“Administering a cholesterol drug alongside an antibiotic eases atypical behavior and restores the signaling balance in the brains of people with fragile X syndrome.

Researchers presented the preliminary results yesterday at the 2018 Society for Neuroscience annual meeting in San Diego, California.

In the wake of failed clinical trials for fragile X syndrome drugs, researchers are increasingly turning to new approaches that combine treatments targeting different biological pathways.

“It’s interesting to combine treatments because we don’t have just one signaling pathway that is defective in fragile X,” says Amal Loudghi, a graduate student in Jean-François Lepage’s lab at the University of Sherbrooke in Quebec, Canada, who presented some of the results.

The new trial, dubbed LovaMiX, combines lovastatin, which treats high cholesterol, with the antibiotic minocycline. Both drugs have proven promising in small trials and animal studies of fragile X syndrome. Minocycline dampens anxiety and eases the severity of traits in people with the syndrome. And lovastatin relieves seizures in fragile X mice.

The researchers gave 23 people with fragile X syndrome, aged 13 to 40 years, either lovastatin or minocycline for eight weeks — first a low dose and then a higher one; roughly half of the participants received lovastatin first, and the other half minocycline first. The team then gave the participants daily doses of both lovastatin and minocycline for 12 weeks.”

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