The potential of GDNF as a treatment for Parkinson's disease

Experimental Neurology

Volumn 185, Issue 1, 2004, Pages 1-6

  Hurelbrink, Carrie B ^a   Barker, Roger A ^a  

^a UNIVERSITY OF CAMBRIDGE   (United Kingdom)

Author keywords

[No Author keywords available]

Indexed keywords

ADENOVIRUS VECTOR; DOPAMINE; DOPAMINE RECEPTOR STIMULATING AGENT; DOXYCYCLINE; GLIAL CELL LINE DERIVED NEUROTROPHIC FACTOR; LENTIVIRUS VECTOR; LEVODOPA; MICROSPHERE; NEUROTROPHIC FACTOR; PARVOVIRUS VECTOR; VIRUS VECTOR;

ANOREXIA; BASAL GANGLION; BEHAVIOR DISORDER; CELL BASED GENE THERAPY; CELL ENCAPSULATION; CELL LOSS; CELL SURVIVAL; CELL TRANSPLANTATION; CLINICAL TRIAL; CORPUS STRIATUM; DISEASE MODEL; DOPAMINE RELEASE; DOPAMINERGIC NERVE CELL; DRUG DELIVERY SYSTEM; DRUG EFFECT; DRUG EFFICACY; DRUG SAFETY; FIBROBLAST; GENE EXPRESSION REGULATION; GENETIC ENGINEERING; GENETIC TRANSDUCTION; HAMSTER; HUMAN; HYPONATREMIA; IMPLANTATION; KIDNEY CELL; LATERAL BRAIN VENTRICLE; MARMOSET; MONKEY; MOTOR DYSFUNCTION; NAUSEA; NERVE FIBER GROWTH; NERVE PROJECTION; NEUROPROTECTION; NIGRONEOSTRIATAL SYSTEM; NONHUMAN; NOTE; PARENCHYMA; PARESTHESIA; PARKINSON DISEASE; PRIORITY JOURNAL; PROMOTER REGION; ROTATION; SIDE EFFECT; STEM CELL; SUBSTANTIA NIGRA; TREATMENT OUTCOME; VIRAL GENE DELIVERY SYSTEM; VIRAL GENE THERAPY; VOMITING; WEIGHT REDUCTION;

EID: 0345732569     PISSN: 00144886     EISSN: None     Source Type: Journal    
DOI: 10.1016/j.expneurol.2003.09.018     Document Type: Note

References (66)

