Potholes and progress on the road to translational treatments in autism spectrum disorder

  • Autism Research
Speaker Jeremy Veenstra-VanderWeele, M.D.
Columbia University
Date & Time


Tea: 4:15pm - 5:00pm
Lecture: 5:00pm - 6:15pm

Location

Gerald D. Fischbach Auditorium
160 5th Avenue
New York, NY 10010 United States

Autism Research

Autism Research lectures bring together scientists and scholars to discuss diverse and important topics related to autism. The lectures are open to the public and are held at the Gerald D. Fischbach Auditorium at the Simons Foundation headquarters in New York City. Tea is served prior to each lecture.

 
On 25 January 2017, Jeremy Veenstra-VanderWeele outlined critical challenges to translating genomic, cellular, and animal model research into new treatments for autism spectrum disorder.

His talk was part of the Simons Foundation Autism Research lecture series.
 

About the Lecture

Emerging genomic and neuroscience findings have delivered hypotheses that are now being tested in autism spectrum disorder (ASD) 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, Jeremy Veenstra-VanderWeele outlined critical challenges, both conceptual and practical, to translating genomic, cellular, and animal model research into new treatments for ASD. He discussed the limitations to conclusions drawn from work in the laboratory as they are extrapolated to the clinic. He also described 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 ASD treatment research, he suggested guideposts as we work toward neurobiologically based treatments for ASD.

About the Speaker

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.

Past Lectures

On the road to precision health: Brain-based biomarkers in autism spectrum disorder

Shafali Spurling Jeste, M.D.Associate Professor in Psychiatry, Neurology and Pediatrics, UCLA David Geffen School of Medicine
Director, Developmental Neurophysiology Lab, UCLA Center for Autism Research and Treatment

On 7 February 2018, Shafali Spurling Jeste provided a topical overview of the current state of research in autism biomarkers. She shared data from studies of autism biomarkers in three key areas: early risk prediction (studies of high-risk infants), heterogeneity within the autism spectrum and genetically defined subgroups within autism. Finally, she discussed the challenges around clinical trial design and development and considered how more objective measures of brain function can improve clinical trials.

Arousal, emotion regulation and challenging behaviors: Insights from the Autism Inpatient Collection

Matthew Siegel, M.D.Associate Professor of Psychiatry and Pediatrics, Tufts University
Vice President, Medical Affairs, Developmental Disorders Service, Maine Behavioral Healthcare

On 24 January 2018, Matthew Siegel drew upon a new resource, the Autism Inpatient Collection data set, to offer preliminary insights into the relationships between physiologic arousal, emotion dysregulation and the occurrence of challenging behaviors. Such behaviors may represent an attempt to modulate physiologic arousal in minimally verbal individuals with autism spectrum disorders.

Autism genetics: Searching for coherence

Daniel Geschwind, M.D., Ph.D.Professor, University of California, Los Angeles

On 28 November 2017, Daniel Geschwind discussed his group’s use of RNA sequencing, chromatin structure and gene networks to help develop an understanding of potential convergent mechanisms in autism spectrum disorders.

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