Home > Science > A new day dawning? First signs of promise for a drug that treats the core symptoms of autism

A new day dawning? First signs of promise for a drug that treats the core symptoms of autism

As many parents know, there currently are no available medical treatments for ASD targeting core autism symptoms.   Available medications target symptoms associated with ASD, such as hyperactivity, irritability, anxiety, or depression. Although the available medicines have helped many who struggle with the challenge of these symptoms, these drugs do not address the difficulties in the areas of social and communication deficits or repetitive behaviors and restricted interests.

Recently, hope has recently been kindled in a new drug for ASD that developed out of basic research on the neural mechanisms of Fragile X syndrome. Back in 2005 research in Dr. Mark Bear’s lab at Harvard showed the Fragile X mutation affects  communication between neurons.  Specifically, the mutation results in an excess of an excitatory neurotransmitter called glutamate, which impairs communication between neurons by making them over-stimulated.  Seeing the potential to help families, a small company called Seaside Therapeutics was started to see if certain drugs could help reduce the level of excitability of neurons.

The drug, arbaclofen, is the first drug being tested. Arbaclofen works by increasing GABA, an inhibitory transmitter, which counteracts the over-excitability of cells.  The preliminary results of a trial conducted with children with Fragile X syndrome looked so promising that Seaside Therapeutics announced the results on this year’s meeting of the International Society for Autism Research (read a review of that meeting’s highlights).  More recently, arbaclofen has been tested in children with ASD without Fragile X. The results of this trial have been reported in the news.  The trial treated 25 children and adolescents with autism for 8 weeks and the preliminary data revealed that arbaclofen was not only well-tolerated but also increase sociability and eye contact, and reduced tantrums and anxiety.

Of course, the testing of this drug continues and a review of the data by independent scientists is essential for evaluating the true benefit of this new drug, however these preliminary results offer good reason for hope and that news is always worth sharing.

  1. September 30, 2010 at 10:04 am

    Very exciting research!

  2. kimberly rivera
    September 30, 2010 at 10:24 am

    It would be so wonderful if we could find something to help make it click in there!! Even though i dont think my child needs medications but if it could help it could be worth a try!!!I have hope that one day we can overcome this disability!

  3. Sharon
    September 30, 2010 at 10:34 am

    when will the study be complete? When will the side effects be available? When will it be approved by the FDA? I am VERY interested in learning more. This would be a miracle for our family. I am not big on medication but honestly at this point if it will increase overall confidence and self worth, it maybe well worth it!

  4. Lydia Bauer
    September 30, 2010 at 10:36 am

    I was wondering when this will be available . My son has all the problems mentioned in the article and we have been trying to find a medication that would help him for the past three years .I would love to give it a try on him . How I would love for him to be able to stay in school one full day and my husband and I to have just one weekend to ourselves. I know it is just as frustrating for him as it is for us not knowing how to help. Please let me know when it will be available. Thanks for Hope, Lydia Bauer

  5. Dawn Sinisi
    September 30, 2010 at 11:39 am

    I myself, an adult with Aspergers would be willing to test/try this medication for research purposes. Please contact me.

  6. Kerri Oastler
    September 30, 2010 at 2:51 pm

    My son takes Gaba in powder form as part of his supplements. I have noticed huge gains in his ability to control his emotions, his anxiety and increases his ability to focus. I would definitely suggest to anyone interested in this drug to try the supplement now. Any natural food store will carry it-ask your naturopath or doctor for dosages. I have found this combined with B12 and Omega 3’s have made my son’s life a lot happier and less stressful. I know this isn’t a “drug” and probably won’t be as effective as the new med but as an option for now and a natural one I would recommend it.

