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Drug To Treat Autism’s Core Symptoms May Soon Be Reality


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In what could signal a turning point, researchers say they’ve identified a drug compound that may address two key facets of autism — repetitive behaviors and socialization.

The compound known as GRN-529 is showing significant promise when tested in mice, giving researchers hope that it could lead to a biomedical treatment for people with autism.

In a study published Wednesday in the journal Science Translational Medicine, researchers tested the compound on a strain of mice known to exhibit autism-like behaviors. They found that mice that received the treatment spent less time repeatedly grooming themselves and more time sniffing noses with other mice as compared to mice who did not receive the drug.

While treatments that are successful in animals don’t always have comparable results in people, scientists say they are particularly optimistic about the potential for this compound. That’s because it is similar to a drug that’s already in clinical trials for treatment of fragile X syndrome, which has many overlapping features with autism.

“Our findings suggest a strategy for developing a single treatment that could target multiple diagnostic symptoms,” said Jacqueline Crawley of the National Institute of Mental Health who worked on the study.

Perhaps even more significant, the findings could alter the way autism is perceived by offering evidence that core symptoms of the disorder may be treatable with medication, the researchers said.

“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,” said Thomas Insel, director of the National Institute of Mental Health.

Currently the only drugs approved specifically for autism are Risperdal and Abilify, antipsychotics that are designed to address irritability associated with the disorder rather than its core issues.

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Comments (18 Responses)

  1. Paula C. Durbin-Westby says:

    Unless the researchers know *exactly* what purpose the repetitive behaviors are serving, they (and you parents who are considering giving your children this drug) should tread with extreme caution. Many repetitive behaviors serve functions that help Autistic people adapt so that we can function in numerous ways, such as being able to concentrate (at school and at work), process visual and auditory information. Since “repeatedly grooming themselves” seems to be a feature of “autistic mice” and NOT of actual Autistic people, once again, I think researchers are perhaps jumping to conclusions about the socialization aspect.

  2. Barb says:

    Another drug, another “cure”. When will people understand that this is a genetic disorder? You can’t “cure” it with drugs, just like you can’t “cure” other genetic disorders like Down syndrome.

    Now, if the drug will help with some of the symptoms such as repetative behavior, all well and good. But will it change the personality of the person with Autism? I hate seeing individuals with Autism on drugs such as Risperdal and they are like walking zombies.

  3. KA101 says:

    I’d just as soon not be non-voluntarily drugged. Thanks much.

  4. Teri says:

    This article makes me sick! Why is it not okay for a child with a so-called disability to display symptoms just because it bothers some people? Those are the people who we should be finding medication for! Look at all the great people with Autism and Asperger’s and their contributions to society and the world. What would have become of them had someone drugged them into submission and turned their brains to mush. I beg schools, parents, and doctors to think about what they do before these children are drugged. Leave these kids alone! Let them be who they are! It’s okay!

  5. cindy says:


  6. Jon K. Evans says:

    I had a classmate declare that Marijuana enables you to get along better with people. That was back in 1970. To this day, I believed that to be a dangerous statement. A few years later, it was suggested that I be on Psychoactive Medications.
    Psychoactive Medications, unless they target a particular neurological function, are stoned dangerous! The idea that a drug can improve socialization is tantamount to prescribing Crystal Meth for symptoms of lethargy!

  7. MomofanAutie says:

    New possible medical treatments are always scary. I would want to see more testing on this in the animal world before it made the jump to humans. Even then, I couldn’t make my child a guinea pig. We have always eschewed drugs that are brand-spanking-new because I think experimental evidence as well as anecdotal from the real world are invaluable.

  8. Kim says:

    My son has severe au and suffers terribly from repetitive behaviors and we have had him on a tight ABA schedule for over 6 years with some improvement but our lives remain challenging and he is often stressed to the point of tears and even aggression. We would welcome some help with finding another source to address this ever challenging behavior as we strive to improve his quality of life.

  9. Jennifer Peterson says:

    So GRN-529 is the newest social lubricant? Alcohol does the same thing. So does pot. I’d try this before giving it to my kid.

