ParkinsonCanadaV1, PascalFrancis, Curation, bibRecord, 000B33

Safety/feasibility of targeting the substantia nigra with AAV2-neurturin in Parkinson patients

Identifieur interne : 000B33 ( PascalFrancis/Curation ); précédent : 000B32; suivant : 000B34

Safety/feasibility of targeting the substantia nigra with AAV2-neurturin in Parkinson patients

Auteurs : Raymond T. Bartus [États-Unis] ; Tiffany L. Baumann [États-Unis] ; Joao Siffert [États-Unis] ; Christopher D. Herzog [États-Unis] ; Ron Alterman [États-Unis] ; Nicholas Boulis [États-Unis] ; Dennis A. Turner [États-Unis] ; Mark Stacy [États-Unis] ; Anthony E. Lang [Canada] ; Andres M. Lozano [Canada] ; C. Warren Olanow [États-Unis]

Source :

Neurology [ 0028-3878 ] ; 2013.

RBID : Pascal:13-0179282

Descripteurs français

Pascal (Inist)
- Pathologie du système nerveux, Sécurité, Faisabilité, Ciblage, Locus niger, Homme.
Wicri :
- topic : Homme.

English descriptors

KwdEn :
- Feasibility, Human, Locus niger, Nervous system diseases, Safety, Targeting.

Abstract

Objective: In an effort to account for deficiencies in axonal transport that limit the effectiveness of neurotrophic factors, this study tested the safety and feasibility, in moderately advanced Parkinson disease (PD), of bilaterally administering the gene therapy vector AAV2-neurturin (CERE-120) to the putamen plus substantia nigra (SN, a relatively small structure deep within the midbrain, in proximity to critical neuronal and vascular structures). Methods: After planning and minimizing risks of stereotactically targeting the SN, an open-label, dose-escalation safety trial was initiated in 6 subjects with PD who received bilateral stereotactic injections of CERE-120 into the SN and putamen. Results: Two-year safety data for all subjects suggest the procedures were well-tolerated, with no serious adverse events. All adverse events and complications were expected for patients with PD undergoing stereotactic brain surgery. Conclusions: Bilateral stereotactic administration of CERE-120 to the SN plus putamen in PD is feasible and this evaluation provides initial empirical support that it is safe and well-tolerated. Classification of evidence: This study provides Class IV evidence that bilateral neurturin gene delivery (CERE-120) to the SN plus putamen in patients with moderately advanced PD is feasible and safe.

