Translational research on disease-modifying therapies for neurodegenerative diseases

Neurology and Clinical Neuroscience

Volumn 1, Issue 1, 2013, Pages 3-10

  Katsuno, Masahisa ^a   Watanabe, Hirohisa ^a   Tanaka, Fumiaki ^a,b   Sobue, Gen ^a  

^a NAGOYA UNIVERSITY GRADUATE SCHOOL OF MEDICINE   (Japan)

^b YOKOHAMA CITY UNIVERSITY MEDICAL CENTER   (Japan)

Author keywords

Biomarker; Clinical trial; Disease modifying therapy; Neurodegenerative disease; Outcome measure

Indexed keywords

ALPHA SYNUCLEIN; ALZHEIMER DISEASE VACCINE; AMYLOID BETA PROTEIN; AMYLOID BETA PROTEIN ANTIBODY; AMYLOID BETA VACCINE; AMYLOID PRECURSOR PROTEIN; ANDROGEN RECEPTOR; ANTIOXIDANT; BAPINEUZUMAB; CREATINE KINASE; DUTASTERIDE; LEUPRORELIN; MONOCLONAL ANTIBODY; POLYGLUTAMINE; SOLANEZUMAB; SOMATOMEDIN BINDING PROTEIN 2; UNCLASSIFIED DRUG;

ALZHEIMER DISEASE; AMYLOID PLAQUE; AMYOTROPHIC LATERAL SCLEROSIS; ARTICLE; DEGENERATIVE DISEASE; DRUG WITHDRAWAL; HUMAN; IMMUNIZATION; KENNEDY DISEASE; LEUKOENCEPHALOPATHY; MENINGOENCEPHALITIS; MOLECULAR PATHOLOGY; MULTICENTER STUDY (TOPIC); MUTATION; NEUROFIBRILLARY TANGLE; NONHUMAN; NUCLEAR MAGNETIC RESONANCE IMAGING; OUTCOME ASSESSMENT; PARKINSON DISEASE; PHASE 1 CLINICAL TRIAL (TOPIC); PHASE 2 CLINICAL TRIAL (TOPIC); PHASE 3 CLINICAL TRIAL (TOPIC); PHOSPHORYLATION; POSITRON EMISSION TOMOGRAPHY; PRIORITY JOURNAL; RANDOMIZED CONTROLLED TRIAL (TOPIC); SCORING SYSTEM; TRANSLATIONAL RESEARCH;

EID: 84902586007     PISSN: None     EISSN: 20494173     Source Type: Journal    
DOI: 10.1002/ncn3.7     Document Type: Article

References (41)

