080210xf's Blog

L'X fragile sera vaincu | Fragile X will be conquered

Mouse study raises questions about fragile X treatment

Deborah Rudacille, Sfari.org |

Blocking a chemical messenger — a much-touted approach to treating fragile X syndrome — is unlikely to completely reverse symptoms of the disorder, according to a provocative new study1.

Multiple drug candidates in clinical trials to treat fragile X syndrome are based on blocking group 1 metabotropic glutamate receptors (mGluR), a set of chemical messenger receptors involved in cell signaling. Animal studies, including an influential 2007 report2, have shown that reining in the runaway signaling by these receptors decreases seizures and other manifestations of over-excitation of the brain.

In the new study, published online in May in Behavioural Brain Research, researchers cut expression of mGluR1 and mGluR5 by half using a genetic approach rather than chemical inhibitors. The 2007 study used the same approach, but this time, the researchers saw an increase in seizures and only modest behavioral improvements.

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