New promise for a treatment for fragile X? Michalon et al. report promising results for treating a mouse model of fragileX syndrom (FXS) with a long-lasting mGlur5 inhibitor, CTEP. FXS is due to repeated genetic elements that slience the FXMR protein, causing global elevations in translation and throghout the brain and other tissues. Despite these widespread effects, many of the neural effects of FXS can be mimicked/blocked by activaiton/deactivation of mGLURs. Several mGLUR antagonists are currently in clinical trials. CTEP is showing remarkable promise, though, as it seems to be able to reverse symptoms of FXS in a mouse model even relatively late in development. Mice also seem to tolerate it well (less weight gain, lower grip strength) None of the other drugs in the development pipeline are expected to work so late in development. Will it pan out? From bench to clinic for a developmental disorder? mGLUR only? I wouldn't have bet on it, but.. (Neuron's preview also added).
Michalon, A., Sidorov, M., Ballard, T. M., Ozmen, L., Spooren, W., Wettstein, J. G., Jaeschke, G., et al. (2012). Chronic Pharmacological mGlu5 Inhibition Corrects Fragile X in Adult Mice. Neuron, 74(1), 49-56. Elsevier Inc. doi:10.1016/j.neuron.2012.03.009. http://linkinghub.elsevier.com/retrieve/pii/S0896627312002723
Neuron's preview: Bhattacharya, A., & Klann, E. (2012). Fragile X Syndrome Therapeutics S(C)TEP through the Developmental Window. Neuron, 74(1), 1-3. Elsevier Inc. doi:10.1016/j.neuron.2012.03.014. http://linkinghub.elsevier.com/retrieve/pii/S0896627312002772
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