New Drug Treatments for Core Symptoms of ASD's On the Way

From www.bioresearchonline.com the following announcement was made regarding the research and development of new drugs that address underlying causes of core autism symptoms:

Roche And Seaside Therapeutics Announce Landmark Alliance To Advance Novel Treatments For Fragile X Syndrome And Autism Spectrum Disorders

June 19, 2012

Roche and Seaside Therapeutics announced recently that they have entered into a collaboration to develop disease modifying treatments for fragile X syndrome (FXS) and autism spectrum disorders (ASD), both neurodevelopmental disorders for which there are currently no effective pharmacological treatments that address core symptoms. The alliance aims to speed up research and development in this field and  lead a fundamental change in the treatment paradigm for FXS and ASD by developing therapeutics that target the molecular basis and, in turn, core symptoms of these neurodevelopmental disorders.

Under the terms of the agreement, Seaside will license patents covering the use of mGluR5 antagonists for the treatment of neurodevelopmental disorders exclusively to Roche. Roche will subsequently lead the development and commercialization of these compounds for the treatment of FXS and ASD. Its mGluR5 drug candidate RG7090, is currently enrolling patients in a Phase 2 clinical trial in FXS.

Seaside will develop its GABA-B agonist program and retains exclusive rights to issued and pending patents covering the use of GABA-B agonists for the treatment of FXS and ASD. Seaside’s lead GABA-B candidate STX209 is currently enrolling patients in Phase 3 trials in FXS and recently completed enrollment in a Phase 2b trial in ASD. Roche may exercise options to commercialize STX209 upon completion of certain clinical development phases in FXS and ASD, but Seaside will continue to lead the clinical development of these programs. Additional terms of the transaction will not be disclosed.

“Roche is committed to finding new treatments in areas of high unmet medical need such as autism spectrum disorders,” comments Luca Santarelli, Global Head of Roche Neuroscience. “Recent discoveries in genetics have shed light on the biological underpinnings of these conditions thus providing a basis for mechanistic drug discovery. To establish a leadership position in this field we sought to build a solid partnership with Seaside Therapeutics, a company that has successfully pioneered the research and development in this novel and uncharted area.”

“This collaboration is a real win for patients and caregivers—aligning leading minds and organizations committed to rapidly advancing transformational drugs to treat autism and fragile X syndrome,” said Randy Carpenter, MD, President and Chief Executive Officer of Seaside Therapeutics. “Importantly, this collaboration also provides Seaside with additional resources to complete late-stage clinical development of STX209, which we believe has the potential to change the treatment paradigm for fragile X and autism and thereby help patients and their families achieve an improved quality of life.”

ASD refers to a group of enigmatic cognitive disorders, including autism and Asperger’s syndrome, which impair social interaction and communication, whereas FXS is a rare genetic disease, whose symptoms closely resemble ASD, and whose underlying mechanism may be similar. With no approved pharmacological therapies that address core symptoms for either condition, the unmet medical need remains high. The compounds that Roche and Seaside Therapeutics are developing hold the promise to become the best-in-class treatments by targeting aberrant glutamate signaling and GABA, thereby restoring synaptic transmission in ASD and FXS patients.

About mGluR5
The most commonly inherited form of autism involves the gene encoding fragile X mental retardation protein (FMRP). Loss of FMRP function disrupts signaling between neurons, leading to widespread brain abnormalities and mental retardation. Normally, FMRP is balanced by mGluR5, an important receptor in the brain that is involved in learning and memory. Without normal FMRP, this balance is lost, leaving mGluR5 function unopposed. Early results from a clinical trial suggest that children with fragile X syndrome can be helped by drugs that inhibit mGluR5 activity. (Source: Sfari)

About Seaside Therapeutics
Seaside Therapeutics is working to correct or improve the course of autism, fragile X syndrome and other neurodevelopmental disorders by translating breakthrough discoveries in neurobiology into therapeutics that will improve the lives of patients and their families. While there are treatments that alleviate some symptoms of neurodevelopmental disorders, there are currently none that address the underlying causes.