1. Akerud P., Alberch J., Eketjall S., Wagner J., Arenas E. Differential effects of glial cell line-derived neurotrophic factor and neurturin on developing and adult substantia nigra dopaminergic neurons. J. Neurochem. 73:1999;70-78.
2. Akerud P., Canals J.M., Snyder E.Y., Arenas E. Neuroprotection through delivery of glial cell line-derived neurotrophic factor by neural stem cells in a mouse model of Parkinson's disease. J. Neurosci. 21:2001;8108-8118.
3. Annett L.E., Rogers D.C., Hernandez T.D., Dunnett S.B. Behavioural analysis of unilateral monoamine depletion in the marmoset. Brain. 115:1992;825-856.
4. Aoi M., Date I., Tomita S., Ohmoto T. GDNF induces recovery of the nigrostriatal dopaminergic system in the rat brain following intracerebroventricular or intraparenchymal administration. Acta Neurochir. (Wien.). 142:2000;805-810.
5. Aoi M., Date I., Tomita S., Ohmoto T. Single administration of GDNF into the striatum induced protection and repair of the nigrostriatal dopaminergic system in the intrastriatal 6-hydroxydopamine injection model of hemiparkinsonism. Restor. Neurol. Neurosci. 17:2001;31-38.
6. Barker R.A. Repairing the brain in Parkinson's disease: where next? Mov. Disord. 17:2002;233-241.
7. Bensadoun J.C., Deglon N., Tseng J.L., Ridet J.L., Zurn A.D., Aebischer P. Lentiviral vectors as a gene delivery system in the mouse midbrain: cellular and behavioral improvements in a 6-OHDA model of Parkinson's disease using GDNF. Exp. Neurol. 164:2000;15-24.
8. Bilang-Bleuel A., Revah F., Colin P., Locquet I., Robert J.J., Mallet J., Horellou P. Intrastriatal injection of an adenoviral vector expressing glial-cell-line-derived neurotrophic factor prevents dopaminergic neuron degeneration and behavioral impairment in a rat model of Parkinson disease. Proc. Natl. Acad. Sci. U. S. A. 94:1997;8818-8823.
9. Bjorklund A., Dunnett S.B., Brundin P., Stoessl A.J., Freed C.R., Breeze R.E., Levivier M., Peschanski M., Studer L., Barker R. Neural transplantation for the treatment of Parkinson's disease. Lancet Neurol. 2:2003;437-445.
10. Blanchet P.J., Konitsiotis S., Mochizuki H., Pluta R., Emerich D.F., Chase T.N., Maral M.M. Complications of a trophic xenotransplant approach in parkinsonian monkeys. Prog. Neuro-Psychopharmacol. Biol. Psychiatry. 27:2003;607-612.
11. Bowenkamp K.E., Lapchak P.A., Hoffer B.J., Miller P.J., Bickford P.C. Intracerebroventricular glial cell line-derived neurotrophic factor improves motor function and supports nigrostriatal dopamine neurons in bilaterally 6-hydroxydopamine lesioned rats. Exp. Neurol. 145:1997;104-117.
12. Chauhan N.B., Siegel G.J., Lee J.M. Depletion of glial cell line-derived neurotrophic factor in substantia nigra neurons of Parkinson's disease brain. J. Chem. Neuroanat. 21:2001;277-288.
13. Choi-Lundberg D.L., Lin Q., Chang Y.N., Chiang Y.L., Hay C.M., Mohajeri H., Davidson B.L., Bohn M.C. Dopaminergic neurons protected from degeneration by GDNF gene therapy. Science. 275:1997;838-841.
14. Choi-Lundberg D.L., Lin Q., Schallert T., Crippens D., Davidson B.L., Chang Y.N., Chiang Y.L., Qian J., Bardwaj L., Bohn M.C. Behavioral and cellular protection of rat dopaminergic neurons by an adenoviral vector encoding glial cell line-derived neurotrophic factor. Exp. Neurol. 154:1998;261-275.
15. Connor B., Kozlowski D.A., Schallert T., Tillerson J.L., Davidson B.L., Bohn M.C. Differential effects of glial cell line-derived neurotrophic factor (GDNF) in the striatum and substantia nigra of the aged Parkinsonian rat. Gene Ther. 6:1999;1936-1951.
16. Costa S., Iravani M.M., Pearce R.K., Jenner P. Glial cell line-derived neurotrophic factor concentration dependently improves disability and motor activity in MPTP-treated common marmosets. Eur. J. Pharmacol. 412:2001;45-50.
17. Cunningham L.A., Su C. Astrocyte delivery of glial cell line-derived neurotrophic factor in a mouse model of Parkinson's disease. Exp. Neurol. 174:2002;230-242.
