Monday, September 17, 2018 11:21:08 PM
Palfi S1,2, Gurru J3, Le H4, Howard K5, Ralph GS6, Mason S7, G G8, Domenech P9, Buttery P10, Hantraye P11, Tuckwell N12, Barker R13, Mitrophanous K14.
Author information
1
AP-HP, Groupe Henri-Mondor Albert-Chenevier , 51 Av Du Marechal de Lattre de Tassigny , Créteil, Neurochirurgie, France , 94100.
2
Universite Paris-Est Creteil Val de Marne, 27010, IMRB, Creteil, France , 94010 ; stephane.palfi@aphp.fr.
3
Universite Paris-Est Creteil Val de Marne, 27010, IMRB, Creteil, France ; jean-marc.gurruchaga@aphp.fr.
4
AP-HP, Groupe Henri-Mondor Albert-Chenevier, Créteil, France ; helene.lepetit@aphp.fr.
5
Oxford Biomedica Plc, 6377, Clinical development, Oxford, Oxfordshire, United Kingdom of Great Britain and Northern Ireland ; K.Howard@oxfordbiomedica.co.uk.
6
Oxford Biomedica Plc, 6377, virology, Oxford, Oxfordshire, United Kingdom of Great Britain and Northern Ireland ; gr7553@hotmail.com.
7
Addenbrooke's Hospital, 89744, John van Geest Centre for Brain Repair , Cambridge, Cambridgeshire, United Kingdom of Great Britain and Northern Ireland ; slm64@cam.ac.uk.
8
AP-HP, Groupe Henri-Mondor Albert-Chenevier, Créteil, Neurochirurgie, France ; gaetane.gouello@aphp.fr.
9
AP-HP, Groupe Henri-Mondor Albert-Chenevier, Créteil, France ; philippe.domenech@inserm.fr.
10
Addenbrooke's Hospital, 89744, John van Geest Centre for Brain Repair , Cambridge, Cambridgeshire, United Kingdom of Great Britain and Northern Ireland ; pcb10@cam.ac.uk.
11
CEA, Institute of Biomedical Imaging (I2BM) and Molecular Imaging Research Center, Nantes, France ; philippe.hantraye@cea.fr.
12
Oxford Biomedica Plc, 6377, Clinical development, Oxford, Oxfordshire, United Kingdom of Great Britain and Northern Ireland ; N.Tuckwell@oxfordbiomedica.co.uk.
13
Addenbrooke's Hospital, 89744, John van Geest Centre for Brain Repair , Cambridge, Cambridgeshire, United Kingdom of Great Britain and Northern Ireland ; rab46@cam.ac.uk.
14
Oxford BioMedica (UK) Ltd, Virology , Medawar Centre , Oxford Science Park , Oxford, United Kingdom of Great Britain and Northern Ireland , OX4 4GA ; k.mitrophanous@oxfordbiomedica.co.uk.
Abstract
Parkinson's disease is typically treated with oral dopamine replacement therapies, however long term use is complicated by motor fluctuations from intermittent stimulation of dopamine receptors and off-target effects. ProSavin, a lentiviral vector based gene therapy that delivers local and continuous dopamine, was previously shown to be well tolerated in a phase 1/2 first-in-human study, with significant improvements in motor behaviour from baseline at 1 year. Here, patients with Parkinson's disease from the open-label trial were followed up in the long term to assess the safety and efficacy of ProSavin after bilateral injection into the putamen. 15 patients who were previously treated with ProSavin have been followed for up for 5 years with some having been seen for 8 years. Eight patients received deep brain stimulation at different time points and their subsequent assessments continued to assess safety. Ninety-six drug-related adverse events were reported (87 mild, 6 moderate, 3 severe) of which more than half occurred in the first year. The most common drug-related events were dyskinesias (33 events, 11 patients) and on-off phenomena (22 events, 11 patients). A significant improvement in the defined "off" UPDRS part III motor scores, compared with baseline, was seen at 2 years (mean score 29·2 vs. 38·4, n=14, p<0.05) and at 4 years in 8 out of 15 patients. ProSavin continued to be safe and well tolerated in patients with Parkinson's disease. Moderate improvements in motor behaviour over baseline continued to be reported in the majority of patients who could still be evaluated up to 5 years of follow up.
https://www.ncbi.nlm.nih.gov/pubmed/30156440
Good luck and GOD bless,
George
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