Boston Children’s receives a $7.5 million grant from Aligning Research to Impact Autism (ARIA) to provide clinical research coordination for the IMPACT Network

(Press-News.org) Boston Children’s Hospital today announced that it has received a grant of $7.5 million from Aligning Research to Impact Autism (ARIA) to support its role in the Clinical Coordinating Center (CCC) for the Innovative Medicine and Precision Approaches to Clinical Trials (IMPACT) Network, an international network of sites designed to accelerate clinical trials for individuals with autism, both with a known rare high-penetrance genetic etiology and those with profound autism without a genetic cause identified.

Selection of Boston Children’s Hospital as a co-lead for this major clinical research program is a testament to the Hospital’s decades-long commitment to, investment in, and global leadership of autism research and clinical care. Boston Children’s is a top center for investigation in neurodevelopmental disorders, neurogenetics, and translational neuroscience, and is ideally positioned to drive the ARIA initiative’s pivotal work.

With the grant, Boston Children’s Hospital’s Rosamund Stone Zander and Hansjoerg Wyss Translational Neuroscience Center, together with UCLA, will serve as the nucleus of the IMPACT Network, providing scientific leadership and management of regulatory compliance and data collection quality control across all sites. The CCC will support the development of study design protocols, as well as recruitment and enrollment strategies, ultimately ensuring standardization of clinical and data processes and efficient workflows for future clinical trials. It will also provide trial coordination, site management, protection of human subjects, and training and education for all ARIA projects. The CCC is jointly led by Mustafa Sahin, MD, PhD, Neurologist-in-Chief at Boston Children’s Hospital, and Shafali Jeste, MD, Chair and Executive Medical Director of the UCLA Department of Pediatrics.

An innovative and highly collaborative physician-scientist, Dr. Sahin leads a world-renowned team committed to ushering in a new era in pediatric neurology focused on genetic diagnoses and targeted therapies. For the past 10 years, Dr. Sahin has led the multi-institutional Developmental Synaptopathies Consortium through the NIH-funded Rare Disease Clinical Research Network. This highly collaborative network advanced understanding of Tuberous Sclerosis Complex (TSC), a genetic disorder associated with autism and epilepsy, as well as PTEN Hamartoma Tumor Syndrome and Phelan McDermid Syndrome. Dr. Sahin’s lab has also been investigating the fundamental mechanisms underlying rare neurogenetic diseases using stem cell and animal models. In recognition of his impact, he was elected to the prestigious National Academy of Medicine in 2023. Now, he brings this expertise to co-lead the ARIA IMPACT Network Clinical Coordinating Center, which will accelerate clinical trial readiness and clinical trial implementation for autism with and without high-penetrance genetic etiologies.

“Our scientific understanding of autism has advanced to where we are now poised to introduce therapies that we hope will transform futures for patients and families everywhere,” said Dr. Sahin. “ARIA’s catalytic funding will allow us to turbocharge the development of the clinical research structures we need to conduct effective clinical trials for autistic people, especially those with profound autism who have the most limited options for care.”

“We are proud to embark on this journey with Dr. Sahin, Dr. Jeste, and future IMPACT Network investigators to systematically approach clinical trial readiness to both enhance our understanding of and develop effective treatments for autism and related neurodevelopmental conditions,” said Dr. Ekemini Riley, Ph.D., Managing Director of ARIA.    

 

About Boston Children’s Hospital:

Boston Children’s Hospital is ranked among the best children’s hospitals in the nation by U.S. News & World Report and is a pediatric teaching affiliate of Harvard Medical School. Home to the world’s largest research enterprise based at a pediatric medical center, Boston Children’s has led the way in life-changing pediatric innovation since its founding in 1869. Today, 3,000 researchers and scientific staff, including 14 members of the National Academy of Sciences, 37 members of the National Academy of Medicine and 13 Howard Hughes Medical Investigators across ~1M square feet of lab space comprise the research community. From bench to bedside, scientists work on preventing, treating, and curing diseases that impact both children and adults, no matter how rare or complex the condition. Founded as a 20-bed hospital for children, Boston Children’s is now a 491-bed comprehensive center for pediatric and adolescent health care. 

With nine satellite locations and the Martha Elliot Health Center, Boston Children’s also provides 24/7 pediatric care at five hospitals including Beverly Hospital, Winchester Hospital, St. Luke’s Hospital, South Shore Hospital and Cape Cod Hospital.

Boston Children’s also includes: Affiliation with Franciscan Children’s Hospital, including 112 beds and 700+ employees; Boston Children’s Primary Care Alliance, a robust network of 33 pediatric practices serving patients and families throughout Massachusetts; The Pediatric Physicians’ Organization at Boston Children’s Hospital (PPOC) with more than 400 physicians, nurse practitioners, and physician assistants devoted exclusively to pediatric primary care, in close collaboration with subspecialists at Boston Children’s in more than 90 locations throughout Massachusetts; Boston Children’s Health Physicians (BCHP) which is the largest pediatric multispecialty group in New York's Metropolitan Area, the Hudson Valley, and Connecticut, including nearly 300 clinicians in more than 60 locations.

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About Aligning Research to Impact Autism:

Aligning Research to Impact Autism (ARIA) is a scientific initiative to accelerate understanding and treatment of autism and related neurodevelopmental conditions through alignment, collaboration, and cutting-edge research. ARIA connects emerging research, insights, and promising technologies from across scientific fields to create more therapeutic opportunities for people with profound autism and people on the spectrum who seek additional support. Its focus areas include building a clinical trial and translational research network to rapidly develop and test new therapies, funding frontier science, and facilitating data sharing. ARIA is led by Managing Director Ekemini Riley, PhD, of the Coalition for Aligning Science and Scientific Director Matthew State, PhD, of the University of California, San Francisco. Learn more at ariaroadmap.org.    

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