Design of clinical trials of gene therapy in Parkinson disease

Experimental Neurology

Volumn 209, Issue 1, 2008, Pages 41-47

  Lewis, Travis B ^a   Standaert, David G ^a  

^a UNIVERSITY OF ALABAMA AT BIRMINGHAM   (United States)

Author keywords

Clinical trials; Endpoints; Gene therapy; Neuroprotection; Parkinson disease; Study design; Surrogate markers

Indexed keywords

BIOLOGICAL MARKER; GLUTAMATE DECARBOXYLASE;

ARTICLE; CLINICAL TRIAL; DISEASE COURSE; DOSE RESPONSE; GENE TARGETING; GENE THERAPY; GENETIC HETEROGENEITY; HUMAN; NEUROPROTECTION; PARKINSON DISEASE; PRIORITY JOURNAL; QUALITY OF LIFE; RISK BENEFIT ANALYSIS; THERAPY EFFECT; TREATMENT DURATION; VIRAL GENE THERAPY;

ANIMALS; BIOLOGICAL MARKERS; CLINICAL TRIALS AS TOPIC; DISEASE PROGRESSION; ENDPOINT DETERMINATION; GENE THERAPY; HUMANS; PARKINSON DISEASE; RESEARCH DESIGN; TREATMENT OUTCOME;

EID: 37549042441     PISSN: 00144886     EISSN: 10902430     Source Type: Journal    
DOI: 10.1016/j.expneurol.2007.08.012     Document Type: Article

References (37)

