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Drug treatment possible for reversing autism

Kathleen Blanchard's picture
Possible drug therapy for autism may be in the future.

Researchers have found a critical and previously unknown link between synthesis of brain proteins and autism spectrum disorders that they say could lead to new treatments. In their studies, scientists were able to reverse ASD behaviors in mice by manipulating brain cell signals, which could lead to the development of drugs to reverse ASD associated behaviors.

McGill University scientists explain mouse studies show symptoms that develop and associated with ASD are the result of abnormally high synthesis of a group of proteins called neuroligins.

Neuroligins are molecules that are involved in sending brain signals. The molecules bind with a ligand (another group of molecules or ions) to transmit and process information in the brain.

Prof. Nahum Sonenberg, from McGill’s Dept. of Biochemistry, Faculty of Medicine, and the Goodman Cancer Research Centre explained in a press release: “We used a mouse model in which a key gene controlling initiation of protein synthesis was deleted.

In these mice, production of neuroligins was increased. Neuroligins are important for the formation and regulation of connections known as synapses between neuronal cells in the brain and essential for the maintenance of the balance in the transmission of information from neuron to neuron.”

When the researchers blocked synthesis of the proteins with gene therapy they were able to reverse autism like behaviors.

Christos Gkogkas, a postdoctoral fellow at McGill and lead author. “Our work is the first to link translational control of neuroligins with altered synaptic function and autism-like behaviors in mice.

Neuroligin mutation was first discovered in 2003 and linked to autism spectrum disorders, but research hasn’t uncovered the exact mechanism of how the mutations lead to ASD symptoms.

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Gkogkas said the researchers reversed autism symptom in mice using existing compounds developed for cancer treatment that reduce protein synthesis. Next they used non-replicating viruses to halt the abnormal synthesis of neuroligins.

The scientists used a computer algorithm to identify structure and regulation of mRNA of neuroligins. They discovered that abnormal synthesis of the proteins is what causes imbalance in single brain cell signals.

The autistic behaviours in mice were prevented by selectively reducing the synthesis of one type of neuroligin and reversing the changes in synaptic excitation in cells,” explained Prof. Jean-Claude Lacaille at the University of Montreal’s Groupe de Recherche sur le Système Nerveux Central and Department of Physiology in a media release.

In other words, he says, the scientists manipulated brain cell signals to see how it affected behavior in the mice. When they manipulated a second neuroligin they were able to reverse autistic behaviors.

“The fact that the balance can be affected suggests that there could be a potential for pharmacological intervention by targeting these mechanisms,” Lacaille concluded.

But Sonenberg cautions that the finding doesn't mean a treatment for autism is close at hand. . “The drug we used would be too toxic to use for ASDs,” he says. “But we’ve shown that this pathway is important, identified potential therapeutic targets and demonstrated that a drug therapy is possible in principle.”

The finding suggests new hope for treating autism spectrum disorders with drugs that block abnormally high synthesis of neuroligins in the brain associated with ASD that affects 1 in 88 children across every ethnic, racial and socioeconomic group, according to statistics from the CDC.

Journal Nature
November 21, 2012

Image credit: Wikimedia commons



A Florida researcher, Robert Cade, and his co-workers and colleagues have identified a milk protein, casomorphin, as the probable cause of attention deficit disorder and autism. They found Beta-casomorphin-7 in high concentrations in the blood and urine of patients with either schizophrenia or autism. Children with ADHD may also be more likely to suffer from other food allergies. They could be losing vital vitamins, iron and even zinc because of their allergy to dairy products, wheat, corn, yeast, soy, citrus, eggs, chocolate, peanuts, chlorine, fluoride and artificial colors and preservatives. Dr. Rimland, who's son is autistic, experimented with eliminating certain kinds of food and found that by eliminating casein and gluten from his son's diet, autistic symptoms were reduced. This gave rise to the notion of the "leaky gut" cause of autism, in which the gastrointestinal walls have been compromised, allowing for incompletely broken down and thereby toxic protein chains to be absorbed into the bloodstream. One thing is for sure! Autism is not caused by a lack of drugs like Ritalin. ADHD medicated children often turn to drug or alcohol use in later life. It has also been reported that selling of Ritalin and other ADHD prescribed drugs has been noticed because of their opiate like qualities.
Cause of Autism is easy to fine. unfortunately scientists are looking wrong direction. I am a father of six children. First four are normal the last two are Autistic. I have looked what happen before the first Autistic child was conceived. The only thing happen was my wife had mercury Amalgam filling for her two of her teeth. Four of my work colleagues have children with Autism they also had mercury amalgam filling as dental treatment. I have also done general survey among the parents with autistic children almost 98% they had dental treatment with mercury amalgam. Why study has not been done animal with Mercury Amalgam filling, if they produce Autistic offspring specially the behavior of their offspring. My understanding is Autism is cause by neorotocxic before birth. that is why detox does not work. Early intervention also has not been quantified. So call biomedical and diet intervention also does not work. My god help as in regarding Autism.