Home > Got Questions?, Science > Are there effective medicines for treating core autism symptoms?

Are there effective medicines for treating core autism symptoms?


This week’s “Got Questions” answer comes from Joseph Horrigan, MD, Autism Speaks assistant vice president, head of medical research.

First, it’s important to note that medicines for treating autism are most effective when used in conjunction with behavioral therapies. Ideally, medicines are a complement to other treatment strategies.

Medicines for treating autism’s three core symptoms—communication difficulties, social challenges and repetitive behavior—have long represented a huge area of unmet need. Unfortunately, few drugs on the market today effectively relieve these symptoms and none of the options most often prescribed by practitioners work well for every individual.

In fact, while the Food and Drug Administration (FDA) has approved two drugs for treating irritability associated with the autism (risperidone and aripiprazole), it has yet to approve a medicine for treating autism’s three core characteristics. Nonetheless, medicines such as risperidone and aripiprazole can be beneficial in ways that can ease these core symptoms, because relieving irritability often improves sociability while reducing tantrums, aggressive outbursts and self-injurious behaviors.

The good news is that the range of medication options may soon change, thanks to recent advances in our understanding of the biology that produces autism’s core symptoms. This has made it possible for researchers to begin testing compounds that may help normalize crucial brain functions involved in autism. Early experiments suggest that several compounds with different mechanisms of action have great potential for clinical use, and many are now in clinical trials. [This link takes you to the search engine of the NIH clinical trial network, with results under the search term “autism.”]

Although these developments are exciting and hold real promise for bettering the lives of people with autism, we will have to wait at least a few more years before we know if any of these drug studies produce enough information on safety and effectiveness to merit FDA approval for the treatment of core symptoms.

Today, most medicines prescribed to ease autism’s disabling symptoms are used “off label,” meaning that their FDA approval is for other, sometimes-related conditions such as attention deficit hyperactivity disorder (ADHD), sleep disturbances or depression. Such off-label use is common in virtually all areas of medicine and is usually done to relieve significant suffering in the absence of sufficiently large and targeted studies.

An example in autism would be the class of medicines known as selective serotonin re-uptake inhibitors (SSRIs), including fluoxetine. Several of these medicines are FDA-approved for the treatment of anxiety disorders and depression, in children as well as adults. Although large clinical trials have yet to demonstrate their effectiveness, parents and clinicians have found that they can  ease social difficulties among some people with autism. However, it has proven to be difficult to predict which medicines in this class may produce the greatest benefit for a given patient with autism. Similarly, determining the best dose can be quite challenging.

Another example would be naltrexone, which is FDA-approved for the treatment of alcohol and opioid addictions. It can ease disabling repetitive and self-injurious behaviors in some children and adults with autism.

These medicines do not work for everyone, and all medicines have side effects. And as noted above, each person may respond differently to medicines. In addition, changes in response to a medicine can occur as time goes on, even when the dose is not changed. Over time, some people develop tolerance (when a drug stops being effective) or sensitization (when side effects worsen).

Because using these medications in children and adolescents can be a difficult decision for parents, you may find it helpful to use our Medication Decision Tool Kit, a guide for actively working with a physician to find the approach that fits best with your values and goals. You can download it free here.

These are exciting times in the development of new medicines for relieving autism’s most disabling symptoms, and Autism Speaks is increasing its funding and focus in this promising area, while placing great emphasis on ensuring the safety of promising new medicines. Please stay tuned!

Read more science news and perspective on the Science Page.

  1. Janice Miller
    December 9, 2011 at 10:01 am

    It may be useful if doctors could understand how serotonin and dopamine function in the brain; but the knowledge may not translate into being able to treat the core pain of 90% of children diagnosed with autism. Most children diagnosed with autism are really dealing with inflammation and infections due to problems with their immune system. Digestive problems can affect the immune system in an adverse way.

    Shouldn’t the advise be to regulate the digestive system before any psychotropic medication would be prescribed? Most psychotropic medications are in a class of medications that inhibit and aggravate the digestive and respiratory systems, and further complicate immune issues. I know children who have had diarrhea for years with no relief… Why prescribe anti-behavioral medication while ignoring the fact that digestion is not occurring? Couldn’t the repetitive behaviors result simply from the child trying to digest their food and rid their body of toxins?

    “The good news is that the range of medication options may soon change, thanks to recent advances in our understanding of the biology that produces autism’s core symptoms. ” Yes, soon scientists will research zinc levels instead of dopamine levels.

    Here is a good example, “Curemark Receives Investigational New Drug Clearance For CM-AT For Autism”

    “With 1 in 150 children diagnosed with autism, we have children who are suffering. We are very excited about the opportunity to enter a Phase III trial for this ever-growing population of children.”

