Improving the Prediction of Response to Therapy in Autism
Open accessAbstract
Autism is a heterogeneous disorder involving complex mechanisms and systems occurring at diverse times. Because an individual child with autism may have only a subset of all possible abnormalities at a specific time, it may be challenging to identify beneficial effects of an intervention in double-blind, randomized, controlled trials, which compare the mean responses to treatments. Beneficial effects in a small subset of children may be obscured by the lack of effect in the majority. We review the evidence for several potential model systems of biochemical abnormalities that may contribute to the etiology of autism, we describe potential biomarkers or treatment targets for each of these abnormalities, and we provide illustrative treatment trials using this methodology. Potential model systems include immune over and under reactivity, inflammation, oxidative stress, free fatty acid metabolism, mitochondrial dysfunction, and excitotoxicity. Including potential biomarkers and targeted treatments in clinical trials for autism provides a potential method for limiting the heterogeneity of enrolled subjects, which may improve the power of studies to identify beneficial effects of treatments while also improving the understanding of the disease.
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Biological abnormalities have been reported in eye tracking, neuroanatomical pathway connectivity, brain regional volumes, cortical activation during social and communication tasks, serotonin levels, and other biological assays, but not with sufficient consistency to constitute uniform diagnostic biomarkers.
“Biological abnormalities have been reported in eye tracking, neuroanatomical pathway connectivity, brain regional volumes, cortical activation during social and communication tasks as measured with functional magnetic resonance imaging and magnetoencephalography, serotonin levels, and other biological assays, but not with sufficient consistency for these to constitute uniform diagnostic biomarkers”
From Negative allosteric modulation of the mGluR5 receptor reduces repetitive behaviors and rescues social deficits in mouse models of autism (2012) · cites this paper as [93]
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An Overview of Autism Spectrum Disorder, Heterogeneity and Treatment Options
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- Authors
- Bent, S., & Hendren, R. L.
- Affiliations
- University of California, San Francisco (US)
- Corresponding author
- Bent, Stephen
- Venue
- Neurotherapeutics
- Publisher
- Elsevier BV
- Published
- 2010-07
- Volume
- 7
- Issue
- 3
- Pages
- 232-240
- ISSN
- 1878-7479 (ISSN-L)
- PMC
- PMC5084226
- OpenAlex
- W1995938206
- Cited by
- 25
- References
- 60
- Citations by year
- 2026: 2 · 2025: 1 · 2020: 1 · 2019: 3 · 2017: 3 · 2016: 1 · 2015: 5 · 2013: 2 · 2012: 3
- FWCI
- 0.21
- Type
- journal article
- Language
- en
- Open access
- bronze
- doi.org
- Licence
- Locations
- Neurotherapeutics (publishedVersion)
- PubMed Central (publishedVersion)
- PubMed (publishedVersion)
- Funders
- Autism Speaks (AS2311)
- Emch Foundation
- Topics
- Life Sciences › Neuroscience › Cognitive Neuroscience › Autism Spectrum Disorder Research
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Genetics › Genetics and Neurodevelopmental Disorders
- Health Sciences › Medicine › Psychiatry and Mental health › Child Nutrition and Feeding Issues
- Keywords
- autism spectrum disorder, treatment response prediction, biomarkers, targeted therapy, treatment effect heterogeneity, immunoreactivity, inflammation, oxidative stress, free fatty acid metabolism, mitochondrial dysfunction, excitotoxicity, biochemical abnormalities, autism etiology, clinical trial design
- MeSH terms
- Complementary Therapies, Autistic Disorder, Humans, Predictive Value of Tests, Biomarkers, Randomized Controlled Trials as Topic
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