Research Webinar Series: Clinical Trial Readiness for Neurodevelopmental Disorders: On the Road to Precision Health

October 28, 2020 12:00 PM - 1:00 PM
The Saban Research Institute Auditorium
4661 Sunset Blvd.
Los Angeles, CA 90027

Webinar (Please email if you wish to register)

Presented by

Shafali Spurling Jeste, MD
Associate Professor-in-Residence
Departments of Psychiatry, Neurology, and Pediatrics
University of California, Los Angeles


We have entered an unprecedented era of discovery in neurodevelopmental disorders (NDD), with precision both in timing of our diagnoses and in the identification of specific genetic etiologies that cause NDDs. As targeted therapies are developed, we must be prepared for the design and implementation of successful clinical trials. Clinical trial readiness includes (1) identification of meaningful clinical endpoints, (2) measurement of biomarkers that inform patient selection and drug target engagement measurement, and (3) methods to improve accessibility of research and treatment for all families. This presentation will begin with an overview of the state of the field in precision health in NDDs followed by data from several studies that have addressed these goals in clinical trial readiness, including prospective studies of high risk infants and endpoint and biomarker studies in syndromic NDDs. We will end with reflections on COVID-19 and its impact on clinical care and research in NDDs.

About the Speaker

Dr. Jeste is a behavioral child neurologist specializing in autism and related neurodevelopmental disorders. She is an Associate Professor-in-Residence in Psychiatry, Neurology and Pediatrics at the UCLA David Geffen School of Medicine, the director of the UCLA CARING Clinic, and a lead investigator in the UCLA Center for Autism Research and Treatment (CART). After earning a BA in philosophy from Yale University in 1997 and her MD from Harvard Medical School in 2002, Dr. Jeste completed a residency in child neurology and a fellowship in behavioral child neurology at Boston Children’s Hospital. She was recruited to UCLA CART in 2010. Dr. Jeste’s research is focused on developing methods to improve precision in the diagnosis and treatment of neurodevelopmental disorders. Her lab studies neurodevelopmental disorders from early infancy through late childhood. Dr. Jeste has designed innovative studies in early predictors of autism in a genetic syndrome called Tuberous Sclerosis Complex (TSC) that integrate biomarkers with behavior to define atypical development prior to the onset of autism. This work in TSC has led to the first randomized controlled clinical trial of behavioral intervention for these infants and has paved the way for other early intervention trials in rare genetic syndromes. Dr. Jeste’s research is directly inspired by her clinical work. To address the many gaps in medical care for rare genetic forms of neurodevelopmental disorders, she founded and directs the CARING Clinic (Care and Research in Neurogenetics). This clinic has become the hub for several new clinical trials for genetic syndromes. Dr. Jeste’s work is funded by the National Institutes of Health, the Department of Defense and the Simons Foundation. She holds several national and international leadership positions including the Board of Directors of the American Brain Foundation, Board of Directors of the National Organization for Rare Disorders, and the Board of Directors of the International Society for Autism Research. In 2019 she became Chair of the International Baby Siblings Research Consortium. In 2019 she was awarded the Presidential Early Career Award for Scientists and Engineers for her innovations in research in early predictors and intervention for genetic neurodevelopmental disorders.

Hosted by

Pat Levitt, PhD
Chief Scientific Officer, Vice President, and Director, The Saban Research Institute Simms/Mann Chair in Developmental Neurogenetics, Children’s Hospital Los Angeles WM Keck Provost Professor in Neurogenetics
Department of Pediatrics
Keck School of Medicine of USC
Editor-in-Chief, Mind, Brain and Education

Additional Information

Brought to you by TSRI Office of Training, Education, Career Planning & Development (TECPAD)

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