Safety/feasibility of targeting the substantia nigra with AAV2-neurturin in Parkinson patients
Identifieur interne : 000637 ( Pmc/Corpus ); précédent : 000636; suivant : 000638Safety/feasibility of targeting the substantia nigra with AAV2-neurturin in Parkinson patients
Auteurs : Raymond T. Bartus ; Tiffany L. Baumann ; Joao Siffert ; Christopher D. Herzog ; Ron Alterman ; Nicholas Boulis ; Dennis A. Turner ; Mark Stacy ; Anthony E. Lang ; Andres M. Lozano ; C. Warren OlanowSource :
- Neurology [ 0028-3878 ] ; 2013.
Abstract
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).
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.
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.
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.
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.
Url:
DOI: 10.1212/WNL.0b013e3182904faa
PubMed: 23576625
PubMed Central: 3716474
Links to Exploration step
PMC:3716474Le document en format XML
<record><TEI><teiHeader><fileDesc><titleStmt><title xml:lang="en">Safety/feasibility of targeting the substantia nigra with AAV2-neurturin in Parkinson patients</title><author><name sortKey="Bartus, Raymond T" sort="Bartus, Raymond T" uniqKey="Bartus R" first="Raymond T." last="Bartus">Raymond T. Bartus</name></author><author><name sortKey="Baumann, Tiffany L" sort="Baumann, Tiffany L" uniqKey="Baumann T" first="Tiffany L." last="Baumann">Tiffany L. Baumann</name></author><author><name sortKey="Siffert, Joao" sort="Siffert, Joao" uniqKey="Siffert J" first="Joao" last="Siffert">Joao Siffert</name></author><author><name sortKey="Herzog, Christopher D" sort="Herzog, Christopher D" uniqKey="Herzog C" first="Christopher D." last="Herzog">Christopher D. Herzog</name></author><author><name sortKey="Alterman, Ron" sort="Alterman, Ron" uniqKey="Alterman R" first="Ron" last="Alterman">Ron Alterman</name></author><author><name sortKey="Boulis, Nicholas" sort="Boulis, Nicholas" uniqKey="Boulis N" first="Nicholas" last="Boulis">Nicholas Boulis</name></author><author><name sortKey="Turner, Dennis A" sort="Turner, Dennis A" uniqKey="Turner D" first="Dennis A." last="Turner">Dennis A. Turner</name></author><author><name sortKey="Stacy, Mark" sort="Stacy, Mark" uniqKey="Stacy M" first="Mark" last="Stacy">Mark Stacy</name></author><author><name sortKey="Lang, Anthony E" sort="Lang, Anthony E" uniqKey="Lang A" first="Anthony E." last="Lang">Anthony E. Lang</name></author><author><name sortKey="Lozano, Andres M" sort="Lozano, Andres M" uniqKey="Lozano A" first="Andres M." last="Lozano">Andres M. Lozano</name></author><author><name sortKey="Olanow, C Warren" sort="Olanow, C Warren" uniqKey="Olanow C" first="C. Warren" last="Olanow">C. Warren Olanow</name></author></titleStmt><publicationStmt><idno type="wicri:source">PMC</idno><idno type="pmid">23576625</idno><idno type="pmc">3716474</idno><idno type="url">http://www.ncbi.nlm.nih.gov/pmc/articles/PMC3716474</idno><idno type="RBID">PMC:3716474</idno><idno type="doi">10.1212/WNL.0b013e3182904faa</idno><date when="2013">2013</date><idno type="wicri:Area/Pmc/Corpus">000637</idno><idno type="wicri:explorRef" wicri:stream="Pmc" wicri:step="Corpus" wicri:corpus="PMC">000637</idno></publicationStmt><sourceDesc><biblStruct><analytic><title xml:lang="en" level="a" type="main">Safety/feasibility of targeting the substantia nigra with AAV2-neurturin in Parkinson patients</title><author><name sortKey="Bartus, Raymond T" sort="Bartus, Raymond T" uniqKey="Bartus R" first="Raymond T." last="Bartus">Raymond T. Bartus</name></author><author><name sortKey="Baumann, Tiffany L" sort="Baumann, Tiffany L" uniqKey="Baumann T" first="Tiffany L." last="Baumann">Tiffany L. Baumann</name></author><author><name sortKey="Siffert, Joao" sort="Siffert, Joao" uniqKey="Siffert J" first="Joao" last="Siffert">Joao Siffert</name></author><author><name sortKey="Herzog, Christopher D" sort="Herzog, Christopher D" uniqKey="Herzog C" first="Christopher D." last="Herzog">Christopher D. Herzog</name></author><author><name sortKey="Alterman, Ron" sort="Alterman, Ron" uniqKey="Alterman R" first="Ron" last="Alterman">Ron Alterman</name></author><author><name sortKey="Boulis, Nicholas" sort="Boulis, Nicholas" uniqKey="Boulis N" first="Nicholas" last="Boulis">Nicholas Boulis</name></author><author><name sortKey="Turner, Dennis A" sort="Turner, Dennis A" uniqKey="Turner D" first="Dennis A." last="Turner">Dennis A. Turner</name></author><author><name sortKey="Stacy, Mark" sort="Stacy, Mark" uniqKey="Stacy M" first="Mark" last="Stacy">Mark Stacy</name></author><author><name sortKey="Lang, Anthony E" sort="Lang, Anthony E" uniqKey="Lang A" first="Anthony E." last="Lang">Anthony E. Lang</name></author><author><name sortKey="Lozano, Andres M" sort="Lozano, Andres M" uniqKey="Lozano A" first="Andres M." last="Lozano">Andres M. Lozano</name></author><author><name sortKey="Olanow, C Warren" sort="Olanow, C Warren" uniqKey="Olanow C" first="C. Warren" last="Olanow">C. Warren Olanow</name></author></analytic><series><title level="j">Neurology</title><idno type="ISSN">0028-3878</idno><idno type="eISSN">1526-632X</idno><imprint><date when="2013">2013</date></imprint></series></biblStruct></sourceDesc></fileDesc><profileDesc><textClass></textClass></profileDesc></teiHeader><front><div type="abstract" xml:lang="en"><sec><title>Objective:</title><p>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).</p></sec><sec><title>Methods:</title><p>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.</p></sec><sec><title>Results:</title><p>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.</p></sec><sec><title>Conclusions:</title><p>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.</p></sec><sec><title>Classification of evidence:</title><p>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.</p></sec></div></front></TEI><pmc article-type="research-article"><pmc-comment>The publisher of this article does not allow downloading of the full text in XML form.</pmc-comment>
