIN; VIRUS VECTOR; ZK 187638;

AMYOTROPHIC LATERAL SCLEROSIS; CLINICAL TRIAL; COMPLEX FORMATION; CONVALESCENCE; CORD BLOOD STEM CELL TRANSPLANTATION; DISEASE COURSE; DRUG DELIVERY SYSTEM; DRUG EFFICACY; DRUG SCREENING; DRUG SELECTIVITY; DRUG TARGETING; EXERCISE; GENE MUTATION; GLIA; HUMAN; LIFESPAN; LIPID DIET; MOLECULAR EVOLUTION; MOLECULAR MECHANICS; MOTONEURON; MOTOR NEURON DISEASE; NERVE CELL LESION; NERVE CELL NECROSIS; NEUROTOXICITY; NONHUMAN; PRIORITY JOURNAL; PROTEIN EXPRESSION; QUALITY OF LIFE; REVIEW; RODENT; TRANSGENIC ANIMAL;

AMYOTROPHIC LATERAL SCLEROSIS; ANIMALS; DISEASE MODELS, ANIMAL; DRUG DELIVERY SYSTEMS; DRUG THERAPY, COMBINATION; HUMANS; MICE; STEM CELL TRANSPLANTATION; SUPEROXIDE DISMUTASE;

EID: 33646230035     PISSN: 01656147     EISSN: None     Source Type: Journal    
DOI: 10.1016/j.tips.2006.03.009     Document Type: Review

References (71)

