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New Autism Drug Study: A Cautious Approach

Low doses of an anti-anxiety medication improved social interaction and decreased repetitive behaviors in mice, according to research recently published in the journal Neuron. The new autism drug study involved mice bred to have autism-like symptoms. Mice that received low doses of the drug clonazepam showed notable behavioral shifts, like enhanced social interaction, decreased repetitive behaviors and better spatial learning.

The theory behind the new autism drug study is that the medicine may have affected gamma-aminobutyric acid (GABA) receptors, which was associated with reduced core autistic symptoms such as repetitive behaviors. This supports the idea that the neurophysiological basis for autism comes from a neurotransmitter imbalance, which results in core behaviors of repetitive actions and decreased social interaction.

Initial Enthusiasm
Experts who treat children on the autism spectrum are excited about this study. One of them told Scientific American that based on these results, she would consider off-label use of the medication. This response is understandable, because clonazepam is a familiar medication that is already in limited use for patients on the autism spectrum and no other drugs target the disorder’s core symptoms.

However, as a clinician who treats autistic children and as the mother of a child on the spectrum, would I start using it now? This is a complicated question. There is not one sole theory to explain the whole autism spectrum. This research is encouraging, but it is hardly appropriate as a treatment model for children. For instance, it isn’t clear what kind of autistic children may benefit from clonazepam or what dosage would be safe. Would the low dose that worked on mice still apply to humans? Since there is potential to develop dependency on clonazepam, how long would it be safe to administer it?

These are important considerations. Still, this autism drug study is promising and should spur further research. I will be excited to see the results in human trials, which may shed light on how to apply this in practice and whether we have discovered a revolutionizing treatment. As a developmental pediatrician and affected mother, I truly hope so.

This post was written by Saidi Clemente, MD, director of pediatric developmental behavior at Staten Island University Hospital.

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