Therapeutic Development in Amyotrophic Lateral Sclerosis

Clinical Therapeutics

Volumn 37, Issue 3, 2015, Pages 668-680

  Bucchia, Monica ^a   Ramirez, Agnese ^a   Parente, Valeria ^a   Simone, Chiara ^a   Nizzardo, Monica ^a   Magri, Francesca ^a   Dametti, Sara ^a   Corti, Stefania ^a  

^a UNIVERSITY OF MILAN   (Italy)

Author keywords

amyotrophic lateral sclerosis; clinical trials; molecular targets; motor neuron disease; small molecules

Indexed keywords

RILUZOLE;

ADULT; AMYOTROPHIC LATERAL SCLEROSIS; DISEASE COURSE; HUMAN; PATHOPHYSIOLOGY; TREATMENT OUTCOME;

ADULT; AMYOTROPHIC LATERAL SCLEROSIS; DISEASE PROGRESSION; HUMANS; RILUZOLE; TREATMENT OUTCOME;

EID: 84939967299     PISSN: 01492918     EISSN: 1879114X     Source Type: Journal    
DOI: 10.1016/j.clinthera.2014.12.020     Document Type: Review

References (65)

1. G.M. Thomsen, G. Gowing, S. Svendsen, and C.N. Svendsen The past, present and future of stem cell clinical trials for ALS Exp Neurol 262 2014 127 137
2. M. DeJesus-Hernandez, I.R. Mackenzie, and B.F. Boeve Expanded GGGGCC hexanucleotide repeat in noncoding region of C9ORF72 causes chromosome 9p-linked FTD and ALS Neuron 72 2011 245 256
3. L. Ferraiuolo, J. Kirby, and A.J. Grierson Molecular pathways of motor neuron injury in amyotrophic lateral sclerosis Nat Rev Neurol 7 2011 616 630
4. R.K. Chaturvedi, and M. Flint Beal Mitochondrial diseases of the brain Free Radic Biol Med 63 2013 1 29
5. K.N. Alavian, S.I. Dworetzky, and L. Bonanni Effects of dexpramipexole on brain mitochondrial conductances and cellular bioenergetic efficiency Brain Res 1446 2012 1 11
6. M.E. Cudkowicz, L.H. van den Berg, and J.M. Shefner Dexpramipexole versus placebo for patients with amyotrophic lateral sclerosis [6]: a randomised, double-blind, phase 3 trial Lancet Neurol 12 2013 1059 1067
7. P.H. Gordon The murky path to drug discovery in ALS becomes clearer Lancet Neurol 12 2013 1037 1038
8. O. Weinreb, T. Amit, and O. Bar-Am Rasagiline: a novel anti-Parkinsonian monoamine oxidase-B inhibitor with neuroprotective activity Prog Neurobiol 92 2010 330 344
9. S. Waibel, A. Reuter, and S. Malessa Rasagiline alone and in combination with riluzole prolongs survival in an ALS mouse model J Neurol 251 2004 1080 1084
10. E.C. Hirsch, and S. Hunot Neuroinflammation in Parkinsons disease: a target for neuroprotection? Lancet Neurol 8 2009 382 397
11. K.M. Lucin, and T. Wyss-Coray Immune activation in brain aging and neurodegeneration: too much or too little? Neuron 64 2009 110 122
12. M.C. Evans, Y. Couch, and N. Sibson Inflammation and neurovascular changes in amyotrophic lateral sclerosis Mol Cell Neurosci 53 2013 34 41
13. P.D. Drew, J. Xu, and P.D. Storer Peroxisome proliferator-activated receptor agonist regulation of glial activation: relevance to CNS inflammatory disorders Neurochem Int 49 2006 183 189
14. A.M. Clement, M.D. Nguyen, and E.A. Roberts Wild-type nonneuronal cells extend survival of SOD1 mutant motor neurons in ALS mice Science 302 2003 113 117
15. T. Philips, and J.D. Rothstein Glial cells in amyotrophic lateral sclerosis Exp Neurol 2014
16. F. Rizzo, G. Riboldi, and S. Salani Cellular therapy to target neuroinflammation in amyotrophic lateral sclerosis Cell Mol Life Sci 71 2014 999 1015
17. J. Chun, and V. Brinkmann A mechanistically novel, first oral therapy for multiple sclerosis: the development of fingolimod (FTY720, Gilenya) Discov Med 12 2011 213 228
18. J.M. Lincecum, F.G. Vieira, and M.Z. Wang From transcriptome analysis to therapeutic anti-CD40L treatment in the SOD1 model of amyotrophic lateral sclerosis Nat Genet 42 2010 392 399
19. M.T. Mizwicki, M. Fiala, and L. Magpantay Tocilizumab attenuates inflammation in ALS patients through inhibition of IL6 receptor signaling Am J Neurodegener Dis 1 2012 305 315
20. M. Fiala, M.T. Mizwicki, and R. Weitzman Tocilizumab infusion therapy normalizes inflammation in sporadic ALS patients Am J Neurodegener Dis 2 2013 129 139
21. C.C. Tan, J.T. Yu, and M.S. Tan Autophagy in aging and neurodegenerative diseases: implications for pathogenesis and therapy Neurobiol Aging 35 2014 941 957
