Research

Faculty within the Division of Child Neurology are engaged in a wide variety of research programs.

  • The Pediatric Stroke and Cerebrovascular Center and the UCSF Stroke Sciences Group, under the direction of Heather Fullerton, MD, MAS, are widely recognized for research on the causes, treatment and prevention of strokes in children. Dr. Fullerton and her collaborators have described stroke related to recent trauma, minor acute infections, sickle cell disease, arteriovenous malformations, and many other conditions. Christine Fox, MD, has studied stroke in the setting of congenital heart disease, and epilepsy as an outcome of pediatric stroke. Nancy Hills, PhD, has studied the relationship between stroke and vasoactive medications, such as therapies for ADHD. Sabine Mueller, MD, PhD, has studied strokes as a late effect of treatment for childhood cancers, and Amy Gelfand, MD, has studied migraine as a risk factor for childhood stroke. Dr. Yvonne Wu has established risk factors for perinatal stroke and cerebral palsy.
  • The Newborn Brain Research Institute is pioneering medical breakthroughs for newborns with neurological complications. The co-directors of the NBRI are Donna Ferriero, MD, a neurologist and leading clinical investigator in neurological problems of newborns, and David Rowitch, MD, PhD, a neonatologist and Howard Hughes Medical Institute investigator whose laboratory research focuses on basic biological regulation of neural stem cells. Hannah Glass, MDCM, MAS, participates in research that uses advanced imaging and brain monitoring to predict outcomes following newborn brain injury. Yvonne Wu, MD, an international expert in the epidemiology of cerebral palsy and newborn brain injury in term infants, conducts clinical trials on a novel therapy for hypoxic-ischemic encephalopathy. She also leads multidisciplinary studies designed to improve our understanding of maternal and perinatal risk factors leading to neonatal brain injury. The laboratory of Zena Vexler, PhD, studies the underlying mechanisms of neonatal and childhood stroke, including the role of neuroinflammation in injury and repair and how blood-brain-barrier permeability is affected by stroke. She is also working to identify biomarkers of injury and recovery from neonatal stroke.
  • The UCSF Epilepsy Center investigates early-onset epilepsies, including their genetic origin, their clinical phenotypes and EEG characteristics. The program works to improve the early recognition of disorders such as Dravet syndrome, PCDH19 epilepsy, CDKL5 epilepsy, as well as rare neonatal-onset epilepsies such as benign familial neonatal seizures, KCNQ2 encephalopathy and SCN2A neonatal epilepsy. The program is a member of the multicenter Pediatric Epilepsy Research Consortium (PERC) that is currently enrolling young children with new onset epilepsy in an observational database. The Pediatric Epilepsy Reserach Program is also recognized for its studies in neonatal EEG monitoring and neonatal seizures. The program has conducted investigational medication trials and is involved in pioneering trials of new medications for intractable epilepsies, including pure cannabidiol and fenfluoramine.
  • The UCSF Regional Pediatric Multiple Sclerosis Center, under the direction of Emmanuelle Waubant, MD, PhD, is a leading group in research on pediatric multiple sclerosis and neuromyelitis optica, and provides care for young patients affected by these diseases. The group has been critical in describing the clinical and radiological features of MS in very young patients. Ongoing research projects of the group include the identification of environmental and genetic risk factors associated with these disorders, as well as finding new treatments. The team also focuses on factors that may worsen disease severity, some of which may be modifiable.
  • The Pediatric Neuromuscular Center is involved in several laboratory and clinical studies of the immunologic mechanisms occurring in the muscular dystrophies. The expectation is that these studies will lead to potentially new therapies for these disorders. Research is conducted under the direction of Jonathan Strober, MD, director of the center, and in conjunction with Julie Saba, MD, PhD, John and Edna Beck Chair in Pediatric Cancer Research at Children’s Hospital Oakland Research Institute.
  • Members of the Pediatric Brain Center have been part of groundbreaking research in stem cell transplantation for a fatal central nervous system disorder, Pelizaeus-Merzbacher Disease (PMD), in which affected children cannot make an important protein, myelin. This is one of the first human stem cell transplant studies and one that has implications for many other conditions affecting myelin.
  • The UCSF Surgical Movement Disorders Center is one of the highest volume clinical centers in the western United States for implantation of deep brain stimulation (DBS) systems. This experience also includes treating children with certain forms of pediatric movement disorders such as primary and secondary forms of dystonia and Tourette’s syndrome. Neurosurgeons Philip Starr, MD, PhD, and Paul Larson, MD, together with radiologist Alastair Martin, PhD, have pioneered a new surgical method for placement of DBS electrodes while children are asleep, using an interventional MRI technique. Outcomes research looking at this new methodology was directed by the Center’s medical director, Jill Ostrem, MD. Neurologist Marta San Luciano, MD, is also researching the genetics of pediatric dystonia and other forms of familial movement disorders that can affect children.
  • The Pediatric Brain Tumor Center at UCSF, under the direction of Sabine Mueller, MD, PhD, investigates new treatment options for children with brain tumors and studies late effects of the disease and therapies in pediatric brain tumor survivors. Dr. Mueller and Michael Prados MD, lead the Pacific Pediatric Neuro-Oncology Consortium (PNOC), which is a clinical trials consortium consisting of 11 children’s hospitals that work together to understand how brain tumors develop in children and conduct studies of advanced, personalized treatment strategies. Many clinical trials offered through PNOC are based on discoveries made in research laboratories at the Pediatric Brain Tumor Research Center housed within the Helen Diller Family Comprehensive Cancer Center. These include the W. A. Weiss laboratory and the Persson laboratory. The center also participates in early phase clinical trials offered through the Children’s Oncology Group and other industry- sponsored trials. There is also a strong research program for late effects in childhood cancer survivors, with a specific focus on vascular effects of radiation therapy (RadART pro study). These studies are done in collaboration with pediatric stroke expert Heather Fullerton MD and director of the Survivor of Childhood Cancer Program Robert Goldsby MD. The program uses web-linked interfaces to monitor long-term side effects in pediatric cancer patients.