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. Commentary
Identifieur interne : 002645 ( Main/Exploration ); précédent : 002644; suivant : 002646Safety and tolerability of gene therapy with an adeno-associated virus (AAV) borne GAD gene for Parkinson's disease : an open label, phase I trial. Commentary
Auteurs : A. Jon Stoessl [Canada] ; Michael G. Kaplitt [États-Unis] ; Andrew Feigin [États-Unis] ; Chengke Tang [États-Unis] ; Helen L. Fitzsimons ; Paul Mattis [États-Unis] ; Patricia A. Lawlor [Nouvelle-Zélande] ; Ross J. Bland ; Deborah Young [Nouvelle-Zélande] ; Kristin Strybing [États-Unis] ; David Eidelberg [États-Unis] ; Matthew J. During [États-Unis, Nouvelle-Zélande]Source :
- Lancet : (British edition) [ 0140-6736 ] ; 2007.
Descripteurs français
- Pascal (Inist)
- Wicri :
- topic : Association, Génétique, Médicament, Médecine.
English descriptors
- KwdEn :
Abstract
Background Dopaminergic neuronal loss in Parkinson's disease leads to changes in the circuitry of the basal ganglia, such as decreased inhibitory GABAergic input to the subthalamic nucleus. We aimed to measure the safety, tolerability, and potential efficacy of transfer of glutamic acid decarboxylase (GAD) gene with adeno-associated virus (AAV) into the subthalamic nucleus of patients with Parkinson's disease. Methods We did an open label, safety and tolerability trial of unilateral subthalamic viral vector (AAV-GAD) injection in 11 men and 1 woman with Parkinson's disease (mean age 58========dot;2, SD=5========dot;7 years). Four patients received low-dose, four medium-dose, and four high-dose AAV-GAD at New York Presbyterian Hospital. Inclusion criteria consisted of Hoehn and Yahr stage 3 or greater, motor fluctuations with substantial off time, and age 70 years or less. Patients were assessed clinically both off and on medication at baseline and after 1, 3, 6, and 12 months at North Shore Hospital. Efficacy measures included the Unified Parkinson's Disease Rating Scale (UPDRS), scales of activities of daily living (ADL), neuropsychological testing, and PET imaging with 18F-fluorodeoxyglucose. The trial is registered with the ClinicalTrials.gov registry, number NCT00195143. Findings All patients who enrolled had surgery, and there were no dropouts or patients lost to follow-up. There were no adverse events related to gene therapy. Significant improvements in motor UPDRS scores (p=0========dot;0015), predominantly on the side of the body that was contralateral to surgery, were seen 3 months after gene therapy and persisted up to 12 months. PET scans revealed a substantial reduction in thalamic metabolism that was restricted to the treated hemisphere, and a correlation between clinical motor scores and brain metabolism in the supplementary motor area. Interpretation AAV-GAD gene therapy of the subthalamic nucleus is safe and well tolerated by patients with advanced Parkinson's disease, suggesting that in-vivo gene therapy in the adult brain might be safe for various neurodegenerative diseases.
