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Fragile X syndrome

Ras; MAP kinase 3 (MAPK3; ERK-1); MAPK1 (ERK-2)

Studies in mice suggest lovastatin could help treat epilepsy in patients with fragile X syndrome. In hippocampus slices from a mouse model for fragile X syndrome, lovastatin decreased Ras-ERK-1/ERK-2 pathway signaling, abnormally high protein synthesis and epilepsy-associated activity bursts compared with vehicle. Lovastatin also decreased seizures. Next steps include dose ranging studies and developmental timing studies to identify a therapeutic window for statin treatment in patients with fragile X syndrome.
The generic lovastatin is an HMG-CoA reductase inhibitor marketed to treat dyslipidemia and coronary artery disease (CAD).
Seaside Therapeutics Inc.'s arbaclofen (STX-209), a GABAB receptor antagonist, is in Phase III testing for fragile X syndrome.
Novartis AG's AFQ056, a metabotropic glutamate receptor subtype 5 (mGluR5; GRM5) antagonist, is in Phase III trials for fragile X syndrome (see Statins for fragile X, page 4).

SciBX 6(6); doi:10.1038/scibx.2013.145
Published online Feb. 14, 2013

Patent pending; available for licensing

Osterweil, E.K. et al. Neuron; published online Jan. 23, 2013;
Contact: Mark F. Bear, Massachusetts Institute of Technology,
Cambridge, Mass.