The use of difluoromethylornithine (DFMO) as a treatment of neurodegenerative disorders

Data from our mouse model of Alzheimer's disease and from humans with AD indicate a disruption in the immune mediated regulation of arginine and ornithine in the brain. Two primary enzyme systems, arginine utilization via arginase and ornithine utilization to form polyamines are key factors in the disease process. Difluoromethylornithine (DFMO) blocks the enzyme activity of ornithine decarboxylase (ODC)) in a primary fashion and arginase (AG) in a secondary fashion to block abnormal polyamine formation and restore metabolic balance in the brain. When used in a mouse model of Alzheimer's disease, DFMO alone compared to vehicle (water) significantly reverses the behavioral and pathological features of AD in this model.

Duke File (IDF) Number

T-005042

Inventor(s)

  • Colton, Carol

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College

School of Medicine (SOM)