There are, therefore, the conditions for studying the effects of drugs acting on the mTOR system targeting autistic symptoms by themselves, also because rapamycin (acting as a selective mTOR inhibitor) has produced encouraging results on animals regardless of the presence of cortical tubers and epilepsy.[ 4 ] Mizuguchi et al .[ 5 ] carried out a randomized, double-blind, placebo-controlled trial, finding a favorable trend in autistic symptoms in a sample of children with tuberous sclerosis treated with everolimus (another selective mTOR inhibitor), but this trend was not supported by a statistical analysis of the data.[ 5 ] Overwater et al .[ 6 ] carried out another randomized, double-blind, placebo-controlled trial, finding no favorable cognitive or behavioral effects of everolimus in a sample of children with tuberous sclerosis.
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