Direct brain infusion of glial cell line-derived neurotrophic factor in Parkinson disease

Nature Medicine

Volumn 9, Issue 5, 2003, Pages 589-595

  Gill, Steven S ^a   Patel, Nikunj K ^a   Hotton, Gary R ^b   O'Sullivan, Karen ^a   McCarter, Renée ^a   Bunnage, Martin ^a   Brooks, David J ^b   Svendsen, Clive N ^c   Heywood, Peter ^a  

^a FRENCHAY HOSPITAL   (United Kingdom)

^b HAMMERSMITH HOSPITAL   (United Kingdom)

^c UNIVERSITY OF WISCONSIN MADISON   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

DOPAMINE; FLUORINE 18; GLIAL CELL LINE DERIVED NEUROTROPHIC FACTOR; NEUROTROPHIC FACTOR;

ADULT; ARTICLE; CATHETER; CLINICAL ARTICLE; CLINICAL TRIAL; DAILY LIFE ACTIVITY; DRUG INFUSION; DRUG PENETRATION; DRUG SAFETY; DYSKINESIA; HEADACHE; HUMAN; HYPERSALIVATION; LHERMITTE DUCLOS DISEASE; MOUTH ULCER; NAUSEA; NERVE REGENERATION; NONHUMAN; NUCLEAR MAGNETIC RESONANCE IMAGING; PARKINSON DISEASE; PHASE 1 CLINICAL TRIAL; POSITRON EMISSION TOMOGRAPHY; PRIORITY JOURNAL; PUMP; PUTAMEN; RATING SCALE; SCINTILLATION CAMERA; SIDE EFFECT; TASTE DISORDER; VIVID DREAM; VOMITING; WEIGHT REDUCTION;

DIHYDROXYPHENYLALANINE; FLUORINE RADIOISOTOPES; GLIAL CELL LINE-DERIVED NEUROTROPHIC FACTOR; HUMANS; MIDDLE AGED; NERVE GROWTH FACTORS; PARKINSON DISEASE; PILOT PROJECTS; PUTAMEN; RESEARCH DESIGN; TOMOGRAPHY, EMISSION-COMPUTED;

PRIMATES; RODENTIA;

EID: 0038249170     PISSN: 10788956     EISSN: None     Source Type: Journal    
DOI: 10.1038/nm850     Document Type: Article

References (37)

