In this issue of JAMA Neurology, Rafii et al1 report the results of a randomized clinical trial of intracerebral gene delivery in patients with Alzheimer disease (AD). Neurodegenerative diseases, of which AD is the most common, remain a challenging frontier for medical treatment. There are no preventive measures, and for the millions of individuals who already have AD, there are no disease-modifying agents to slow, stop, or reverse progression. The primary efforts of the field include a diverse assortment of ongoing drug trials attempting to alleviate or reduce various AD pathophysiologic processes: amyloidogenesis, neurofibrillary change, synaptic losses, and neuronal loss.2 While these efforts may be getting closer to success, they have not yet reached fruition.