<front><journal-meta><journal-id journal-id-type="nlm-ta">Neurology</journal-id><journal-id journal-id-type="iso-abbrev">Neurology</journal-id><journal-id journal-id-type="hwp">neurology</journal-id><journal-id journal-id-type="publisher-id">neur</journal-id><journal-id journal-id-type="pmc">neurology</journal-id><journal-id journal-id-type="publisher-id">WNL</journal-id><journal-title-group><journal-title>Neurology</journal-title></journal-title-group><issn pub-type="ppub">0028-3878</issn><issn pub-type="epub">1526-632X</issn><publisher><publisher-name>Lippincott Williams & Wilkins</publisher-name><publisher-loc>Hagerstown, MD</publisher-loc></publisher></journal-meta><article-meta><article-id pub-id-type="pmid">23576625</article-id><article-id pub-id-type="pmc">3716474</article-id><article-id pub-id-type="publisher-id">WNL204989</article-id><article-id pub-id-type="doi">10.1212/WNL.0b013e3182904faa</article-id><article-categories><subj-group subj-group-type="hwp-journal-coll"><subject>19</subject><subject>21</subject><subject>165</subject><subject>292</subject></subj-group><subj-group subj-group-type="heading"><subject>Article</subject></subj-group></article-categories><title-group><article-title>Safety/feasibility of targeting the substantia nigra with AAV2-neurturin in Parkinson patients</article-title></title-group><contrib-group><contrib contrib-type="author" corresp="yes"><name><surname>Bartus</surname><given-names>Raymond T.</given-names></name><degrees>PhD</degrees><author-comment content-type="disclosure"><p><list list-type="order"><title>Scientific Advisory Boards:</title><list-item><p>NONE</p></list-item></list></p><p><list list-type="order"><title>Gifts:</title><list-item><p>NONE</p></list-item></list></p><p><list list-type="order"><title>Funding for Travel or Speaker Honoraria:</title><list-item><p>NONE</p></list-item></list></p><p><list list-type="order"><title>Editorial Boards:</title><list-item><p>1) Non-paid Section Editor for Neurobiology of Aging 2) Non-paid Editorial Board member for Brain Research; Drug Delivery and Translational Research</p></list-item></list></p><p><list list-type="order"><title>Patents:</title><list-item><p>Several patents related to use of AAV-NRTN for neurodegenerative diseases, including PD. However, all rights assigned to Ceregene, Inc.</p></list-item></list></p><p><list list-type="order"><title>Publishing Royalties:</title><list-item><p>Several patents related to AAV-NRTN as therapy for neurodgenerative diseases, including PD, but all rights assigned to Ceregene.</p></list-item></list></p><p><list list-type="order"><title>Employment, Commercial Entity:</title><list-item><p>Chief Scientific Officer and Exec Vice President of Ceregene, Inc. a for-profit company that has been developing CERE-120 for PD and CERE-110 for AD for past 11+ years</p></list-item></list></p><p><list list-type="order"><title>Consultancies:</title><list-item><p>Officer for Ceregene, a non-profitable, for-profit company that has been developing CERE-120 for PD and CERE- 110 for AD for past 11+ years</p></list-item></list></p><p><list list-type="order"><title>Speakers' Bureaus:</title><list-item><p>NONE</p></list-item></list></p><p><list list-type="order"><title>Other activities:</title><list-item><p>As Officer of Ceregene, have been awared stock options</p></list-item></list></p><p><list list-type="order"><title>Clinical Procedures or Imaging Studies:</title><list-item><p>NONE</p></list-item></list></p><p><list list-type="order"><title>Research Support, Commercial Entities:</title><list-item><p>received salary from Ceregene, a commerical entity, as listed earlier</p></list-item></list></p><p><list list-type="order"><title>Research Support, Government Entities:</title><list-item><p>NONE</p></list-item></list></p><p><list list-type="order"><title>Research Support, Academic Entities:</title><list-item><p>Adjunct Professor, Tufts University Medical School</p></list-item></list></p><p><list list-type="order"><title>Research Support, Foundations and Societies:</title><list-item><p>NONE</p></list-item></list></p><p><list list-type="order"><title>Stock/Stock Options/Board of Directors Compensation:</title><list-item><p>NONE</p></list-item></list></p><p><list list-type="order"><title>License Fee Payments, Technology or Inventions:</title><list-item><p>NONE</p></list-item></list></p><p><list list-type="order"><title>Royalty Payments, Technology or Inventions:</title><list-item><p>NONE</p></list-item></list></p><p><list list-type="order"><title>Stock/Stock Options, Research Sponsor:</title><list-item><p>Yes, stock optinos from Ceregene, as stated above</p></list-item></list></p><p><list list-type="order"><title>Stock/Stock Options, Medical Equipment & Materials:</title><list-item><p>NONE</p></list-item></list></p><p><list list-type="order"><title>Legal Proceedings:</title><list-item><p>Expert witness for legal litigation involving biotech industry.</p></list-item></list></p></author-comment></contrib><contrib contrib-type="author"><name><surname>Baumann</surname><given-names>Tiffany L.