Neuroscience Research

Under the umbrellas of neurology and neurosurgery, our team conducts research studies to develop innovative treatments and therapies for rare neurological conditions. Our areas of neurological research include concussion, epilepsy, hydrocephalus, movement disorders, neuromuscular diseases, neuro-oncology & brain tumors, and stroke.

Concussion

• Neurology and the Emergency Department have partnered with NeurOptics to evaluate pupillometry as an objective biomarker of persistent post-concussive symptoms in children and adolescents.
• Neurology and Gastroenterology have partnered to conduct a prospective, randomized controlled trial examining the effectiveness of an auricular neurostimulation device called Percutaneous Electrical Nerve Field Stimulation (PENFS) in treating adolescents with persistent post-concussive symptoms. This device targets the autonomic nervous system which is may be dysregulated after concussion or mild traumatic brain injury.

Epilepsy

• Our Comprehensive Level 4 Epilepsy Program is a national leader in pediatric epilepsy care, offering cutting-edge diagnostics, innovative medical approaches and advanced surgical interventions from the nation’s foremost epilepsy experts.
• We have active participants in many national clinical trials that allow us to offer investigational therapies to patients with rare epilepsy syndromes.
• We partner with colleagues around the nation to exchange treatment strategies and outcomes, and to aggregate patient data to increase sample size and statistical power.
• Our research has led to less invasive methods for Epilepsy surgery, such as Deep Brain Stimulation (DBS), vagus nerve stimulation and use of mini-grid technology. These procedures eliminate the need to remove parts of the patient’s brain that cause seizures. These innovative care solutions lead to minimized seizure activity, as well as lowered complication rates post-operation and decreased recovery time compared to typical epilepsy surgery.

Hydrocephalus

• We are the region’s leader in complex multicystic hydrocephalus research. Our research focuses on avoiding shunt failure and lesions that occur from multiple surgeries involved with a hydrocephalus diagnosis.
• We have helped pioneer two neurosurgeries to remove cerebrospinal fluid in a child’s or baby’s brain, eliminating the need for a valve. Our research advancements in minimally invasive neurosurgery have helped decrease pain after surgery and increase recovery time for patients.
• Research is underway to learn more about what causes shunt failures, develop catheter-free mechanical solutions and develop pharmacological treatments to prevent hydrocephalus.
• In the long-term, innovative care coming from our research helps to combat the neurological conditions that result from hydrocephalus, such as developmental delays and cranial bulging.

Movement Disorders

• Dr. Sanger, pediatric neurologist and Chief Scientific Officer at CHOC Research Institute, pioneered a more precise three-stage surgical approach using deep brain stimulation (DBS) to treat secondary dystonia in pediatric patients.

Neuromuscular Disease

• CHOC has earned a national reputation for excellence in pediatric neuromuscular diseases, specializing in spina bifida, muscular dystrophy (MD), scoliosis, cerebral palsy, spasticity and other pediatric neuromuscular diseases and rehabilitation.
• CHOC was chosen as a center for an intrathecal catheter study for the administration of Spinraza for the treatment of spinal muscular atrophy.
• We participated in a Managed Access Program that allowed us to offer an investigational therapy to patients with an extremely rare genetic neuromuscular disorder.
• CHOC is the only pediatric clinic in Orange County designated by the Muscular Dystrophy Association, giving us access to additional access to national research.

Neuro-oncology & Brain Tumors

• These areas focus on genomic research and genome sequencing
• Genomic Research allows us to identify individualized treatments for children and adolescents based on the molecular profile of their cancer. Pediatric cancer has typically seen an 80% cure rate. Advancements in precision medicine through neuro-oncology research gives us hope to reach the 20% of pediatric cancer patients who have yet to find a cure.
• Our connection to Children’s Oncology Group (COG) allows us to be the only medical center in the southwestern U.S. that offers COG Phase 1 clinical trials, providing access to research studies that may lead to improved survival rates or reduction of side effects in neuro-oncology patients.

Stroke

• Dr. Rachel Pearson, Director of Brain Injury Medicine at CHOC, leads a study aimed at characterizing stroke prevalence and barriers to the diagnosis and treatment of acute ischemic stroke. The goal of this research is to optimize health systems and improve quality of care.
• CHOC is a member organization of the International Pediatric Stroke Study (IPSS), the largest stroke registry study in the world, with a global network of over 100 participating institutions in 34 countries. Research efforts focus on improving care for infants and children suffering from strokes.

A Prospective, Long-Term Registry of Patients with a Diagnosis of Spinal Muscular Atrophy (SMA) “Restore”

A Multicenter, Open-Label, Randomized, Active Comparator Study to Evaluate the Efficacy, Safety, and Pharmacokinetics of Lacosamide in Neonates with Repeated Electroencephalographic Neonatal Seizures (“LENS”)

Detecting Seizure and Loss of Consciousness from Eye Movements (SLoCEM) (Eysz, Inc.)

A double-blind, randomized, placebo-controlled study to evaluate the safety, tolerability, efficacy, and pharmacokinetics of intravenous “investigational product” (IP) as adjunctive therapy to treat participants with status epilepticus (Ganaxolone) (“RAISE”)

Phase 2 Multicenter, Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study to Assess the Efficacy, Safety, Tolerability, and Pharmacokinetics of NBI-827104 in Pediatric Subjects with Epileptic Encephalopathy with Continuous Spike-and-Wave During Sleep

A multi-center, double-blind, placebo-controlled parallel group study with adaptive design to evaluate the efficacy, safety, and tolerability of brivaracetam as monotherapy in patients 4-25 years of age with Childhood Absence Epilepsy (CAE) or Juvenile Absence Epilepsy (JAE) (“EXPAND”) (protocol NO1269)

Epilepsy Adherence in Children and Technology