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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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Healing Mental Illness with Chemistry

The Cutting Edge of Health and Wellness Today
Friday, July 31, at 2 PM Pacific

Healing Mental Illness with Chemistry

Today, I am delighted to be joined by Dr. William Walsh, the author of “Nutrient Power“, a ground-breaking book which reviews his 30 years of research and clinical experience. Dr. Walsh has authored more than 200 scientific articles and reports and directs an international physician-training program and has developed biochemical therapies used by doctors throughout the world to help patients with depression, anxiety, bipolar disorder, schizophrenia, and autism. Please join us for this important program.
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Optimal Health, Detoxification and Autism: A Conversation with Sidney Baker, MD

The Cutting Edge of Health and Wellness Today
Friday, April 17, at 2 PM Pacific

Optimal Health, Detoxification and Autism: A Conversation with Sidney Baker, MD

Today I will be joined by Dr. Sidney Baker, who has pioneered the understanding of the biochemical factors that lead to Autism and continues to provide the cutting-edge research into that condition. Dr. Baker’s grasp of chronic illness and how to approach it has been taught to countless physicians, enable them to provide better medical care for their patients.

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FOR SCIENTISTS AT UC SAN DIEGO, THE EFFORT TO CONQUER AUTISM IS PERSONAL

Published in Triton, a UC San Diego Alumni Publication

By Scott LaFee
Scott LaFee is a public information officer at UC San Diego Health Sciences.

Heart disease and cancer kill. Chronic conditions like diabetes and arthritis plague millions. Alzheimer’s disease robs the elderly of their memories. But none are more terrifying than autism, which afflicts children and whose symptoms, seeming to appear without warning between the ages of two and three, can dramatically erase a child’s previous development and personality. ..

“Autism has a different kind of history,” says Laura Schreibman, Ph.D., distinguished professor emeritus of psychology who founded the groundbreaking Autism Intervention Research Program at UC San Diego in 1984. “For example, cancer never started off with parents being blamed. People got defensive. We know a lot about cancer, but relatively little about autism, which can result in some very strange or wrong-headed ideas. It’s sad, confusing and unfortunate . . . But I’ve never felt there was a better career for me.”
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ROBERT NAVIAUX

Robert NaviauxRobert Naviaux, M.D. ’86, Ph.D., is a relative newcomer to autism research.

A professor in the departments of Medicine, Pediatrics and Pathology, Naviaux is a highly regarded authority on mitochondria—the tiny power plants in all cells, whose dysfunction can result in an alarming array of metabolic diseases.

For decades, Naviaux and colleagues have pioneered genetic research in mitochondrial conditions. Some are well-known afflictions, such as diabetes, but others are not. Leigh’s disease, for example, is a rare inherited disorder that typically strikes without warning in a child’s first year of life, triggering seizures and rapid development regression. Patients rarely survive to adolescence.

Naviaux got seriously involved in autism research after being invited to a meeting of autism scientists. He listened, pondered and soon had the glimmerings of a new hypothesis. Different from classical forms of mitochondrial diseases, which are solely genetic, Naviaux concluded that autism was the result of multiple converging causes: genetic, environmental and a phenomenon dubbed the “cell danger response” (CDR).

The CDR hypothesis posits that when genes and environmental factors interact adversely, cells that feel threatened or become damaged react defensively. Their protective membranes stiffen. Internal metabolic processes change, most notably those involving mitochondria. Communications with other cells are dramatically reduced.

“Cells behave like countries at war,” Naviaux said. “When a threat begins, they harden their borders. They don’t trust their neighbors. But without constant communication with the outside, cells begin to function differently. In the case of neurons, it might be by making fewer or too many connections. One way to look at this related to autism is this: When cells stop talking to each other, children stop talking.”

Naviaux’s work pushes the limits of the prevailing autism paradigm, but he insists it is actually complementary. Autism results from genetics, environmental factors and dysfunctional metabolic processes.

In the last two years, he has published papers showing that when CDR is tamped down, allowing cells to restore normal communications and functions, autism-like symptoms in a mouse model are reversed.

The particular remedy uses a century-old drug for treating sleeping sickness. While its beneficial effects are temporary and adverse side effects make it unsuitable for long-term use, Naviaux thinks it could lead to new insights and therapies not yet imagined.

“We can only be observers and ask questions of nature. We can use the tools of the scientific method to test new ideas. We must have the courage to follow where the data leads us without bias.”

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From Eric Gordon, MD at Gordon Medical:

Gordon Medical  has just completed collecting the blood and urine samples on 40 patients with CFIDS/ME and 40 controls for a new study. In this study Dr Naviaux will apply the same technology he used in his study of autism to define the activation state of the cell danger response (CDR) in people with CFIDS/ME compared to people without symptoms. Treatment options will depend on the results and our ability to fund the next step in research. Treatments that are new require significant investment in not just the science but also in fulfilling governmental regulations designed to protect patients. We are hoping once we have our preliminary results back to begin raising money to pay for the next steps. Look for an upcoming post where we talk about CDR and metabolomics.

(The results are now in, the study is under review, and is expected to be published in fall of 2016. We are now raising funds for a replication study starting June 2016. See more at the website below. All sizes of donations are gratefully accepted.  Editor- 6/2/2016)

FIND OUT MORE ABOUT CDR AND METABOLOMICS AT GORDON MEDICAL RESEARCH CENTER.

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The Autism Epidemic: Understanding the Problems and Exciting New Treatments

Fridays at 2 PM Pacific Time
The Cutting Edge of Health and Wellness Today

The Autism Epidemic: Understanding the Problems and Exciting New Treatments

We will be discussing the spectrum of Autism which has now reached epidemic proportions. We now understand more about the causes for these diverse neurological conditions (which range from severe autism to Asperger’s Syndrome to ADHD) which allows us to provide valuable treatments. This will include our presentation about how NAET can make a big difference for many of these unfortunate children.