  7. Kate
    September 30, 2010 at 3:19 pm

    Hello. I was prescribed Baclofen last year for severe muscle spasms & spasticity (tightening of the skin). I can relate to this study. I received chemotherapy as a child, which in my opinion is the root of stiffness that I have. The stiffness was so bad I couldn’t make eye contact, understand what I was being taught in school, couldn’t sit still, and I even had some repetition and sensory issues. With the use of Baclofen MANY of my symptoms improved dramatically. Also, my neurologist has prescribed other muscle relaxers that have even further helped with the stiffness and now I can pay attention, etc. My life has taken a 180 since last year and I am so thankful for my neurologist being proactive and sticking by me.

    For those with severe symptoms if oral baclofen is not enough, baclofen can be prescribed in a cream usually combined with another medication. Mine is combined with Amitriptylene (spelling?).

    I have full faith that this medication is the beginning of relief for patients with Fragile X and Autism. If anyone has questions, please email me at cmarts4@gmail.com

  8. Loramath
    October 1, 2010 at 6:10 am

    I must say that most drugs that enter first stage clinical trials usually don’t end up being approved due to different reasons (too many secondary effects, does not work well in humans, a large percentage of patients don’t seem to be helped by it and we don’t know why), etc…

  9. RAJ
    October 1, 2010 at 7:33 am

    Randi Hagerman the principal author of this study receives funding from Seaside Therapeutics, the patent holder and manufacturer of this drug.

    There is a long history of of ties between various autism researchers and the manufacturers of drugs claimed to be a treatment or even ‘cure’ for autism.

    The first being Loretta Bender who claimed dramatic improvement in autistic children given LSD.


    Edward Ritvo a highly respected autism researcher found fenfluramine to induce dramatic improvement in a clinical trial.


    The effects were so dramatic that parents were confronting their pediatricians demanding they prescribe fenfluramine for their children. Worldwide independant clinical trials were conducted and found fenflouromine to be ineffective in the treatment of autistic children.

    Finally, the FDA banned the use of fenfluramine for any use after it was determined that the drug was associated with an inreased risk for inducing heart valve defects.

    More recently Eric Hollander of Autism Speaks claimed that the use of Prozac was an effective autism treatemnt in children as young as two years old. Hollander holds the patent on a formulation of Prozac specifically for treatment of autism. His clinical study noted marked improvement. Large scale independant clinical trial trial were held and found Prozac was no more effective in the treatment of autism than placebo.


    The association between autism researchers and the pharmaceutical industry has been exapnding and drug treatments for autism is a three billion dollar a year industry and is rapidly growing.

    Caution should be taken when clinical trials reporting dramatic imrovements in autistic children when those trials are conducted by autism researchers with a financial interest in those drug treatments.

    Below is the statement in the article by Randi Hagerman and her financial interests with the manufacturer of this drug:

    Competing Interests
    RH has received funding from Seaside Therapeutics, Novartis, Roche, Forest, Johnson & Johnson and Curemark for clinical trials, and also consults with Novartis and Roche regarding clinical trials in fragile X syndrome.

  10. autismspeaksfan
    October 2, 2010 at 3:40 am

    It’s a bit puzzing why some neurodiversity advocates attack kgaccount on you tube when the mum is clearly not in the same camp as vaccines caused my son’s autism group. I’ve seen her videos. This is severe autism. Why wouldn’t the neurodiverity advocates be behing this child?

  11. Patti Russell
    October 2, 2010 at 8:03 pm

    I am interested in finding out more about this new drug to help those with autism. My son is 4 and has autism. We are now doing ABA and some Hypobaric Thereapy with him. We have seen improvement in him but there still alot of the core symtoms we are still seeing. If this new drug would work to improve the core symtoms we would be interested to give it a try. I would like to learn more as it becomes available.

  12. Loramath
    October 4, 2010 at 4:26 am

    Please be careful about these news, you shouldn’t be publishing on this website at this stage, there are desperate parents that will do anything to help their children, even buying experimental drugs on the internet. This article sounds as this was the autism cure we had been looking for, which is probably not.

    We don’t really know if this substance has any effect at all, or worse, it might even be harmful. Or they might sell you something else and pretend it’s this thing. I just want to ask every parent reading this article to be careful with what they give to their children.

  1. September 30, 2010 at 10:06 am

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