Pioneering new discovery and the acceleration of shared knowledge through data-driven research is at the cornerstone of the CHOP Neuroscience Center’s research mission. Researchers from the divisions of Neurology and Neurosurgery — united under the umbrella of the CHOP Neuroscience Center — are leading innovative, bench-to-bedside focused research that allows ever increasing opportunities to develop precision medicine treatment plans for infants, children and adolescents with acute or chronic neurological or neurosurgical conditions. We partner across multiple groups to accelerate clinical neuroscience discovery, including biomarker discovery, tissue genomics, outcomes research, clinical trials, and advanced neurodiagnostics, to improve treatment options and outcomes for children affected by neurological disorders.

Our envisioned future for pediatric neuroscience is a world where prevention, precision therapeutics, and protection are optimized for pediatric brain health. We fundamentally believe that the concept of “optimization” is informed by the priorities and beliefs of our patients and families, our shared communities, and society as a whole. Having treated more children than anywhere else in the country, our Neuroscience Center at CHOP has borne witness to this directly through advances within a number of our programs.

Precision therapeutics is far more than marrying an accurate diagnosis with an approved therapy. It is the science of studying how disease happens and determining a specific place to focus treatment in each unique child. Discoveries across our research teams in the Neuroscience Center have resulted in different contributions to improved childhood health outcomes.

Center for Data-Driven Discovery in Biomedicine (D3b)

Led by Jay Storm, MD, and Adam Resnick, PhD, the Center for Data Driven Discovery in Biomedicine (D3b) harnesses and broadly shares biomedical information to quickly reach and benefit patients. D3b has the goal of advancing precision medicine — helping to match the most appropriate treatment to individual patients.

D3b was launched in January of 2016 to drive innovation in translational research discoveries, clinical trials, and standards of care for children and adults diagnosed with rare and devastating diseases. D3b’s scientific expertise and diverse research platforms target pediatric cancers and diseases of childhood development, with pediatric brain tumors as a driving area of research emphasis.

Applying D3b’s Research Methodology (from Data to Information to Knowledge to Impact), the center and its partners are reducing the time to discovery, clinical trial delivery, and improved care for children. Tasks that once took researchers years to complete can now be completed in minutes, made possible through D3b’s research platforms and tools. These innovative platforms help to remove the current barriers to research across CHOP’s Department of Surgery, Neuroscience Center, Research Institute, and throughout the entire scientific community. By breaking down silos, D3b is empowering and facilitating researchers everywhere to accelerate translational impact from the bench to the bedside for the next breakthrough.

D3b seeks to bridge the current discovery gap across pediatrics, adolescents, and adult cancers, with a focus on the underrepresented areas of young adults, as well as for diseases with no curative standard of care. D3b’s current and future collaborative research programs are underpinned by the D3b Center’s commitment to redefine the traditional research model and provide solutions across the entire lifespan of each child diagnosed with a pediatric disease.

Neuroscience Center Biorepository (NCB)

Led by Ingo Helbig, MD, Adam Resnick, PhD, and Amit Bar-Or, MD, with support from clinicians and research partners across Neurology and Neurosurgery, the Neuroscience Center Biorepository (NCB) is a prospective, neuroscience-focused, disease-agnostic biorepository that collects biological samples and linked clinical data for long-term storage and future analyses from patients (and relatives) seen in the Neuroscience Unit and outpatient spaces at CHOP.

The NCB was officially launched in late winter of 2022 to target the center’s goal for expansive collection of biological samples and linked health data from patients. The project addresses the pressing need for comparative research and biomarker discovery in neuroscience.

The NCB project collects samples and data in partnership with other disease-specific biobanks, such as epilepsy or leukodystrophy. This partnership is unique and enables within-patient comparative, longitudinal explorations of biological samples and health data collected prior to formal diagnosis and treatment to those collected post-treatment and long-term follow-up. The repository serves as a resource for investigators in Neurology, Neurosurgery, Neuro-oncology, Psychiatry, and Developmental Pediatrics and is supported through robustly-integrated data platforms and standard operating procedures (SOPs) to maximize consistency and long-term outcomes.

The NCB provides a “disease agnostic” opportunity to collect and store biological samples and paired data that is collected and collated through advanced bioinformatics programming across multiple platforms. All patients seen by a care provider in the Neuroscience Center are invited to participate in the project.

Bench Research

Our laboratories and research programs are uncovering the causes of pediatric neurologic conditions and developing treatments that have improved the prognosis for children with these diseases.

Stewart Anderson Lab – Focus: Relationship between 22q11.2 deletion syndrome (22q11DS) and schizophrenia

Douglas Coulter Lab – Focus: How specific hippocampal circuits may contribute to the symptoms of temporal lobe epilepsy

Ethan Goldberg Lab – Focus: Develop new treatments and work toward a cure for epilepsy

Judith Grinspan Lab – Focus: Regulation of the myelin sheath

Ingo Helbig Lab – Focus: Harnessing data to better identify and treat childhood brain disorders such as epilepsy

David Lynch Lab – Focus: Efficacy of omaveloxolone to treat Friedreich’s ataxia

Eric Marsh Lab – Focus: Identify the genetic, cellular and network mechanisms of seizures and cognitive dysfunction in children

Xilma Ortiz-Gonzalez Lab – Focus: TBCK encephaloneuronopathy

Michael Robinson Lab – Focus: Clearance of glutamate, the predominant excitatory neurotransmitter in the human brain

Adeline Vanderver Lab – Focus: Leukodystrophy

Fetal and Neonatal Neuroscience Clinical and Translational Research

Our fetal and neonatal neuroscience research integrates the efforts of neurologists, neurosurgeons, anesthesiologists, cardiologists, physicists, and engineers to reduce the burden of brain injury. We offer all families the opportunity to participate in our fetal registry which is designed to correlate neurological findings identified pre-birth with clinical outcomes during childhood and into adulthood (PI IRB – Dr. Juliana Gebb, MFM). Counseling of families is informed by real-world outcomes data, and such data is essential if we are to more comprehensively understand relationships between fetal imaging features, prenatal genetic testing and childhood neurological health. Families are also offered the opportunity to participate in one of many fetal/neonatal/surgical research studies. These collaborations have led to multiple accepted abstracts on genetic diagnoses of prenatal findings.

Wolfson Family Laboratory for Biomedical and Clinical Optics

The Wolfson Lab, led by Wes Baker, PhD, is a world leader in innovation and discovery in clinical and optical imaging. Our team aims to develop and translate optical brain monitoring tools for clinical use, and to study the interplay between the delivery of pediatric critical care and the patient’s long-term brain health. We have recently advanced the development of exciting new clinical indications for non-invasive diffuse optics technologies.