Research ArticleNIEMANN-PICK TYPE C DISEASE

Chronic administration of an HDAC inhibitor treats both neurological and systemic Niemann-Pick type C disease in a mouse model

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Science Translational Medicine  17 Feb 2016:
Vol. 8, Issue 326, pp. 326ra23
DOI: 10.1126/scitranslmed.aad9407

Crossing the Rubicon

Histone deacetylase inhibitors (HDACi) are a class of compounds that have shown promise for treating certain types of cancer. Because HDACi do not cross the blood-brain barrier (BBB), they cannot enter the brain and therefore are of limited value for treating neurological diseases. Now, Alam et al. have developed a new formulation that enables the FDA-approved HDACi vorinostat to cross the BBB and enter the brain tissue of mice with Niemann-Pick type C disease. The authors show that long-term treatment with the formulation improves brain function and prolongs mouse survival, suggesting that this new HDACi formulation will be of benefit for treating Niemann-Pick type C disease and perhaps other types of neurological diseases as well.

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