Mission Statement: The Rasmussen’s Encephalitis (RE) BRI Affinity Group aims to increase awareness regarding RE for the primary purpose of supporting multidisciplinary scientific research directed towards a cure.
Background: Rasmussen’s encephalitis (RE) is a rare inflammatory brain disease that causes intractable focal seizures, and progressive motor, sensory and cognitive deficits. Additional features of RE include progressive cerebral atrophy, hemiparesis, severe epilepsy and visual field defect. RE typically affects previously normal children aged 4-12, but may sometimes occur earlier or later. There is evidence for autoimmune etiology in which T-cells attack a specific unidentified antigen or set of antigens in a patient’s brain. An unusual feature of the disease that sets it apart from other inflammatory diseases of the central nervous system is that it is usually confined to one cerebral hemisphere. Resection of large areas of the brain, including cerebral hemispherectomy, is currently the only effective treatment option for children with RE. The purpose of the proposed BRI Affinity Group is to identify the “cause” for RE and develop new treatments that prevent the brain inflammation associated with this devastating disease to reduce or eliminate the need for brain surgery. To accomplish this goal the Affinity Group will develop an animal model and cell culture system for Rasmussen encephalitis. This work will set the framework for a larger initiative to make UCLA the preeminent center for translational research into Rasmussen’s encephalitis and, ultimately, other rare brain diseases.
Methods: Through a collaboration with the Rasmussen’s encephalitis (RE) Children’s Project, a 501 (c) (3) non-profit organization dedicated to supporting scientific research directed towards a cure (www.rechildrens.org), a team of UCLA investigators have been awarded seed funding to establish the protocols and mechanisms to collect RE brain tissue. The RE repository will collect RE brain tissue, cerebrospinal fluid (CSF), peripheral lymphocytes from blood and saliva from donors at hemispherectomy surgery using IRB-approved standard operating procedures. The RE Children’s Project obtains RE brain tissue from centers around the world and ships that material to UCLA. Once fresh tissue arrives neuroglial cells, neural stem cells, brain infiltrating lymphocytes, and other cell types are isolated and banked along with flash frozen and fixed brain tissue, whole blood, CSF and DNA extracted from saliva. Brain tissue has already been received from over 10 RE patients over the past 18 months. The cell culture system will use these cells isolated from RE patients and stored in the tissue bank to create an in-vitro environment mimicking the cellular environment of the human brain. The animal model system will use cells isolated from RE patients and stored in the tissue bank for injection into immune-deficient mice to study seizure activity utilizing video and EEG. Tissue from these mice will be removed and examined under the microscope to look at the histopathology of RE in-vivo.
UCLA Faculty/Staff Participants:
Gary Mathern (Neurosurgery and Psychiatry and Biobehavioral Sciences)
Aria Fallah (Neurosurgery)
Geoffrey Owens (Neurosurgery)
Harry Vinters (Pathology and Laboratory Medicine and Neurology)
Harley Kornblum (Psychiatry and Biobehavioral Sciences, Pharmacology and Pediatrics)
James Cherry (Pediatrics Infectious Diseases)
Robert Prins (Neurosurgery)
Anatol Bragin (Neurology)
Julia Chang (Neurology)
Carlos Cepeda (Psychiatry and Behavioral Sciences)
Michael Levine (Psychiatry and Biobehavioral Sciences)
Noriko Salamon (Radiological Sciences)
Scientific Coordinator: Geoffrey Owens, PhD and Associate Project Scientist, UCLA Departments of Neurosurgery and Radiology
Clinical Coordinators: Gary W. Mathern, MD and Professor, UCLA Departments of Neurosurgery and Psychiatry and Biobehavioral Sciences
Harry Vinters, MD and Professor, UCLA Departments of Pathology and Laboratory Medicine and Neurology
For more information, contact Julia Chang, Assistant Resident, Department of Neurosurgery