Combining cell transplants or gene therapy with deep brain stimulation for Parkinson's disease.
Identifieur interne : 000286 ( Main/Exploration ); précédent : 000285; suivant : 000287Combining cell transplants or gene therapy with deep brain stimulation for Parkinson's disease.
Auteurs : Nathan C. Rowland [États-Unis] ; Philip A. Starr ; Paul S. Larson ; Jill L. Ostrem ; William J. Marks ; Daniel A. LimSource :
- Movement disorders : official journal of the Movement Disorder Society [ 1531-8257 ] ; 2015.
English descriptors
- KwdEn :
- MESH :
- drug effects : Subthalamic Nucleus.
- surgery : Subthalamic Nucleus.
- therapy : Parkinson Disease.
- Animals, Cell Transplantation, Electric Stimulation Therapy, Genetic Therapy, Humans, Treatment Outcome.
Abstract
Cell transplantation and gene therapy each show promise to enhance the treatment of Parkinson's disease (PD). However, because cell transplantation and gene therapy generally require direct delivery to the central nervous system, clinical trial design involves unique scientific, ethical, and financial concerns related to the invasive nature of the procedure. Typically, such biologics have been tested in PD patients who have not received any neurosurgical intervention. Here, we suggest that PD patients undergoing deep brain stimulation (DBS) device implantation are an ideal patient population for the clinical evaluation of cell transplantation and gene therapy. Randomizing subjects to an experimental group that receives the biologic concurrently with the DBS implantation-or to a control group that receives the DBS treatment alone-has several compelling advantages. First, this study design enables the participation of patients likely to benefit from DBS, many of whom simultaneously meet the inclusion criteria of biologic studies. Second, the need for a sham neurosurgical procedure is eliminated, which may reduce ethical concerns, promote patient recruitment, and enhance the blinding of surgical trials. Third, testing the biologic by "piggybacking" onto an established, reimbursable procedure should reduce the cost of clinical trials, which may allow a greater number of biologics to reach this critical stage of research translation. Finally, this clinical trial design may lead to combinatorial treatment strategies that provide PD patients with more durable control over disabling motor symptoms. By combining neuromodulation with biologics, we may also reveal important treatment paradigms relevant to other diseases of the brain.
DOI: 10.1002/mds.26083
PubMed: 25521796
Affiliations:
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Le document en format XML
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Larson</name></author><author><name sortKey="Ostrem, Jill L" sort="Ostrem, Jill L" uniqKey="Ostrem J" first="Jill L" last="Ostrem">Jill L. Ostrem</name></author><author><name sortKey="Marks, William J" sort="Marks, William J" uniqKey="Marks W" first="William J" last="Marks">William J. Marks</name></author><author><name sortKey="Lim, Daniel A" sort="Lim, Daniel A" uniqKey="Lim D" first="Daniel A" last="Lim">Daniel A. 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Rowland</name><affiliation wicri:level="2"><nlm:affiliation>Department of Neurological Surgery, University of California, San Francisco, CA, USA.</nlm:affiliation><country xml:lang="fr">États-Unis</country><wicri:regionArea>Department of Neurological Surgery, University of California, San Francisco, CA</wicri:regionArea><placeName><region type="state">Californie</region></placeName></affiliation></author><author><name sortKey="Starr, Philip A" sort="Starr, Philip A" uniqKey="Starr P" first="Philip A" last="Starr">Philip A. Starr</name></author><author><name sortKey="Larson, Paul S" sort="Larson, Paul S" uniqKey="Larson P" first="Paul S" last="Larson">Paul S. Larson</name></author><author><name sortKey="Ostrem, Jill L" sort="Ostrem, Jill L" uniqKey="Ostrem J" first="Jill L" last="Ostrem">Jill L. Ostrem</name></author><author><name sortKey="Marks, William J" sort="Marks, William J" uniqKey="Marks W" first="William J" last="Marks">William