22. F. Fornai, P. Longone, and L. Cafaro Lithium delays progression of amyotrophic lateral sclerosis Proc Natl Acad Sci U S A 105 2008 2052 2057
23. J.H. Shin, S.I. Cho, and H.R. Lim Concurrent administration of Neu2000 and lithium produces marked improvement of motor neuron survival, motor function, and mortality in a mouse model of amyotrophic lateral sclerosis Mol Pharmacol 71 2007 965 975
24. UKMND-LiCALS Study Group K.E. Morrison, and S. Dhariwal Lithium in patients with amyotrophic lateral sclerosis [24]: a phase 3 multicentre, randomised, double-blind, placebo-controlled trial Lancet Neurol 12 2013 339 345
25. S.P. Aggarwal, L. Zinman, and E. Simpson Northeast and Canadian Amyotrophic Lateral Sclerosis consortia. Safety and efficacy of lithium in combination with riluzole for treatment of amyotrophic lateral sclerosis: a randomised, double-blind, placebo-controlled trial Lancet Neurol 9 2010 481 488
26. A. Chiò, G. Borghero, and A. Calvo Lithium carbonate in amyotrophic lateral sclerosis: lack of efficacy in a dose-finding trial Neurology 75 2010 619 625
27. R.G. Miller, D.H. Moore, and D.A. Forshew Phase II screening trial of lithium carbonate in amyotrophic lateral sclerosis: examining a more efficient trial design Neurology 77 2011 973 979
28. E. Verstraete, J.H. Veldink, and M.H. Huisman Lithium lacks effect on survival in amyotrophic lateral sclerosis: a phase IIb randomised sequential trial J Neurol Neurosurg Psychiatry 83 2012 557 564
29. P. Wicks, T.E. Vaughan, M.P. Massagli, and J. Heywood Accelerated clinical discovery using self-reported patient data collected online and a patient-matching algorithm Nat Biotechnol 29 2011 411 414
30. P. Dubreuil, S. Letard, and M. Ciufolini Masitinib (AB1010), a potent and selective tyrosine kinase inhibitor targeting KIT PLoS One 4 2009 e7258
31. L. Tönges, R. Günther, and M. Suhr Rho kinase inhibition modulates microglia activation and improves survival in a model of amyotrophic lateral sclerosis Glia 62 2014 217 232
32. M. Takata, H. Tanaka, and M. Kimura Fasudil, a rho kinase inhibitor, limits motor neuron loss in experimental models of amyotrophic lateral sclerosis Br J Pharmacol 170 2013 341 351
33. J.A. Moreno, M. Halliday, and C. Molloy Oral treatment targeting the unfolded protein response prevents neurodegeneration and clinical disease in prion-infected mice Sci Transl Med 5 2013 (206ra138)
34. H.J. Kim, A.R. Raphael, and E.S. LaDow Therapeutic modulation of eIF2α phosphorylation rescues TDP-43 toxicity in amyotrophic lateral sclerosis disease models Nat Genet 46 2014 152 160
35. S.H. Koh, Y. Kim, and H.Y. Kim Inhibition of glycogen synthase kinase-3 suppresses the onset of symptoms and disease progression of G93A-SOD1 mouse model of ALS Exp Neurol 205 2007 336 346
36. F.P. Di Giorgio, M.A. Carrasco, and M.C. Siao Non-cell autonomous effect of glia on motor neurons in an embryonic stem cell-based ALS model Nat Neurosci 10 2007 608 614
37. Z. Yao, G. Zhou, and X.S. Wang A novel human STE20-related protein kinase, HGK, that specifically activates the c-Jun N-terminal kinase signaling pathway J Biol Chem 274 1999 2118 2125
38. Y.M. Yang, S.K. Gupta, and K.J. Kim A small molecule screen in stem-cell-derived motor neurons identifies a kinase inhibitor as a candidate therapeutic for ALS Cell Stem Cell 12 2013 713 726
39. M. van Blitterswijk, S. Gulati, and E. Smoot Anti-superoxide dismutase antibodies are associated with survival in patients with sporadic amyotrophic lateral sclerosis Amyotroph Lateral Scler 12 2011 430 438
40. R.A. Smith, T.M. Miller, and K. Yamanaka Antisense oligonucleotide therapy for neurodegenerative disease J Clin Invest 116 2006 2290 2296
41. T.M. Miller, A. Pestronk, and W. David An antisense oligonucleotide against SOD1 delivered intrathecally for patients with SOD1 familial amyotrophic lateral sclerosis: a phase 1, randomised, first-in-man study Lancet Neurol 12 2013 435 442
42. C.J. Donnelly, P.W. Zhang, and J.T. Pham RNA toxicity from the ALS/FTD C9ORF72 expansion is mitigated by antisense intervention Neuron 80 2013 415 420
