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.
Honig LS. Gene Therapy in Alzheimer Disease—It May Be Feasible, but Will It Be Beneficial?. JAMA Neurol. 2018;75(7):791–793. doi:10.1001/jamaneurol.2017.4029
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