# Center for the Development of Phenotype-Based Treatments of Autism Spectrum Disorder

> **NIH NIH P50** · UNIVERSITY OF CALIFORNIA AT DAVIS · 2020 · $2,278,831

## Abstract

PROJECT SUMMARY – OVERALL
If there is one issue that unites the diverse and vocal community of families affected by autism spectrum
disorder (ASD), it is the frustration that despite the hundreds of millions of dollars that have been spent on
autism research, there are so few treatment options available to decrease the disabilities of their loved ones.
The overarching goal of our proposed Center for the Development of Phenotype-Based Treatments of
Autism Spectrum Disorder is to discover targets for effective treatments in groups of children with ASD with
rigorously defined phenotypic characteristics. It has become abundantly clear that there are many causes and
trajectories of ASD. Moreover, some of the most debilitating aspects of ASD are due to the serious co-morbid
conditions such as anxiety, seizures and intellectual disability. Considering the broad range of clinical and
behavioral features of ASD, it is unlikely that a single treatment will correct all of these problems. This proposal
is based on the premise that identifying clinically meaningful subtypes of ASD will facilitate the analysis of
etiologies and the development of more effective therapeutics. The Specific Aims for the Center include:
Aim #1: To use enhanced clinical evaluations of children with ASD, particularly those with intellectual disability,
to better characterize the sub-group that exhibits clinically significant anxiety. Proper diagnosis and effective
treatment holds the promise of a much-improved quality of life for these children.
Aim #2: To conduct a 16-week randomized comparative treatment trial of
Behavioral Intervention for Anxiety in
Children with Autism
(BIACA), sertraline, and pill placebo in youth with ASD.
Aim #3: To use fMRI to investigate neural predictors of treatment efficacy, markers of treatment-induced
change, and signatures of anxiety sub-types defined in Aim 1.
Aim #4: To carry out behavioral, neuroimaging and electrophysiological analyses of a newly recruited group of
children (2-3 1/2-years-old) with ASD and brains that are disproportionately enlarged relative to body size. The
major goal of this aim is to increase our understanding of the cognitive functions and brain systems that are so
impacted as to lead to a poorer prognosis for these children. This would inform the design of more targeted
behavioral interventions. While there is no evidence that these children have less access to standard
behavioral therapies, it has become clear that they constitute an ASD phenotype that benefits less from
standard interventions. How to treat these children is not yet clear and the Center endeavors to fill this gap.
Aim #5: To generate an iPSC patient resource from a subset of the children that are investigated in Aim #4.
Lines of iPSCs for each subject will be differentiated into neural progenitor cells, oligodendrocytes and
microglial cells to identify gray and white matter contributions to the development of enlarged brains. The cell
lines will also be st...

## Key facts

- **NIH application ID:** 9984468
- **Project number:** 5P50HD093079-04
- **Recipient organization:** UNIVERSITY OF CALIFORNIA AT DAVIS
- **Principal Investigator:** David G Amaral
- **Activity code:** P50 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2020
- **Award amount:** $2,278,831
- **Award type:** 5
- **Project period:** 2017-09-07 → 2022-07-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/9984468

## Citation

> US National Institutes of Health, RePORTER application 9984468, Center for the Development of Phenotype-Based Treatments of Autism Spectrum Disorder (5P50HD093079-04). Retrieved via AI Analytics 2026-05-22 from https://api.ai-analytics.org/grant/nih/9984468. Licensed CC0.

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