Emerging genomic and neuroscience findings have delivered hypotheses that are now being tested in autism spectrum disorder and related genetic syndromes. Unfortunately, these clinical trials have not yet yielded positive results, suggesting a need to step back and evaluate the science of testing new treatments for neurodevelopmental disorders.

In this lecture, Dr. Jeremy Veenstra-VanderWeele will outline critical challenges, both conceptual and practical, to translating genomic, cellular and animal model research into new treatments in autism spectrum disorder (ASD). He will discuss the limitations to conclusions drawn from work in the laboratory as they are extrapolated to the clinic. He will also describe common pitfalls in 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 autism treatment research, he will suggest guideposts as we work toward neurobiologically based treatments for autism.

Jeremy Veenstra-VanderWeele is the Mortimer D. Sackler, M.D., Associate Professor in Psychiatry at Columbia University Medical Center and the New York State Psychiatric Institute. He completed his M.D. and residency training at the University of Chicago, postdoctoral training in neuroscience at Vanderbilt University, and moved to Columbia in 2014. As a child psychiatrist and developmental neuroscientist, his primary motivation is to deliver new treatments to children with autism spectrum disorder and related neurodevelopmental disorders.