</given-names></name><degrees>BS</degrees><author-comment content-type="disclosure"><p><list list-type="order"><title>Scientific Advisory Boards:</title><list-item><p>NONE</p></list-item></list></p><p><list list-type="order"><title>Gifts:</title><list-item><p>NONE</p></list-item></list></p><p><list list-type="order"><title>Funding for Travel or Speaker Honoraria:</title><list-item><p>NONE</p></list-item></list></p><p><list list-type="order"><title>Editorial Boards:</title><list-item><p>NONE</p></list-item></list></p><p><list list-type="order"><title>Patents:</title><list-item><p>NONE</p></list-item></list></p><p><list list-type="order"><title>Publishing Royalties:</title><list-item><p>NONE</p></list-item></list></p><p><list list-type="order"><title>Employment, Commercial Entity:</title><list-item><p>Receives salary and stock options as employee at Ceregene since 2004. Current position = Director of Clinical Operations & Manager of Regulatory Affairs</p></list-item></list></p><p><list list-type="order"><title>Consultancies:</title><list-item><p>NONE</p></list-item></list></p><p><list list-type="order"><title>Speakers' Bureaus:</title><list-item><p>NONE</p></list-item></list></p><p><list list-type="order"><title>Other Activities:</title><list-item><p>NONE</p></list-item></list></p><p><list list-type="order"><title>Clinical Procedures or Imaging Studies:</title><list-item><p>NONE</p></list-item></list></p><p><list list-type="order"><title>Research Support, Commercial Entities:</title><list-item><p>NONE</p></list-item></list></p><p><list list-type="order"><title>Research Support, Government Entities:</title><list-item><p>NONE</p></list-item></list></p><p><list list-type="order"><title>Research Support, Academic Entities:</title><list-item><p>NONE</p></list-item></list></p><p><list list-type="order"><title>Research Support, Foundations and Societies:</title><list-item><p>NONE</p></list-item></list></p><p><list list-type="order"><title>Stock/Stock Options/Board of Directors Compensation:</title><list-item><p>NONE</p></list-item></list></p><p><list list-type="order"><title>License Fee Payments, Technology or Inventions:</title><list-item><p>NONE</p></list-item></list></p><p><list list-type="order"><title>Royalty Payments, Technology or Inventions:</title><list-item><p>NONE</p></list-item></list></p><p><list list-type="order"><title>Stock/Stock Options, Research Sponsor:</title><list-item><p>NONE</p></list-item></list></p><p><list list-type="order"><title>Stock/Stock Options, Medical Equipment & Materials:</title><list-item><p>NONE</p></list-item></list></p><p><list list-type="order"><title>Legal Proceedings:</title><list-item><p>NONE</p></list-item></list></p></author-comment></contrib><contrib contrib-type="author"><name><surname>Siffert</surname><given-names>Joao</given-names></name><degrees>MD</degrees><author-comment content-type="disclosure"><p><list list-type="order"><title>Scientific Advisory Boards:</title><list-item><p>NONE</p></list-item></list></p><p><list list-type="order"><title>Gifts:</title><list-item><p>NONE</p></list-item></list></p><p><list list-type="order"><title>Funding for Travel or Speaker Honoraria:</title><list-item><p>NONE</p></list-item></list></p><p><list list-type="order"><title>Editorial Boards:</title><list-item><p>NONE</p></list-item></list></p><p><list list-type="order"><title>Patents:</title><list-item><p>NONE</p></list-item></list></p><p><list list-type="order"><title>Publishing Royalties:</title><list-item><p>NONE</p></list-item></list></p><p><list list-type="order"><title>Employment, Commercial Entity:</title><list-item><p>1) Avanir Pharmaceuticals, SVP, R&D and Chief Scientific Officer (August, 2011 - Present) 2) Ceregene, Inc., VP, Chief Medical Officer (9/2007-8/2011)</p></list-item></list></p><p><list list-type="order"><title>Consultancies:</title><list-item><p>NONE</p></list-item></list></p><p><list list-type="order"><title>Speakers' Bureaus:</title><list-item><p>NONE</p></list-item></list></p><p><list list-type="order"><title>Other Activities:</title><list-item><p>NONE</p></list-item></list></p><p><list list-type="order"><title>Clinical Procedures or Imaging Studies:</title><list-item><p>NONE</p></list-item></list></p><p><list list-type="order"><title>Research Support, Commercial Entities:</title><list-item><p>Full time employee of Avanir Pharmaceuticals (see above)</p></list-item></list></p><p><list list-type="order"><title>Research Support, Government Entities:</title><list-item><p>NONE</p></list-item></list></p><p><list list-type="order"><title>Research Support, Academic Entities:</title><list-item><p>NONE</p></list-item></list></p><p><list list-type="order"><title>Research Support, Foundations and Societies:</title><list-item><p>The Michael J Fox Foundation for PD Research (J. Siffert, PI; grant awarded to Avanir Pharmaceuticals, Inc.)</p></list-item></list></p><p><list list-type="order"><title>Stock/Stock Options/Board of Directors Compensation:</title><list-item><p>NONE</p></list-item></list></p><p><list list-type="order"><title>License Fee Payments, Technology or Inventions:</title><list-item><p>NONE</p></list-item></list></p><p><list list-type="order"><title>Royalty Payments, Technology or Inventions:</title><list-item><p>NONE</p></list-item></list></p><p><list list-type="order"><title>Stock/Stock Options, Research Sponsor:</title><list-item><p>1) 5 thousand stock options from Ceregene 2) Stock options and stocks from Avanir Pharmaceuticals</p></list-item></list></p><p><list list-type="order"><title>Stock/Stock Options, Medical Equipment & Materials:</title><list-item><p>NONE</p></list-item></list></p><p><list list-type="order"><title>Legal Proceedings:</title><list-item><p>NONE</p></list-item></list></p></author-comment></contrib><contrib contrib-type="author"><name><surname>Herzog</surname><given-names>Christopher D.