Seaside Therapeutics is using a scientific approach in attempt to address this unmet need and changing the landscape of drug development by focusing on single-gene disorders with a high prevalence of autism, such as fragile X syndrome. For more information, visit www.seasidetherapeutics.com

About Seaside Therapeutics’ STX209:
STX209 is an oral selective gamma-amino butyric acid type B (GABA-B) receptor agonist. Pathologies observed in certain neurodevelopmental disorders, including autism spectrum disorders and fragile X syndrome are believed to be caused by excessive activation of glutamate receptors and abnormally high ratios of excitatory to inhibitory neurotransmission in the brain. GABA-B receptors play an important role in modulating the release of glutamate and optimizing the ratio of excitatory to inhibitory neurotransmission. STX209 has demonstrated efficacy in preclinical models, suggesting that it may improve function in individuals with autism spectrum disorders and fragile X syndrome. With STX209, Seaside has successfully completed the largest, randomized, blinded, placebo-controlled trial (Phase 2) in patients with fragile X syndrome and an open-label Phase 2a exploratory trial in patients with autism spectrum disorders. Two Phase 3 studies, one in adolescents and adults (ages 12 to 50) and one in children (ages 5 to 11) with fragile X syndrome, are currently enrolling participants. A Phase 2b study in children, adolescents and adults (ages 5 to 21) with autism spectrum disorders recently completed enrollment.

About Roche’s involvement in autism spectrum disorders
Roche has a strong commitment in the area of neurodevelopmental disorders, in particular in ASD with currently three compounds in clinical development. A Phase 2 trial is currently ongoing in adult and adolescent fragile X patients through a wide age range to investigate the safety and efficacy of Roche mGluR5 antagonist RG7090 (read more about the study under http://clinicaltrial.gov/). In parallel Roche is exploring biomarkers to identify patients most likely to benefit from this treatment.

A recent study1 demonstrated that chronic treatment with Roche’s mGluR5 antagonist corrected fragile X symptoms in a mouse model, even if started after the disease onset. These new data suggest beneficial effects in a wide range of symptoms and a disease-modifying potential for mGluR5 antagonists in fragile X syndrome.

Roche is also committed to establishing connections with leading academic institutions and biotechnology companies. Recently, an international consortium of scientists, led by Roche and King’s College London, has launched the largest single grant for autism research in the world and the largest for the study of any mental health disorder in Europe. Read more about European Autism Interventions – A Multicentre Study for Developing New Medications (EU-AIMS).

Roche also signed an agreement with the Harvard Stem Cell Institute, Harvard Medical School, Children’s Hospital Boston and Massachusetts General Hospital to develop cell models for autism spectrum disorders.

Key players in the field of ASD research across the industry, including Seaside Therapeutics, academia and patient advocacy groups recently gathered at the fourth edition of Roche - Nature Medicine Translational Neuroscience Symposium, to share the latest advances in the field of “Autism Spectrum Disorders: From Biological Understanding to Therapeutic Strategies”.

About Roche
Headquartered in Basel, Switzerland, Roche is a leader in research-focused healthcare with combined strengths in pharmaceuticals and diagnostics. Roche is the world’s largest biotech company with truly differentiated medicines in oncology, virology, inflammation, metabolism and CNS. Roche is also the world leader in in-vitro diagnostics, tissue-based cancer diagnostics and a pioneer in diabetes management. Roche’s personalized healthcare strategy aims at providing medicines and diagnostic tools that enable tangible improvements in the health, quality of life and survival of patients. In 2011, Roche had over 80’000 employees worldwide and invested over 8 billion Swiss francs in R&D. The Group posted sales of 42.5 billion Swiss francs. Genentech, United States, is a wholly owned member of the Roche Group. Roche has a majority stake in Chugai Pharmaceutical, Japan. For more information, visit www.roche.com.

