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Tetrahydrobiopterin as a treatment for autism spectrum disorders: a double-blind, placebo-controlled trial

Cheryl Klaiman1, Lynne Huffman, Lauren Masaki

  • 1Marcus Autism Center, Children's Healthcare of Atlanta and Department of Pediatrics, Emory University School of Medicine, Atlanta, Georgia, USA. cheryl.klaiman@emory.edu

Journal of Child and Adolescent Psychopharmacology
|June 21, 2013
PubMed

Insights

Tetrahydrobiopterin (BH4) showed promise in reducing core symptoms of autism spectrum disorder (ASD) in children. While global measures showed no change, BH4 improved social awareness, mannerisms, and hyperactivity.

Area of Science:

  • Neuroscience
  • Developmental Pediatrics
  • Pharmacology

Background:

  • Autism spectrum disorder (ASD) is a complex neurodevelopmental condition.
  • Core symptoms of ASD include social deficits, repetitive behaviors, and communication challenges.
  • Investigating novel therapeutic targets for ASD is crucial.

Purpose of the Study:

  • To evaluate the efficacy of tetrahydrobiopterin (BH4) in ameliorating core symptoms of autism spectrum disorder (ASD).
  • To assess the impact of BH4 on global functioning and specific behavioral domains in children with ASD.

Main Methods:

  • A randomized, double-blind, placebo-controlled trial involving 46 children aged 3-7 years with ASD.
  • Participants received either 20 mg/kg/day of BH4 or a placebo for 16 weeks.
  • Outcomes were assessed using Clinical Global Impressions (CGI-I, CGI-S), Preschool Language Scale-4 (PLS-4), Social Responsiveness Scale (SRS), Aberrant Behavior Checklist (ABC), and Vineland Adaptive Behavior Scales (Vineland).

Main Results:

  • No significant differences were observed between BH4 and placebo groups on the primary outcome measures (CGI-I and CGI-S).
  • Secondary analyses revealed statistically significant improvements in social awareness, autism mannerisms, hyperactivity, and inappropriate speech in the BH4 group compared to placebo.
  • Adverse events were infrequent and comparable between the BH4 and placebo groups.

Conclusions:

  • Tetrahydrobiopterin (BH4) demonstrates potential as a therapeutic agent for reducing specific symptoms associated with autism spectrum disorder.
  • Further research is warranted to confirm these findings and explore the underlying mechanisms of BH4 action in ASD.
  • ClinicalTrials.gov Identifier: NCT00850070.
Abstract

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