Hypothesis-driven investigations of diverse pharmacological targets in two mouse models of autism.
Maya A Rhine, Jennifer M Parrott, Maria N Schultz and 2 others
PMID 30653853WHAT IT FOUND
In mice, gaboxadol reduced repetitive grooming without lowering activity, while other drugs had mixed effects.
No human patients were tested.
Key findings
01Gaboxadol reduced repetitive self-grooming in BTBR mice in replicated cohorts without lowering general activity.
02The TrkB agonist restored sociability in BTBR mice on a social approach assay but did not improve male-female social interaction.
03High-dose d-cycloserine increased social contact in Shank mutant mice, but hyperlocomotion appeared responsible.
STILL TO COME
How it was doneWhat they found
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What it does not show
The study was done in mice, not people, so it does not show what would happen in a clinic. The outcomes were mouse behaviors such as grooming and sniffing, not symptoms in people. Some effects appeared in a cohort but not in another, so the findings need replication. Drug effects on social or learning tasks were sometimes explained by increased movement or activity changes. The BTBR mice unexpectedly showed normal sociability in some experiments, which makes interpretation harder. The paper did not state the total number of mice.
Declared interests
The authors declared no competing interests. No industry involvement or funding supported these studies. The work was supported by NIH extramural funding.
The easy way to misread this
Do not read this as evidence that a drug can improve autism symptoms in patients. The study tested mice and behavioral outcomes, not people in therapy.