Prerequisites to launch neuroprotective trials in Parkinson's disease: an industry perspective.
Identifieur interne : 000C54 ( Main/Exploration ); précédent : 000C53; suivant : 000C55Prerequisites to launch neuroprotective trials in Parkinson's disease: an industry perspective.
Auteurs : Johannes R. Streffer [Belgique] ; Igor D. Grachev ; Cheryl Fitzer-Attas ; Baltazar Gomez-Mancilla ; Babak Boroojerdi ; Juliana Bronzova ; Susanne Ostrowitzki ; Stephen J. Victor ; Paulo Fontoura ; Robert AlexanderSource :
- Movement disorders : official journal of the Movement Disorder Society [ 1531-8257 ] ; 2012.
English descriptors
- KwdEn :
- MESH :
- chemical , metabolism : Biological Markers.
- diagnosis : Parkinson Disease.
- metabolism : Parkinson Disease.
- prevention & control : Parkinson Disease.
- standards : Clinical Trials as Topic, Research Design.
- chemical , therapeutic use : Neuroprotective Agents.
- Humans.
Abstract
Realizing that 60% to 80% of dopaminergic nigrostriatal neurons are nonfunctional at the time of clinical diagnosis, there is an emerging consensus that disease-modifying treatments should be initiated in the earliest stages of Parkinson's disease (PD). To date, clinical trial designs and metrics in PD have been focused on motor symptoms as the core feature of the clinical disease. To identify earlier or "pre-motor" populations in PD, new markers have been proposed. We address the prerequisites needed to use these pre-motor markers in clinical trials for the selection of subjects, definition of populations, and monitoring of disease progression. This may require the development of new diagnostic criteria potentially based on non-motor clinical signs, imaging techniques, or biological features, all requiring discussion in a regulatory framework. Questions addressed include: Which steps must be taken to gain a broad consensus in the field from academic opinion leaders, patient advocacy groups, regulatory bodies, and industry? How do we prevent the selection of subgroups, which may not be representative of the full disease spectrum? Is there a way forward in personalized medicine? How do we balance risk and benefit in an at-risk population? While many tools are available, a concerted effort is required to develop integrated data sets, as well as to achieve the necessary standardization for multicenter clinical trials. To this end, public-private consortia (including academic centers, patient advocacy groups, and industry) will be of crucial importance to prospectively investigate and define the best tools and treatment paradigms.
DOI: 10.1002/mds.25017
PubMed: 22508283
Affiliations:
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Le document en format XML
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Grachev</name></author><author><name sortKey="Fitzer Attas, Cheryl" sort="Fitzer Attas, Cheryl" uniqKey="Fitzer Attas C" first="Cheryl" last="Fitzer-Attas">Cheryl Fitzer-Attas</name></author><author><name sortKey="Gomez Mancilla, Baltazar" sort="Gomez Mancilla, Baltazar" uniqKey="Gomez Mancilla B" first="Baltazar" last="Gomez-Mancilla">Baltazar Gomez-Mancilla</name></author><author><name sortKey="Boroojerdi, Babak" sort="Boroojerdi, Babak" uniqKey="Boroojerdi B" first="Babak" last="Boroojerdi">Babak Boroojerdi</name></author><author><name sortKey="Bronzova, Juliana" sort="Bronzova, Juliana" uniqKey="Bronzova J" first="Juliana" last="Bronzova">Juliana Bronzova</name></author><author><name sortKey="Ostrowitzki, Susanne" sort="Ostrowitzki, Susanne" uniqKey="Ostrowitzki S" first="Susanne" last="Ostrowitzki">Susanne Ostrowitzki</name></author><author><name sortKey="Victor, Stephen J" sort="Victor, Stephen J" uniqKey="Victor S" first="Stephen J" last="Victor">Stephen J. Victor</name></author><author><name sortKey="Fontoura, Paulo" sort="Fontoura, Paulo" uniqKey="Fontoura P" first="Paulo" last="Fontoura">Paulo Fontoura</name></author><author><name