43. C. Lagier-Tourenne, M. Baughn, and F. Rigo Targeted degradation of sense and antisense C9orf72 RNA foci as therapy for ALS and frontotemporal degeneration Proc Natl Acad Sci 110 2013 E4530 E4539
44. D. Sareen, J.G. ORourke, and P. Meera Targeting RNA foci in iPSC-derived motor neurons from ALS patients with a C9ORF72 repeat expansion Sci Transl Med 5 2013 (208ra149)
45. T. Zu, Y. Liu, and M. Bañez-Coronel RAN proteins and RNA foci from antisense transcripts in C9ORF72 ALS and frontotemporal dementia Proc Natl Acad Sci 110 2013 E4968 E4977
46. M.D. Disney, I. Yildirim, and J.L. Childs-Disney Methods to enable the design of bioactive small molecules targeting RNA Org Biomol Chem 12 2014 1029 1039
47. N.M. Boulis, T. Federici, and J.D. Glass Translational stem cell therapy for amyotrophic lateral sclerosis Nat Rev Neurol 8 2011 172 176
48. A.C. Lepore, J. ODonnell, and A.S. Kim Human glial-restricted progenitor transplantation into cervical spinal cord of the SOD1 mouse model of ALS PLoS One 6 2011 e25968
49. A. Serio, B. Bilican, and S.J. Barmada Astrocytes pathology and the absence of non-cell autonomy in an induced pluripotent stem cells model of TDP-43 proteinopathy Proc Natl Acad Sci U S A 110 2013 4697 4702
50. D.B. Re, V. Le Verche, and C. Yu Necroptosis Drives Motor Neuron Death in Models of Both Sporadic and Familial ALS Neuron 81 2014 1001 1008
51. M. Suzuki, and C.N. Svendsen Combining growth factor and stem cell therapy for amyotrophic lateral sclerosis Trends Neurosci 31 2008 192 198
52. F.H. Gage, and S. Temple Neural stem cells: generating and regenerating the brain Neuron 80 2013 588 601
53. M. Nizzardo, C. Simone, and F. Rizzo Minimally invasive transplantation of iPSC-derived ALDHhiSSCloVLA4+ neural stem cells effectively improves the phenotype of an amyotrophic lateral sclerosis model Hum Mol Genet 23 2014 342 354
54. Y.D. Teng, S.C. Benn, and S.N. Kalkanis Multimodal actions of neural stem cells in a mouse model of ALS: a meta-analysis Sci Transl Med 4 2012 (165ra164)
55. J. Riley, T. Federici, and M. Polak Intraspinal stem cell transplantation in amyotrophic lateral sclerosis: a phase I safety trial, technical note, and lumbar safety outcomes Neurosurgery 71 2012 405 416
56. M. Johannessen, S. Ramachandran, and L. Riemer Voltage-gated sodium channel modulation by sigma-receptors in cardiac myocytes and heterologous systems Am J Physiol Cell Physiol 296 2009 C1049 C1057
57. P.H. Gordon Amyotrophic Lateral Sclerosis: An update for 2013 Clinical Features, Pathophysiology, Management and Therapeutic Trials Aging Dis 4 2013 295 310
58. V. Meininger, P.F. Pradat, and A. Corse Safety, pharmacokinetic, and functional effects of the nogo-a monoclonal antibody in amyotrophic lateral sclerosis: a randomized, first-in-human clinical trial PloS One 9 2014 e97803
59. H. Kwieciński, B. Ryniewicz, and A. Ostrzycki Treatment of myotonia with antiarrhythmic drugs Acta Neurol Scand 86 1992 371 375
60. M. Nagai, D.B. Re, and T. Nagata Astrocytes expressing ALS-linked mutated SOD1 release factors selectively toxic to motor neurons Nat Neurosci 10 2007 615 622
61. E. Fritz, P. Izaurieta, and A. Weiss Mutant SOD1-expressing astrocytes release toxic factors that trigger motoneuron death by inducing hyperexcitability J Neurophysiol 109 2013 2803 2814
62. A.J. Russell, J.J. Hartman, and A.C. Hinken Activation of fast skeletal muscle troponin as a potential therapeutic approach for treating neuromuscular diseases Nat Med 18 2012 452 455
63. J.M. Shefner, M.L. Watson, and L. Meng A study to evaluate safety and tolerability of repeated doses of tirasemtiv in patients with amyotrophic lateral sclerosis Amyotroph Lateral Scler Frontotemporal Degener 14 2013 574 581
64. A. Van Hoecke, L. Schoonaert, and R. Lemmens EPHA4 is a disease modifier of amyotrophic lateral sclerosis in animal models and in humans Nat Med 18 2012 1418 1422
65. I. Lamberto, H. Qin, and R. Noberini Distinctive binding of three antagonistic peptides to the ephrin-binding pocket of the EphA4 receptor Biochem J 445 2012 47 56