SOURCE: Roche

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Long Term Effects of Teaching Social Skills to Autistic Teens

From Medical News Today:

Teaching Social Skills To Autistic Teens Proves Effective Over Long Term

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Teenagers with autism spectrum disorder are in a bind. The disorder is characterized by impairments in communication and social interaction, but it's a continuum, so some teens diagnosed with ASD are considered high functioning and healthy enough to be "mainstreamed" in school. 

But without the proper social skills, even mainstreamed teens don't quite fit into the general social milieu of middle school or high school. As a result, they suffer from all the slings and arrows of that world. 

Since 2006, however, the UCLA PEERS (Program for the Education and Enrichment of Relational Skills) clinic has assisted high-functioning teens with ASD by literally teaching them the strategies they need to fit in better with their peers. And while previous research demonstrated that the program was effective, it wasn't known whether the new skills "stuck" with these teens after they completed the PEERS classes. 

In the current edition of the Journal of Autism and Developmental Disorders, Elizabeth Laugeson, director of the PEERS Clinic and a UCLA assistant clinical professor of psychiatry, and colleagues report that in a long-term follow-up study, they found that the skills taught and learned stayed with the kids - and in some cases even improved. 

ASD includes a range of pervasive developmental disorders characterized by problems with communication and socialization; it's estimated that one in 88 children born in the United States has some form of ASD. 

The researchers' findings show that the PEERS intervention resulted in significant improvements in social skills, as reported by parents and teachers using standardized measurements of social functioning. Reports from parents also suggested that teens' ASD symptoms related to social responsiveness decreased significantly by the end of the class and even at the long-term, 14-week follow-up. In addition, the teens' knowledge of social skills improved, as did the frequency of their get-togethers with their peers. 

Teacher ratings of the teens' social functioning in class also showed significant improvements at the long-term follow-up - an important finding, since the teachers did not know the teens had participated in the PEERS class. 

Both parents and teachers also reported there were fewer problem behaviors with the teens 14 weeks after the program was over. 

"Teens not only showed better social competence and greater understanding of social skills, but they were having more frequent get-togethers with their peers because they had developed the critical skills needed to make and keep friends," said Laugeson, who also directs The Help Group-UCLA Autism Research Alliance. 

Studies on the effectiveness of social-skills training for individuals with ASD indicate that intervention during childhood and adolescence is critical. However, very few evidence-based interventions focus on improving the social competence of teens with ASD, which makes the present findings unique and important, Laugeson said. 

"This is exciting news," she said. "It shows that teens with autism can learn social skills and that the tools stick even after the program is over, improving their quality of life and helping them to develop meaningful relationships and to feel more comfortable within their social world. The fact that these social skills are sticking is critical, because we need them to thrive throughout our lives." 

Laugeson attributes the power of the program to the parents. The PEERS classes, which focus on teaching the rules of social etiquette to teens, require parents to participate as well. In separate meetings, the parents are also provided with information on how to be social coaches for their teens in the real world. Many of the social skills taught are those most of us know intuitively: how to have a conversation (by trading information), showing good sportsmanship ("Hey, nice shot!"), and how to avoid bullying or deflect taunts ("Yeah, whatever"). 

The classes meet for 90 minutes once a week for 14 weeks and include brief didactic instruction, role-playing demonstrations, behavioral rehearsal exercises for teens to practice newly learned skills, in-class coaching with performance feedback, and weekly "homework" assignments, supervised by parents, such as inviting a friend over for a get-together at home. 

"The class is very structured, and the skills are broken down into small rules and steps of social etiquette that give the teens specific actions they can take in response to a social situation," Laugeson said. "This method of instruction is very appealing to teens with autism because they tend to think concretely and literally and often learn by rote." 

What makes this program even more unique, Laugeson said, is that it teaches the skills used by socially accepted teens - not what adults think teens should do. For example, if teens with ASD are teased, "most adults will tell teens to ignore the person, walk away or tell an adult," she said. "But when you ask teens if this works, they say no. So we want to teach our teens to do what kids that are socially accepted are naturally doing. In this case, that would be to give a short comeback that shows what the person said didn't bother them - like saying 'whatever' or 'yeah, and?' They learn not to take the bait." 