1. Bendotti C., and Carri M.T. Lessons from models of SOD1-linked familial ALS. Trends Mol. Med. 10 (2004) 393-400
2. Valentine J.S., et al. Copper-zinc superoxide dismutase and amyotrophic lateral sclerosis. Annu. Rev. Biochem. 74 (2005) 563-593
3. Turner M.R., et al. Clinical trials in ALS: an overview. Semin. Neurol. 21 (2001) 167-175
4. Miller R.G., et al. Riluzole for amyotrophic lateral sclerosis (ALS)/motor neuron disease (MND). Amyotroph. Lateral Scler. Other Motor Neuron Disord. 4 (2003) 191-206
5. Gordon P.H. Advances in clinical trials for amyotrophic lateral sclerosis. Curr. Neurol. Neurosci. Rep. 5 (2005) 48-54
6. Shibata N. Transgenic mouse model for familial amyotrophic lateral sclerosis with superoxide dismutase-1 mutation. Neuropathology 21 (2001) 82-92
7. Gurney M.E., et al. Motor neuron degeneration in mice that express a human Cu,Zn superoxide dismutase mutation. Science 264 (1994) 1772-1775
8. Shaw P.J. Molecular and cellular pathways of neurodegeneration in motor neurone disease. J. Neurol. Neurosurg. Psychiatry 76 (2005) 1046-1057
9. Andersen P.M., et al. EFNS task force on management of amyotrophic lateral sclerosis: guidelines for diagnosing and clinical care of patients and relatives. Eur. J. Neurol. 12 (2005) 921-938
10. Heath P.R., and Shaw P.J. Update on the glutamatergic neurotransmitter system and the role of excitotoxicity in amyotrophic lateral sclerosis. Muscle Nerve 26 (2002) 438-458
11. Ludolph, A.C. et al. (2000) The role of excitotoxicity in ALS - what is the evidence? J. Neurol. 247 (Suppl. 1) I7-16
12. Guo H., et al. Increased expression of the glial glutamate transporter EAAT2 modulates excitotoxicity and delays the onset but not the outcome of ALS in mice. Hum. Mol. Genet. 12 (2003) 2519-2532
13. Rothstein J.D., et al. β-Lactam antibiotics offer neuroprotection by increasing glutamate transporter expression. Nature 433 (2005) 73-77
14. Kawahara Y., et al. Glutamate receptors: RNA editing and death of motor neurons. Nature 427 (2004) 801
15. Tortarolo M., et al. Glutamate AMPA receptors change in motor neurons of SOD1(G93A) transgenic mice and their inhibition by a noncompetitive antagonist ameliorates the progression of amytrophic lateral sclerosis-like disease. J. Neurosci. Res. 8 (2006) 134-146
16. Spalloni A., et al. Cu/Zn-superoxide dismutase (GLY93→ALA) mutation alters AMPA receptor subunit expression and function and potentiates kainate-mediated toxicity in motor neurons in culture. Neurobiol. Dis. 15 (2004) 340-350
17. Rembach A., et al. Antisense peptide nucleic acid targeting GluR3 delays disease onset and progression in the SOD1 G93A mouse model of familial ALS. J. Neurosci. Res. 77 (2004) 573-582
18. Kuner R., et al. Late-onset motoneuron disease caused by a functionally modified AMPA receptor subunit. Proc. Natl. Acad. Sci. U. S. A. 102 (2005) 5826-5831
19. Canton T., et al. RPR 119990, a novel α-amino-3-hydroxy-5-methyl-4-isoxazolepropionic acid antagonist: synthesis, pharmacological properties, and activity in an animal model of amyotrophic lateral sclerosis. J. Pharmacol. Exp. Ther. 299 (2001) 314-322
20. Van Damme P., et al. The AMPA receptor antagonist NBQX prolongs survival in a transgenic mouse model of amyotrophic lateral sclerosis. Neurosci. Lett. 343 (2003) 81-84
21. Bendotti C., et al. Early vacuolization and mitochondrial damage in motor neurons of FALS mice are not associated with apoptosis or with changes in cytochrome oxidase histochemical reactivity. J. Neurol. Sci. 191 (2001) 25-33
22. Dupuis L., et al. Evidence for defective energy homeostasis in amyotrophic lateral sclerosis: benefit of a high-energy diet in a transgenic mouse model. Proc. Natl. Acad. Sci. U. S. A. 101 (2004) 11159-11164
23. Siklos L., et al. Ultrastructural evidence for altered calcium in motor nerve terminals in amyotropic lateral sclerosis. Ann. Neurol. 39 (1996) 203-216
24. Cozzolino M., et al. Apaf1 mediates apoptosis and mitochondrial damage induced by mutant human SOD1s typical of familial amyotrophic lateral sclerosis. Neurobiol. Dis. 21 (2006) 69-79
25. Liu J., et al. Toxicity of familial ALS-linked SOD1 mutants from selective recruitment to spinal mitochondria. Neuron 43 (2004) 5-17
26. Vijayvergiya C., et al. Mutant superoxide dismutase 1 forms aggregates in the brain mitochondrial matrix of amyotrophic lateral sclerosis mice. J. Neurosci. 25 (2005) 2463-2470
27. Pasinelli P., et al. Amyotrophic lateral sclerosis-associated SOD1 mutant proteins bind and aggregate with Bcl-2 in spinal cord mitochondria. Neuron 43 (2004) 19-30
28. Kirkinezos I.G., et al. Cytochrome c association with the inner mitochondrial membrane is impaired in the CNS of G93A-SOD1 mice. J. Neurosci. 25 (2005) 164-172
29. Kieran D., et al. Treatment with arimoclomol, a coinducer of heat shock proteins, delays disease progression in ALS mice. Nat. Med. 10 (2004) 402-405
30. Carri M.T., et al. Neurodegeneration in amyotrophic lateral sclerosis: the role of oxidative stress and altered homeostasis of metals. Brain Res. Bull. 61 (2003) 365-374
31. Kaspar B.K., et al. Retrograde viral delivery of IGF-1 prolongs survival in a mouse ALS model. Science 301 (2003) 839-842
32. Dobrowolny G., et al. Muscle expression of a local Igf-1 isoform protects motor neurons in an ALS mouse model. J. Cell Biol. 168 (2005) 193-199
33. Azzouz M., et al. VEGF delivery with retrogradely transported lentivector prolongs survival in a mouse ALS model. Nature 429 (2004) 413-417
34. Storkebaum E., et al. Treatment of motoneuron degeneration by intracerebroventricular delivery of VEGF in a rat model of ALS. Nat. Neurosci. 8 (2005) 85-92
35. Lambrechts D., et al. VEGF is a modifier of amyotrophic lateral sclerosis in mice and humans and protects motoneurons against ischemic death. Nat. Genet. 34 (2003) 383-394
36. Streit W.J., et al. Role of microglia in the central nervous system's immune response. Neurol. Res. 27 (2005) 685-691