J. Marks</name></author><author><name sortKey="Lim, Daniel A" sort="Lim, Daniel A" uniqKey="Lim D" first="Daniel A" last="Lim">Daniel A. Lim</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="2015" type="published">2015</date></imprint></series></biblStruct></sourceDesc></fileDesc><profileDesc><textClass><keywords scheme="KwdEn" xml:lang="en"><term>Animals</term><term>Cell Transplantation</term><term>Electric Stimulation Therapy</term><term>Genetic Therapy</term><term>Humans</term><term>Parkinson Disease (therapy)</term><term>Subthalamic Nucleus (drug effects)</term><term>Subthalamic Nucleus (surgery)</term><term>Treatment Outcome</term></keywords><keywords scheme="MESH" qualifier="drug effects" xml:lang="en"><term>Subthalamic Nucleus</term></keywords><keywords scheme="MESH" qualifier="surgery" xml:lang="en"><term>Subthalamic Nucleus</term></keywords><keywords scheme="MESH" qualifier="therapy" xml:lang="en"><term>Parkinson Disease</term></keywords><keywords scheme="MESH" xml:lang="en"><term>Animals</term><term>Cell Transplantation</term><term>Electric Stimulation Therapy</term><term>Genetic Therapy</term><term>Humans</term><term>Treatment Outcome</term></keywords></textClass></profileDesc></teiHeader><front><div type="abstract" xml:lang="en">Cell transplantation and gene therapy each show promise to enhance the treatment of Parkinson's disease (PD). However, because cell transplantation and gene therapy generally require direct delivery to the central nervous system, clinical trial design involves unique scientific, ethical, and financial concerns related to the invasive nature of the procedure. Typically, such biologics have been tested in PD patients who have not received any neurosurgical intervention. Here, we suggest that PD patients undergoing deep brain stimulation (DBS) device implantation are an ideal patient population for the clinical evaluation of cell transplantation and gene therapy. Randomizing subjects to an experimental group that receives the biologic concurrently with the DBS implantation-or to a control group that receives the DBS treatment alone-has several compelling advantages. First, this study design enables the participation of patients likely to benefit from DBS, many of whom simultaneously meet the inclusion criteria of biologic studies. Second, the need for a sham neurosurgical procedure is eliminated, which may reduce ethical concerns, promote patient recruitment, and enhance the blinding of surgical trials. Third, testing the biologic by "piggybacking" onto an established, reimbursable procedure should reduce the cost of clinical trials, which may allow a greater number of biologics to reach this critical stage of research translation. Finally, this clinical trial design may lead to combinatorial treatment strategies that provide PD patients with more durable control over disabling motor symptoms. By combining neuromodulation with biologics, we may also reveal important treatment paradigms relevant to other diseases of the brain.</div></front></TEI><affiliations><list><country><li>États-Unis</li></country><region><li>Californie</li></region></list><tree><noCountry><name sortKey="Larson, Paul S" sort="Larson, Paul S" uniqKey="Larson P" first="Paul S" last="Larson">Paul S. Larson</name><name sortKey="Lim, Daniel A" sort="Lim, Daniel A" uniqKey="Lim D" first="Daniel A" last="Lim">Daniel A. Lim</name><name sortKey="Marks, William J" sort="Marks, William J" uniqKey="Marks W" first="William J" last="Marks">William J. Marks</name><name sortKey="Ostrem, Jill L" sort="Ostrem, Jill L" uniqKey="Ostrem J" first="Jill L" last="Ostrem">Jill L. Ostrem</name><name sortKey="Starr, Philip A" sort="Starr, Philip A" uniqKey="Starr P" first="Philip A" last="Starr">Philip A. Starr</name></noCountry><country name="États-Unis"><region name="Californie"><name sortKey="Rowland, Nathan C" sort="Rowland, Nathan C" uniqKey="Rowland N" first="Nathan C" last="Rowland">Nathan C. Rowland</name></region></country></tree></affiliations></record>Pour manipuler ce document sous Unix (Dilib)
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