Children’s Healthcare of Atlanta’s Marcus Autism Center has received a $21.9 million grant from the late Bernie Marcus and The Marcus Foundation to launch what it describes as the largest study to date on behavior, brain, and genomic biomarkers in children with autism spectrum disorder (ASD) and related genetic neurodevelopmental conditions. The research aims to better understand the causes of autism severity and treatment responses, particularly in children with profound autism.
The initiative will be conducted in collaboration with Children’s Behavioral and Mental Health and Neurosciences Research Programs, Emory University School of Medicine’s Department of Human Genetics, and the Tri-Institutional Center for Translational Research in Neuroimaging and Data Science (TReNDS). It plans to enroll 7,500 children from birth through age 12.
Ami Klin, PhD, Principal Investigator and Director of the Marcus Autism Center, said: “The goal is to enable precision medicine interventions that will accelerate learning, make symptoms less severe and improve response to treatment in children with profound autism, and possibly even prevent profound disability from emerging in the first place. If successful, our research could usher in a new era of behavior-brain-genomic precision medicine to optimize outcomes of children in a community that cannot wait.”
Researchers led by Dr. Klin will embed their clinical trial within regular clinical practice at Marcus Autism Center. The findings are intended for integration into standard care services. The study will follow participants from infancy—before symptoms appear—and continue through treatment stages using multi-omic and behavioral neuroscience tools to find modifiable factors contributing to profound autism.
“Most treatments we have right now are behaviorally based,” Dr. Klin said. “By studying profound autism at multiple levels—in behavior, brain networks, and basic biology—one of the key goals is to identify new biological targets for drugs and other therapies: to support learning and adaptability, to make symptoms less severe, and to promote better quality of life for children and families affected by profound autism.”
Dr. Klin also stated: “This represents the largest scientific effort to date to study children with profound autism from infancy to early adolescence, and to develop actionable predictors that can improve treatment response, while personalizing treatments and developing new ones. We hope to generate a moonshot factory of solutions for a community that carries the most severe symptoms of autism and has been under-represented in autism research.”
In the United States there are more than 2.3 million children diagnosed with ASD; over 620,000 have profound autism—a condition marked by significant intellectual disabilities, limited or no verbal communication abilities, extreme challenges with daily living skills, often requiring continuous care.
