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Updated: Jul 8, 2025
Testing Sensory and Multisensory Function in Children with Autism Spectrum Disorder
Published on: April 22, 2015
Brief Report: A Double-Blind, Placebo-Controlled, Crossover, Proof-of-Concept Study of Minocycline in Autism Spectrum
Craig A Erickson1,2, Rebecca C Shaffer3,4, Meredith Will3,4
1Division of Child and Adolescent Psychiatry, Cincinnati Children's Hospital Medical Center, 3333 Burnet Avenue MLC 4002, Cincinnati, OH, 45229, USA. craig.erickson@cchmc.org.
Abstract:
Neuroinflammatory mechanisms have been implicated in the pathophysiology of autism spectrum disorder (ASD). Minocycline is a matrix metalloproteinase inhibitor 9 (MMP9) inhibitor tetracycline antibiotic with known anti-inflammatory properties. In preclinical animal models of ASD, minocycline has demonstrated potential positive effects on phenotypes that may have relevance to ASD. We conducted the first placebo-controlled study of minocycline in ASD. This double-blind, placebo-controlled crossover trial employed four week treatment periods with a two week washout period. Twenty-four 12-22 year olds (mean age 17.4 years; range 12.9-22.5 years) with ASD were enrolled. Overall minocycline was well tolerated. No minocycline-associated clinical changes were noted with treatment on any performance or clinician or caregiver completed measures were noted. We hypothesize that either minocycline does not have potential therapeutic effects in ASD or our project was underpowered to define potential subject subgroups who may potentially respond positively to this drug.
Insights
Minocycline, an anti-inflammatory drug, showed no significant clinical benefits in adolescents with autism spectrum disorder (ASD) in a placebo-controlled trial. Further research may be needed to identify potential subgroups who could benefit from this autism treatment.
Area of Science:
- Neuroscience
- Psychiatry
- Pharmacology
Background:
- Neuroinflammation is increasingly recognized as a contributing factor in the development of autism spectrum disorder (ASD).
- Minocycline, a tetracycline antibiotic, possesses anti-inflammatory properties and has shown promise in preclinical models relevant to ASD.
- Previous research suggests minocycline's potential to modulate matrix metalloproteinase 9 (MMP9), an enzyme implicated in neuroinflammation.
Purpose of the Study:
- To investigate the efficacy of minocycline as a therapeutic intervention for autism spectrum disorder (ASD) in adolescents.
- To conduct the first placebo-controlled trial evaluating minocycline's effects on ASD phenotypes.
- To assess the safety and tolerability of minocycline in this population.
Main Methods:
- A double-blind, placebo-controlled crossover trial was conducted.
- Twenty-four adolescents aged 12-22 years with ASD were enrolled.
- Participants received either minocycline or placebo for four-week periods, separated by a two-week washout phase.
Main Results:
- Minocycline was generally well-tolerated by the participants.
- No significant clinical improvements were observed across performance, clinician, or caregiver-reported measures during minocycline treatment.
- The study did not identify any specific subgroups that responded positively to minocycline.
Conclusions:
- Minocycline did not demonstrate therapeutic efficacy in this adolescent ASD cohort.
- The study may have been underpowered to detect subtle treatment effects or identify responsive subgroups.
- Future research should explore alternative therapeutic strategies or refined patient stratification for minocycline in ASD.
