A forward look at therapy for pediatric movement disorders.
The mainstay of current therapy for pediatric movement disorders is oral symptomatic medication, unless a reversible etiology can be found. However, this approach is apt to pale in comparison with innovative strategies on the clinical forefront. Classical pharmacotherapy is restricted by the blood-brain barrier, which prevents access to the brain of potentially therapeutic molecules. Recent developments in molecular biotechnology include antibody-mediated drug release, feedback-responsive delivery systems, carrier-mediated transport, microspheres composed of polymers and liposomes, permeabilizers, and selective delivery to localized sites and vectors. Neuroprotective strategies for delivering neurotrophic factors and antiapoptotic and antioxidant molecules in neurodegenerative disorders are currently under study in clinical trials. Stem cell transplantation has great potential for tissue engineering and also as a carrier for gene therapy, although its use raises complex societal issues. These approaches, together with a plethora of transgenic knockout animal models of neurodegenerative disorders, offer real promise for a previously untreatable group of movement disorders.
Pranzatelli
Michael R
MR
Department of Neurology, Southern Illinois University School of Medicine, Springfield, IL 62702, USA.
D000818:Animals; D030801:Animals, Genetically Modified; D002648:Child; D006801:Humans; D009069:Movement Disorders / Q000628:therapy*; D010372:Pediatrics