My professional goal is to develop an effective therapeutic for human prion disease, a fatal neurodegenerative disease, and to build the tools that will help such a therapeutic succeed, including biomarkers and natural history data.

At the Broad, Vallabh is focused on developing preventive drugs for prion disease. Lowering of prion protein (PrP) is a genetically well-validated strategy for delaying the onset of prion disease, and it lends itself to measurement of PrP as a biomarker available before the disease process has begun. Vallabh is working on the discovery and preclinical development of PrP-lowering drugs, credentialing of PrP in cerebrospinal fluid as a pharmacodynamic biomarker for such drugs, and establishment of a clinical pathway for preventive drug development. She co-leads a natural history study at Massachusetts General Hospital devoted to the assessment of fluid biomarkers in individuals at risk for genetic prion disease, and helped to launch the Prion Registry to promote and enable research participation in her community.