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- Title
A phase 1 clinical trial of nerve growth factor gene therapy for Alzheimer disease.
- Authors
Tuszynski, Mark H; Thal, Leon; Pay, Mary; Salmon, David P; U, Hoi Sang; Bakay, Roy; Patel, Piyush; Blesch, Armin; Vahlsing, H Lee; Ho, Gilbert; Tong, Gang; Potkin, Steven G; Fallon, James; Hansen, Lawrence; Mufson, Elliott J; Kordower, Jeffrey H; Gall, Christine; Conner, James
- 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.
- Publication
Nature medicine, 2005, Vol 11, Issue 5, p551
- ISSN
1078-8956
- Publication type
Journal Article
- DOI
10.1038/nm1239