Columbia and Weill Cornell Medicine Researchers Awarded $17.25 Million to Study Profound Autism

Researchers at Columbia University Irving Medical Center and Weill Cornell Medicine have won a $17.25 million grant from the Aligning Research to Understand Autism (ARIA) initiative to study profound autism, which affects over one quarter of individuals who are on the autism spectrum. People with profound autism are often nonverbal and require 24-hour support and assistance with daily tasks throughout their lives.

With this new funding from ARIA, the Columbia team, along with collaborators at New York State Psychiatric Institute and Weill Cornell Medicine, will join an international network of research and clinical sites called the ARIA IMPACT Network, which is working to accelerate clinical trial readiness and therapeutic development for autism.

Over the next five years, the researchers will contribute to the ARIA IMPACT-Ready study, a short-term natural history and clinical endpoint study. This study aims to better understand how autism and related neurodevelopmental conditions develop and change over time and gather crucial insights about clinical endpoints and outcome measures that may boost the development of new treatments and future intervention opportunities.

“The grant will allow us to draw on collective expertise in pediatric psychiatry, neurology, and genomic research at Columbia University Irving Medical Center, New York State Psychiatric Institute, and Weill Cornell Medicine to expand our understanding of the development of people with profound autism,” says Jeremy Veenstra-VanderWeele, MD, the Ruane Professor of Psychiatry and Pediatrics at Columbia University Vagelos College of Physicians and Surgeons, director of the Division of Child & Adolescent Psychiatry at NewYork-Presbyterian Morgan Stanley Children’s Hospital of Children’s Hospital of New York and New York State Psychiatric Institute, and the site lead for the study. “Together with information we have about genetic risk for profound autism, the study may identify the best opportunities to intervene—be it with medicines, behavioral interventions, or communication tools.”

The research team aims to enroll children with profound autism and follow them over a period of two years. “All too often, children with profound autism are excluded from research studies because of the many challenges they and their families face,” says William Fifer, PhD, a professor of medical psychology (in Psychiatry and Pediatrics) and the biomarker lead for the study at Columbia University Vagelos College of Physicians and Surgeons. “This study will identify the particular needs of people living with profound autism so that we can develop more effective therapies.”

“The data collected through this program can support the future launch of clinical trials pertaining toprecision medicines, and we are eager to bring new treatments to children and their families,” says Zachary Grinspan, MD, MS, associate professor of pediatrics, neurology, and population health sciences at Weill Cornell Medicine and interim chief of child neurology at NewYork-Presbyterian Komansky Children’s Hospital of Children’s Hospital of New York and Weill Cornell Medicine, and the clinical lead for the study.

Co-investigators from Columbia, Jennifer Bain, MD, PhD, associate professor of neurology and pediatrics, and Nicolo Pini, PhD, assistant professor of medical psychology, will complement the team to bring their international expertise in rare disease clinical trials and natural history studies.

This grant reflects growing research and clinical trial collaborations that span Columbia University, Weill Cornell Medicine, New York State Psychiatric Institute, and NewYork-Presbyterian.

Since 2013, the NewYork-Presbyterian Center for Autism and the Developing Brain (CADB), a collaborative program with Columbia and Weill Cornell Medicine, has served as a clinical, training, and research hub in White Plains, New York. CADB provides comprehensive care for people of all ages living with autism and other developmental challenges.

In 2022, the National Institutes of Health designated Columbia, in collaboration with Weill Cornell Medicine, as an Autism Center of Excellence. Since then, the team has undertaken a separate study to understand the genetic underpinnings of autism and related conditions and correlate genetic risk factors with emerging features of autism and related disorders in early development.