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New Therapies in Autism: Part One

Solomon Behar, MD and Shafali Jeste, MD

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Autism expert Shafali Jeste helps us sort out legitimate from unproven autism therapies and manage symptoms associated with autism. She also give tips on how to vet new proposed autism therapies.


  • When discussing autism treatments, it is important to clarify whether the treatment is directed at the the core symptoms of autism or at the potential comorbidities.  Behavioral intervention is the only known therapy for improving the core symptoms.

  • Insomnia should be aggressively treated in a patient with autism.  Treatment includes a strict bedtime routine +/- melatonin, +/- an alpha-agonist.

  • Chelation therapy, hyperbaric oxygen and/or the ketogenic diet should not be recommended; additionally, these therapies can also be harmful.


  • How is autism defined?  Autism is defined by having core issues, problems and/or challenges in social skills and communication; as well as having repetitive behaviors and restricted interests.  This is important to keep in mind as there are therapies directed at these core symptoms and other therapies that target other comorbidities (i.e. insomnia, anxiety) that come with autism.

  • What therapies are widely accepted in the management of autism?  While it may not sound satisfying per se, the gold standard intervention and treatment for the core symptoms of autism is behavioral intervention.  In 2001, the National Academy published recommendations for educational interventions in children with autism and these recommendations are still used as first line today.  The committee recommends that all children with autism have 30-40 hours of school based interventional services targeting the core symptoms.  There is not one medicine that treats the core symptoms of autism.

    • The behavioral interventions are varied, but all go under the realm of Applied Behavior Analysis (ABA).  ABA is often misunderstood in that many think of ABA as discrete trial training; that is structured intervention when there is an adult with the child and the adult is coaching the child on certain skills over and over.

    • After the diagnosis is made and behavioral intervention is recommended, it can be difficult for families to navigate the school resources and understand the insurance coverage.

      • In the face of an overwhelming body of information, navigating these resources and therapies can be very challenging and families should not be faulted for asking about “experimental” treatments.

  • Are there pharmacologic therapies being studied?  Autism is not one condition and the clinical presentation is extremely varied.  Having one medication that targets all the variable symptoms and ideologies is tough and likely not to be reached.  However, the field is moving towards precision therapies in which specific subtypes (based on symptoms) are recognized and drug treatments may be available by sub-type.   

  • Are there vitamins, supplements or diets that are used in the treatment of autism?  The way to frame this question should be in terms of cost and benefit and, of course, the supplements should not be harmful.  There is a middle ground for these dietary supplements and the gluten-free or casein-free diet. Taking a balanced approach may be useful and can be stated as “we do not have data that support these therapies.  However, if the dietary approach is to remove preservatives and simple sugars and provide a healthy balanced diet, that may help behaviors.” The indirect costs of a gluten-free diet, for example, are in the quality of life it may impose on the family.  A recent meta-analysis did not show a true benefit of the gluten-free diet in treating the core symptoms of autism and don’t have enough evidence to support it.  Secondary analysis in some of the smaller trials have shown that sometimes irritability and behavior dysregulation can actually improve.  

Elder et al.  A review of gluten- and casein-free diets for treatment of autism: 2005-2015.  Nutr Diet Suppl. 2015; 7:87-101. PMCID: PMC522335

  • What therapies are harmful?  

    • Chelation therapy has been suggested in the treatment of autism and this is a harmful treatment (has lead to deaths) and should not be recommended.  This therapy is marketed as “pulling toxins out of the child’s blood that might be causing developmental delays.”  

    • Hyperbaric oxygen is another treatment that is not entirely clear if it is medically harmful, but it is incredibly expensive and can cause great strain on the family.  

    • The ketogenic diet can be dangerous because children can become acidotic and limit intake.

  • What therapies are FDA approved?  The atypical psychotic Risperidone (Risperdal) and Aripiprazole (Abilify) were both studied in large clinical trials and shown to improve specifically the symptom of irritability, as defined by a subscale of the Aberrant Behavior Checklist.

    • Risperidone has been used longer.  The main side effects are weight gain and sedation.  There have been reports that it can cause hyperprolactinemia.

    • Aripiprazole can also cause weight gain and sedation, although the side effects appear to be less severe.

  • How can insomnia be treated in autism?  Insomnia affects up to 80% of children with autism and, not surprisingly has a negative impact on the quality of life of the child and family.  Therefore, it should always be asked about (some families are reluctant to bring it up) and should be be treated aggressively.  Insomnia in autism is a pattern of difficulty falling asleep and difficulty staying asleep, with multiple night time wakings. This is very challenging for families; unlike a newborn who may be rocked back to sleep, for example, a child with autism who wakes in the middle of the night because of sensory sensitivities or anxiety, or for another neurobiological reason can be very hard to get back to sleep. In addition to behavioral strategies to help develop a nighttime routine, medications can be used.

    • Melatonin, a natural neuro hormone helps the body know when it is time to go to sleep.  There have been many large clinical trials that have shown good effects in helping to improve the onset of sleep.  It should be given about 2 hours prior to bedtime and after it is given, screen time should be avoided as the body does not produce natural melatonin when it’s seeing a lot of light.  

      • A good starting dose is 3 mg for a small child and can be started as young as 2 ½-3 years old.  Clinical trials have looked at doses of 6-9 mg and generally start at age 6.

        • Autism Speaks has a helpful toolkit on sleep issues and insomnia in autism.

        • Note, melatonin is not FDA approve as it is a dietary supplement.  There is no one perfect brand; if a family finds a brand that is working, stick with that.  Many brands, such as Nature’s Best, have liquid and gummy formulations too. Labels should be read carefully as each brand may have a different formulation.

          • Ask the parents to bring in the the supplements they are giving to their child and make sure that there are no harmful additives.  Asking a pharmacist about interactions may be helpful.

    • Alpha-agonists, clonidine or guanfacine, may be used to help the child stay asleep and may be used in combination with melatonin.  Clonidine has been widely studied and is the most commonly used and well studied. It should be given about an hour before bedtime.  Guanfacine is a longer acting and can be used in conjunction; however, it has not been as widely studied as clonidine.

Malow, BA et al.  A practice pathway for the identification, evaluation, and management of insomnia in children and adolescents with autism spectrum disorders.  Pediatrics 2012 Nov; 130 Suppl2:S106-24. PMID 23118242


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Can I Get A Witness? Full episode audio for MD edition 178:02 min - 84 MB - M4AHippo Peds RAP April 2018 Written Summary 403 KB - PDF