Therapeutic potential of nervous system growth factors for neurodegenerative disease

Fifty years after the discovery of nerve growth factor, the first gene therapy trial for the treatment of Alzheimer's disease utilizing nerve growth factor has begun. This review focuses on the history of nerve growth factor and the rationale behind targeting nerve growth factor therapy to rescue degenerating neurons of the cholinergic basal forebrain system in Alzheimer's disease. Further, the future steps necessary to turn the promise of gene therapy into a practical approach for treating Alzheimer's disease and other neurodegenerative disorders are discussed, including key issues, such as regulatable gene expression and making the transition from an ex vivo to an in vivo gene therapy approach.