sortKey="Alexander, Robert" sort="Alexander, Robert" uniqKey="Alexander R" first="Robert" last="Alexander">Robert Alexander</name></author></titleStmt><publicationStmt><idno type="wicri:source">PubMed</idno><date when="2012">2012</date><idno type="doi">10.1002/mds.25017</idno><idno type="RBID">pubmed:22508283</idno><idno type="pmid">22508283</idno><idno type="wicri:Area/PubMed/Corpus">000E04</idno><idno type="wicri:Area/PubMed/Curation">000E04</idno><idno type="wicri:Area/PubMed/Checkpoint">000C79</idno><idno type="wicri:Area/Ncbi/Merge">003626</idno><idno type="wicri:Area/Ncbi/Curation">003626</idno><idno type="wicri:Area/Ncbi/Checkpoint">003626</idno><idno type="wicri:Area/Main/Merge">000C69</idno><idno type="wicri:Area/Main/Curation">000C54</idno><idno type="wicri:Area/Main/Exploration">000C54</idno></publicationStmt><sourceDesc><biblStruct><analytic><title xml:lang="en">Prerequisites to launch neuroprotective trials in Parkinson's disease: an industry perspective.</title><author><name sortKey="Streffer, Johannes R" sort="Streffer, Johannes R" uniqKey="Streffer J" first="Johannes R" last="Streffer">Johannes R. Streffer</name><affiliation wicri:level="1"><nlm:affiliation>Janssen Research & Development, Janssen-Pharmaceutical Companies of Johnson & Johnson, Beerse, Belgium. jstreffe@its.jnj.com</nlm:affiliation><country xml:lang="fr">Belgique</country><wicri:regionArea>Janssen Research & Development, Janssen-Pharmaceutical Companies of Johnson & Johnson, Beerse</wicri:regionArea><wicri:noRegion>Beerse</wicri:noRegion></affiliation></author><author><name sortKey="Grachev, Igor D" sort="Grachev, Igor D" uniqKey="Grachev I" first="Igor D" last="Grachev">Igor D. Grachev</name></author><author><name sortKey="Fitzer Attas, Cheryl" sort="Fitzer Attas, Cheryl" uniqKey="Fitzer Attas C" first="Cheryl" last="Fitzer-Attas">Cheryl Fitzer-Attas</name></author><author><name sortKey="Gomez Mancilla, Baltazar" sort="Gomez Mancilla, Baltazar" uniqKey="Gomez Mancilla B" first="Baltazar" last="Gomez-Mancilla">Baltazar Gomez-Mancilla</name></author><author><name sortKey="Boroojerdi, Babak" sort="Boroojerdi, Babak" uniqKey="Boroojerdi B" first="Babak" last="Boroojerdi">Babak Boroojerdi</name></author><author><name sortKey="Bronzova, Juliana" sort="Bronzova, Juliana" uniqKey="Bronzova J" first="Juliana" last="Bronzova">Juliana Bronzova</name></author><author><name sortKey="Ostrowitzki, Susanne" sort="Ostrowitzki, Susanne" uniqKey="Ostrowitzki S" first="Susanne" last="Ostrowitzki">Susanne Ostrowitzki</name></author><author><name sortKey="Victor, Stephen J" sort="Victor, Stephen J" uniqKey="Victor S" first="Stephen J" last="Victor">Stephen J. Victor</name></author><author><name sortKey="Fontoura, Paulo" sort="Fontoura, Paulo" uniqKey="Fontoura P" first="Paulo" last="Fontoura">Paulo Fontoura</name></author><author><name sortKey="Alexander, Robert" sort="Alexander, Robert" uniqKey="Alexander R" first="Robert" last="Alexander">Robert Alexander</name></author></analytic><series><title level="j">Movement disorders : official journal of the Movement Disorder Society</title><idno type="eISSN">1531-8257</idno><imprint><date when="2012" type="published">2012</date></imprint></series></biblStruct></sourceDesc></fileDesc><profileDesc><textClass><keywords scheme="KwdEn" xml:lang="en"><term>Biological Markers (metabolism)</term><term>Clinical Trials as Topic (standards)</term><term>Humans</term><term>Neuroprotective Agents (therapeutic use)</term><term>Parkinson Disease (diagnosis)</term><term>Parkinson Disease (metabolism)</term><term>Parkinson Disease (prevention & control)</term><term>Research Design (standards)</term></keywords><keywords scheme="MESH" type="chemical" qualifier="metabolism" xml:lang="en"><term>Biological Markers</term></keywords><keywords scheme="MESH" qualifier="diagnosis" xml:lang="en"><term>Parkinson Disease</term></keywords><keywords scheme="MESH" qualifier="metabolism" xml:lang="en"><term>Parkinson