</given-names></name><degrees>PhD</degrees><author-comment content-type="disclosure"><p><list list-type="order"><title>Scientific Advisory Boards:</title><list-item><p>NONE</p></list-item></list></p><p><list list-type="order"><title>Gifts:</title><list-item><p>NONE</p></list-item></list></p><p><list list-type="order"><title>Funding for Travel or Speaker Honoraria:</title><list-item><p>NONE</p></list-item></list></p><p><list list-type="order"><title>Editorial Boards:</title><list-item><p>NONE</p></list-item></list></p><p><list list-type="order"><title>Patents:</title><list-item><p>NONE</p></list-item></list></p><p><list list-type="order"><title>Publishing Royalties:</title><list-item><p>NONE</p></list-item></list></p><p><list list-type="order"><title>Employment, Commercial Entity:</title><list-item><p>Received salary and stock options as an employee of Ceregene, Inc.</p></list-item></list></p><p><list list-type="order"><title>Consultancies:</title><list-item><p>NONE</p></list-item></list></p><p><list list-type="order"><title>Speakers' Bureaus:</title><list-item><p>NONE</p></list-item></list></p><p><list list-type="order"><title>Other Activities:</title><list-item><p>NONE</p></list-item></list></p><p><list list-type="order"><title>Clinical Procedures or Imaging Studies:</title><list-item><p>NONE</p></list-item></list></p><p><list list-type="order"><title>Research Support, Commercial Entities:</title><list-item><p>NONE</p></list-item></list></p><p><list list-type="order"><title>Research Support, Government Entities:</title><list-item><p>NONE</p></list-item></list></p><p><list list-type="order"><title>Research Support, Academic Entities:</title><list-item><p>NONE</p></list-item></list></p><p><list list-type="order"><title>Research Support, Foundations and Societies:</title><list-item><p>NONE</p></list-item></list></p><p><list list-type="order"><title>Stock/Stock Options/Board of Directors Compensation:</title><list-item><p>NONE</p></list-item></list></p><p><list list-type="order"><title>License Fee Payments, Technology or Inventions:</title><list-item><p>NONE</p></list-item></list></p><p><list list-type="order"><title>Royalty Payments, Technology or Inventions:</title><list-item><p>NONE</p></list-item></list></p><p><list list-type="order"><title>Stock/Stock Options, Research Sponsor:</title><list-item><p>NONE</p></list-item></list></p><p><list list-type="order"><title>Stock/Stock Options, Medical Equipment & Materials:</title><list-item><p>NONE</p></list-item></list></p><p><list list-type="order"><title>Legal Proceedings:</title><list-item><p>NONE</p></list-item></list></p></author-comment></contrib><contrib contrib-type="author"><name><surname>Alterman</surname><given-names>Ron</given-names></name><degrees>MD</degrees><author-comment content-type="disclosure"><p><list list-type="order"><title>Scientific Advisory Boards:</title><list-item><p>Medtronic, Inc. Codman, Inc.</p></list-item></list></p><p><list list-type="order"><title>Gifts:</title><list-item><p>NONE</p></list-item></list></p><p><list list-type="order"><title>Funding for Travel or Speaker Honoraria:</title><list-item><p>NONE</p></list-item></list></p><p><list list-type="order"><title>Editorial Boards:</title><list-item><p>NONE</p></list-item></list></p><p><list list-type="order"><title>Patents:</title><list-item><p>NONE</p></list-item></list></p><p><list list-type="order"><title>Publishing Royalties:</title><list-item><p>NONE</p></list-item></list></p><p><list list-type="order"><title>Employment, Commercial Entity:</title><list-item><p>NONE</p></list-item></list></p><p><list list-type="order"><title>Consultancies:</title><list-item><p>Ceregene, Inc.</p></list-item></list></p><p><list list-type="order"><title>Speakers' Bureaus:</title><list-item><p>NONE</p></list-item></list></p><p><list list-type="order"><title>Other Activities:</title><list-item><p>NONE</p></list-item></list></p><p><list list-type="order"><title>Clinical Procedures or Imaging Studies:</title><list-item><p>NONE</p></list-item></list></p><p><list list-type="order"><title>Research Support, Commercial Entities:</title><list-item><p>NONE</p></list-item></list></p><p><list list-type="order"><title>Research Support, Government Entities:</title><list-item><p>NONE</p></list-item></list></p><p><list list-type="order"><title>Research Support, Academic Entities:</title><list-item><p>NONE</p></list-item></list></p><p><list list-type="order"><title>Research Support, Foundations and Societies:</title><list-item><p>NONE</p></list-item></list></p><p><list list-type="order"><title>Stock/Stock Options/Board of Directors Compensation:</title><list-item><p>NONE</p></list-item></list></p><p><list list-type="order"><title>License Fee Payments, Technology or Inventions:</title><list-item><p>NONE</p></list-item></list></p><p><list list-type="order"><title>Royalty Payments, Technology or Inventions:</title><list-item><p>NONE</p></list-item></list></p><p><list list-type="order"><title>Stock/Stock Options, Research Sponsor:</title><list-item><p>NONE</p></list-item></list></p><p><list list-type="order"><title>Stock/Stock Options, Medical Equipment & Materials:</title><list-item><p>NONE</p></list-item></list></p><p><list list-type="order"><title>Legal Proceedings:</title><list-item><p>NONE</p></list-item></list></p></author-comment></contrib><contrib contrib-type="author"><name><surname>Boulis</surname><given-names>Nicholas</given-names></name><degrees>MD</degrees><author-comment content-type="disclosure"><p><list list-type="order"><title>Scientific Advisory Boards:</title><list-item><p>NONE</p></list-item></list></p><p><list list-type="order"><title>Gifts:</title><list-item><p>NONE</p></list-item></list></p><p><list list-type="order"><title>Funding for travel or speaker honoraria:</title><list-item><p>1. NeuralStem paid for a trip to Mexico so that i could evaluate the surgical facilities available to support an ALS stem cell transplantation trial.