An Easy Way to Contribute to Autism Research

CDSweep Makes it Easy to Clean Out Your Unwanted CDs & Support Autism Research!

June 5, 2012 by autismsciencefoundation

You know you have piles of dusty CDs and DVDs somewhere in your house. You’ve long since converted everything to digital, so now what do you do with all those jewel cases? Donate them, of course!

CDSweep (www.cdsweep.com) launches today as an easy way to do a clean sweep of all your unwanted CDs and DVDs and help fund critical autism research. Just box up all of your old discs, ship them free with prepaid FedEx labels, and for each disc received a donation will be made to ASF.

Go to www.CDSweep.com, enter the amount of discs you’d like to send, and you’ll be emailed a prepaid FedEx label to ship them all for free. Your donation is tax-deductible and provides funding directly to scientists conducting innovative autism research and supports the needs of people with autism and their families. Each disk will be valued at up to 50 cents each! You can even request that your CDs be digitized and sent back to you as MP3s for free.

A HUGE THANK YOU goes to the volunteer committee who put this project together. Thank you team!

Jason Roth, Founder

Ken Waagner, Smartley-Dunn Solutions, Ltd.

Seth Mnookin, author, “The Panic Virus”

Debbi Dougherty-Roth, ad:tech

Sarah Wefald, graphic designer

Kris Schrey, iPodmeister

Better Diagnosis Through DNA Testing

DNA Scans Can Spot Cause Of Brain Issue In 25% Of Kids

By Robert Langreth - Jun 13, 2012 2:00 PM ET

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Scanning the genes of children with inherited brain disorders pinpoints the precise cause more than a quarter of the time, often changing the diagnosis, according to one of the largest studies of child DNA sequencing.

The study published today in Science Translational Medicine examined the genes in 118 people with child-onset brain development disorders where obvious causes had been excluded. Researchers found new disease-causing mutations in 22 patients. In 10 more people, the scans found rare genetic diseases that had been misdiagnosed because symptoms were atypical. In two cases, the new diagnosis led to changes in treatment.

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The study “is a remarkable demonstration of the power of sequencing in the clinic -- precise, molecular, root cause diagnosis,” said Eric Topol, a professor of genomics at the Scripps Research Institute in San Diego in an e-mail. “Virtually all diseases” will be more precisely defined through gene sequencing in the not-too-distant future, he wrote.

The research shows how often DNA scans can improve diagnosis in kids with brain disorders such as autism, severe epilepsy, or intellectual disability, said Joseph Gleeson, a child neurologist and study author. Brain development disorders afflict as much as 4 percent of children and are often genetic, yet the precise causes can be elusive, he said. Sequencing lets researchers examine all genes for abnormalities, instead of testing for one genetic disorder at a time as done now.

Biggest Surprise

The biggest surprise is that some children with genetic brain disorders have diseases that have been known for a long time, said Gleeson, of the University of California, San Diego. The kids weren’t diagnosed correctly because “frequently their symptoms don’t match those in the textbook.”

As a consequence, doctors may not have thought of ordering gene tests for those rare diseases, Gleeson said.

“This is taking the whole medical field by surprise,” Gleeson said. “It used to be that the gray-haired professor was the gold standard, and if patient didn’t respond to treatment then patient was an anomaly.”

Gene sequencing eliminates the problem of misleading or vague symptoms by testing all genes, Gleeson said. “It will change the way we practice medicine,” he said.

The study follows research published in the June issue of the Journal of Medical Genetics, where scientists at Duke University used sequencing to diagnose six of 12 kids with genetic disease of unknown origin. In five of the cases, the children had had mutations in genes known to cause disease, said David Goldstein, the study leader and director of the Center for Human Genome Variation at Duke’s School of Medicine in Durham, North Carolina. One child had been diagnosed with autism, yet turned out to actually have Pitt-Hopkins Syndrome.

Absolutely Transformational

For diagnosing pediatric genetic disease, sequencing “is an absolutely transformational technology,” Goldstein said in a telephone interview.