    “As part of Curemark’s ENCAPTASE(TM) technology platform, CM-AT is based upon the observation that many children with autism do not digest protein. CM-AT is a proprietary therapy formulated to be released in the small intestine of children with autism. Designed as an easily utilized powder taken three times a day, CM-AT was developed with the children in the forefront. The administration of CM-AT with meals allows for an increase in protein digestion thus potentially reducing allergy and increasing the availability of essential amino acids. With proper protein digestion in a subset of children with autism, the need for protein restricted diets, such as gluten-free and casein-free diets, to which many children with autism have become accustomed, may no longer be necessary. Being able to digest protein rather than avoid it will allow children with autism greater access to the building blocks for the manufacture of new proteins such as neurotransmitters. A partial or complete lack of protein digestion could further lead to other gastrointestinal and digestive dysfunctions.”

    http://autismspeaksnetwork.ning.com/forum/topics/curemark-receives-investigational-new-drug-clearance-for-cm-at

    • Jordan
      December 9, 2011 at 6:14 pm

      A lot of what you describe (persistent diarrhea, infections) seem to describe what my son goes through. Do you have any suggestions that might help? We are new to this. Thank you for any help you can give.

  2. becki
    December 9, 2011 at 10:35 am

    my son has aspbergers and he has his days where he flaps his hands like crazy and days where he barley does it.is there anything i can do to help him control it?

    • December 9, 2011 at 4:38 pm

      I also have a 9 yr old son with Asperger’s, when he gets restless he cannot stop moving his hands. There is stuff called tacky tape, it hangs posters on the wall without damage, its like a putty. I buy it at Walmart and he loves it. Whenever he gets fidgety he keeps it in his hands and it works!

  3. becki showers
    December 9, 2011 at 10:38 am

    my son has days where he flaps his hand like crazy and there are days he barley does it. is there anything i can do to help him control it?

  4. Katie Wright
    December 9, 2011 at 1:59 pm

    An over-emphasis on meds here concerns me. Sometimes meds are necessary and do help but all too often doctors have no idea that there are other safer interventions available today for ASD kids. Meanwhile Risperdal, commonly prescribed to ASD kids causes significant weight gain and increases risk for diabetes.

    I have also been saddened seeing a number of ASD boys have to be temporarily hospitalized in order to get off powerful psychiatric meds.

    We need a more holistic approach to ASD kids. TACA.com or talk about curing autism has phenomenal website. Great here and now concrete advice/ info on biological interventions. Nourishing Hope is, in my opinion, the best ASD dietary intervention resource.

  5. Andie
    December 9, 2011 at 4:51 pm

    Please treat the underlying medical issues first, find a physician that recognizes the gut brain connection and knows how to medically test for all physiological issues. Putting a band-aide on a broken arm is not going to set it straight – same thing with band-aiding our kiddos behaviors issues will not take away any of the medical issues. Best to all you warrior moms and dads. http://autism.com

  6. Peter Sinclair
    December 9, 2011 at 5:22 pm

    Do any medicines show promise in addressing core deficits (speech, sociability)? Seems to focus of the article is more about reducing “problem behaviors” rather than “filling gaps”.

  7. Kathleen Ward
    December 9, 2011 at 9:29 pm

    My granddaughter who I adopted is 9 yrs old and is Autistic. For a few years I was trying the bio-meds on my own but then realized there has to be more I can do. I took her to a D.A.N Doctor and they did all the test and put her on a AM/PM Compound, L-Taurine, DMAE, Therabiotics, Nystantin for 6 mos, Omega Swirl and Methyl B-12 shots. I can not believe she is the same child. She is not totally over some of the Autistic traits but she is so much better. I know there is big time controverse over Bio-Meds and D.A.N Doctors but I know what they did for my granddaughter is wonderful so far. I think more research into natural compounds should be studied. There is to much prescription drugs given to kids just to mask the problem.

  8. December 10, 2011 at 12:24 pm

    Autism obviously is a multifactorial and heterogeneous disorder. As has been stated, most drugs used for treating have strikingly little evidence to support their use. I believe that adysfunctional gut and nutrition play a cricial role in the pathogenesis of initiating and sustaining the disorders. While well meaning people may disagree, it is my opinion (based on limited evidence and my expertize in GI and nutrition) that a multipronged strategy likely to have the best chance of success in many and not necessarily all patients. My suggestions based on above hypothesis are included in this article http://goo.gl/amSwZ

  9. BJBlackler
    December 11, 2011 at 9:10 am

    Big-Pharma loves Autism Speaks and their pro-med approach. Why not at least, once in a while, shed a light on some of the dietary and/or biomedical interventions that parents are finding successful?

  10. Poohmommy
    December 16, 2011 at 2:28 pm

    I’ve read alot about the dietary solutions, but the problem I have with them is getting my son to eat what’s on the diet. Picky does not even begin to describe him and his step-mother, who has primary physical custody at the moment, doesn’t care what he eats. He’s almost 16 and weighs over 200 lbs. He’s been on Risperdal, but had to be taken off of it due to issues with his liver and has been trapsing through the other gambit of drugs as none seem to work as well as the Risperdal.

    I never wanted him on drugs in the first place, but was bullied into it by his school district. Now I’m worried sick about his weight because his father died of complications from Type II diabetes and I don’t want my son going the same way.

    All I want for him is to be able to live as normal a life as I can get him because he has no family except me and there’s going to come a time when I’m not here to watch out for him.

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