Evidence map›Paper›PMID 25705365›Full record

ArticleMolecular autism2015

Antipurinergic therapy corrects the autism-like features in the Fragile X (Fmr1 knockout) mouse model.

Jane C Naviaux, Lin Wang, Kefeng Li, A Taylor Bright, William A Alaynick, Kenneth R Williams, Susan B Powell, Robert K Naviaux

2 registry-linked trialsOpen access · goldAbstract read
In one paragraph

Article in Molecular autism, 2015. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to 2 registered trials, which are not on this map. Cited by 52 papers.

0numbers the graph read from it
0cells of the map it votes in
52citing papers in PubMed
22.4field-weighted citation impact, top 1% of its field
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

NCT02508259 phase1 / phase2completednot on this map

The UCSD Suramin Autism Treatment-1 (SAT1) Trial

TypeinterventionalSponsorUniversity of California, San DiegoRan2015 to 2016Enrolled10ConditionsAutism Spectrum DisordersArmsSuramin, Saline
NCT06866275 phase2recruitingnot on this mapstarted 2025, after this paper: background citation

Suramin for the Treatment of Autism Trial (STAT): A Randomized, Double Blind, Crossover Trial of KZ101 in a Male Pediatric Population With Autism Spectrum Disorder

TypeinterventionalSponsorChildren's Hospital of Orange CountyRan2025 to 2028Enrolled45ConditionsAutism Spectrum Disorder (ASD)ArmsKZ101, Placebo
3 · Its place in the literature

Who cites it

52 citing papers in PubMed, 281 citations in OpenAlex.

  1. Trial
  2. Article
  3. Novel therapeutics in autism spectrum disorder.Neurotherapeutics : the journal of the American Society for Experimental NeuroTherapeutics · 2026
    Review
  4. Article
  5. Review
  6. Review
  7. Article
  8. Article
  9. Article
  10. Article
  11. Article
  12. Review
  13. Article
  14. Article
  15. Review
  16. Mouse models of fragile X-related disorders.Disease models & mechanisms · 2023
    Review
  17. Article
  18. Article
  19. Article
  20. Article
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

8 authors at 3 institutions in 1 country.

Jane C NaviauxDepartment of Psychiatry, University of California, San Diego School of Medicine, 214 Dickinson St., Bldg CTF, Rm C102, San Diego, CA 92103-8467 USA.
Lin WangThe Mitochondrial and Metabolic Disease Center, University of California, San Diego School of Medicine, 214 Dickinson St., Bldg CTF, Rm C102, San Diego, CA 92103-8467 USA ; Department of Medicine, University of California, San Diego School of Medicine, 214 Dickinson St., Bldg CTF, Rm C102, San Diego, CA 92103-8467 USA.
Kefeng LiThe Mitochondrial and Metabolic Disease Center, University of California, San Diego School of Medicine, 214 Dickinson St., Bldg CTF, Rm C102, San Diego, CA 92103-8467 USA ; Department of Medicine, University of California, San Diego School of Medicine, 214 Dickinson St., Bldg CTF, Rm C102, San Diego, CA 92103-8467 USA.
A Taylor BrightThe Mitochondrial and Metabolic Disease Center, University of California, San Diego School of Medicine, 214 Dickinson St., Bldg CTF, Rm C102, San Diego, CA 92103-8467 USA ; Department of Medicine, University of California, San Diego School of Medicine, 214 Dickinson St., Bldg CTF, Rm C102, San Diego, CA 92103-8467 USA.
William A AlaynickThe Mitochondrial and Metabolic Disease Center, University of California, San Diego School of Medicine, 214 Dickinson St., Bldg CTF, Rm C102, San Diego, CA 92103-8467 USA ; Department of Medicine, University of California, San Diego School of Medicine, 214 Dickinson St., Bldg CTF, Rm C102, San Diego, CA 92103-8467 USA.
Kenneth R WilliamsThe Mitochondrial and Metabolic Disease Center, University of California, San Diego School of Medicine, 214 Dickinson St., Bldg CTF, Rm C102, San Diego, CA 92103-8467 USA ; Department of Medicine, University of California, San Diego School of Medicine, 214 Dickinson St., Bldg CTF, Rm C102, San Diego, CA 92103-8467 USA ; General Atomics, Inc, San Diego, CA USA.
Susan B PowellDepartment of Psychiatry, University of California, San Diego School of Medicine, 214 Dickinson St., Bldg CTF, Rm C102, San Diego, CA 92103-8467 USA ; Research Service, VA San Diego Healthcare System, La Jolla, CA USA.
Robert K NaviauxThe Mitochondrial and Metabolic Disease Center, University of California, San Diego School of Medicine, 214 Dickinson St., Bldg CTF, Rm C102, San Diego, CA 92103-8467 USA ; Department of Medicine, University of California, San Diego School of Medicine, 214 Dickinson St., Bldg CTF, Rm C102, San Diego, CA 92103-8467 USA ; Department of Pediatrics, University of California, San Diego School of Medicine, 214 Dickinson St., Bldg CTF, Rm C102, San Diego, CA 92103-8467 USA ; Department of Pathology, University of California, San Diego School of Medicine, 214 Dickinson St., Bldg CTF, Rm C102, San Diego, CA 92103-8467 USA ; Veterans Affairs Center for Excellence in Stress and Mental Health (CESAMH), La Jolla, CA USA.
University of California, San Diego · USGeneral Atomics (United States) · USVA San Diego Healthcare System · US

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

backgroundThis study was designed to test a new approach to drug treatment of autism spectrum disorders (ASDs) in the Fragile X (Fmr1) knockout mouse model.

methodsWe used behavioral analysis, mass spectrometry, metabolomics, electron microscopy, and western analysis to test the hypothesis that the disturbances in social behavior, novelty preference, metabolism, and synapse structure are treatable with antipurinergic therapy (APT).

resultsWeekly treatment with the purinergic antagonist suramin (20 mg/kg intraperitoneally), started at 9 weeks of age, restored normal social behavior, and improved metabolism, and brain synaptosomal structure. Abnormalities in synaptosomal glutamate, endocannabinoid, purinergic, and IP3 receptor expression, complement C1q, TDP43, and amyloid β precursor protein (APP) were corrected. Comprehensive metabolomic analysis identified 20 biochemical pathways associated with symptom improvements. Seventeen pathways were shared with human ASD, and 11 were shared with the maternal immune activation (MIA) model of ASD. These metabolic pathways were previously identified as functionally related mediators of the evolutionarily conserved cell danger response (CDR).

conclusionsThe data show that antipurinergic therapy improves the multisystem, ASD-like features of both the environmental MIA, and the genetic Fragile X models. These abnormalities appeared to be traceable to mitochondria and regulated by purinergic signaling.

Indexed as

Antipurinergic therapy (APT)Autism spectrum disordersCell danger response (CDR)EnvironmentFragile X syndromeGeneticsMaternal immune activation (MIA)MetabolismMetabolomicsMitochondriaPurinergic signaling

Identifiers

PMID25705365
PMCPMC4334917
OpenAlexW2124120525

What OpenQuestion holds

Textmetadata
LicenceCC BY
Read underepoch 390

Registered trials

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.