Clinical Trial Investigates Drug for Autism A clinical trial at the University of California, San Diego School of Medicine is investigating the safety and efficacy of an unprecedented drug therapy for autism spectrum disorder (ASD). The phase 1 clinical trial will evaluate suramin, a century-old drug still used for African sleeping sickness, as a novel treatment ... News in Brief
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News in Brief  |   September 01, 2015
Clinical Trial Investigates Drug for Autism
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Special Populations / Autism Spectrum / News in Brief
News in Brief   |   September 01, 2015
Clinical Trial Investigates Drug for Autism
The ASHA Leader, September 2015, Vol. 20, 15. doi:10.1044/leader.NIB8.20092015.15
The ASHA Leader, September 2015, Vol. 20, 15. doi:10.1044/leader.NIB8.20092015.15
A clinical trial at the University of California, San Diego School of Medicine is investigating the safety and efficacy of an unprecedented drug therapy for autism spectrum disorder (ASD).
The phase 1 clinical trial will evaluate suramin, a century-old drug still used for African sleeping sickness, as a novel treatment for children with an ASD diagnosis. Previous research at the school found that a single injection of suramin reversed symptoms of ASD in mouse models.
This trial, which will include 20 qualifying participants, is the first to test suramin in children. No medication is available that cures ASD, and very few can improve its core symptoms.
In the trial, half of the participating children will receive suramin intravenously; half will receive a placebo (saline infusion). Researchers will conduct behavioral and medical tests, including blood and urine analyses, before and after treatment.
The trial is the first clinical investigation of the theory that ASD may result from abnormal activation of the cell danger response. Under this theory, cells threatened or damaged by microbes (such as viruses or bacteria), physical forces or chemicals (such as pollutants) react defensively and their membranes stiffen as part of the normal immune response. Cells’ internal metabolic processes are altered—most notably those of mitochondria, the cells’ critical “power plants.” The cell danger response activates and communication between cells decreases.
Suramin, as currently constituted, can be used only for a few months because of its toxicity risk, and is not available as an ongoing treatment.
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September 2015
Volume 20, Issue 9