Identifying a Disease-Modifying Treatment for Alzheimer’s
MentorVirginia Lee, PhD The University of Pennsylvania
This project will evaluate potential disease-modifying effects of a clinically approved drug in an Alzheimer’s mouse model generated by human-derived pathology.
This project uses human-derived Alzheimer’s disease (AD) pathology injections into mice to generate a clinically relevant model. Mice will be treated with a clinically approved drug that modulates the synaptic location of a receptor, whose mislocalization is associated with AD symptoms. Aim 1 will assess aggregated tau, inflammation, and synaptic abnormalities. Aim 2 will investigate changes in cognitive and behavioral function.
This project involves the use of Alzheimer’s patient-derived pathology to yield a clinically relevant model ideal for testing the therapeutic efficacy of drug treatments. Most pharmacological treatments used in Alzheimer’s research have not been tested in humans. The compound used in these studies is currently in phase II clinical trials, designating it as safe for use in humans. The animal model and treatment approach in this project have the potential to identify a safe and effective treatment for Alzheimer’s patients. Notably, these clinically relevant studies may generate results that do not necessitate further pre-clinical studies. The findings from this project will inherently identify a safe drug that may quickly progress to an FDA approved disease-modifying treatment.