An article in the early edition (December 12, 2011) of the Proceedings of the National Academy of Sciences USA, reported that Lixte's investigational drug, LB-205, was shown to have therapeutic potential in a laboratory model of Gaucher disease (http://www.pnas.org/content/early/2011/12/08/1119181109.full.pdf+html).
John Kovach, MD, founder and president of Lixte said, "This is a novel use of the class of drugs known as histone deacetylase inhibitors (HDACi's). HDACi's have been used clinically primarily as an anticancer treatments. To our knowledge, this is the first time HDACi's have been shown to have therapeutic potential in a model of Gaucher disease. We were pleased that the effectiveness of LB-205 compared favorably with a clinically approved HDACi. Primary treatment for many Gaucher disease patients is injection of the enzyme, glucocerebrosidase, which decreases the toxic accumulation of glucocerebroside in many tissues. Unfortunately, in some patients, the brain is significantly affected by glucocerebroside accumulation and the injected enzyme does enter the brain readily. Although the effectiveness of HDACi's for Gaucher treatment remains to be proven in clinical studies, the discovery is intriguing because some HDACi's, like LB-205, penetrate the brain and may be effective given by mouth rather than injection, simplifying long term management."
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