Research Projects
Drug Discovery for Newborn Brain Disorders: We have developed a preclinical therapeutic pipeline for identification and testing of potential treatments for newborn brain disorders. Our major focus is currently on 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 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. We are using a high-throughput, cell culture-based screening platform combined with a novel neonatal feasibility scoring system to identify compounds that show high potential for both efficacy and safety in newborn brain disorders. Screen hits are validated and further tested in disease models to confirm efficacy and determine the best dosing strategy for future clinical trials.
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 and Community Engagement: We are working to bring new treatments to patients for pediatric brain disorders. We are working on strategies to identify the patients who are most likely to benefit from specific therapies targeting known disease mechanisms. We are also pioneering efforts such as adaptive, pharmacokinetic-guided clinical trial design to improve clinical trial efficiency. 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. Dr. Ostrem is also a site P.I. for clinical trials testing new treatments for acquired and genetic disorders affecting the white matter, such as Angelman Syndrome.