Affiliations:
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Le document en format XML
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<author><name sortKey="Lawlor, Patricia A" sort="Lawlor, Patricia A" uniqKey="Lawlor P" first="Patricia A." last="Lawlor">Patricia A. Lawlor</name>
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<author><name sortKey="Young, Deborah" sort="Young, Deborah" uniqKey="Young D" first="Deborah" last="Young">Deborah Young</name>
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<author><name sortKey="Strybing, Kristin" sort="Strybing, Kristin" uniqKey="Strybing K" first="Kristin" last="Strybing">Kristin Strybing</name>
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<author><name sortKey="Eidelberg, David" sort="Eidelberg, David" uniqKey="Eidelberg D" first="David" last="Eidelberg">David Eidelberg</name>
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<author><name sortKey="During, Matthew J" sort="During, Matthew J" uniqKey="During M" first="Matthew J." last="During">Matthew J. During</name>
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<profileDesc><textClass><keywords scheme="KwdEn" xml:lang="en"><term>Association</term>
<term>Clinical trial</term>
<term>Drug</term>
<term>Gene</term>
<term>Gene therapy</term>
<term>Genetics</term>
<term>Medicine</term>
<term>Parkinson disease</term>
<term>Phase I trial</term>
<term>S Phase</term>
<term>Safety</term>
<term>Toxicity</term>
<term>Treatment</term>
<term>Virus</term>
</keywords>
<keywords scheme="Pascal" xml:lang="fr"><term>Thérapie génique</term>
<term>Toxicité</term>
<term>Sécurité</term>
<term>Parkinson maladie</term>
<term>Association</term>
<term>Virus</term>
<term>Gène</term>
<term>Génétique</term>
<term>Phase S</term>
<term>Essai clinique</term>
<term>Médicament</term>
<term>Essai clinique phase I</term>
<term>Médecine</term>
<term>Traitement</term>
<term>Essai thérapeutique</term>
<term>Essai ouvert</term>
</keywords>
<keywords scheme="Wicri" type="topic" xml:lang="fr"><term>Association</term>
<term>Génétique</term>
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<front><div type="abstract" xml:lang="en">Background Dopaminergic neuronal loss in Parkinson's disease leads to changes in the circuitry of the basal ganglia, such as decreased inhibitory GABAergic input to the subthalamic nucleus. We aimed to measure the safety, tolerability, and potential efficacy of transfer of glutamic acid decarboxylase (GAD) gene with adeno-associated virus (AAV) into the subthalamic nucleus of patients with Parkinson's disease. Methods We did an open label, safety and tolerability trial of unilateral subthalamic viral vector (AAV-GAD) injection in 11 men and 1 woman with Parkinson's disease (mean age 58<sub>========dot;</sub>
2, SD=5<sub>========dot;</sub>
7 years). Four patients received low-dose, four medium-dose, and four high-dose AAV-GAD at New York Presbyterian Hospital. Inclusion criteria consisted of Hoehn and Yahr stage 3 or greater, motor fluctuations with substantial off time, and age 70 years or less. Patients were assessed clinically both off and on medication at baseline and after 1, 3, 6, and 12 months at North Shore Hospital. Efficacy measures included the Unified Parkinson's Disease Rating Scale (UPDRS), scales of activities of daily living (ADL), neuropsychological testing, and PET imaging with <sup>18</sup>
F-fluorodeoxyglucose. The trial is registered with the ClinicalTrials.gov registry, number NCT00195143. Findings All patients who enrolled had surgery, and there were no dropouts or patients lost to follow-up. There were no adverse events related to gene therapy. Significant improvements in motor UPDRS scores (p=0<sub>========dot;</sub>
0015), predominantly on the side of the body that was contralateral to surgery, were seen 3 months after gene therapy and persisted up to 12 months. PET scans revealed a substantial reduction in thalamic metabolism that was restricted to the treated hemisphere, and a correlation between clinical motor scores and brain metabolism in the supplementary motor area. Interpretation AAV-GAD gene therapy of the subthalamic nucleus is safe and well tolerated by patients with advanced Parkinson's disease, suggesting that in-vivo gene therapy in the adult brain might be safe for various neurodegenerative diseases.</div>
</front>
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<li>Nouvelle-Zélande</li>
<li>États-Unis</li>
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<tree><noCountry><name sortKey="Bland, Ross J" sort="Bland, Ross J" uniqKey="Bland R" first="Ross J." last="Bland">Ross J. Bland</name>
<name sortKey="Fitzsimons, Helen L" sort="Fitzsimons, Helen L" uniqKey="Fitzsimons H" first="Helen L." last="Fitzsimons">Helen L. Fitzsimons</name>
</noCountry>
<country name="Canada"><noRegion><name sortKey="Stoessl, A Jon" sort="Stoessl, A Jon" uniqKey="Stoessl A" first="A. Jon" last="Stoessl">A. Jon Stoessl</name>
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