In June 2020, cannabidiol hit what has become a familiar hurdle in fragile X research: Like many previous drug candidates, it missed its primary target in clinical testing. Among 109 young people with fragile X syndrome who took the drug and 101 who took a placebo, researchers saw no meaningful difference in a rating of social avoidance.A secondary analysis factored into the trial's original design offered a glimmer of hope, though. A subset of 91 participants showed marked improvement on the same measure after treatment with the drug, according to unpublished findings. Among that group, the gene underlying fragile X syndrome, called FMR1, is awash with methyl groups, which block its expression of the protein FMRP. By contrast, the remaining participants have only partially methylated copies of the gene and may make more FMRP."Looking at methylation was pretty important to find that subgroup that had the most optimal response," says Randi Hagerman, medical director of the MIND Institute at the University of California, Davis, who led one of the trial sites.
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