37. Weydt P., and Moller T. Neuroinflammation in the pathogenesis of amyotrophic lateral sclerosis. Neuroreport 16 (2005) 527-531
38. Hensley K., et al. Message and protein-level elevation of tumor necrosis factor α (TNF α) and TNF α-modulating cytokines in spinal cords of the G93A-SOD1 mouse model for amyotrophic lateral sclerosis. Neurobiol. Dis. 14 (2003) 74-80
39. Veglianese P., et al. Activation of the p38MAPK cascade is associated with upregulation of TNF α receptors in the spinal motor neurons of mouse models of familial ALS. Mol. Cell. Neurosci. 31 (2006) 218-231
40. Ackerley S., et al. p38α stress-activated protein kinase phosphorylates neurofilaments and is associated with neurofilament pathology in amyotrophic lateral sclerosis. Mol. Cell. Neurosci. 26 (2004) 354-364
41. Tortarolo M., et al. Persistent activation of p38 mitogen-activated protein kinase in a mouse model of familial amyotrophic lateral sclerosis correlates with disease progression. Mol. Cell. Neurosci. 23 (2003) 180-192
42. Barbeito L.H., et al. A role for astrocytes in motor neuron loss in amyotrophic lateral sclerosis. Brain Res. Brain Res. Rev. 47 (2004) 263-274
43. Hafezparast M., et al. Mutations in dynein link motor neuron degeneration to defects in retrograde transport. Science 300 (2003) 808-812
44. Puls I., et al. Distal spinal and bulbar muscular atrophy caused by dynactin mutation. Ann. Neurol. 57 (2005) 687-694
45. Kieran D., et al. A mutation in dynein rescues axonal transport defects and extends the life span of ALS mice. J. Cell Biol. 169 (2005) 561-567
46. Gurney M.E., et al. Riluzole preserves motor function in a transgenic model of familial amyotrophic lateral sclerosis. Neurology 50 (1998) 62-66
47. Beghi E., et al. Merits of a new drug trial for ALS?. Science 308 (2005) 632-633
48. Klivenyi P., et al. Neuroprotective effects of creatine in a transgenic animal model of amyotrophic lateral sclerosis. Nat. Med. 5 (1999) 347-350
49. Klivenyi P., et al. Additive neuroprotective effects of creatine and cyclooxygenase 2 inhibitors in a transgenic mouse model of amyotrophic lateral sclerosis. J. Neurochem. 88 (2004) 576-582
50. Bruijn L.I., et al. Unraveling the mechanisms involved in motor neuron degeneration in ALS. Annu. Rev. Neurosci. 27 (2004) 723-749
51. Zhang W., et al. Additive neuroprotective effects of minocycline with creatine in a mouse model of ALS. Ann. Neurol. 53 (2003) 267-270
52. Ralph G.S., et al. Silencing mutant SOD1 using RNAi protects against neurodegeneration and extends survival in an ALS model. Nat. Med. 11 (2005) 429-433
53. Raoul C., et al. Lentiviral-mediated silencing of SOD1 through RNA interference retards disease onset and progression in a mouse model of ALS. Nat. Med. 11 (2005) 423-428
54. Silani V., and Leigh N. Stem therapy for ALS: hope and reality. Amyotroph. Lateral Scler. Other Motor Neuron Disord. 4 (2003) 8-10
55. Garbuzova-Davis S., et al. Intravenous administration of human umbilical cord blood cells in a mouse model of amyotrophic lateral sclerosis: distribution, migration, and differentiation. J. Hematother. Stem Cell Res. 12 (2003) 255-270
56. Klein S.M., et al. GDNF delivery using human neural progenitor cells in a rat model of ALS. Hum. Gene Ther. 16 (2005) 509-521
57. Hemendinger R., et al. Sertoli cells improve survival of motor neurons in SOD1 transgenic mice, a model of amyotrophic lateral sclerosis. Exp. Neurol. 196 (2005) 235-243
58. Kaspar B.K., et al. Synergy of insulin-like growth factor-1 and exercise in amyotrophic lateral sclerosis. Ann. Neurol. 57 (2005) 649-655
59. Nagai M., et al. Rats expressing human cytosolic copper-zinc superoxide dismutase transgenes with amyotrophic lateral sclerosis: associated mutations develop motor neuron disease. J. Neurosci. 21 (2001) 9246-9254
60. Wang J., et al. Fibrillar inclusions and motor neuron degeneration in transgenic mice expressing superoxide dismutase 1 with a disrupted copper-binding site. Neurobiol. Dis. 10 (2002) 128-138
61. Wang J., et al. Coincident thresholds of mutant protein for paralytic disease and protein aggregation caused by restrictively expressed superoxide dismutase cDNA. Neurobiol. Dis. 20 (2005) 943-952
62. Oosthuyse B., et al. Deletion of the hypoxia-response element in the vascular endothelial growth factor promoter causes motor neuron degeneration. Nat. Genet. 28 (2001) 131-138
63. Schmitt-John T., et al. Mutation of Vps54 causes motor neuron disease and defective spermiogenesis in the wobbler mouse. Nat. Genet. 37 (2005) 1213-1215
64. Cai H., et al. Loss of ALS2 function is insufficient to trigger motor neuron degeneration in knock-out mice but predisposes neurons to oxidative stress. J. Neurosci. 25 (2005) 7567-7574
65. Angelov D.N., et al. Therapeutic vaccine for acute and chronic motor neuron diseases: implications for amyotrophic lateral sclerosis. Proc. Natl. Acad. Sci. U. S. A. 100 (2003) 4790-4795
66. Ryu H., et al. Sodium phenylbutyrate prolongs survival and regulates expression of anti-apoptotic genes in transgenic amyotrophic lateral sclerosis mice. J. Neurochem. 93 (2005) 1087-1098
67. Li M., et al. Functional role of caspase-1 and caspase-3 in an ALS transgenic mouse model. Science 288 (2000) 335-339
68. Liu R., et al. Increased mitochondrial antioxidative activity or decreased oxygen free radical propagation prevent mutant SOD1-mediated motor neuron cell death and increase amyotrophic lateral sclerosis-like transgenic mouse survival. J. Neurochem. 80 (2002) 488-500
69. 10 administration increases brain mitochondrial concentrations and exerts neuroprotective effects. Proc. Natl. Acad. Sci. U. S. A. 95 (1998) 8892-8897
70. Crow J.P., et al. Manganese porphyrin given at symptom onset markedly extends survival of ALS mice. Ann. Neurol. 58 (2005) 258-265
71. Kriz J., et al. Efficient three-drug cocktail for disease induced by mutant superoxide dismutase. Ann. Neurol. 53 (2003) 429-436