1. Rascol, O., Goetz, C., Koller, W., Poewe, W. & Sampaio, C. Treatment interventions for Parkinson's disease: an evidence based assessment. Lancet 359, 1589-1598 (2002).
2. 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, 1130-1132 (1993).
3. Beck, K.D. et al. Mesencephalic dopaminergic neurons protected by GDNF from axotomy-induced degeneration in the adult brain. Nature 373, 339-341 (1995).
4. Tomac, A. et al. Protection and repair of the nigrostriatal dopaminergic system by GDNF in vivo. Nature 373, 335-339 (1995).
5. Bjorklund, A., Rosenblad, C., Winkler, C. & Kirik, D. Studies on neuroprotective and regenerative effects of GDNF in a partial lesion model of Parkinson's disease. Neurobiol. Dis. 4, 186-200 (1997).
6. Gash, D.M. et al. Functional recovery in parkinsonian monkeys treated with GDNF. Nature 380, 252-255 (1996).
7. Gash, D.M., Zhang, Z. & Gerhardt, G. Neuroprotective and neurorestorative properties of GDNF. Ann. Neurol. 44, S121-S125 (1998).
8. Grondin, R. et al. Chronic, controlled GDNF infusion promotes structural and functional recovery in advanced parkinsonian monkeys. Brain 125, 1-11 (2002).
9. Kordower, J.H. et al. Neurodegeneration prevented by lentiviral vector delivery of GDNF in primate models of Parkinson's disease. Science 290, 767-773 (2000).
10. Kordower, J.H. et al. Clinicopathological findings following intraventricular glial-derived neurotrophic factor treatment in a patient with Parkinson's disease. Ann. Neurol. 46, 419-424 (1999).
11. Nutt, J.G. et al. Randomized, double-blind trial of glial cell line-derived neurotrophic factor (GDNF) in PD. Neurology 60, 69-73 (2003).
12. Golden, J.P. et al. Expression of neurturin, GDNF, and their receptors in the adult mouse CNS. J. Comp. Neurol. 398, 139-150 (1998).
13. Langston, J.W. et al. Core assessment program for intracerebral transplantations (CAPIT). Mov. Disord. 7, 2-13 (1992).
14. Peto, V., Jenkinson, C., Fitzpatrick, R. & Greenhall, R. The development and validation of a short measure of functioning and well being for individuals with Parkinson's disease. Qual. Life Res. 4, 241-248 (1995).
15. McCarter, R.J., Walton, N.H., Rowan, A.F., Gill, S.S., & Palomo, M. Cognitive functioning after subthalamic nucleotomy for refractory Parkinson's disease. J. Neurol. Neurosurg. Psychiatry 69, 60-66 (2000).
16. 18F-dopa-PET and clinical study of the rate of progression in Parkinson's disease. Brain 119 (Pt 2), 585-591 (1996).
17. Brooks, D.J. et al. The relationship between locomotor disability, autonomic dysfunction, and the integrity of the striatal dopaminergic system in patients with multiple system atrophy, pure autonomic failure, and Parkinson's disease, studied with PET. Brain 113 (Pt 5), 1539-1552 (1990).
18. 18F-dopa-PET study. Brain 122 (Pt 9), 1637-1650 (1999).
19. Whone, A.L. et al. The REAL-PET study: slower progression in early Parkinson's disease treated with ropinirole compared with L-dopa. Neurology 58, A82-A83 (2002).
20. Miyoshi, Y. et al. Glial cell line-derived neurotrophic factor-levodopa interactions and reduction of side effects in parkinsonian monkeys. Ann. Neurol. 42, 208-214 (1997).
21. Freed, C.R. et al. Transplantation of embryonic dopamine neurons for severe Parkinson's disease. N. Engl. J. Med. 344, 710-719 (2001).
22. Hagell, P. et al. Dyskinesias following neural transplantation in Parkinson's disease. Nat. Neurosci. 5, 627-628 (2002).
23. Bhatia, K.P. & Marsden, C.D. The behavioural and motor consequences of focal lesions of the basal ganglia in man. Brain 117 (Pt 4), 859-876 (1994).
24. Goetz, C.G., Leurgans, S., Raman, R. & Stebbins, G.T. Objective changes in motor function during placebo treatment in PD. Neurology 54, 710-714 (2000).
25. The Deep-Brain Stimulation for Parkinson's Disease Study Group. Deep brain stimulation of the subthalamic nucleus or the pars interna of the globus palidus in Parkinson's Disease. N. Engl. J. Med. 345, 956-963 (2001).
26. Lindvall, O. & Hagell, P. Clinical observations after neural transplantation in Parkinson's disease. Prog. Brain Res. 127, 299-320 (2000).
27. Quinn, N.P., Rossor, M.N. & Marsden, C.D. Olfactory threshold in Parkinson's disease. J. Neurol. Neurosurg. Psychiatry 50, 88-89 (1987).
28. Daniel, S.E. & Hawkes, C.H. Preliminary diagnosis of Parkinson's disease by olfactory bulb pathology. Lancet 340, 186 (1992).
29. Wenning, G.K. et al. Short- and long-term survival and function of unilateral intrastriatal dopaminergic grafts in Parkinson's disease. Ann. Neurol. 42, 95-107 (1997).
30. Hoffman, A.F., van Horne, C.G., Eken, S., Hoffer, B.J. & Gerhardt, G.A. In vivo microdialysis studies on somatodendritic dopamine release in the rat substantia nigra: effects of unilateral 6-OHDA lesions and GDNF. Exp. Neurol. 147, 130-141 (1997).
31. Zurn, A.D., Widmer, H.R. & Aebischer, P. Sustained delivery of GDNF: towards a treatment for Parkinson's disease. Brain Res. Rev. 36, 222-229 (2001).
32. 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, 8108-8118 (2001).
33. Ostenfeld, T. et al. Neurospheres modified to produce glial cell line-derived neurotrophic factor increase the survival of transplanted dopamine neurons. J. Neurosci. Res. 69, 955-965 (2002).
34. Sofroniew, M.V., Howe, C.L. & Mobley, W.C. Nerve growth factor signaling, neuroprotection, and neural repair. Annu. Rev. Neurosci. 24, 1217-1281 (2001).
35. Beck, M., Karch, C., Wiese, S. & Sendtner, M. Motoneuron cell death and neurotrophic factors: basic models for development of new therapeutic strategies in ALS. Amyotroph. Lateral Scler. Other Motor Neuron Disord. 2 (Suppl. 1), S55-S68 (2001).
36. Kordower, J.H., Isacson, O. & Emerich, D.F. Cellular delivery of trophic factors for the treatment of Huntington's disease: is neuroprotection possible? Exp. Neurol. 159, 4-20 (1999).
37. Patlak, C.S. & Blasberg, R.G. Graphical evaluation of blood-to-brain transfer constants from multiple-time uptake data. Generalizations. J. Cereb. Blood Flow Metab. 5, 584-590 (1985).