1. Katsuno M, Tanaka F, Sobue G. Perspectives on molecular targeted therapies and clinical trials for neurodegenerative diseases. J. Neurol. Neurosurg. Psychiatry. 2012; 83: 329-35.
2. Ballard C, Khan Z, Clack H, Corbett A. Nonpharmacological treatment of Alzheimer disease. Can. J. Psychiatry. 2011; 56: 589-95.
3. Zinman L, Cudkowicz M. Emerging targets and treatments in amyotrophic lateral sclerosis. Lancet Neurol. 2011; 10: 481-90.
4. Weiner MW, Veitch DP, Aisen PS et al. The Alzheimer's Disease Neuroimaging Initiative: a review of papers published since its inception. Alzheimers Dement. 2012; 8: S1-68.
5. Morris R, Mucke L. Alzheimer's disease: a needle from the haystack. Nature 2006; 440: 284-5.
6. Lemere CA, Masliah E. Can Alzheimer disease be prevented by amyloid-beta immunotherapy? Nat Rev Neurol 2010; 6: 108-19.
7. Mackenzie IR, Rademakers R, Neumann M. TDP-43 and FUS in amyotrophic lateral sclerosis and frontotemporal dementia. Lancet Neurol 2010; 9: 995-1007.
8. Shulman JM, De Jager PL, Feany MB. Parkinson's disease: genetics and pathogenesis. Annu. Rev. Pathol. 2011; 6: 193-222.
9. Di Prospero NA, Fischbeck KH. Therapeutics development for triplet repeat expansion diseases. Nat. Rev. Genet. 2005; 6: 756-65.
10. Schenk D, Barbour R, Dunn W et al. Immunization with amyloid-beta attenuates Alzheimer-disease-like pathology in the PDAPP mouse. Nature 1999; 400: 173-7.
11. DeMattos RB, Bales KR, Cummins DJ, Dodart JC, Paul SM, Holtzman DM. 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 2001; 98: 8850-5.
12. Hampel H, Frank R, Broich K et al. Biomarkers for Alzhei-mer's disease: academic, industry and regulatory perspectives. Nat Rev Drug Discov 2010; 9: 560-74.
13. Huang Y, Mucke L. Alzheimer mechanisms and therapeutic strategies. Cell 2012; 148: 1204-22.
14. Holmes C, Boche D, Wilkinson D et al. Long-term effects of Abeta42 immunisation in Alzheimer's disease: follow-up of a randomised, placebo-controlled phase I trial. Lancet 2008; 372: 216-23.
15. Masliah E, Hansen L, Adame A et al. Abeta vaccination effects on plaque pathology in the absence of encephalitis in Alzheimer disease. Neurology 2005; 64: 129-31.
16. Gilman S, Koller M, Black RS et al. Clinical effects of Abeta immunization (AN1792) in patients with AD in an interrupted trial. Neurology 2005; 64: 1553-62.
17. Fagan T, Strobel G. The solanezumab benefit: oh, so small, but probably real. [homepage on the internet]. Alzheimer Research Forum Cited 9 Oct 2012; 1 Nov 2012. Available from: URL http://www.alzforum.org/new/detail.asp?id=3288
18. Salloway S, Sperling R, Gilman S et al. A phase 2 multiple ascending dose trial of bapineuzumab in mild to moderate Alzheimer disease. Neurology 2009; 73: 2061-70.
19. Mangialasche F, Solomon A, Winblad B, Mecocci P, Kivipelto M. Alzheimer's disease: clinical trials and drug development. Lancet Neurol. 2010; 9: 702-16.
20. Jack CR, Jr, Knopman DS, Jagust WJ et al. Hypothetical model of dynamic biomarkers of the Alzheimer's pathological cascade. Lancet Neurol, 2010; 9: 119-28.
21. Ridha BH, Barnes J, Bartlett JW et al. Tracking atrophy progression in familial Alzheimer's disease: a serial MRI study. Lancet Neurol. 2006; 5: 828-34.
22. Orr HT, Zoghbi HY. Trinucleotide repeat disorders. Annu. Rev. Neurosci. 2007; 30: 575-621.
23. Katsuno M, Adachi H, Doyu M et al. Leuprorelin rescues polyglutamine-dependent phenotypes in a transgenic mouse model of spinal and bulbar muscular atrophy. Nat. Med. 2003; 9: 768-73.
24. Banno H, Katsuno M, Suzuki K et al. Phase 2 trial of leuprorelin in patients with spinal and bulbar muscular atrophy. Ann. Neurol. 2009; 65: 140-50.
25. Katsuno M, Banno H, Suzuki K et al. Efficacy and safety of leuprorelin in patients with spinal and bulbar muscular atrophy (JASMITT study): a multicentre, randomised, double-blind, placebo-controlled trial. Lancet Neurol. 2010; 9: 875-84.
26. Fernandez-Rhodes LE, Kokkinis AD, White MJ et al. Efficacy and safety of dutasteride in patients with spinal and bulbar muscular atrophy: a randomised placebo-controlled trial. Lancet Neurol. 2011; 10: 140-7.
27. Arrasate M, Mitra S, Schweitzer ES, Segal MR, Finkbeiner S. Inclusion body formation reduces levels of mutant huntingtin and the risk of neuronal death. Nature 2004; 431: 805-10.
28. Tomiyama T, Nagata T, Shimada H et al. A new amyloid beta variant favoring oligomerization in Alzheimer's-type dementia. Ann. Neurol. 2008; 63: 377-87.
29. Nagai Y, Inui T, Popiel HA et al. A toxic monomeric conformer of the polyglutamine protein. Nat. Struct. Mol. Biol. 2007; 14: 332-40.
30. Miller J, Arrasate M, Brooks E et al. Identifying polyglutamine protein species in situ that best predict neurodegeneration. Nat. Chem. Biol. 2011; 7: 925-34.
31. Pitteri SJ, JeBailey L, Faça VM et al. Integrated proteomic analysis of human cancer cells and plasma from tumor bearing mice for ovarian cancer biomarker discovery. PLoS ONE 2009; 4: e7916.
32. Ludolph AC, Bendotti C, Blaugrund E et al. Guidelines for preclinical animal research in ALS/MND: a consensus meeting. Amyotroph. Lateral. Scler. 2010; 11: 38-45.
33. Miller G. Alzheimer's research Stopping Alzheimer's before it starts. Science 2012; 337: 790-2.
34. Tabrizi SJ, Langbehn DR, Leavitt BR et al. Biological and clinical manifestations of Huntington's disease in the longitudinal TRACK-HD study: cross-sectional analysis of baseline data. Lancet Neurol. 2009; 8: 791-801.
35. Hashizume A, Katsuno M, Banno H et al. Difference in chronological changes of outcome measures between untreated and placebo-treated patients of spinal and bulbar muscular atrophy. J. Neurol. 2012; 259: 712-19.
36. Lesko LJ. Paving the Critical Path: how can clinical pharmacology help achieve the vision? Clin. Pharmacol. Ther. 2007; 81: 170-7.
37. Cudkowicz ME, Katz J, Moore DH et al. Toward more efficient clinical trials for amyotrophic lateral sclerosis. Amyotroph. Lateral. Scler. 2010; 11: 259-65.
38. Meissner WG, Frasier M, Gasser T et al. Priorities in Parkinson's disease research. Nat. Rev. Drug. Discov. 2011; 10: 377-93.
39. Gordon PH, Moore DH, Miller RG et al. Western ALS Study Group. Efficacy of minocycline in patients with amyotrophic lateral sclerosis: a phase III randomised trial. Lancet. Neurol. 2007; 6: 1045-53.
40. Wang SJ, Hung HM, O'Neill R. Adaptive design clinical trials and trial logistics models in CNS drug development. Eur. Neuropsychopharmacol. 2011; 21: 159-66.
41. Miller RG, Moore DH, Forshew DA et al. Phase II screening trial of lithium carbonate in amyotrophic lateral sclerosis: exam-ining a more efficient trial design. Neurology 2011; 77: 973-9.