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Drug used for sleeping sickness now being seen as tool against autism

By Catholic Online (NEWS CONSORTIUM)
6/18/2014 (3 years ago)
Catholic Online (www.catholic.org)

Trials conducted on laboratory mice prove successful; human trial to begin next year

An old drug used in the treatment of sleeping sickness has also temporarily reversed symptoms of autism in mice, researchers have claimed. Researchers say the drug restored normal cellular signaling in a mouse model of autism in animals that were the human biological age equivalent of 30 years old. The results came with a caveat: the effects in the laboratory only lasted for around five weeks.

Suramin, a well-known inhibitor of purinergic signaling, first synthesized in 1916 and is commonly used to treat trypanosomiasis or African sleeping sickness, a parasitic disease was used in trial runs on the mice.

Suramin, a well-known inhibitor of purinergic signaling, first synthesized in 1916 and is commonly used to treat trypanosomiasis or African sleeping sickness, a parasitic disease was used in trial runs on the mice.

Highlights

By Catholic Online (NEWS CONSORTIUM)
Catholic Online (www.catholic.org)
6/18/2014 (3 years ago)

Published in Health

Keywords: Suramin, autism, sleeping sickness, trial tests, mice


LOS ANGELES, CA (Catholic Online) - "Obviously correcting abnormalities in a mouse is a long way from a cure in humans, but we think this approach is a new and fresh way to think about and address the challenge of autism," Robert K. Naviaux at the University of California, San Diego School of Medicine says.

One of the universal symptoms of autism, Naviaux says, is metabolic disturbances.

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"Cells have a halo of metabolites (small molecules involved in metabolism, the set of chemical processes that maintain life) and nucleotides surrounding them.

"These create a sort of chemical glow that broadcasts the state of health of the cell."

Cells that are threatened or damaged by microbes, such as viruses or bacteria, or by physical forces or by chemicals, such as pollutants, react defensively, a part of the normal immune response. In this case, communications between cells are dramatically reduced.

If this occurs during childhood, for example, neuro-development is delayed. "Cells behave like countries at war," he says. "When a threat begins, they harden their borders. They don't trust their neighbors.

"One way to look at this related to autism is this: When cells stop talking to each other, children stop talking.

Suramin, a well-known inhibitor of purinergic signaling, first synthesized in 1916 and is commonly used to treat trypanosomiasis or African sleeping sickness, a parasitic disease was used in trial runs on the mice.
 
"The discovery that a single dose of medicine can fundamentally reset metabolism for weeks means that newer and safer drugs might not need to be given chronically," he said.

"Members of this new class of medicines might need to be given only intermittently during sensitive developmental windows to unblock metabolism and permit improved development in response to many kinds of behavioral and occupational therapies, and to natural play."

These recent findings dovetail with the idea that autism is caused by a multitude of interconnected factors: "Twenty percent of the known factors associated with autism are genetic, but most are not.

"It's wrong to think of genes and the environment as separate and independent factors. Genes and environmental factors interact. The net result of this interaction is metabolism."

Researchers found that suramin blocked the extracellular signaling pathway used by ATP and other mitokines in a mouse model of autism spectrum disorder (ASD), ending this cell danger response and related inflammation.

A single dose remained effective in the mice for about five weeks - but then washed out. Suramin also cannot be taken long-term since it can result in anemia and adrenal gland dysfunction.

Cells subsequently began behaving normally and autism-like behaviors and metabolism in the mice were corrected.

Naviaux said these and earlier findings are sufficiently encouraging to soon launch a small phase 1 clinical trial with children who have ASD later this year.

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