“It is a much larger, more important application than people were anticipating,” Goldstein said. Today’s study confirms that sequencing can revise the diagnosis in many cases, he said.

Gene sequencing produces huge volumes of data that can be hard to interpret, UCSD’s Gleeson said. In the Science Translational Medicine study released today, Gleeson and colleagues simplified the analysis by studying sick children of married cousins in countries in the Middle East, North Africa, and Central Asia, where such relationships are common. The families had multiple relatives afflicted by genetic disease, making it easier to find inherited mutations, he said.

Exome Sequencing

The California researchers worked with scientists at Broad Institute in Cambridge,Massachusetts, to scan the genes in the 118 patients. This technology is called exome sequencing and looks at the 1 percent of the DNA that contains genes that create proteins. The researchers used equipment from San Diego- based Illumina Inc. (ILMN) and elsewhere to perform the sequencing.

Translating the sequencing technology into everyday medical practice will be a challenge, Gleeson said, as doctors are “sorely prepared” for the data onslaught. “We don’t have computational algorithms that can manage the analysis and we don’t have doctors that can interpret the data,” he said.

Duke’s Goldstein said the technology can be applied rapidly. ‘There is nothing that prohibits this from being done on scale,’’ he said.

The technology may save money because the raw cost of sequencing the 1 to 2 percent of the genome that contains genes now cost less than $1,000, not including cost of interpreting the data, Goldstein said.

For some patients “it is clearly less than has been spent on gene-by-gene tests” that cost several thousand dollars per gene, Goldstein said.

To contact the reporters on this story: Robert Langreth in New York atrlangreth@bloomberg.net;

To contact the editor responsible for this story: Reg Gale at rgale5@bloomberg.net

Financial Advice for Parents of Autistic Individuals

I have not read this book, and can not endorse it. I would be interested in comments from anyone who may have read it.

Adviser Creates Guide to Financial Side of Raising an Autistic Child

A financial adviser and father of an autistic son, Greg Zibricky has taken his firsthand knowledge and written a book—"F.A.M.I.L.Y. Autism Guide: Your Financial Blueprint to Autism"—to help similar parents navigate money, insurance and legal issues.

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• June 4, 2012

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Greg Zibricky knows that feeling of being told your child is autistic. It was 15 years ago when he and his wife, Dawn, found out their then-3-year-old son Aaron was diagnosed with the disorder.

Zibricky knows how overwhelming it can be to keep track of the day-to-day tasks involved with treatment, like medications and therapy appointments and doctor visits. In those situations, parents are thinking about the present physical health of their child, not his or her financial future.

That's why Zibricky, a certified financial adviser for 27 years and president of Provider Group in Frankfort, has written a book, F.A.M.I.L.Y. Autism Guide: Your Financial Blueprint to Autism, to give parents of autistic children a head start when it comes to financial planning, from the perspective of a father who has been there.

"Families have enough coming at them whether their kids are 3, 13 or 23, that it's very oppressive for them to think, 'I have to see an attorney.' 'I have to see a financial adviser," said Zibricky, a New Lenox resident. "I wanted them to have a real-life guide to kind of give them a jump-start. ... If a family has access to this material, that cuts down the amount of insecurity and the amount of unknowns they have."

Although Zibricky had never written a book ("I wrote a screenplay once. It wasn't very good."), he said he thought it was the best way to distill the knowledge he had developed over the past 10 years he's been doing this type of financial planning for his family, as well as for his Provider Group clients in similar situations. He's taken that information—culled from the individual timelines and plans he's created for dozens of families—and streamlined it for a larger audience, he said.

But the book doesn't simply answer questions like how to handle health insurance or apply for government benefits, it also lays out coping strategies for parents. For instance, the "F" in F.A.M.I.L.Y. stands for "Flexibility," and that chapter deals more with adjusting attitudes and outlook than it does cold, hard financial facts. Zibricky illustrates his ideas in that chapter—and throughout the book—with personal anecdotes of his family's experiences. (The other letters in the title, by the way, stand for Access, Money, Insurance and Yap, which stresses the importance of communication.)

