critical aspects of clinical trial design for novel cell and gene therapies

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ID: 233013
2011
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Abstract
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).
Reference Key
kefalopoulou2011parkinson'scritical Use this key to autocite in the manuscript while using SciMatic Manuscript Manager or Thesis Manager
Authors ;Zinovia Kefalopoulou;Iciar Aviles-Olmos;Thomas Foltynie
Journal heat and mass transfer/waerme- und stoffuebertragung
Year 2011
DOI
10.4061/2011/804041
URL
http://dx.doi.org/10.4061/2011/804041
Keywords
neurology diseases of the nervous system

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