18. Date I., Shingo T., Yoshida H., Fujiwara K., Kobayashi K., Takeuchi A., Ohmoto T. Grafting of encapsulated genetically modified cells secreting GDNF into the striatum of parkinsonian model rats. Cell Transplant. 10:2001;397-401.
19. Davidson B.L., Breakefield X.O. Viral vectors for gene delivery to the nervous system. Nat. Rev., Neurosci. 4:2003;353-364.
20. Deglon N., Tseng J.L., Bensadoun J.C., Zurn A.D., Arsenijevic Y., Pereira D.A., Zufferey R., Trono D., Aebischer P. Self-inactivating lentiviral vectors with enhanced transgene expression as potential gene transfer system in Parkinson's disease. Hum. Gene Ther. 11:2000;179-190.
21. Eslamboli A., Cummings R.M., Ridley R.M., Baker H.F., Muzyczka N., Burger C., Mandel R.J., Kirik D., Annett L.E. Recombinant adeno-associated viral vector (rAAV) delivery of GDNF provides protection against 6-OHDA lesion in the common marmoset monkey (Callithrix jacchus). Exp. Neurol. 184:2004;536-548.
22. Fearnley J.M., Lees A.J. Ageing and Parkinson's disease: substantia nigra regional selectivity. Brain. 114:1991;2283-2301.
23. Freed C.R., Greene P.E., Breeze R.E., Tsai W.Y., DuMouchel W., Kao R., Dillon S., Winfield H., Culver S., Trojanowski J.Q., Eidelberg D., Fahn S. Transplantation of embryonic dopamine neurons for severe Parkinson's disease. N. Engl. J. Med. 344:2001;710-719.
24. Gash D.M., Zhang Z., Ovadia A., Cass W.A., Yi A., Simmerman L., Russell D., Martin D., Lapchak P.A., Collins F., Hoffer B.J., Gerhardt G.A. Functional recovery in parkinsonian monkeys treated with GDNF. Nature. 380:1996;252-255.
25. Georgievska B., Kirik D., Bjorklund A. Aberrant sprouting and downregulation of tyrosine hydroxylase in lesioned nigrostriatal dopamine neurons induced by long-lasting overexpression of glial cell line derived neurotrophic factor in the striatum by lentiviral gene transfer. Exp. Neurol. 177:2002;461-474.
26. Georgievska B., Kirik D., Rosenblad C., Lundberg C., Bjorklund A. Neuroprotection in the rat Parkinson model by intrastriatal GDNF gene transfer using a lentiviral vector. NeuroReport. 13:2002;75-82.
27. Gerhardt G.A., Cass W.A., Huettl P., Brock S., Zhang Z., Gash D.M. GDNF improves dopamine function in the substantia nigra but not the putamen of unilateral MPTP-lesioned rhesus monkeys. Brain Res. 817:1999;163-171.
28. Gill S.S., Patel N.K., Hotton G.R., O'Sullivan K., McCarter R., Bunnage M., Brooks D.J., Svendsen C.N., Heywood P. Direct brain infusion of glial cell line-derived neurotrophic factor in Parkinson disease. Nat. Med. 9:2003;589-595.
29. Gouhier C., Chalon S., Aubert-Pouessel A., Venier-Julienne M.C., Jollivet C., Benoit J.P., Guilloteau D. Protection of dopaminergic nigrostriatal afferents by GDNF delivered by microspheres in a rodent model of Parkinson's disease. Synapse. 44:2002;124-131.
30. Grondin R., Zhang Z., Yi A., Cass W.A., Maswood N., Andersen A.H., Elsberry D.D., Klein M.C., Gerhardt G.A., Gash D.M. Chronic, controlled GDNF infusion promotes structural and functional recovery in advanced parkinsonian monkeys. Brain. 125:2002;2191-2201.
31. Hagell P., Piccini P., Bjorklund A., Brundin P., Rehncrona S., Widner H., Crabb L., Pavese N., Oertel W.H., Quinn N., Brooks D.J., Lindvall O. Dyskinesias following neural transplantation in Parkinson's disease. Nat. Neurosci. 5:2002;627-628.
32. Hoffer B.J., Hoffman A., Bowenkamp K., Huettl P., Hudson J., Martin D., Lin L.F., Gerhardt G.A. Glial cell line-derived neurotrophic factor reverses toxin-induced injury to midbrain dopaminergic neurons in vivo. Neurosci. Lett. 182:1994;107-111.
33. Hurelbrink C.B., Barker R.A. Prospects for the treatment of Parkinson's disease using neurotrophic factors. Expert Opin. Pharmacother. 2:2001;1531-1543.
34. Iravani M.M., Costa S., Jackson M.J., Tel B.C., Cannizzaro C., Pearce R.K., Jenner P. GDNF reverses priming for dyskinesia in MPTP-treated, L-DOPA-primed common marmosets. Eur. J. Neurosci. 13:2001;597-608.
