Investigators at the RIKEN Brain Science Institute in Saitama, Japan have published encouraging results in this month's Nature Medicine. Studies involving both cellular and murine models of HD demonstrated a decrease in protein aggregation following treatment with a simple sugar called trehalose. Trehalose is nontoxic and can be administered orally, two characteristics that make it particularly intriguing to HD investigators. Huntington's Disease is caused by an extension of a glutamine region in the huntingtin protein.
This insertion destabilizes the protein and enables it to aggregate with other mutant huntingtins, forming clumps within an HD cell's cytoplasm. Investigators hypothesize that trehalose stabilizes mutant huntingtin and thereby prevents it from aggregating. Trehalose was added to water given to transgenic HD mice. Decreases in protein aggregation were observed in both liver and brain cells. Additionally, trehalose-treated mice demonstrated improvements in motor dysfunction and experienced extended life spans.
The results of this study are quite encouraging, although preliminary. Further studies in both mice and humans are planned to clarify the benefits of trehalose and to determine its effectiveness, dose, and safety for HD patients. Additionally, investigators are searching for another compound similar in structure but more effective so as to maximize the benefits of this form of treatment.
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Created: Aug. 11, 2004
Last updated: Nov. 14, 2010