Critical Aspects of Clinical Trial Design for Novel Cell and Gene Therapies
Joint Authors
Foltynie, Thomas
Aviles-Olmos, Iciar
Kefalopoulou, Zinovia
Source
Evidence-Based Complementary and Alternative Medicine
Issue
Vol. 2011, Issue 2011 (31 Dec. 2011), pp.1-10, 10 p.
Publisher
Hindawi Publishing Corporation
Publication Date
2011-12-27
Country of Publication
Egypt
No. of Pages
10
Main Subjects
Abstract EN
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).
American Psychological Association (APA)
Kefalopoulou, Zinovia& Aviles-Olmos, Iciar& Foltynie, Thomas. 2011. Critical Aspects of Clinical Trial Design for Novel Cell and Gene Therapies. Evidence-Based Complementary and Alternative Medicine،Vol. 2011, no. 2011, pp.1-10.
https://search.emarefa.net/detail/BIM-499352
Modern Language Association (MLA)
Kefalopoulou, Zinovia…[et al.]. Critical Aspects of Clinical Trial Design for Novel Cell and Gene Therapies. Evidence-Based Complementary and Alternative Medicine No. 2011 (2011), pp.1-10.
https://search.emarefa.net/detail/BIM-499352
American Medical Association (AMA)
Kefalopoulou, Zinovia& Aviles-Olmos, Iciar& Foltynie, Thomas. Critical Aspects of Clinical Trial Design for Novel Cell and Gene Therapies. Evidence-Based Complementary and Alternative Medicine. 2011. Vol. 2011, no. 2011, pp.1-10.
https://search.emarefa.net/detail/BIM-499352
Data Type
Journal Articles
Language
English
Notes
Includes bibliographical references
Record ID
BIM-499352