Nerve growth factor gene therapy: activation of neuronal responses in Alzheimer disease

MH Tuszynski, JH Yang, D Barba, U Hoi-Sang… - JAMA …, 2015 - jamanetwork.com
Importance Alzheimer disease (AD) is the most common neurodegenerative disorder and
lacks effective disease-modifying therapies. In 2001, we initiated a clinical trial of nerve
growth factor (NGF) gene therapy in AD, the first effort at gene delivery in an adult
neurodegenerative disorder. This program aimed to determine whether a nervous system
growth factor prevents or reduces cholinergic neuronal degeneration in patients with AD. We
present postmortem findings in 10 patients with survival times ranging from 1 to 10 years …