  10. Edward says:

    Barb said “When will people understand that this is a genetic disorder? You can’t “cure” it with drugs, just like you can’t “cure” other genetic disorders like Down syndrome.” The notion that genetic disorders cannot be treated with drugs is just plain wrong. It may be true that these diseases cannot be “cured” by drugs but they can be treated. Recent developments in the treatment of diseases such as hemophilia, phenylketonurea and cystic fibrosis are the most obvious examples. That being said, caution is always warranted when new “breakthroughs” are announced (seemingly about once every 5 years) and even more skepticism is required with such preliminary animal studies.

  11. Johannes says:

    Will it enable people with autism to leave happier lives? May be not looking strange will be an advantage, or may be society should just get used to it that some of us have quirks. The word cure is used too quickly. Happy lives and function are more important.

  12. Rosella A. Alm says:

    I wonder how such a world-changing medication would affect a person who is older, like my son. He will be 47 in September, and has no verbal ability, even though I believe he understands most, if not all conversation around him. I think changing his perceptions would be highly traumatic to him. He can be exactly the way he is, and enjoy life on his own terms, and have his own world view.

  13. Jackie says:

    I agree with Kim. I didn’t read anything about the rats turning into zombies. No one thinks this is a cure. But, when you have a headache you take Advil. It would help to treat the symptoms. Opinions are like noses everbody has one. Don’t judge or give advice or recommendations when it’s truly bias. As a parent with an AU child… This is encouraging!!

  14. Vikki Stefans says:

    Great! Now if any autistic mice come to my clinic, I’ll know where to refer them! Of course, I’m still waiting for the mdx mice to drop in…I fantasize about telling a mommy and daddy mouse on their first visit after diagnosis, don’t worry, it’s just muscular dystrophy and we’ve got ever so many treatments that work really well for that nowadays! Too bad for those humans! Good article, just wishing we’d have something new sooner rather than later. I’ve got a couple kids on very low dose risperidone and its helpful, but they are going to start requiring intensive monitoring for insulin resistance issues apparently per a letter from Medicaid.

  15. Vikki Stefans says:

    “Another drug, another “cure”. When will people understand that this is a genetic disorder? You can’t “cure” it with drugs, just like you can’t “cure” other genetic disorders like Down syndrome.”

    Good point, you can’t necessariy fix genes directly, but if you understand the molecular mechanisms of what’s going on, you can know what to do to make your life or your ability to function better. Hopefully without being zombified or otherwise messed up by side effects. I’m facing that myself with a bad diabesity gene I found out about – its exciting to have a better idea of what’s wrong and what’s likely to help or harm, but also scary to know there is nothing that will remove the problem entirely.

  16. Justen says:

    It’s seems like in every case, Big Pharma looks to prey on people to make more money with their synthetic concoctions. One drug always leads to another. They are feeding for a temporary solution, in which to gain the trust of progress, and get our kids hooked on something else so they can counter act it again with another one their clever substances. Why not spend the money on things that work such as ABA programs, speech therapy, natural remedies, Stem Cell Research, etc… Because it works, it takes Human involvement and none of these ‘So called Concerned’ Lab Monkeys are in it for a cure, they are in it to prolong the disability, and to make their $$$.

  17. Tammi says:

    As a parent of a childwith Asperger’s and a professional who works with other parents and children I can gaurantee there are parents who are asking how they can get in on the study, just to get it sooner. No I am not judging, because I know. I see some comments here that just shows how important social function really is. My own child has a few friends, but is often made fun of at school, often has problems with getting work done, functioning at her age level and even being able to dress herself properly. No biggie right? She is almost 12, looks perfectly normal and acts like she is on illegal drugs (quoting someone else). As a parent it is frustrating to watch her stumble, it is frustrating to have to deny her many of the freedoms of other kids her age. It is scary to leave her home alone at all, and when I do I make sure there is no-cook food and something to occupy her. To have a day that she can pick appropriate clothes, without the sensory issues, go to a friend’s house without driving their mother to ask her to go home, a day that she can stop talking for a while, or even have some interest in more than one or two subjects, where homework isn’t such a struggle just because she isn’t interested in the subject matter. I can understand, especially when I catch myself watching her for socially unacceptable behavior, to stop her before she embarasses herself and to try to explain why she can’t do that and make her understand. I love my child, but I want her to be able to truly live and explore.

  18. Marie Eugene says:

    I am a parent with an autistic child. When will GRN-529 be ready? will it be something afforadble only to the rich and famous? You can feel by the tone of my writing that i am a frustrated parent who does not think that enough has been done to address this hurricane that ravaging our lives and destroying our children’ future.

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