1. Albin R.L. Sham surgery controls: intracerebral grafting of fetal tissue for Parkinson's disease and proposed criteria for use of sham surgery controls. J. Med. Ethics 28 (2002) 322-325
2. Boeve B.F., Silber M.H., and Ferman T.J. REM sleep behavior disorder in Parkinson's disease and dementia with Lewy bodies. J. Geriatr. Psychiatry Neurol. 17 (2004) 146-157
3. Bowenkamp K.E., David D., Lapchak P.L., Henry M.A., Granholm A.C., Hoffer B.J., and Mahalik T.J. 6-Hydroxydopamine induces the loss of the dopaminergic phenotype in substantia nigra neurons of the rat. A possible mechanism for restoration of the nigrostriatal circuit mediated by glial cell line-derived neurotrophic factor. Exp. Brain Res. 111 (1996) 1-7
4. Braak H., de Vos R.A., Bohl J., and Del Tredici K. Gastric alpha-synuclein immunoreactive inclusions in Meissner's and Auerbach's plexuses in cases staged for Parkinson's disease-related brain pathology. Neurosci. Lett. 396 (2006) 67-72
5. Brooks D.J. Neuroimaging in Parkinson's disease. NeuroRx 1 (2004) 243-254
6. Clark L.N., Wang Y., Karlins E., Saito L., Mejia-Santana H., Harris J., Louis E.D., Cote L.J., Andrews H., Fahn S., Waters C., Ford B., Frucht S., Ottman R., and Marder K. Frequency of LRRK2 mutations in early- and late-onset Parkinson disease. Neurology 67 (2006) 1786-1791
7. Den Oudsten B.L., Van Heck G.L., and De Vries J. The suitability of patient-based measures in the field of Parkinson's disease: a systematic review. Mov. Disord. 22 (2007) 1390-1401
8. Fahn S., Oakes D., Shoulson I., Kieburtz K., Rudolph A., Lang A., Olanow C.W., Tanner C., and Marek K. Levodopa and the progression of Parkinson's disease. N. Engl. J. Med. 351 (2004) 2498-2508
9. Ferrari F.K., Samulski T., Shenk T., and Samulski R.J. Second-strand synthesis is a rate-limiting step for efficient transduction by recombinant adeno-associated virus vectors. J. Virol. 70 (1996) 3227-3234
10. Fox N., and Growdon J.H. Biomarkers and surrogates. NeuroRx 1 (2004) 181
11. Frank S., Kieburtz K., Holloway R., and Kim S.Y. What is the risk of sham surgery in Parkinson disease clinical trials? A review of published reports. Neurology 65 (2005) 1101-1103
12. Fukuchi K., Tahara K., Kim H.D., Maxwell J.A., Lewis T.L., Accavitti-Loper M.A., Kim H., Ponnazhagan S., and Lalonde R. Anti-Abeta single-chain antibody delivery via adeno-associated virus for treatment of Alzheimer's disease. Neurobiol. Dis. 23 (2006) 502-511
13. Goetz C.G., Fahn S., Martinez-Martin P., Poewe W., Sampaio C., Stebbins G.T., Stern M.B., Tilley B.C., Dodel R., Dubois B., Holloway R., Jankovic J., Kulisevsky J., Lang A.E., Lees A., Leurgans S., LeWitt P.A., Nyenhuis D., Olanow C.W., Rascol O., Schrag A., Teresi J.A., Van Hilten J.J., and LaPelle N. Movement Disorder Society-sponsored revision of the Unified Parkinson's Disease Rating Scale (MDS-UPDRS): process, format, and clinimetric testing plan. Mov. Disord. 22 (2007) 41-47
14. Goldstein D.S., Sharabi Y., Karp B.I., Bentho O., Saleem A., Pacak K., and Eisenhofer G. Cardiac sympathetic denervation preceding motor signs in Parkinson disease. Clin. Auton. Res. 17 (2007) 118-121
15. Hardy J., Cai H., Cookson M.R., Gwinn-Hardy K., and Singleton A. Genetics of Parkinson's disease and parkinsonism. Ann. Neurol. 60 (2006) 389-398
16. Hauck B., Zhao W., High K., and Xiao W. Intracellular viral processing, not single-stranded DNA accumulation, is crucial for recombinant adeno-associated virus transduction. J. Virol. 78 (2004) 13678-13686
17. Heffernan C., and Jenkinson C. Measuring outcomes for neurological disorders: a review of disease-specific health status instruments for three degenerative neurological conditions. Chronic Illn. 1 (2005) 131-142
18. Hughes A.J., Daniel S.E., Ben-Shlomo Y., and Lees A.J. The accuracy of diagnosis of parkinsonian syndromes in a specialist movement disorder service. Brain 125 (2002) 861-870
19. Kaplitt M.G., Feigin A., Tang C., Fitzsimons H.L., Mattis P., Lawlor P.A., Bland R.J., Young D., Strybing K., Eidelberg D., and During M.J. Safety and tolerability of gene therapy with an adeno-associated virus (AAV) borne GAD gene for Parkinson's disease: an open label, phase I trial. Lancet 369 (2007) 2097-2105
20. Kieburtz K. Designing neuroprotection trials in Parkinson's disease. Ann. Neurol. 53 Suppl 3 (2003) S100-S107 (discussion S107-109)
21. Kim S.Y., Frank S., Holloway R., Zimmerman C., Wilson R., and Kieburtz K. Science and ethics of sham surgery: a survey of Parkinson disease clinical researchers. Arch. Neurol. 62 (2005) 1357-1360
22. Langston J.W. The Parkinson's complex: parkinsonism is just the tip of the iceberg. Ann. Neurol. 59 (2006) 591-596
23. Miller D.H. Biomarkers and surrogate outcomes in neurodegenerative disease: lessons from multiple sclerosis. NeuroRx 1 (2004) 284-294
24. Movement Disorder Society Task Force on Rating Scales for Parkinson's Disease. The Unified Parkinson's Disease Rating Scale (UPDRS): status and recommendations. Mov. Disord 18 (2003) 738-750
25. National Commission for the Protection of Human Subjects of Biomedical and Behavioral Research. Belmont Report: Ethical Principles and Guidelines for the Protection of Human Subjects of Research (1979), US Government Printing Office, Washington, DC
26. NINDS NET-PD Investigators. A randomized, double-blind, futility clinical trial of creatine and minocycline in early Parkinson disease. Neurology 66 (2006) 664-671
27. Olanow C.W., Schapira A.H., LeWitt P.A., Kieburtz K., Sauer D., Olivieri G., Pohlmann H., and Hubble J. TCH346 as a neuroprotective drug in Parkinson's disease: a double-blind, randomised, controlled trial. Lancet Neurol. 5 (2006) 1013-1020
28. Parkinson's Study Group. Effect of deprenyl on the progression of disability in early Parkinson's disease. N. Engl. J.Med. 321 (1989) 1364-1371
29. Parkinson's Study Group. Effects of tocopherol and deprenyl on the progression of disability in Parkinson's disease. N. Engl. J. Med. 328 (1993) 176-183
30. Ravina B., Eidelberg D., Ahlskog J.E., Albin R.L., Brooks D.J., Carbon M., Dhawan V., Feigin A., Fahn S., Guttman M., Gwinn-Hardy K., McFarland H., Innis R., Katz R.G., Kieburtz K., Kish S.J., Lange N., Langston J.W., Marek K., Morin L., Moy C., Murphy D., Oertel W.H., Oliver G., Palesch Y., Powers W., Seibyl J., Sethi K.D., Shults C.W., Sheehy P., Stoessl A.J., and Holloway R. The role of radiotracer imaging in Parkinson disease. Neurology 64 (2005) 208-215
31. Schapira A.H., and Olanow C.W. Neuroprotection in Parkinson disease: mysteries, myths, and misconceptions. JAMA 291 (2004) 358-364
32. Scherfler C., Schwarz J., Antonini A., Grosset D., Valldeoriola F., Marek K., Oertel W., Tolosa E., Lees A.J., and Poewe W. Role of DAT-SPECT in the diagnostic work up of Parkinsonism. Mov Disord. 22 (2007) 1229-1238
33. Scherzer C.R., Eklund A.C., Morse L.J., Liao Z., Locascio J.J., Fefer D., Schwarzschild M.A., Schlossmacher M.G., Hauser M.A., Vance J.M., Sudarsky L.R., Standaert D.G., Growdon J.H., Jensen R.V., and Gullans S.R. Molecular markers of early Parkinson's disease based on gene expression in blood. Proc. Natl. Acad. Sci. U. S. A. 104 (2007) 955-960
34. Shoulson I. DATATOP: a decade of neuroprotective inquiry. Parkinson Study Group. Deprenyl and tocopherol antioxidative therapy of parkinsonism. Ann. Neurol. 44 (1998) S160-S166
35. Tolosa E., Wenning G., and Poewe W. The diagnosis of Parkinson's disease. Lancet Neurol. 5 (2006) 75-86
36. Welsh M., McDermott M.P., Holloway R.G., Plumb S., Pfeiffer R., and Hubble J. Development and testing of the Parkinson's disease quality of life scale. Mov. Disord. 18 (2003) 637-645
37. Xia C.F., Chu C., Li J., Wang Y., Zhang Y., Boado R.J., and Pardridge W.M. Comparison of cDNA and genomic forms of tyrosine hydroxylase gene therapy of the brain with Trojan horse liposomes. J. Gene Med. 9 (2007) 605-612