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Le document en format XML

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<sourceDesc><biblStruct><analytic><title xml:lang="en" level="a">Safety/feasibility of targeting the substantia nigra with AAV2-neurturin in Parkinson patients</title>
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<author><name sortKey="Boulis, Nicholas" sort="Boulis, Nicholas" uniqKey="Boulis N" first="Nicholas" last="Boulis">Nicholas Boulis</name>
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<author><name sortKey="Turner, Dennis A" sort="Turner, Dennis A" uniqKey="Turner D" first="Dennis A." last="Turner">Dennis A. Turner</name>
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<author><name sortKey="Stacy, Mark" sort="Stacy, Mark" uniqKey="Stacy M" first="Mark" last="Stacy">Mark Stacy</name>
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<author><name sortKey="Lang, Anthony E" sort="Lang, Anthony E" uniqKey="Lang A" first="Anthony E." last="Lang">Anthony E. Lang</name>
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<author><name sortKey="Lozano, Andres M" sort="Lozano, Andres M" uniqKey="Lozano A" first="Andres M." last="Lozano">Andres M. Lozano</name>
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<author><name sortKey="Warren Olanow, C" sort="Warren Olanow, C" uniqKey="Warren Olanow C" first="C." last="Warren Olanow">C. Warren Olanow</name>
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<profileDesc><textClass><keywords scheme="KwdEn" xml:lang="en"><term>Feasibility</term>
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<term>Locus niger</term>
<term>Nervous system diseases</term>
<term>Safety</term>
<term>Targeting</term>
</keywords>
<keywords scheme="Pascal" xml:lang="fr"><term>Pathologie du   système nerveux</term>
<term>Sécurité</term>
<term>Faisabilité</term>
<term>Ciblage</term>
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<front><div type="abstract" xml:lang="en">Objective: In an effort to account for deficiencies in axonal transport that limit the effectiveness of neurotrophic factors, this study tested the safety and feasibility, in moderately advanced Parkinson disease (PD), of bilaterally administering the gene therapy vector AAV2-neurturin (CERE-120) to the putamen plus substantia nigra (SN, a relatively small structure deep within the midbrain, in proximity to critical neuronal and vascular structures). Methods: After planning and minimizing risks of stereotactically targeting the SN, an open-label, dose-escalation safety trial was initiated in 6 subjects with PD who received bilateral stereotactic injections of CERE-120 into the SN and putamen. Results: Two-year safety data for all subjects suggest the procedures were well-tolerated, with no serious adverse events. All adverse events and complications were expected for patients with PD undergoing stereotactic brain surgery. Conclusions: Bilateral stereotactic administration of CERE-120 to the SN plus putamen in PD is feasible and this evaluation provides initial empirical support that it is safe and well-tolerated. Classification of evidence: This study provides Class IV evidence that bilateral neurturin gene delivery (CERE-120) to the SN plus putamen in patients with moderately advanced PD is feasible and safe.</div>
</front>
</TEI>
<inist><standard h6="B"><pA><fA01 i1="01" i2="1"><s0>0028-3878</s0>
</fA01>
<fA02 i1="01"><s0>NEURAI</s0>
</fA02>
<fA03 i2="1"><s0>Neurology</s0>
</fA03>
<fA05><s2>80</s2>
</fA05>
<fA06><s2>18</s2>
</fA06>
<fA08 i1="01" i2="1" l="ENG"><s1>Safety/feasibility of targeting the substantia nigra with AAV2-neurturin in Parkinson patients</s1>
</fA08>
<fA11 i1="01" i2="1"><s1>BARTUS (Raymond T.)</s1>
</fA11>
<fA11 i1="02" i2="1"><s1>BAUMANN (Tiffany L.)</s1>
</fA11>
<fA11 i1="03" i2="1"><s1>SIFFERT (Joao)</s1>
</fA11>
<fA11 i1="04" i2="1"><s1>HERZOG (Christopher D.)</s1>
</fA11>
<fA11 i1="05" i2="1"><s1>ALTERMAN (Ron)</s1>
</fA11>
<fA11 i1="06" i2="1"><s1>BOULIS (Nicholas)</s1>
</fA11>
<fA11 i1="07" i2="1"><s1>TURNER (Dennis A.)</s1>
</fA11>
<fA11 i1="08" i2="1"><s1>STACY (Mark)</s1>
</fA11>
<fA11 i1="09" i2="1"><s1>LANG (Anthony E.)</s1>
</fA11>
<fA11 i1="10" i2="1"><s1>LOZANO (Andres M.)</s1>
</fA11>
<fA11 i1="11" i2="1"><s1>WARREN OLANOW (C.)</s1>
</fA11>
<fA14 i1="01"><s1>Ceregene, Inc.</s1>
<s2>San Diego, CA</s2>
<s3>USA</s3>
<sZ>1 aut.</sZ>
<sZ>2 aut.</sZ>
<sZ>3 aut.</sZ>
<sZ>4 aut.</sZ>
</fA14>
<fA14 i1="02"><s1>Mount Sinai School of Medicine</s1>
<s2>New York, NY</s2>
<s3>USA</s3>
<sZ>5 aut.</sZ>
<sZ>11 aut.</sZ>
</fA14>
<fA14 i1="03"><s1>Emory University</s1>
<s2>Atlanta, GA</s2>
<s3>USA</s3>
<sZ>6 aut.</sZ>
</fA14>
<fA14 i1="04"><s1>Duke University</s1>
<s2>Durham, NC</s2>
<s3>USA</s3>
<sZ>7 aut.</sZ>
