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Researchers Studying Century-Old Drug in Potential New Approach to Autism

Excerpts from article originally posted May 26, 2017 | Scott LaFee and Heather Buschman, PhD in Newsroom

Robert Naviaux, MD PhDIn a small, randomized Phase I/II clinical trial (SAT1), researchers at University of California San Diego School of Medicine say a 100-year-old drug called suramin, originally developed to treat African sleeping sickness, was safely administered to children with autism spectrum disorder (ASD), who subsequently displayed measurable, but transient, improvement in core symptoms of autism.

“The purpose of CDR is to help protect the cell and jump-start the healing process,” said Naviaux, by essentially causing the cell to harden its membranes, cease interaction with neighbors and withdraw within itself until the danger has passed.

“But sometimes CDR gets stuck,” Naviaux said. “This prevents completion of the natural healing cycle and can permanently alter the way the cell responds to the world. When this happens, cells behave as if they are still injured or in imminent danger, even though the original cause of the injury or threat has passed.”

At the molecular level, cellular homeostasis or equilibrium is altered, creating an abnormal cellular response that leads to chronic disease. “When this happens during early child development,” said Naviaux, “it causes autism and many other chronic childhood disorders.”

Suramin works by inhibiting the signaling function of adenosine triphosphate (ATP), a nucleotide or small molecule produced by cellular mitochondria and released from the cell as a danger signal. When CDR is activated, the effect of extracellular ATP is similar to a warning siren that never stops. Suramin inhibits the binding of ATP and similar molecules to key purinergic receptors, according to Naviaux. It silences the siren, “signaling the cellular war is over, the danger has passed and cells can return to ‘peacetime’ jobs like normal neurodevelopment, growth and healing.”

Robert Naviaux, MD, PhD

All five boys who received the suramin infusion displayed improvements in language and social behavior, restricted or repetitive behaviors and coping skills. Assessment of improvements was based upon observational examinations and interviews using standardized tests and questionnaires, such as the Autism Diagnostic Observation Schedule, 2nd edition (ADOS-2), the Expressive One Word Picture Vocabulary Testing (EOWPWT), the Aberrant Behavior Checklist (ABC), the Autism Treatment Evaluation Checklist (ATEC), the Repetitive Behavior Questionnaire (RBQ) and the Clinical Global Impression (CGI) questionnaire. To minimize misinterpretation of natural day-to-day variations in symptoms, parents were asked to mark a symptom as changed in the 6-week CGI only if the symptom lasted for at least one week.

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Low-dose suramin in autism spectrum disorder: a small, phase I/II, randomized clinical trial

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Update on AIMS 12-21-16

Science in Service of HumanityFrom Asha Baxter and Susan Friedl
AIMS Research Coordinators

In research the turtle wins the race. Things always move more slowly than anticipated. The AIMS study is simultaneously recruiting participants and collecting funding. If you have sent in a form on the website or talked to us directly then you have been put in the queue for the study. Don’t worry, we won’t forget you!

This third study through Science in Service of Humanity (SISOH) will be the first to look at how individual as opposed to population based metabolomics data can be used to evaluate CFS/ME and other poorly defined illness. That means that if you have something other than CFS, you can also participate in the study. Remember we are also looking for age, sex, and illness matched controls who can participate. Encourage those you know to sign up, even if they are not a match for you. Controls are essential to being sure the study results are valid. [Read more…]