</p></list-item></list></p><p><list list-type="order"><title>Editorial Boards:</title><list-item><p>NONE</p></list-item></list></p><p><list list-type="order"><title>Patents:</title><list-item><p>1. I received a 170,000 dollar payment as part of payment for my share of IntElect Medical after its sale to Boston Scientific. 2. NeuralStem licensed 2 patents from the Cleveland Clinic that i own a share of. I received an $80,000 this in 2011.</p></list-item></list></p><p><list list-type="order"><title>Publishing Royalties:</title><list-item><p>NONE</p></list-item></list></p><p><list list-type="order"><title>Employment, Commercial Entity:</title><list-item><p>1. i work for Emory University and Emory Healthcare. 2. I received income for reviewing a lawsuit against medtronic and a pain surgeon.</p></list-item></list></p><p><list list-type="order"><title>Consultancies:</title><list-item><p>1. I served as a consultant for Ceregene receiving $9999/year. This relationship ended in 2012. 2. I receive $9999 from NeuralStem/year.</p></list-item></list></p><p><list list-type="order"><title>Speakers' Bureaus:</title><list-item><p>NONE</p></list-item></list></p><p><list list-type="order"><title>Other Activities:</title><list-item><p>NONE</p></list-item></list></p><p><list list-type="order"><title>Clinical Procedures or Imaging Studies:</title><list-item><p>NONE</p></list-item></list></p><p><list list-type="order"><title>Research Support, Commercial Entities:</title><list-item><p>NONE</p></list-item></list></p><p><list list-type="order"><title>Research Support, Government Entities:</title><list-item><p>NONE</p></list-item></list></p><p><list list-type="order"><title>Research Support, Academic Entities:</title><list-item><p>NONE</p></list-item></list></p><p><list list-type="order"><title>Research Support, Foundations and Societies:</title><list-item><p>NONE</p></list-item></list></p><p><list list-type="order"><title>Stock/Stock Options/Board of Directors Compensation:</title><list-item><p>NONE</p></list-item></list></p><p><list list-type="order"><title>License fee payments, Technology or Inventions:</title><list-item><p>1. see above reference to payment for Cleveland Clinic Patents by NeuralStem.</p></list-item></list></p><p><list list-type="order"><title>Royalty Payments, Technology or Inventions:</title><list-item><p>NONE</p></list-item></list></p><p><list list-type="order"><title>Stock/Stock Options, Research Sponsor:</title><list-item><p>NONE</p></list-item></list></p><p><list list-type="order"><title>Stock/Stock Options, Medical Equipment & Materials:</title><list-item><p>NONE</p></list-item></list></p><p><list list-type="order"><title>Legal Proceedings:</title><list-item><p>1. Case review for law firm defending medtronic about a sacral stimulation case.</p></list-item></list></p></author-comment></contrib><contrib contrib-type="author"><name><surname>Turner</surname><given-names>Dennis A.</given-names></name><degrees>MD</degrees><author-comment content-type="disclosure"><p><list list-type="order"><title>Scientific Advisory Boards:</title><list-item><p>Ceregene advisory position for study plan reviews</p></list-item></list></p><p><list list-type="order"><title>Gifts:</title><list-item><p>NONE</p></list-item></list></p><p><list list-type="order"><title>Funding for Travel or Speaker Honoraria:</title><list-item><p>NONE</p></list-item></list></p><p><list list-type="order"><title>Editorial Boards:</title><list-item><p>NONE</p></list-item></list></p><p><list list-type="order"><title>Patents:</title><list-item><p>NONE</p></list-item></list></p><p><list list-type="order"><title>Publishing Royalties:</title><list-item><p>NONE</p></list-item></list></p><p><list list-type="order"><title>Employment, Commercial Entity:</title><list-item><p>NONE</p></list-item></list></p><p><list list-type="order"><title>Consultancies:</title><list-item><p>Medtronics Neurological Consultant for Alnylam/Medtronics planned siRNA study for Huntington's Disase</p></list-item></list></p><p><list list-type="order"><title>Speakers' Bureaus:</title><list-item><p>NONE</p></list-item></list></p><p><list list-type="order"><title>Other Activities:</title><list-item><p>NONE</p></list-item></list></p><p><list list-type="order"><title>Clinical Procedures or Imaging Studies:</title><list-item><p>NONE</p></list-item></list></p><p><list list-type="order"><title>Research Support, Commercial Entities:</title><list-item><p>NONE</p></list-item></list></p><p><list list-type="order"><title>Research Support, Government Entities:</title><list-item><p>NIH Grants R01AG037599 (PI) 2010-2014,R21NS066115 (PI) 2010-2013, and RO1NS079312 (Coll Inv) 2012-1016; VA Merit Review funding (PI) 1982-2012</p></list-item></list></p><p><list list-type="order"><title>Research Support, Academic Entities:</title><list-item><p>NONE</p></list-item></list></p><p><list list-type="order"><title>Research Support, Foundations and Societies:</title><list-item><p>NONE</p></list-item></list></p><p><list list-type="order"><title>Stock/Stock Options/Board of Directors Compensation:</title><list-item><p>NONE</p></list-item></list></p><p><list