35. Kearns C.M., Gash D.M. GDNF protects nigral dopamine neurons against 6-hydroxydopamine in vivo. Brain Res. 672:1995;104-111.
36. Kirik D., Rosenblad C., Björklund A. Preservation of a functional nigrostriatal dopamine pathway by GDNF in the intrastriatal 6-OHDA lesion model depends on the site of administration of the trophic factor. Eur. J. Neurosci. 12:2000;3871-3882.
37. Kirik D., Rosenblad C., Björklund A., Mandel R.J. Long-term rAAV-mediated gene transfer of GDNF in the rat Parkinson's model: intrastriatal but not intranigral transduction promotes functional regeneration in the lesioned nigrostriatal system. J. Neurosci. 20:2000;4686-4700.
38. Kirik D., Georgievska B., Rosenblad C., Bjorklund A. Delayed infusion of GDNF promotes recovery of motor function in the partial lesion model of Parkinson's disease. Eur. J. Neurosci. 13:2001;1589-1599.
39. Kordower J.H. In vivo gene delivery of glial cell line-derived neurotrophic factor for Parkinson's disease. Ann. Neurol. 53(Suppl. 3):2003;S120-S134.
40. Kordower J.H., Palfi S., Chen E.Y., Ma S.Y., Sendera T., Cochran E.J., Mufson E.J., Penn R., Goetz C.G., Comella C.D. Clinicopathological findings following intraventricular glial-derived neurotrophic factor treatment in a patient with Parkinson's disease. Ann. Neurol. 46:1999;419-424.
41. Kordower J.H., Emborg M.E., Bloch J., Ma S.Y., Chu Y., Leventhal L., McBride J., Chen E.Y., Palfi S., Roitberg B.Z., Brown W.D., Holden J.E., Pyzalski R., Taylor M.D., Carvey P., Ling Z., Trono D., Hantraye P., Deglon N., Aebischer P. Neurodegeneration prevented by lentiviral vector delivery of GDNF in primate models of Parkinson's disease. Science. 290:2000;767-773.
42. Kozlowski D.A., Connor B., Tillerson J.L., Schallert T., Bohn M.C. Delivery of a GDNF gene into the substantia nigra after a progressive 6-OHDA lesion maintains functional nigrostriatal connections. Exp. Neurol. 166:2000;1-15.
43. Lapchak P.A., Araujo D.M., Hilt D.C., Sheng J., Jiao S. Adenoviral vector-mediated GDNF gene therapy in a rodent lesion model of late stage Parkinson's disease. Brain Res. 777:1997;153-160.
44. Lapchak P.A., Miller P.J., Collins F., Jiao S. Glial cell line-derived neurotrophic factor attenuates behavioural deficits and regulates nigrostriatal dopaminergic and peptidergic markers in 6-hydroxydopamine-lesioned adult rats: comparison of intraventricular and intranigral delivery. Neuroscience. 78:1997;61-72.
45. Lin L.F., Doherty D.H., Lile J.D., Bektesh S., Collins F. GDNF: a glial cell line-derived neurotrophic factor for midbrain dopaminergic neurons. Science. 260:1993;1130-1132.
46. Lindner M.D., Winn S.R., Baetge E.E., Hammang J.P., Gentile F.T., Doherty E., McDermott P.E., Frydel B., Ullman M.D., Schallert T. Implantation of encapsulated catecholamine and GDNF-producing cells in rats with unilateral dopamine depletions and parkinsonian symptoms. Exp. Neurol. 132:1995;62-76.
47. Ma Y., Feigin A., Dhawan V., Fukuda M., Shi Q., Greene P., Breeze R., Fahn S., Freed C., Eidelberg D. Dyskinesia after fetal cell transplantation for parkinsonism: a PET study. Ann. Neurol. 52:2002;628-634.
48. Mandel R.J., Spratt S.K., Snyder R.O., Leff S.E. Midbrain injection of recombinant adeno-associated virus encoding rat glial cell line-derived neurotrophic factor protects nigral neurons in a progressive 6-hydroxydopamine-induced degeneration model of Parkinson's disease in rats. Proc. Natl. Acad. Sci. U. S. A. 94:1997;14083-14088.
49. Mandel R.J., Snyder R.O., Leff S.E. Recombinant adeno-associated viral vector-mediated glial cell line-derived neurotrophic factor gene transfer protects nigral dopamine neurons after onset of progressive degeneration in a rat model of Parkinson's disease. Exp. Neurol. 160:1999;205-214.
