Research Projects
Drug Discovery for For White Matter Disorders: We have established a preclinical therapeutic pipeline for identification and testing of potential treatments for white matter and newborn brain disorders. Our approach involves high-throughput, cell culture-based screening platforms for compound identification followed by validation in disease models. We also have built collaborations with chemical biologists to develop on the next generation of small molecules. We are particularly interested in white matter injury of prematurity. Preterm birth, defined as birth before 37 weeks of completed gestation, is a major source of pediatric morbidity and mortality worldwide. White matter injury is the most common cause of brain injury in premature infants and is associated with a high risk of future disability and other neurological symptoms, such as muscle stiffness and weakness (cerebral palsy) and seizures. There are no specific treatments for babies with this type of brain injury. Patients are monitored closely for associated symptoms, and are referred to rehabilitative therapies such as physical therapy to help maximize their developmental potential. We are working to develop treatments that directly promote recovery and repair in patients with white matter injury.
Biomarker Development: We are working to identify better ways to predict future neurological symptoms and development in pediatric patients with white matter disorders. Areas of interest include blood biomarkers, including high-throughput proteomic analysis on very small volume blood samples. We are also working to develop quantitative imaging (brain MRI) biomarkers, with a focus on advanced neuroimaging techniques that allow for a high-resolution, objective evaluation of the maturation of white matter microstructure and myelination in babies with white matter injury.
Clinical Trials for Rare Pediatric Neurological Disorders: We are working to bring new treatments to patients for pediatric brain disorders, using adaptive, pharmacokinetic-guided protocols to improve clinical trial efficiency. The WRAP (White Matter Injury Repair with Clemastine in Preterm Neonates) trial is a Phase I trial testing a new potential treatment for white matter injury. We are currently enrolling in the Neonatal Intensive Care Nursery at UCSF. We also participate as a clinical trial site for several industry-sponsored clinical trials, including for Angelman Syndrome and Dravet Syndrome. Finally, by working directly with families and caregivers, we aim to better understand the priorities of patient communities and to receive input on clinical trial design, such as through the UCSF VOICE Study.