Critical Aspects of Clinical Trial Design for Novel Cell and Gene Therapies
Neural cell transplantation and gene therapy have attracted considerable interest as promising therapeutic alternatives for patients with Parkinson's disease (PD). Preclinical and open-label studies have suggested that grafted fetal neural tissue or viral vector gene transfer can achieve consid...
Saved in:
| Main Authors: | , , |
|---|---|
| Format: | Article |
| Language: | English |
| Published: |
Wiley
2011-01-01
|
| Series: | Parkinson's Disease |
| Online Access: | http://dx.doi.org/10.4061/2011/804041 |
| Tags: |
Add Tag
No Tags, Be the first to tag this record!
|
| _version_ | 1832567979623055360 |
|---|---|
| author | Zinovia Kefalopoulou Iciar Aviles-Olmos Thomas Foltynie |
| author_facet | Zinovia Kefalopoulou Iciar Aviles-Olmos Thomas Foltynie |
| author_sort | Zinovia Kefalopoulou |
| collection | DOAJ |
| description | Neural cell transplantation and gene therapy have attracted considerable interest as promising therapeutic alternatives for patients with Parkinson's disease (PD). Preclinical and open-label studies have suggested that grafted fetal neural tissue or viral vector gene transfer can achieve considerable biochemical and clinical improvements, whereas subsequent double-blind, placebo-controlled protocols have produced rather more modest and variable results. Detailed evaluation of these discordant findings has highlighted several crucial issues such as patient selection criteria, details surrounding transplantation or gene therapy methodologies, as well as the study designs themselves that ought to be carefully considered in the planning phases of future clinical trials. Beyond the provision of symptomatic efficacy and safety data, it also remains to be identified whether the possibilities offered by stem cell and gene therapy technological advances might translate to meaningful neuroprotection and/or disease-modifying effects or alleviate the nonmotor aspects of PD and thus offer additional benefits beyond those achieved through conventional pharmacotherapy or deep brain stimulation (DBS). |
| format | Article |
| id | doaj-art-f71972d540294152b0fd4ed7163b2042 |
| institution | Kabale University |
| issn | 2042-0080 |
| language | English |
| publishDate | 2011-01-01 |
| publisher | Wiley |
| record_format | Article |
| series | Parkinson's Disease |
| spelling | doaj-art-f71972d540294152b0fd4ed7163b20422025-02-03T01:00:09ZengWileyParkinson's Disease2042-00802011-01-01201110.4061/2011/804041804041Critical Aspects of Clinical Trial Design for Novel Cell and Gene TherapiesZinovia Kefalopoulou0Iciar Aviles-Olmos1Thomas Foltynie2Sobell Department of Motor Neuroscience and Movement Disorders, UCL Institute of Neurology, Queen Square, London WC1N 3BG, UKSobell Department of Motor Neuroscience and Movement Disorders, UCL Institute of Neurology, Queen Square, London WC1N 3BG, UKSobell Department of Motor Neuroscience and Movement Disorders, UCL Institute of Neurology, Queen Square, London WC1N 3BG, UKNeural cell transplantation and gene therapy have attracted considerable interest as promising therapeutic alternatives for patients with Parkinson's disease (PD). Preclinical and open-label studies have suggested that grafted fetal neural tissue or viral vector gene transfer can achieve considerable biochemical and clinical improvements, whereas subsequent double-blind, placebo-controlled protocols have produced rather more modest and variable results. Detailed evaluation of these discordant findings has highlighted several crucial issues such as patient selection criteria, details surrounding transplantation or gene therapy methodologies, as well as the study designs themselves that ought to be carefully considered in the planning phases of future clinical trials. Beyond the provision of symptomatic efficacy and safety data, it also remains to be identified whether the possibilities offered by stem cell and gene therapy technological advances might translate to meaningful neuroprotection and/or disease-modifying effects or alleviate the nonmotor aspects of PD and thus offer additional benefits beyond those achieved through conventional pharmacotherapy or deep brain stimulation (DBS).http://dx.doi.org/10.4061/2011/804041 |
| spellingShingle | Zinovia Kefalopoulou Iciar Aviles-Olmos Thomas Foltynie Critical Aspects of Clinical Trial Design for Novel Cell and Gene Therapies Parkinson's Disease |
| title | Critical Aspects of Clinical Trial Design for Novel Cell and Gene Therapies |
| title_full | Critical Aspects of Clinical Trial Design for Novel Cell and Gene Therapies |
| title_fullStr | Critical Aspects of Clinical Trial Design for Novel Cell and Gene Therapies |
| title_full_unstemmed | Critical Aspects of Clinical Trial Design for Novel Cell and Gene Therapies |
| title_short | Critical Aspects of Clinical Trial Design for Novel Cell and Gene Therapies |
| title_sort | critical aspects of clinical trial design for novel cell and gene therapies |
| url | http://dx.doi.org/10.4061/2011/804041 |
| work_keys_str_mv | AT zinoviakefalopoulou criticalaspectsofclinicaltrialdesignfornovelcellandgenetherapies AT iciaravilesolmos criticalaspectsofclinicaltrialdesignfornovelcellandgenetherapies AT thomasfoltynie criticalaspectsofclinicaltrialdesignfornovelcellandgenetherapies |