Research Areas

DEVELOPMENTAL ORIGINS OF PEDIATRIC BRAIN TUMORS

      Our group has a longstanding interest in developmental signaling pathways and their relationship to pediatric brain tumor formation and progression. We use sophisticated mosaic mouse genetics approaches to model pediatric brain tumors and study tumor cell and tumor microenvironment at single cell resolution throughout the course of tumorigenesis including following treatment interventions.

 ENHANCING THERAPEUTIC DELIVERY PAST BIOLOGICAL BARRIERS

      We in collaboration with chemical biology colleagues are advancing a P-selectin targeting nanoparticle platform technology to deliver anti-cancer therapeutics including small molecule drugs and PROTAC degraders past intact blood-brain barrier (BBB) specifically at the site of intracranial disease. We have found that this technology increases therapeutic index up to 10-fold thus minimizing treatment-related toxicities. We are currently assessing various classes of drugs in several pediatric and adult brain tumor models including targeting of leptomeningeal disease. In addition, we are leveraging use of the technology in focal neurological disorders including stroke, multiple sclerosis, focal epilepsy, and neurodegenerative disease models.

      Of note, the eye has been shown to have a similar barrier to therapeutic delivery as the BBB known as the blood-retinal barrier, or BRB. We are exploring whether this drug delivery technology can enhance therapeutic delivery for ophthalmic conditions including pediatric cancers (retinoblastoma, NF1-related optic pathway glioma) as well as other inflammatory eye diseases.