list-type="order"><title>License Fee Payments, Technology or Inventions:</title><list-item><p>NONE</p></list-item></list></p><p><list list-type="order"><title>Royalty Payments, Technology or Inventions:</title><list-item><p>NONE</p></list-item></list></p><p><list list-type="order"><title>Stock/Stock Options, Research Sponsor:</title><list-item><p>NONE</p></list-item></list></p><p><list list-type="order"><title>Stock/Stock Options, Medical Equipment & Materials:</title><list-item><p>NONE</p></list-item></list></p><p><list list-type="order"><title>Legal Proceedings:</title><list-item><p>NONE</p></list-item></list></p></author-comment></contrib><contrib contrib-type="author"><name><surname>Stacy</surname><given-names>Mark</given-names></name><degrees>MD</degrees><author-comment content-type="disclosure"><p><list list-type="order"><title>Scientific Advisory Boards:</title><list-item><p>Scientific Advisory Committee: Allergan</p></list-item></list></p><p><list list-type="order"><title>Gifts:</title><list-item><p>NONE</p></list-item></list></p><p><list list-type="order"><title>Funding for Travel or Speaker Honoraria:</title><list-item><p>NONE</p></list-item></list></p><p><list list-type="order"><title>Editorial Boards:</title><list-item><p>Editorial Board: Journal of Clinical Investigation</p></list-item></list></p><p><list list-type="order"><title>Patents:</title><list-item><p>NONE</p></list-item></list></p><p><list list-type="order"><title>Publishing Royalties:</title><list-item><p>1) Handbook of Dystonia, Informa Press, 2012.</p></list-item></list></p><p><list list-type="order"><title>Employment, Commercial Entity:</title><list-item><p>NONE</p></list-item></list></p><p><list list-type="order"><title>Consultancies:</title><list-item><p>Johnson & Johnson, Merz Pharmaceuticals, Osmotica, Neuronova, Pfizer, ProStrakan, Psychogenics, SKLSI.</p></list-item></list></p><p><list list-type="order"><title>Speakers' Bureaus:</title><list-item><p>NONE</p></list-item></list></p><p><list list-type="order"><title>Other Activities:</title><list-item><p>NONE</p></list-item></list></p><p><list list-type="order"><title>Clinical Procedures or Imaging Studies:</title><list-item><p>NONE</p></list-item></list></p><p><list list-type="order"><title>Research Support, Commercial Entities:</title><list-item><p>Ceregene, EMD Serono, Merck, Neuraltus, Novartis, Parkinson Study Group.</p></list-item></list></p><p><list list-type="order"><title>Research Support, Government Entities:</title><list-item><p>NONE</p></list-item></list></p><p><list list-type="order"><title>Research Support, Academic Entities:</title><list-item><p>NONE</p></list-item></list></p><p><list list-type="order"><title>Research Support, Foundations and Societies:</title><list-item><p>Michael J. Fox Foundation</p></list-item></list></p><p><list list-type="order"><title>Stock/Stock Options/Board of Directors Compensation:</title><list-item><p>NONE</p></list-item></list></p><p><list list-type="order"><title>License Fee Payments, Technology or Inventions:</title><list-item><p>NONE</p></list-item></list></p><p><list list-type="order"><title>Royalty Payments, Technology or Inventions:</title><list-item><p>NONE</p></list-item></list></p><p><list list-type="order"><title>Stock/Stock Options, Research Sponsor:</title><list-item><p>NONE</p></list-item></list></p><p><list list-type="order"><title>Stock/Stock Options, Medical Equipment & Materials:</title><list-item><p>NONE</p></list-item></list></p><p><list list-type="order"><title>Legal Proceedings:</title><list-item><p>NONE</p></list-item></list></p></author-comment></contrib><contrib contrib-type="author"><name><surname>Lang</surname><given-names>Anthony E.</given-names></name><degrees>MD</degrees><author-comment content-type="disclosure"><p><list list-type="order"><title>Scientific Advisory Boards:</title><list-item><p>Dr. Lang has served as an advisor for Abbott, Allon Therapeutics, Biovail, Boerhinger-Ingelheim, Cephalon, Ceregene, Eisai, Medtronic, Lundbeck A/S, NeuroMolecular, Novartis, Merck Serono, Solvay, and Teva, and received grants from Canadian Institutes of Health Research, Dystonia Medical Research Foundation, Michael J. Fox Foundation, National Parkinson Foundation, Ontario Problem Gambling Research Centre, Parkinson's Disease Foundation.</p></list-item></list></p><p><list list-type="order"><title>Gifts:</title><list-item><p>NONE</p></list-item></list></p><p><list list-type="order"><title>Funding for Travel or Speaker Honoraria:</title><list-item><p>NONE</p></list-item></list></p><p><list list-type="order"><title>Editorial Boards:</title><list-item><p>NONE</p></list-item></list></p><p><list list-type="order"><title>Patents:</title><list-item><p>NONE</p></list-item></list></p><p><list list-type="order"><title>Publishing Royalties:</title><list-item><p>Schapira A, Lang AE, Fahn S. Movement Disorders 4. Saunders, Elsevier, Philadelphia, PA 2009. Olanow CW, Stocchi F, Lang AE. The Non-motor and Non- Dopaminergic Features of Parkinson's Disease. Wiley- Blackwell, UK. 2011. Weiner WJ, Shulman LM, Lang AE. Parkinson's Disease: A Complete Guide for Patients and Families. The Johns Hopkins University Press, Baltimore. 