Autism Research: A Breath of Fresh Air

With all the words that are spilled and all the debates that rage over autism cause, cure, treatment and research there is little discussion, let alone effort, to actually find and prove new treatments and cures for autism disorders. It is a breath of fresh air to learn of some autism research that is actually being conducted with a view to HELPING autistic persons FUNCTION better. Such news is seldom heard.

One of the funders of the study is Autism Speaks which often takes heat from all sides in the world's autism communities. Well done Autism Speaks and other funding agencies involved in this effort. Hopefully more research aimed at actually helping autistic persons function better will be conducted in the near future.

Jill Cornfield at Autism Vox has highlighted research by Seaside Therapeutics LLC of a drug that may help autistic persons communicate better. The Seaside Therapeutics LLC press release indicates that it has received $30 million dollars in funding to conduct the study, which builds on the work of Seaside scientific founder Mark Bear of MIT, and explains the theory and aim of the study:

Historically, drug discovery in disorders of brain development has been unproductive largely due to the lack of mechanistic understanding of these disorders, as well as the absence of predictive animal models. Seaside Therapeutics is changing this paradigm through scientific exploration that focuses on identifying the fundamental pathophysiology of brain development disorders and applying this knowledge to develop targeted therapeutics. Recent discoveries by the Company's scientific founder, Mark Bear, Ph.D., Howard Hughes Medical Institute Investigator and Professor of Neuroscience at M.I.T., have revealed a molecular pathway, the mGluR5 signaling cascade, that is disrupted in a specific disorder of brain development - Fragile X Syndrome. With this knowledge, further research has provided insights for developing novel medications to normalize the function of this pathway, which Seaside believes may extend beyond Fragile X into a number of other developmental disorders, including autism.

STX209 is a selective gamma-amino butyric acid type B (GABA-B) receptor agonist. STX209 inhibits glutamate signaling in the brain and should thereby indirectly inhibit the excessive metabotropic glutamate receptor (mGluR) mediated protein synthesis implicated in Fragile X Syndrome. Preclinical studies using STX209 and other prototypic GABA agonists have demonstrated efficacy in animal models of Fragile X, suggesting that GABA agonists may provide significant benefits to people with Fragile X Syndrome and other disorders of brain development. STX209 entered a Phase 2 clinical study in adults and adolescents with Fragile X in December 2008 and a second trial in adolescents with autism spectrum disorders was initiated in March 2009. Seaside intends to expand both studies to include children as young as 6 years old during 2009. Data from both Phase 2 studies is expected in the first quarter of 2010.

STX107 is a highly potent, selective mGluR5 antagonist that was licensed from Merck & Company, Inc. STX107 was selected for development based on Dr. Bear's discovery of the connection between mGluR5 signaling and Fragile X Syndrome. Specifically, the evidence suggests that most, if not all, of the neurological and psychiatric consequences of Fragile X can be accounted for by exaggerated signaling through mGluR5 receptors. Preclinical research indicates that normalizing this exaggerated mGluR5 signaling reverses most of the anatomic, behavioral and synaptic abnormalities associated with Fragile X. By directly inhibiting exaggerated mGluR5 signaling, STX107 provides a compelling opportunity to treat core symptoms and disabilities of Fragile X Syndrome, autism and other developmental disorders. Seaside has been awarded translational research grants to support the development of STX107 from the National Institute of Mental Health, the National Institute of Child Health and Human Development, the National Institute of Neurological Disorders and Stroke, Autism Speaks, FRAXA and the Best Pharmaceuticals for Children Act. STX107 is expected to enter Phase 1 clinical studies in healthy volunteers in October 2009.

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Autism at School: Smaller Classrooms for Autistic Children

Neuroscientists at a meeting of the International Multisensory Research Forum held held at The City College of New York (CCNY) have argued that children with autism require smaller class sizes because of sensory integration dysfunction. I agree with the learned neuroscientists although I would add that some autistic children also have Intellectual Disabilities and serious lack of understanding and communication skills. Some require ABA based instruction to learn. For all these reasons the larger mainstream classroom is not appropriate for some autistic children. I have stated that position many times on this blog site, in the Wayne MacKay review of inclusive education here in New Brunswick and in other public meetings with education officials.

My son, at our request, receives his academic instruction in a small room with a teacher assistant. He visits common areas of the school such as the gym, kitchen, library, pool etc where he is around other children. Several children say hi to Conor when they see him. One of these students who lives nearby even visits him at our home now and plays with water balloons on the step with Conor. Prior to being removed from the mainstream classroom Conor was coming home with bite marks on his hands the result of over stimulation in the classroom.

Science Daily quotes Dr. John J. Foxe, Professor of Neuroscience at CCNY :

“Sensory integration dysfunction has long been speculated to be a core component of autism spectrum disorder (ASD) but there has been precious little hard empirical evidence to support this notion. Viewing a speaker’s articulatory movements can greatly improve a listener’s ability to understand spoken words, and this is especially the case under noisy environmental conditions.”

“These results are the first of their kind to verify that children with autism have substantial difficulties in these situations, and this has major implications for how we go about teaching these children in the classroom,” he continued. “Children with autism may become distressed in large classroom settings simply because they are unable to understand basic speech if the environment is sufficiently noisy.

“We should start to pay attention to the need for smaller numbers in the classroom and we need to carefully control the levels of background noise that these kids are exposed to. Imagine how frustrating it must be to sit in a classroom without being able to properly understand what the teacher or your classmates are saying to you.

“Being able to detect speech in noise plays a vital role in how we communicate with each other because our listening environments are almost never quiet. Even the hum of air conditioners or fans that we can easily ignore may adversely impact these children’s ability to understand speech in the classroom.

“Our data show that the multisensory speech system develops relatively slowly across the childhood years and that considerable tuning of this system continues to occur even into early adolescence. Our data suggest that children with Autism lag almost 5 years behind typically developing children in this crucial multisensory ability.”

Actually this research simply confirms what many parents, including me, have observed directly for many years.

When your child comes home every day with bite marks on his hands it is evidence that can not be ignored by a parent.

When your child is moved to a quieter, less stressful environment and the biting ceases the conclusion to be drawn is obvious and all the feel good rhetoric of the mainstream classroom for all crowds such as we have here in New Brunswick will not cause you to change your mind.

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Dr. Laurent Mottron's Work With Low Functioning, Cognitively Impaired Autistic Persons

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