Tauroursodeoxycholic acid, a bile acid, is neuroprotective in a transgenic animal model of Huntington's disease

CD Keene, CMP Rodrigues, T Eich… - Proceedings of the …, 2002 - National Acad Sciences
CD Keene, CMP Rodrigues, T Eich, MS Chhabra, CJ Steer, WC Low
Proceedings of the National Academy of Sciences, 2002National Acad Sciences
Huntington's disease (HD) is an untreatable neurological disorder caused by selective and
progressive degeneration of the caudate nucleus and putamen of the basal ganglia.
Although the etiology of HD pathology is not fully understood, the observed loss of neuronal
cells is thought to occur primarily through apoptosis. Furthermore, there is evidence in HD
that cell death is mediated through mitochondrial pathways, and mitochondrial deficits are
commonly associated with HD. We have previously reported that treatment with …
Huntington's disease (HD) is an untreatable neurological disorder caused by selective and progressive degeneration of the caudate nucleus and putamen of the basal ganglia. Although the etiology of HD pathology is not fully understood, the observed loss of neuronal cells is thought to occur primarily through apoptosis. Furthermore, there is evidence in HD that cell death is mediated through mitochondrial pathways, and mitochondrial deficits are commonly associated with HD. We have previously reported that treatment with tauroursodeoxycholic acid (TUDCA), a hydrophilic bile acid, prevented neuropathology and associated behavioral deficits in the 3-nitropropionic acid rat model of HD. We therefore examined whether TUDCA would also be neuroprotective in a genetic mouse model of HD. Our results showed that systemically administered TUDCA led to a significant reduction in striatal neuropathology of the R6/2 transgenic HD mouse. Specifically, R6/2 mice began receiving TUDCA at 6 weeks of age and exhibited reduced striatal atrophy, decreased striatal apoptosis, as well as fewer and smaller size ubiquitinated neuronal intranuclear huntingtin inclusions. Moreover, locomotor and sensorimotor deficits were significantly improved in the TUDCA-treated mice. In conclusion, TUDCA is a nontoxic, endogenously produced hydrophilic bile acid that is neuroprotective in a transgenic mouse model of HD and, therefore, may provide a novel and effective treatment in patients with HD.
National Acad Sciences