2001</p></list-item></list></p><p><list list-type="order"><title>Employment, Commercial Entity:</title><list-item><p>NONE</p></list-item></list></p><p><list list-type="order"><title>Consultancies:</title><list-item><p>NONE</p></list-item></list></p><p><list list-type="order"><title>Speakers' Bureaus:</title><list-item><p>NONE</p></list-item></list></p><p><list list-type="order"><title>Other Activities:</title><list-item><p>NONE</p></list-item></list></p><p><list list-type="order"><title>Clinical Procedures or Imaging Studies:</title><list-item><p>NONE</p></list-item></list></p><p><list list-type="order"><title>Research Support, Commercial Entities:</title><list-item><p>NONE</p></list-item></list></p><p><list list-type="order"><title>Research Support, Government Entities:</title><list-item><p>Canadian Institutes of Health Research,</p></list-item></list></p><p><list list-type="order"><title>Research Support, Academic Entities:</title><list-item><p>NONE</p></list-item></list></p><p><list list-type="order"><title>Research Support, Foundations and Societies:</title><list-item><p>Dystonia Medical Research Foundation, Michael J. Fox Foundation, National Parkinson Foundation, Ontario Problem Gambling Research Centre, Parkinson's Disease Foundation, and</p></list-item></list></p><p><list list-type="order"><title>Stock/Stock Options/Board of Directors Compensation:</title><list-item><p>Ceregene</p></list-item></list></p><p><list list-type="order"><title>License Fee Payments, Technology or Inventions:</title><list-item><p>NONE</p></list-item></list></p><p><list list-type="order"><title>Royalty Payments, Technology or Inventions:</title><list-item><p>NONE</p></list-item></list></p><p><list list-type="order"><title>Stock/Stock Options, Research Sponsor:</title><list-item><p>NONE</p></list-item></list></p><p><list list-type="order"><title>Stock/Stock Options, Medical Equipment & Materials:</title><list-item><p>NONE</p></list-item></list></p><p><list list-type="order"><title>Legal Proceedings:</title><list-item><p>Welding rod companies</p></list-item></list></p></author-comment></contrib><contrib contrib-type="author"><name><surname>Lozano</surname><given-names>Andres M.</given-names></name><degrees>MD</degrees><author-comment content-type="disclosure"><p><list list-type="order"><title>Scientific Advisory Boards:</title><list-item><p>Johnson and Johnson (Corporate Office of Science and Technology) Codman Ceregene Neurologix Functional Neuroscience</p></list-item></list></p><p><list list-type="order"><title>Gifts:</title><list-item><p>NONE</p></list-item></list></p><p><list list-type="order"><title>Funding for Travel or Speaker Honoraria:</title><list-item><p>NONE</p></list-item></list></p><p><list list-type="order"><title>Editorial Boards:</title><list-item><p>Canadian Journal of Neurological Science (Associate Editor 2001-2006) Brain Stimulation (Deputy Editor) Journal of Neurosurgery Neurosurgery Movement Disorders World Neurosurgery Neurological Research Stereotactic and Functional Neurosurgery Operative Neurosurgery NeuroRx Surgical Neurology Parkinsonism and Related Disorders Neurobiology of disease (Guest Editor) Neurosurgical focus (Guest Editor)</p></list-item></list></p><p><list list-type="order"><title>Patents:</title><list-item><p>1) Lozano AM and Mayberg HS. Methods of treating depression, mood disorders and anxiety using neuromodulation, 2010, US patent No. 7,653,433. 2) Lozano AM and Mayberg HS. Methods of treating depression, mood disorders and anxiety using neuromodulation, 2008, US patent No. 7,346,395. 3) Velasco F, Jimenez F, Velasco M and Lozano AM. Method of treating mood disorders and/or anxiety disorders by brain stimulation. 2007, US patent No. 7,313, 442. 4) Lozano AM. Brain Stimulation lead used for lesioning. 2007, US patent No. 7,285,118. 5) Lozano AM and Rise M. Method of treating movement disorders by electrical stimulation and/or drug infusion of the pendunculopontine nucleus. 2002, US patent No. 6,356,784.</p></list-item></list></p><p><list list-type="order"><title>Publishing Royalties:</title><list-item><p>1) Tarsy D, Vitek JL, Lozano AM (Eds). Surgical Treatment of Parkinson's Disease and Other Movement Disorders. 2002 Humana Press. 2) Lozano AM, Tasker RR, Gildenberg P (Eds). Textbook of Stereotactic and Functional Neurosurgery (2nd edition). 195 Chapters and 3285 pages, Springer. Published May 2009</p></list-item></list></p><p><list list-type="order"><title>Employment, Commercial Entity:</title><list-item><p>Board of Functional Neuromodulation Inc.</p></list-item></list></p><p><list list-type="order"><title>Consultancies:</title><list-item><p>Medtronic St-Jude Boston Scientific Amgen Eli Lilly Bristol Myers Elekta Bayer Schering-Plough QIC Functional Neuroscience</p></list-item></list></p><p><list list-type="order"><title>Speakers' Bureaus:</title><list-item><p>NONE</p></list-item></list></p><p><list list-type="order"><title>Other Activities:</title><list-item><p>NONE</p></list-item></list></p><p><list list-type="order"><title>Clinical Procedures or Imaging Studies:</title><list-item><p>NONE</p></list-item></list></p><p><list list-type="order"><title>Research Support, Commercial Entities:</title><list-item><p>NONE</p></list-item></list></p><p><list list-type="order"><title>Research Support, Government Entities:</title><list-item><p>NONE</p></list-item></list></p><p><list list-type="order"><title>Research Support, Academic Entities:</title><list-item><p>NONE</p></list-item></list></p><p><list list-type="order"><title>Research Support, Foundations and Societies:</title><list-item><p>NONE</p></list-item></list></p><p><list list-type="order"><title>Stock/Stock Options/Board of Directors Compensation:</title><list-item><p>Ceregene</p></list-item></list></p><p><list list-type="order"><title>License fee payments, Technology or Inventions:</title><list-item><p>Functional Neuroscience</p></list-item></list></p><p><list list-type="order"><title>Royalty Payments, Technology or