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Expert Opin Investig Drugs. 2019 Aug;28(8):709-718. doi: 10.1080/13543784.2019.1649656. Epub 2019 Aug 12.

Investigational drugs in early-stage clinical trials for autism spectrum disorder.

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a Division of Psychiatry, Cincinnati Children's Hospital Medical Center , Cincinnati , OH , USA.
b College of Medicine, University of Cincinnati , Cincinnati , Oh , USA.


Introduction: Pharmacologic interventions in Autism Spectrum Disorder (ASD) have historically focused on symptom-based approaches. However, a treatment for the core social deficits has remained unidentified. While a definitive theory for the cause of ASD is not yet known, recent advances in our understanding of ASD pathophysiology have opened the door for research on new pharmaceutical methods to target core symptomology. Areas covered: Herein, we review the novel pharmacologic therapies undergoing early-stage clinical trials for the treatment of the social symptoms associated with ASD. Specifically, these strategies center on altering neurologic excitatory and inhibitory imbalance, neuropeptide abnormalities, immunologic dysfunction, and biochemical deficiencies in ASD. Expert opinion: Utilizing the growing field of knowledge regarding the pathological mechanisms and altered neurobiology of individuals with ASD has led to the development of many innovative pharmaceutical interventions. Clinical trials for neurobiologic and immunologic targets show promise in impacting the social behavior and processing deficits in ASD but need evaluation in larger clinical trials and continued biomarker development to more effectively and consistently assess pharmacologic effects. Additionally, evaluating patient-specific drug responsivity and integrating behavioral intervention in conjunction with pharmacologic treatment is crucial to developing a successful approach to ASD treatment.


Autism; clinical trials; drug development; social deficits

[Indexed for MEDLINE]

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