Disease</term></keywords><keywords scheme="MESH" qualifier="prevention & control" xml:lang="en"><term>Parkinson Disease</term></keywords><keywords scheme="MESH" qualifier="standards" xml:lang="en"><term>Clinical Trials as Topic</term><term>Research Design</term></keywords><keywords scheme="MESH" type="chemical" qualifier="therapeutic use" xml:lang="en"><term>Neuroprotective Agents</term></keywords><keywords scheme="MESH" xml:lang="en"><term>Humans</term></keywords></textClass></profileDesc></teiHeader><front><div type="abstract" xml:lang="en">Realizing that 60% to 80% of dopaminergic nigrostriatal neurons are nonfunctional at the time of clinical diagnosis, there is an emerging consensus that disease-modifying treatments should be initiated in the earliest stages of Parkinson's disease (PD). To date, clinical trial designs and metrics in PD have been focused on motor symptoms as the core feature of the clinical disease. To identify earlier or "pre-motor" populations in PD, new markers have been proposed. We address the prerequisites needed to use these pre-motor markers in clinical trials for the selection of subjects, definition of populations, and monitoring of disease progression. This may require the development of new diagnostic criteria potentially based on non-motor clinical signs, imaging techniques, or biological features, all requiring discussion in a regulatory framework. Questions addressed include: Which steps must be taken to gain a broad consensus in the field from academic opinion leaders, patient advocacy groups, regulatory bodies, and industry? How do we prevent the selection of subgroups, which may not be representative of the full disease spectrum? Is there a way forward in personalized medicine? How do we balance risk and benefit in an at-risk population? While many tools are available, a concerted effort is required to develop integrated data sets, as well as to achieve the necessary standardization for multicenter clinical trials. To this end, public-private consortia (including academic centers, patient advocacy groups, and industry) will be of crucial importance to prospectively investigate and define the best tools and treatment paradigms.</div></front></TEI><affiliations><list><country><li>Belgique</li></country></list><tree><noCountry><name sortKey="Alexander, Robert" sort="Alexander, Robert" uniqKey="Alexander R" first="Robert" last="Alexander">Robert Alexander</name><name sortKey="Boroojerdi, Babak" sort="Boroojerdi, Babak" uniqKey="Boroojerdi B" first="Babak" last="Boroojerdi">Babak Boroojerdi</name><name sortKey="Bronzova, Juliana" sort="Bronzova, Juliana" uniqKey="Bronzova J" first="Juliana" last="Bronzova">Juliana Bronzova</name><name sortKey="Fitzer Attas, Cheryl" sort="Fitzer Attas, Cheryl" uniqKey="Fitzer Attas C" first="Cheryl" last="Fitzer-Attas">Cheryl Fitzer-Attas</name><name sortKey="Fontoura, Paulo" sort="Fontoura, Paulo" uniqKey="Fontoura P" first="Paulo" last="Fontoura">Paulo Fontoura</name><name sortKey="Gomez Mancilla, Baltazar" sort="Gomez Mancilla, Baltazar" uniqKey="Gomez Mancilla B" first="Baltazar" last="Gomez-Mancilla">Baltazar Gomez-Mancilla</name><name sortKey="Grachev, Igor D" sort="Grachev, Igor D" uniqKey="Grachev I" first="Igor D" last="Grachev">Igor D. Grachev</name><name sortKey="Ostrowitzki, Susanne" sort="Ostrowitzki, Susanne" uniqKey="Ostrowitzki S" first="Susanne" last="Ostrowitzki">Susanne Ostrowitzki</name><name sortKey="Victor, Stephen J" sort="Victor, Stephen J" uniqKey="Victor S" first="Stephen J" last="Victor">Stephen J. Victor</name></noCountry><country name="Belgique"><noRegion><name sortKey="Streffer, Johannes R" sort="Streffer, Johannes R" uniqKey="Streffer J" first="Johannes R" last="Streffer">Johannes R. Streffer</name></noRegion></country></tree></affiliations></record>Pour manipuler ce document sous Unix (Dilib)
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