In fact, Zibricky considers his personal perspective to be the book's most valuable asset. Even with his specialized financial know-how and his wife's nursing background, the Zibrickys faced—and still face—many of the same challenges and questions families without their training encounter.

"What shapes the book is the first-hand experience," he said. "As a parent you're approached quite often by folks who say they're familiar with your plight. But unless you talk to another parent, the reality is that's not the case."

"Here's a family that had the benefits of a mother who was a nurse, a mother ... who was active in the disability community before Aaron was diagnosed," he added. "And you know, here's the father who's a certified financial planner. ... If you're going to have two parents who could help, we were the ones, and we recognized that. And we find that to be powerful, because it's coming from the point of view that we had advantages. And there are other families who didn't have a medical background, didn't have a financial background, and had to deal with the same stuff we did, and even (with our advantages), it's hard enough."

The next step for Zibricky is to start to promote the book, which is on sale at outlets like Amazon andBarnes & Noble. In fact, he's already using social media, such as Facebook and LinkedIn, to spread the word.

As the book hits shelves, Aaron is just turning 19 and has graduated high school. It's a bit of synchronicity that isn't lost on Zibricky.

"The last 10 years have been everything," he said. "They've been trying, frustrating, exciting. But in a sense, we're just getting started."

Exercise DVD for Autistic Individuals

I have not used or viewed the following product, and so can not endorse it. If anyone tries it and would like to submit feedback about it, it would be welcome here.

New DVD Showcases Fitness Activities That Improve Socialization Skills For Children With Autism

WILLOWBROOK, Ill., June 7, 2012 /PRNewswire/ -- More than half of the nation's children with autism are overweight, at risk for diabetes or cardiovascular disease, have bone and joint problems or suffer from depression, anxiety and gastrointestinal problems. That alarming statistic...

 7 JUN 2012 WDM Group PR Network

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WILLOWBROOK, Ill., June 7, 2012 /PRNewswire/ -- More than half of the nation's children with autism are overweight, at risk for diabetes or cardiovascular disease, have bone and joint problems or suffer from depression, anxiety and gastrointestinal problems. That alarming statistic was the impetus for a new exercise video for children with autism, hosted by nationally recognized fitness expert Suzanne Gray.  

Gray, who is CEO of Right Fit Sports Fitness Wellness, Willowbrook, has spent more than 30 years developing fitness programs for children with special needs.  Over time, she has seen how exercise works in addressing obesity, improving motor skills and muscle tone, eliminating self-destructive and self-stimulating behaviors and enhancing socialization skills for children with autism.  The DVD, entitled, Raising the Bar: Fitness & Movement Exercises for Youth and Adults with Autism, was produced by Healthy Learning and is now available to order online at www.Amazon.com, www.Right-Fit.com and www.healthylearning.com. 

The specific developmental exercises and movement activities featured in the DVD are included in Suzanne's innovative "Raise the Bar©" program, which are included in the book 101 Games and Activities for Youth with Autism. Her program received the Club Industry "Best of the Best Award" in the category of "Best Children's Fitness Program."

"The DVD demonstrates specific exercises parents and teachers can do with their children every day," says Gray.  "A structured routine is essential to improving the muscular imbalances and compensations, deficient motor skills, poor posture and other fitness pitfalls common among youth with autism. When you combine a fitness program with music and play, it enhances the way a child with autism responds to sensory information and it will boost independence, happiness and self-esteem."

ABOUT SUZANNE M. GRAY

Gray's programs have been implemented by community centers, schools, and park districts across the country. They have been deployed at the nationally recognized Argonne National Laboratory and with several private and not-for-profit organizations, such as Charlie's Gift, Hinsdale, IL; and Turning Pointe Autism Foundation, Naperville, IL.  These organizations/schools support and bring awareness to special needs children who struggle with various issues, such as socialization, motor skills and obesity.

For more information on Suzanne M. Gray or Right-Fit, log on to www.Right-Fit.com, or www.suzannemgray.com.

SOURCE Suzanne M. Gray