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Histone Deacetylase Inhibitors - A Possible Treatment for HD
report by Lisa J. Bain
Abstract:
At a workshop held in August 2001 at the Memorial Sloan Kettering Cancer Center, more than 20 scientists from cancer and HD research fields met to discuss the potential for conducting clinical trials of histone deacetylase (HDAC) inhibitors in patients with HD. Paul Marks' group at MSKCC presented data regarding the evolution of a class of these agents called hybrid polar compounds, their mechanistic and structural properties, and results from both preclinical and clinical trials. These studies indicate that the agents inhibit tumor growth and have low toxicity. HD researchers presented data from studies investigating the role of HDACs in perturbations of gene expression, seen in models of polyglutamine disease. In two Drosophila models and a yeast model, HDAC inhibitors appear to interfere with the neurodegenerative process. Workshop participants urged additional studies in other HD animal models in preparation for human clinical trials. In order to begin human trials of HDAC inhibitors in HD patients, researchers will need to define the optimal study population and identify appropriate clinical and biological endpoints.
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