ATLANTA — The Marcus Autism Center announced it will conduct the largest study of behavior, brain and genomic biomarkers in children across the autism spectrum following a $21.9 million grant from the late Bernie Marcus and The Marcus Foundation.
The research will include 7,500 children from birth to 12 years of age. The study aims to better understand what causes autism severity and how to improve treatment responses in children with profound autism.
The center, a subsidiary of Children’s Healthcare of Atlanta, plans to use the study to identify biological and behavioral factors that can be modified to optimize treatment outcomes. The research goal is to develop new therapies and precision medicine interventions that can be integrated into standard-of-care services.
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In the United States, more than 2.3 million children have autism spectrum disorder.
According to the center, more than a quarter of those children, approximately 620,000, have what’s known as profound autism.
Children with profound autism face significant challenges, including severe intellectual disabilities and limited or no verbal communication. These individuals often experience extreme difficulties with daily living skills and typically require around-the-clock care and support.
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Dr. Ami Klin serves as the principal investigator for the study and the director of the Marcus Autism Center. He also serves as a Georgia Research Alliance Eminent Scholar and a professor at the Emory University School of Medicine.
“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,” Klin said in a statement. “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.”
Led by Klin, researchers will conduct an embedded pragmatic clinical trial during regular clinical practice. This methodology allows findings to be integrated directly into standard-of-care services.
The research will utilize behavioral neuroscience measures to discover modifiable factors that lead to profound autism. Participants will be studied from birth, before symptoms emerge, as well as before and after receiving treatment.
Klin said current treatment options for families are often limited.
“Most treatments we have right now are behaviorally based,” 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.”
The study involves a collaboration between several organizations, including the Emory University School of Medicine’s Department of Human Genetics and the Tri-Institutional Center for Translational Research in Neuroimaging and Data Science.
Klin described the project as the largest scientific effort to date to study children with profound autism from infancy to early adolescence.
“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,” Klin said.
The Marcus Autism Center is a not-for-profit organization and a subsidiary of Children’s Health care of Atlanta. It currently treats more than 5,000 children with autism and related disorders each year.
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