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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

In 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

Health Rising – Recovery Potentially Possible: Naviaux Talks on Chronic Fatigue Syndrome (ME/CFS)

by Cort Johnson | Dec 13, 2016

Personalized treatment plans will require addressing the core metabolic abnormalities found in most ME/CFS patients plus the individual metabolic issues found of each patient.

Treatments that work for a time and then stop could be the result of not addressing all the metabolic needs of an individual.

Cort Johnson – “Recovery Potentially Possible: Naviaux Talks on Chronic Fatigue Syndrome (ME/CFS)”

The day after my brother’s wedding I shot down to San Diego to meet Rachel Riggs and a doctor with ME/CFS. Rachel, who has turned into a volunteer patient coordinator had enrolled me in Naviaux’s next metabolomics study. (Resistance, I quickly surmised, was futile – not that I was putting up any.) Rachel chatted away on the phone with another potential participant as we drove down to Naviaux’s lab. I was one of the last to give blood. editor’s note: Cort actualy enrolled in the 2nd Metabolomics study. SISOH is now recruiting for a 3rd study.

After I gave a surprising small amount of blood we tromped down the hall to meet with Dr. Naviaux in his workroom, the industrial looking pipes overhead bringing back memories of college labs in the past. Ducking into one lab Rachel showed me two $500,000 dollar mass spectometer machines each the size of a large microwave.

Gracious, as always, Dr. Naviaux offered us some coffee or tea. A bit spacey from my fast I tried out some green tea – at which point my nose immediately stopped up. At the first sound of my sniffles Naviaux turned to me and said we would have to note that for the study. (No one with a cold is allowed in the study.) Those sniffles cleared up later. (Dr. Naviaux, if you read this I promise it was from the tea…)

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Support CFS/ME Research by Shopping at AmazonSmile

Now you can support Gordon Medical’s research into CFS/ME and other chronic illness by shopping at AmazonSmile. Just use the link here in the post and your purchases will help support Gordon Medical Research Center’s fundraising efforts. For eligible purchases at AmazonSmile, the AmazonSmile Foundation will donate 0.5% of the purchase price to the customer’s selected charitable organization. AmazonSmile is the same Amazon you know. Same products, same prices, same service.

We hope you select us!

 

We are currently fundraising for the Analyzing Individual Metabolomics Study (AIMS), our third research study on the use of metabolomics in CFS/ME. Our first study resulted in the ground breaking research, “Metabolic features of chronic fatigue syndrome,” published in the Proceedings of the National Academy of Sciences. Our second study, a replication study with a larger group of CFS patients, has completed enrollment, and is processing the data collected. Our thirds study, AIMS, is now recruiting participants and fundraising. We will be looking at how comprehensive metabolomics analysis can be used to evaluate CFS/ME. AIMS builds on our previous studies, which have demonstrated there is a clear metabolomic profile in patients with CFS/ME. The more funds we are able to raise, the more participants we can include in this important research. To find out more about our research, or to sign up to participate, go to our research website at Science in Service of Humanity (SISOH). You can also make a tax deductible donation directly to the Gordon Medical Research Center.

The Most Important and Groundbreaking Study of ME/CFS to Date

 in Clinical Pain Advisor
September 27, 2016

Metabolomic Deficiencies Characteristic of Chronic Fatigue Syndrome

In a study designed to test the utility of targeted metabolomics in the diagnosis of myalgic encephalomyelitis/chronic fatigue syndrome (ME/CFS), researchers identified a unique chemical signature that differentiates affected patients from healthy individuals.

Ronald W. Davis, PhD, director of Stanford Chronic Fatigue Syndrome Research Center of Stanford University School of Medicine commented on the findings of Dr. Naviaux’s group on the the Open Medicine Foundation website, where he serves as director of the Scientific Advisory Board.

“It is the most important and groundbreaking study of ME/CFS to date. Extending recent indications of metabolic alterations in ME/CFS, this study provides the first comprehensive, quantitative demonstration of the metabolomic deficiencies that characterize the disease.”

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