Multi-Organ Drug Exploration (MODE) Platform

Project Summary:

New clinical trials have a lowly success rate of 7.9%. A major limitation to successful translation of preclinical trials in mice-to-human clinical trials is simply that mice are not humans. Human patient populations are diverse and heterogenous and insufficiently represented by mouse models that are often inbred to be genetically homogenous. In addition, many human diseases do not have mouse models that represent the human disease at all. To address this gap, human induced pluripotent stem cells (hiPSC) can be reprogrammed from skin, blood, and urine samples of real human patients. These hiPSC can then be further reprogrammed into representative organ cells for disease modeling and experimental therapeutic testing. The use of hiPSC are limited in part due to the high cost and single organ focus of commercial systems.

This project team developed a customizable and modular design for a new multi-organ drug exploration (MODE) platform that uses hiPSC to model a wide variety of different cell types. Their goal is to enable high-throughput analysis of multiple stem cell-derived cell types in a cost-effective manner using a novel barcoding method. In addition, they will use hiPSC in their MODE platform and in animal models to test the organ-specific targeting of two novel therapeutic delivery systems. The ultimate goal is to use stem cells from real Wisconsin patients to create an accessible platform that represents the genetics of specific patients from the local population for experimental diagnostic and drug screening approaches.