Experimental drug reduces autism symptoms in mice, gov't study shows

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(CBS News) Autism affects one out of every 88 American children and while there are available treatments for early intervention, there is no cure. A new government-funded study has found an experimental treatment is effective at reversing symptoms of autism in mice.

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For the study, published in the April 25 issue of Science and Translational Medicine, researchers from the National Institutes of Health bred a strain of mice to display autism-like behaviors. Similar to how children with autism have social deficits and engage in repetitive behaviors, these mice did not interact and communicate with each other and spent an inordinate amount of time engaging in repetitive behavior - in this case self-grooming.

Cue the experimental drug called GRN-529. The drug was designed to inhibit a type of brain cell receptor that receives the neurotransmitter glutamate. Glutamate is typically involved in learning and memory processes and stimulates other areas of the brain and nervous system.

When mice with the autism-like behaviors were injected with the experimental compound, they reduced the frequency of their repetitive self-grooming and spent more time around strange mice, even sniffing them nose to nose. When tested on a different strain of mice, the experimental compound stopped all repetitive jumping behavior.

"These new results in mice support NIMH-funded research in humans to create treatments for the core symptoms of autism," Dr. Thomas R. Insel, director of the National Institute of Mental Health, said in a statement. "While autism has been often considered only as a disability in need of rehabilitation, we can now address autism as a disorder responding to biomedical treatments."

The researchers said although most mouse brain findings often don't translate to humans, the fact that these compounds are already being tested for an overlapping condition strengthens the case for the drug's effectiveness. This class of compounds is currently being studied in patients with the genetic disease Fragile X syndrome, the most common inherited form of intellectual disability. About one third of patients with Fragile X syndrome also meet criteria for autism.

"These inbred strains of mice are similar, behaviorally, to individuals with autism for whom the responsible genetic factors are unknown, which accounts for about three fourths of people with the disorders," noted study author Dr. Jacqueline Crawley of the NIMH. "Given the high costs - monetary and emotional - to families, schools, and health care systems, we are hopeful that this line of studies may help meet the need for medications that treat core symptoms."

Some experts exercised caution with the new findings. In an accompanying editorial in the same journal issue, Baltazar Gomez-Mancilla, executive director of translational medicine neuroscience at Novartis, wrote, "It is too early to speculate as to whether or not autism spectrum disorders can be reversed by small molecules."

Dr. Uta Frith, a professor of cognitive development at University College London, told BBC News that neurotransmitter problems have long been suspected as an origin of autism, "However, it will be a long time until these findings can be translated for human patients. Tampering with the synapse may well result in undesirable side effects," he said.

Here is a video of the improved sociability in mice on the experimental treatment:

[Omegasseekerr]

We Autistics are already using glutamate blockers to stop melt downs and brain damage. Another thing we can do that works for me is just eat very carefully. The FDA allows glutamate to hide in a huge number of foods under about 30 different names. Go to truthinlabeling.org to learn these names. Every live virus vaccine contains glutamate under the name hydrolyzed protein which sounds innocent but water is used to split the protein so only the glutamate part of it is left to add to the foods. The contents of IVs often alsy have "hydrolyzed protein" in them and the brother of a friend of mine had a rage reaction after he went into a hospital for a non-autism related condition and was given an IV with this brain cell killing and rage reation causing poison in it and ended up locked up in the psyche ward for a while because of it. So Big Pharma is making money selling us glutamate blockers that help us but at the same time claim injecting the same poison into us won't hurt us!

[mikies123]

It would be interesting to see if this "new drug" would work on the primates who experienced delayed acquisition of neonatal reflexes as newborns who were given the birth dose of the TCV hepatitis b vaccine. Mercury is a well-known neurotoxicant affecting glutamate receptors and it study http://www.whale.to/vaccine/primates_hep_b-1.pdf the primates lost key survival skills

I wonder why the researchers just didn't inject these mice with mercury or the hepatitis b vaccine to affect their behavior. They already had a good primate model of vaccine-mercury induced brain damage.

[bpatient]

"Excuse me, but just how did scientists 'bred a strain of mice to display autism-like behaviors.'"

Please see my reply to maurine9. That information has been published.

[maurine9]

This article tells us nothing about how the animals were induced to become autistic. Wouldn't that be crucial to telling us something about how autism is caused--and i am not talking about fragile X or genetics. Besides, there is already a drug on the market used for alzheimers to reduce excess glutamates by acting on ceertain glutamate receptors. This doesn't look like any new drug to me.
Interesting though they say that they plan to treat autism biomedically. Parents of children with autism have been treating them biomedically for many years. Autism is not a mental disability but a metabolic one that affects every organ in the body. Those affected suffer from such things as oxidative stress, immune dysfunction, inflammatory bowel disease and encephalopathy. maybe the parents should be teaching the doctors, don't you think?
Maurine Meleck, SC
grandmother to 2 vaccine injured boys-one recovered with bio-medical treatments.

[bpatient]

"This article tells us nothing about how the animals were induced to become autistic."

Did you miss the link provided in this article to the original research article, which of course includes citations of the work that answers your question?

[bpatient]

It's customary to read the original research article (linked in the article above) and the references cited therein if you want to understand how the research was performed.

[amdachel]

"[R]esearchers from the National Institutes of Health bred a strain of mice to display autism-like behaviors. Similar to how children with autism have social deficits and engage in repetitive behaviors, these mice did not interact and communicate with each other and spent an inordinate amount of time engaging in repetitive behavior - in this case self-grooming."

Excuse me, but just how did scientists "bred a strain of mice to display autism-like behaviors."

For two decades all the experts have been clueless over autism. There's no known cause, cure, or universally effective treatment. There's nothing a mainstream doctor can tell a new mom so that her healthy baby doesn't also end up on the autism spectrum, where currently we find one in every 88 children and one in every 54 boys.

Incredibly, we now hear that researchers can actually induce autism when there's the possibility of making lots of money treating the symptoms.

Anne Dachel, Media editor: Age of Autism