<sZ>8 aut.</sZ>
</fA14>
<fA14 i1="05"><s1>University of Toronto</s1>
<s2>Toronto</s2>
<s3>CAN</s3>
<sZ>9 aut.</sZ>
<sZ>10 aut.</sZ>
</fA14>
<fA14 i1="06"><s1>The Edmond J. Safra Program in Parkinson's Disease Toronto Western Hospital</s1>
<s2>Toronto</s2>
<s3>CAN</s3>
<sZ>9 aut.</sZ>
</fA14>
<fA14 i1="07"><s1>Avanir Pharmaceuticals</s1>
<s2>Aliso Viejo, CA</s2>
<s3>USA</s3>
<sZ>3 aut.</sZ>
</fA14>
<fA14 i1="08"><s1>Beth Israel Deaconess Medical Center</s1>
<s2>Boston, MA</s2>
<s3>USA</s3>
<sZ>5 aut.</sZ>
</fA14>
<fA20><s1>1698-1701</s1>
</fA20>
<fA21><s1>2013</s1>
</fA21>
<fA23 i1="01"><s0>ENG</s0>
</fA23>
<fA43 i1="01"><s1>INIST</s1>
<s2>6345</s2>
<s5>354000504129080140</s5>
</fA43>
<fA44><s0>0000</s0>
<s1>© 2013 INIST-CNRS. All rights reserved.</s1>
</fA44>
<fA45><s0>13 ref.</s0>
</fA45>
<fA47 i1="01" i2="1"><s0>13-0179282</s0>
</fA47>
<fA60><s1>P</s1>
</fA60>
<fA61><s0>A</s0>
</fA61>
<fA64 i1="01" i2="1"><s0>Neurology</s0>
</fA64>
<fA66 i1="01"><s0>USA</s0>
</fA66>
<fC01 i1="01" l="ENG"><s0>Objective: In an effort to account for deficiencies in axonal transport that limit the effectiveness of neurotrophic factors, this study tested the safety and feasibility, in moderately advanced Parkinson disease (PD), of bilaterally administering the gene therapy vector AAV2-neurturin (CERE-120) to the putamen plus substantia nigra (SN, a relatively small structure deep within the midbrain, in proximity to critical neuronal and vascular structures). Methods: After planning and minimizing risks of stereotactically targeting the SN, an open-label, dose-escalation safety trial was initiated in 6 subjects with PD who received bilateral stereotactic injections of CERE-120 into the SN and putamen. Results: Two-year safety data for all subjects suggest the procedures were well-tolerated, with no serious adverse events. All adverse events and complications were expected for patients with PD undergoing stereotactic brain surgery. Conclusions: Bilateral stereotactic administration of CERE-120 to the SN plus putamen in PD is feasible and this evaluation provides initial empirical support that it is safe and well-tolerated. Classification of evidence: This study provides Class IV evidence that bilateral neurturin gene delivery (CERE-120) to the SN plus putamen in patients with moderately advanced PD is feasible and safe.</s0>
</fC01>
<fC02 i1="01" i2="X"><s0>002B17</s0>
</fC02>
<fC02 i1="02" i2="X"><s0>002B17G</s0>
</fC02>
<fC03 i1="01" i2="X" l="FRE"><s0>Pathologie du   système nerveux</s0>
<s5>01</s5>
</fC03>
<fC03 i1="01" i2="X" l="ENG"><s0>Nervous system diseases</s0>
<s5>01</s5>
</fC03>
<fC03 i1="01" i2="X" l="SPA"><s0>Sistema nervioso patología</s0>
<s5>01</s5>
</fC03>
<fC03 i1="02" i2="X" l="FRE"><s0>Sécurité</s0>
<s5>09</s5>
</fC03>
<fC03 i1="02" i2="X" l="ENG"><s0>Safety</s0>
<s5>09</s5>
</fC03>
<fC03 i1="02" i2="X" l="SPA"><s0>Seguridad</s0>
<s5>09</s5>
</fC03>
<fC03 i1="03" i2="X" l="FRE"><s0>Faisabilité</s0>
<s5>10</s5>
</fC03>
<fC03 i1="03" i2="X" l="ENG"><s0>Feasibility</s0>
<s5>10</s5>
</fC03>
<fC03 i1="03" i2="X" l="SPA"><s0>Practicabilidad</s0>
<s5>10</s5>
</fC03>
<fC03 i1="04" i2="X" l="FRE"><s0>Ciblage</s0>
<s5>11</s5>
</fC03>
<fC03 i1="04" i2="X" l="ENG"><s0>Targeting</s0>
<s5>11</s5>
</fC03>
<fC03 i1="04" i2="X" l="SPA"><s0>Blancado</s0>
<s5>11</s5>
</fC03>
<fC03 i1="05" i2="X" l="FRE"><s0>Locus niger</s0>
<s5>12</s5>
</fC03>
<fC03 i1="05" i2="X" l="ENG"><s0>Locus niger</s0>
<s5>12</s5>
</fC03>
<fC03 i1="05" i2="X" l="SPA"><s0>Locus níger</s0>
<s5>12</s5>
</fC03>
<fC03 i1="06" i2="X" l="FRE"><s0>Homme</s0>
<s5>13</s5>
</fC03>
<fC03 i1="06" i2="X" l="ENG"><s0>Human</s0>
<s5>13</s5>
</fC03>
<fC03 i1="06" i2="X" l="SPA"><s0>Hombre</s0>
<s5>13</s5>
</fC03>
<fC07 i1="01" i2="X" l="FRE"><s0>Encéphale</s0>
<s5>37</s5>
</fC07>
<fC07 i1="01" i2="X" l="ENG"><s0>Encephalon</s0>
<s5>37</s5>
</fC07>
<fC07 i1="01" i2="X" l="SPA"><s0>Encéfalo</s0>
<s5>37</s5>
</fC07>
<fC07 i1="02" i2="X" l="FRE"><s0>Système nerveux central</s0>
<s5>38</s5>
</fC07>
<fC07 i1="02" i2="X" l="ENG"><s0>Central nervous system</s0>
<s5>38</s5>
</fC07>
<fC07 i1="02" i2="X" l="SPA"><s0>Sistema nervioso central</s0>
<s5>38</s5>
</fC07>
<fN21><s1>161</s1>
</fN21>
<fN44 i1="01"><s1>OTO</s1>
</fN44>
<fN82><s1>OTO</s1>
</fN82>
</pA>
</standard>
</inist>
</record>

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	La maladie de Parkinson au Canada (serveur d'exploration)
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La maladie de Parkinson au Canada (serveur d'exploration)

Safety/feasibility of targeting the substantia nigra with AAV2-neurturin in Parkinson patients

Safety/feasibility of targeting the substantia nigra with AAV2-neurturin in Parkinson patients

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