Inventions:</title><list-item><p>NONE</p></list-item></list></p><p><list list-type="order"><title>Stock/Stock Options, Research Sponsor:</title><list-item><p>NONE</p></list-item></list></p><p><list list-type="order"><title>Stock/Stock Options, Medical Equipment & Materials:</title><list-item><p>NONE</p></list-item></list></p><p><list list-type="order"><title>Legal Proceedings:</title><list-item><p>NONE</p></list-item></list></p></author-comment></contrib><contrib contrib-type="author"><name><surname>Olanow</surname><given-names>C. Warren</given-names></name><degrees>MD</degrees><author-comment content-type="disclosure"><p><list list-type="order"><title>Scientific Advisory Boards:</title><list-item><p>NONE</p></list-item></list></p><p><list list-type="order"><title>Gifts:</title><list-item><p>NONE</p></list-item></list></p><p><list list-type="order"><title>Funding for Travel or Speaker Honoraria:</title><list-item><p>NONE</p></list-item></list></p><p><list list-type="order"><title>Editorial Boards:</title><list-item><p>NONE</p></list-item></list></p><p><list list-type="order"><title>Patents:</title><list-item><p>NONE</p></list-item></list></p><p><list list-type="order"><title>Publishing Royalties:</title><list-item><p>NONE</p></list-item></list></p><p><list list-type="order"><title>Employment, Commercial Entity:</title><list-item><p>NONE</p></list-item></list></p><p><list list-type="order"><title>Consultancies:</title><list-item><p>NONE</p></list-item></list></p><p><list list-type="order"><title>Speakers' Bureaus:</title><list-item><p>NONE</p></list-item></list></p><p><list list-type="order"><title>Other Activities:</title><list-item><p>NONE</p></list-item></list></p><p><list list-type="order"><title>Clinical Procedures or Imaging Studies:</title><list-item><p>NONE</p></list-item></list></p><p><list list-type="order"><title>Research Support, Commercial Entities:</title><list-item><p>NONE</p></list-item></list></p><p><list list-type="order"><title>Research Support, Government Entities:</title><list-item><p>NONE</p></list-item></list></p><p><list list-type="order"><title>Research Support, Academic Entities:</title><list-item><p>NONE</p></list-item></list></p><p><list list-type="order"><title>Research Support, Foundations and Societies:</title><list-item><p>NONE</p></list-item></list></p><p><list list-type="order"><title>Stock/Stock Options/Board of Directors Compensation:</title><list-item><p>NONE</p></list-item></list></p><p><list list-type="order"><title>License Fee Payments, Technology or Inventions:</title><list-item><p>NONE</p></list-item></list></p><p><list list-type="order"><title>Royalty Payments, Technology or Inventions:</title><list-item><p>NONE</p></list-item></list></p><p><list list-type="order"><title>Stock/Stock Options, Research Sponsor:</title><list-item><p>NONE</p></list-item></list></p><p><list list-type="order"><title>Stock/Stock Options, Medical Equipment & Materials:</title><list-item><p>NONE</p></list-item></list></p><p><list list-type="order"><title>Legal Proceedings:</title><list-item><p>NONE</p></list-item></list></p></author-comment></contrib><aff>From Ceregene, Inc. (R.T.B., T.L.B., J.S., C.D.H.), San Diego, CA; Mount Sinai School of Medicine (R.A., C.W.O.), New York, NY; Emory University (N.B.), Atlanta, GA; Duke University (D.A.T., M.S.), Durham, NC; University of Toronto (A.E.L., A.M.L.), Toronto; and The Edmond J. Safra Program in Parkinson's Disease (A.E.L.), Toronto Western Hospital, Toronto, Canada; Avanir Pharmaceuticals (J.S.), Aliso Viejo, CA; Beth Israel Deaconess Medical Center (R.A.), Boston, MA.</aff></contrib-group><author-notes><corresp>Correspondence to Dr. Bartus: <email>rtbartus@ceregene.com</email></corresp><fn fn-type="financial-disclosure"><p>Go to <ext-link ext-link-type="uri" xlink:href="http://neurology.org/">Neurology.org</ext-link> for full disclosures. Funding information and disclosures deemed relevant by the authors, if any, are provided at the end of the article.</p></fn></author-notes><pub-date pub-type="ppub"><day>30</day><month>4</month><year>2013</year></pub-date><pub-date pub-type="pmc-release"><day>30</day><month>4</month><year>2014</year></pub-date><pmc-comment> PMC Release delay is 12 months and
0 days and was based on the
<volume>80</volume><issue>18</issue><fpage>1698</fpage><lpage>1701</lpage><history><date date-type="received"><day>10</day><month>10</month><year>2012</year></date><date date-type="accepted"><day>22</day><month>1</month><year>2013</year></date></history><permissions><copyright-statement>© 2013 American Academy of Neurology</copyright-statement><copyright-year>2013</copyright-year><copyright-holder>American Academy of Neurology</copyright-holder></permissions><self-uri xlink:title="pdf" xlink:type="simple" xlink:href="WNL204989.pdf"></self-uri><abstract><sec><title>Objective:</title><p>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).</p></sec><sec><title>Methods:</title><p>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.</p></sec><sec><title>Results:</title><p>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.</p></sec><sec><title>Conclusions:</title><p>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.</p></sec><sec><title>Classification of evidence:</title><p>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.</p></sec></abstract><custom-meta-group><custom-meta><meta-name>special-property</meta-name><meta-value>triangle</meta-value></custom-meta></custom-meta-group></article-meta></front></pmc></record>Pour manipuler ce document sous Unix (Dilib)
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