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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.