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Sodium phenylbutyrate in Huntington's disease: A dose-finding study

✍ Scribed by Penelope Hogarth; Luca Lovrecic; Dimitri Krainc

Publisher
John Wiley and Sons
Year
2007
Tongue
English
Weight
57 KB
Volume
22
Category
Article
ISSN
0885-3185

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✦ Synopsis

Abstract

Transcriptional dysregulation in Huntington's disease (HD) is mediated in part by aberrant patterns of histone acetylation. We performed a dose‐finding study in human HD of sodium phenylbutyrate (SPB), a histone deacetylase inhibitor that ameliorates the HD phenotype in animal models. We used a dose‐escalation/de‐escalation design, using prespecified toxicity criteria and standard clinical and laboratory safety measures. The maximum tolerated dose was 15 g/day. At higher doses, toxicity included vomiting, lightheadedness, confusion, and gait instability. We saw no significant laboratory or electrocardiographic abnormalities. Gene expression changes in blood suggested an inverse dose‐response. In conclusion, SPB at 12 to 15 g/day appears to be safe and well‐tolerated in human HD. © 2007 Movement Disorder Society

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