50. Marek K., Innis R., van Dyck C., Fussell B., Early M., Eberly S., Oakes D., Seibyl J. [123I] beta-CIT SPECT imaging assessment of the rate of Parkinson's disease progression. Neurology. 57:2001;2089-2094.
51. McGrath J., Lintz E., Hoffer B.J., Gerhardt G.A., Quintero E.M., Granholm A.C. Adeno-associated viral delivery of GDNF promotes recovery of dopaminergic phenotype following a unilateral 6-hydroxydopamine lesion. Cell Transplant. 11:2002;215-227.
52. Morrish P.K., Sawle G.V., Brooks D.J. An [18F] dopa-PET and clinical study of the rate of progression in Parkinson's disease. Brain. 119(Pt. 2):1996;585-591.
53. Nakao N., Yokote H., Nakai K., Itakura T. Promotion of survival and regeneration of nigral dopamine neurons in a rat model of Parkinson's disease after implantation of embryonal carcinoma-derived neurons genetically engineered to produce glial cell line-derived neurotrophic factor. J. Neurosurg. 92:2000;659-670.
54. Nutt J.G., Burchiel K.J., Comella C.L., Jankovic J., Lang A.E., Laws E.R.J., Lozano A.M., Penn R.D., Simpson R.K.J., Stacy M., Wooten G.F. Randomized, double-blind trial of glial cell line-derived neurotrophic factor (GDNF) in PD. Neurology. 60:2003;69-73.
55. Olanow C.W., Freeman T., Kordower J. Transplantation of embryonic dopamine neurons for severe Parkinson's disease. N. Engl. J. Med. 345:2001;146-147.
56. Olanow C.W., Goetz C.G., Kordower J.H., Stoessl A.J., Sossi V., Brin M.F., Shannon K.M., Nauert G.M., Perl D.P., Godbold J., Freeman T.B. A double-blind controlled trial of bilateral fetal nigral transplantation in Parkinson's disease. Ann. Neurol. 54:2003;403-414.
57. Opacka-Juffry J., Ashworth S., Hume S.P., Martin D., Brooks D.J., Blunt S.B. GDNF protects against 6-OHDA nigrostriatal lesion: in vivo study with microdialysis and PET. NeuroReport. 7:1995;348-352.
58. Palfi S., Leventhal L., Chu Y., Ma S.Y., Emborg M., Bakay R., Deglon N., Hantraye P., Aebischer P., Kordower J.H. Lentivirally delivered glial cell line-derived neurotrophic factor increases the number of striatal dopaminergic neurons in primate models of nigrostriatal degeneration. J. Neurosci. 22:2002;4942-4954.
59. Park K.W., Eglitis M.A., Mouradian M.M. Protection of nigral neurons by GDNF-engineered marrow cell transplantation. Neurosci. Res. 40:2001;315-323.
60. Rosenblad C., Gronborg M., Hansen C., Blom N., Meyer M., Johansen J., Dago L., Kirik D., Patel U.A., Lundberg C., Trono D., Björklund A., Johansen T.E. In vivo protection of nigral dopamine neurons by lentiviral gene transfer of the novel GDNF-family member neublastin/artemin. Mol. Cell. Neurosci. 15:2000;199-214.
61. Samulski R.J., Sally M., Muzyczka N. Adeno-associated viral vectors. Friedman T. The Development of Human Gene Therapy. 1999;131-172 Cold Spring Harbor Laboratory Press, Cold Spring Harbor, NY.
62. Shingo T., Date I., Yoshida H., Ohmoto T. Neuroprotective and restorative effects of intrastriatal grafting of encapsulated GDNF-producing cells in a rat model of Parkinson's disease. J. Neurosci. Res. 69:2002;946-954.
63. Tomac A., Lindqvist E., Lin L.F., Ogren S.O., Young D., Hoffer B.J., Olson L. Protection and repair of the nigrostriatal dopaminergic system by GDNF in vivo. Nature. 373:1995;335-339.
64. Tseng J.L., Baetge E.E., Zurn A.D., Aebischer P. GDNF reduces drug-induced rotational behavior after medial forebrain bundle transection by a mechanism not involving striatal dopamine. J. Neurosci. 17:1997;325-333.
65. Vigna E., Cavalieri S., Ailles L., Geuna M., Loew R., Bujard H., Naldini L. Robust and efficient regulation of transgene expression in vivo by improved tetracycline-dependent lentiviral vectors. Molec. Ther. 5:2002;252-261.
66. Wang L., Muramatsu S., Lu Y., Ikeguchi K., Fujimoto K., Okada T., Mizukami H., Hanazono Y., Kume A., Urano F., Ichinose H., Nagatsu T., Nakano I., Ozawa K. Delayed delivery of AAV-GDNF prevents nigral neurodegeneration and promotes functional recovery in a rat model of Parkinson's disease. Gene Ther. 9:2002;381-389.