Old drug sheds a new light on autism

Posted May 31, 2017 by Tim Sandle
University of California San Diego School of Medicine have completed an early phase clinical trial to evaluate the effectiveness of an established drug for the treatment of autism.
One in 68 children has autism  a 30 percent rise over the last estimate released in 2012  US health ...
One in 68 children has autism, a 30 percent rise over the last estimate released in 2012, US health authorities said
Sergei Supinsky, AFP/File
What's most remarkable about the study is the drug. It is a 100-year-old drug called suramin. The medication was first developed to treat African sleeping sickness. Specifically, the medication is used for treatment of first-stage African trypanosomiasis caused by Trypanosoma brusei rhodesiense without involvement of central nervous system.
Based on pharmacological research, the drug was found to be a potential candidate for the treatment of autism. In the new trial, the medication was administered to children with autism spectrum disorder. The findings showed that the children went onto display a measurable improvement in core symptoms of autism. The only downside was the improvement being only transitory.
Autism spectrum disorder is a neurodevelopmental disorder that affects social interaction, communication, interests and behavior. The cause of autism is unknown, although a combination of genetic and environmental factors are thought to act together. Autism appears in childhood, although not until a child is around three years of age.
The new study involved involving 10 boys, ages five to 14 years, each diagnosed with autism. Five of the boys were administered intravenous infusion of suramin and the other five given a placebo. Within a short space of time. the five boys who received the suramin infusion showed marked improvements in language and social behavior, restricted or repetitive behaviors and coping skills. The effects lasted for around one week following infusion.
As to why the drug worked, Bioscience Technology states that suramin inhibits the signaling function of adenosine triphosphate, a nucleotide or small molecule produced by cellular mitochondria and released from the cell as a danger signal. This tallies with one factor with autism, which is with children with autism exhibiting oxidative stress, which is an outcome of the cell danger response.
Further work will be undertaken with the view of confirming the findings. If proven, later research will assess whether the timescale for the beneficial effect can be improved. The findings are published in the Annals of Clinical and Translation Neurology, in a paper under the title "Low-dose suramin in autism spectrum disorder: a small, phase I/II, randomized clinical trial."