Of monkeys and men: Analysis of the phase 2 double-blind, sham-surgery controlled, randomized trial of AAV2-neurturin gene therapy for parkinson's disease

Current Neurology and Neuroscience Reports

Volumn 11, Issue 4, 2011, Pages 345-348

  Huddleston, Daniel E ^a   Factor, Stewart A ^a  

^a EMORY UNIVERSITY SCHOOL OF MEDICINE   (United States)

Author keywords

AAV2; Animal models; Ceregene; GDNF; Gene delivery; Gene therapy; MPTP; Neuroimaging; Neurosurgical; Neurotrophic factors; Neurturin; Parkinson's; Randomized control trial; Sham surgery; Stereotactic; Viral vector

Indexed keywords

CERE 120; GLUTAMATE DECARBOXYLASE; NEURTURIN; PARVOVIRUS VECTOR; UNCLASSIFIED DRUG;

ARTICLE; HAPLORHINI; HISTOPATHOLOGY; HUMAN; INFLAMMATION; NERVE FIBER TRANSPORT; NONHUMAN; PARKINSON DISEASE; PHASE 2 CLINICAL TRIAL (TOPIC); POSITRON EMISSION TOMOGRAPHY; PUTAMEN; RANDOMIZED CONTROLLED TRIAL (TOPIC); SHAM PROCEDURE; SPECIES DIFFERENCE; TREATMENT RESPONSE; UNSPECIFIED SIDE EFFECT; VIRAL GENE DELIVERY SYSTEM; VIRAL GENE THERAPY;

EID: 85027957635     PISSN: 15284042     EISSN: 15346293     Source Type: Journal    
DOI: 10.1007/s11910-011-0206-y     Document Type: Article

References (10)

1. Lang AE, Gill S, Patel NK, et al. Randomized controlled trial of intraputamenal glial cell line-derived neurotrophic factor infusion in Parkinson disease. Ann Neurol. 2006;59(3):459-66. (Pubitemid 43358066)
2. LeWitt PA, Rezai AR, Leehey MA, et al. AAV2-GAD gene therapy for advanced Parkinson's disease: a double-blind, shamsurgery controlled, randomised trial. Lancet Neurology. 2011.
3. Gasmi M, Herzog CD, Brandon EP, et al. Striatal delivery of neurturin by CERE-120, an AAV2 vector for the treatment of dopaminergic neuron degeneration in Parkinson's Disease. Mol Ther. 2007;15(1):62-8. (Pubitemid 46043650)
4. Gash DM, Zhang Z, Ovadia A, et al. Functional recovery in parkinsonian monkeys treated with GDNF. Nature. 1996;380 (6571):252-5. (Pubitemid 26090648)
5. Kordower JH, Herzog CD, Dass B, et al. Delivery of neurturin by AAV2 (CERE-120)-mediated gene transfer provides structural and functional neuroprotection and neurorestoration in MPTP-treated monkeys. Ann Neurol. 2006;60(6):706-15. (Pubitemid 46048488)
6. Herzog CD, Dass B, Holden JE, et al. Striatal delivery of CERE- 120, an AAV2 vector encoding human neurturin, enhances activity of the dopaminergic nigrostriatal system in aged monkeys. Mov Disord. 2007;22(8):1124-32. (Pubitemid 47067222)
7. Bartus RT, Herzog CD, Chu Y, et al. Bioactivity of AAV2- neurturin gene therapy (CERE-120): differences between Parkinson's disease and nonhuman primate brains. Mov Disord. 2011;26 (1):27-36.
8. Kordower JH, Olanow CW. Regulatable promoters and gene therapy for Parkinson's disease: is the only thing to fear, fear itself? Exp Neurol. 2008;209(1):34-40.
9. Lewis TB, Standaert DG. Design of clinical trials of gene therapy in Parkinson disease. Exp Neurol. 2008;209(1):41-7.
10. Rommelfanger KS, Weinshenker D. Norepinephrine: the redheaded stepchild of Parkinson's disease. Biochem Pharmacol. 2007;74 (2):177-90. (Pubitemid 46890808)