Two main approaches are being pursued to identify new medication treatments that may benefit children with Autism Spectrum Disorder. The first and most common approach is to evaluate a treatment in the total group of people affected by ASD. With limited support for common genetic or environmental risk factors that contribute to risk in ASD, treatment studies in the overall group of children with ASD largely attempt to address individual symptom domains by targeting brain systems and pathways that are not necessarily implicated in autism risk. The second approach is almost the exact opposite, to study a medication for ASD-related symptoms in a defined genetic syndrome that confers substantial risk of ASD but comprises <2% of individuals with ASD. Since cellular and animal models are providing an understanding of the underlying neurobiology in these populations, treatments targeted to the root causes of these syndromes may be possible. With emerging knowledge of brain systems and intersections with genetic data, we can hope for a third approach that is somewhere in the middle, with a treatment being studied in a larger subgroup of individuals with ASD that share a common biomarker or biomarkers. As we embark on these different pathways to new treatments, we are also discovering common pitfalls in ASD clinical trials, including mismatches between hypotheses and study populations, substantial “placebo” effects, and subjective outcome measures. Framing these challenges in the context of past successes in ASD treatment research, I will highlight some guideposts as we work toward neurobiologically-based treatments for ASD.
Target Audience: Physicians (psychiatrists, pediatricians, child neurologists), psychologists, social workers, other mental health clinicians and researchers, and students and trainees.
Upon completion of this activity, participants will be able to:
- Describe the evidence base for psychopharmacology in autism spectrum disorder.
- Distinguish symptom-based versus genetic-based interventions in autism spectrum disorder.