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Nat Med. 2005 May;11(5):551-5. Epub 2005 Apr 24.

A phase 1 clinical trial of nerve growth factor gene therapy for Alzheimer disease.

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  • 1Department of Neurosciences, University of California at San Diego, La Jolla 92093, USA. mtuszynski@ucsd.edu <mtuszynski@ucsd.edu>

Abstract

Cholinergic neuron loss is a cardinal feature of Alzheimer disease. Nerve growth factor (NGF) stimulates cholinergic function, improves memory and prevents cholinergic degeneration in animal models of injury, amyloid overexpression and aging. We performed a phase 1 trial of ex vivo NGF gene delivery in eight individuals with mild Alzheimer disease, implanting autologous fibroblasts genetically modified to express human NGF into the forebrain. After mean follow-up of 22 months in six subjects, no long-term adverse effects of NGF occurred. Evaluation of the Mini-Mental Status Examination and Alzheimer Disease Assessment Scale-Cognitive subcomponent suggested improvement in the rate of cognitive decline. Serial PET scans showed significant (P < 0.05) increases in cortical 18-fluorodeoxyglucose after treatment. Brain autopsy from one subject suggested robust growth responses to NGF. Additional clinical trials of NGF for Alzheimer disease are warranted.

PMID:
15852017
[PubMed - indexed for MEDLINE]
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