Autism What about Drugs?

Beth Hodges, MD

What role can traditional medications play in the treatment of autism spectrum disorders (ASD)? The fact of the matter is…it depends. Make no mistake--autism cannot be cured with a single pill. The major role of medication is to control anxiety, agitation, and other disruptive feelings and behaviors that make it harder for autistic children to integrate into the family and school and to benefit from therapy. Think about it like this: a guy who has just had a nerve-shattering argument with his girlfriend cannot sit down immediately and study in an effective manner for a midterm test. Autistic children can feel unsettled like that all the time. The goal for using medication is not to sedate the child, but simply to provide a tool to help the child block out disruptive stimuli and focus on the task at hand, whether it be school, therapy, family time, or sleep.

Which medication/s to choose depends on the main symptoms being addressed. For a child with sleep cycle disruption, something like Tofranil (imipramine), a sedating antidepressant, may be the most sensible agent to use. Hyperactivity and an inability to settle into the school routine or therapy may benefit from a stimulant such as you would use for a child with attention deficit disorder (ADD/ADHD), though these drugs are certainly not as effective for the ASD child as for an ADD/ADHD child. A child prone to horrific temper tantrums may need an atypical antipsychotic, such as Abilify or Risperdal or a mood stabilizer, such as lithium or Depakote (valproic acid.) An autistic child who also suffers from tics (ie Tourette’s syndrome) can benefit from Orap (pimozide.) Clonidine, an alpha blocker developed to treat high blood pressure, can be helpful for calming mood when used at a low dose and has fewer side effects than some other medications.

The bottom line when choosing medication for ASD is communication with the health care provider. First, make sure you have picked a provider with a lot of knowledge and skill in treating autism and in using the medications under considerations. This person could be a child psychiatrist, a pediatric developmental specialist, or even a family physician or pediatrician with a special interest in the area.

Be clear about your goal. What symptom are you treating? How long should it take to see results? What benign or dangerous side effects could occur? What to do if a dose is missed or inadvertently given twice?

If you agree with the provider to start a medication, then start it. If you see a potential problem, report it to the provider. That person needs all the information to make treatment decisions. Many times parents leave the office with a prescription they never fill or fill and only give once. Most of the medications include an adjustment period, and some have initial side effects that will dissipate with time or require weeks to months to become effective.

Most of the drugs used in ASD are not approved for that disorder specifically or for the age group involved and are being used “off label.” Why? Well, it is very difficult to get children enrolled in drug studies, which means approval can only be obtained “down the road” when drug manufacturers can collect and submit retrospective data. As it sounds, this takes many years to compile and considerable expense on the part of the drug manufacturer. If a drug has already gone generic, there is no pharmaceutical company that can afford to spend the millions to apply a receive a “new indication” from the Food and Drug Administration. Because of this, some drugs (like generic imipramine) will never be “approved” for use in ASD children, but that does not alter the fact that it is used and can be beneficial. The keys to the use of medication in ASD are communication with the provider, trust in the recommendations from the provider, and close follow up with the provider. Also expect that as your child grows, the medication needs, both in type and dose, will change with him, so what works this year may not be the best fit for him the next.

It is also important to emphasize that these medications in general get very low scores (2-4/10) on a 1-10 scale from parents in terms of effectiveness. This means that these type of behavior modifying drugs (unlike, say, a dose of morphine for severe pain, which can get an 8/10) do not generate dramatic results or dramatic behavior changes. Expectations with medications tend to be for complete resolution of the symptom being treated. With ASD, that level of expectation from a medication is bound to lead to dissatisfaction. The best pharmacologic intervention can do is to blunt disruptive behavior to allow for more focus, attention, or integration. There is no magic pill for autism. Therapy is the best treatment, along with control of the environment. The greatest need for behavior control drugs in ASD is typically in the teenage boy, and often they are necessary during that time, but they are not a cure-all.