I-Corps: Integrated Multi-Tissue Human Models for Drug Development

Columbia University, New York NY

Investigators

Abstract

The broader impact/commercial potential of this I-Corps project is the ability to predict how humans will respond to therapeutic treatment options before the clinical trial stage. By testing drug toxicity and efficacy in a human model, drugs can get to market more quickly, with less risk and lower costs, while potentially reducing the use of animal testing. Currently it takes an average of 10 years and $2.8B to get a drug to market, with the majority of the cost due to late-stage drug failures. The ability to screen drugs in human organ systems that are predictive of in vivo conditions will better inform "go/no-go" decision-making throughout the stages of drug development. Broader impacts include the ability to create clinical trials "in-a-dish" where patient populations that will benefit the most from the drug versus those that would be at risk can be determined before clinical testing. Use of patient-specific cells may enable the development of disease models where animal models failed to recapitulate the disease, as well as drug testing for rare diseases, where the limited number of affected individuals prevents the standard clinical trial format. This I-Corps project utilizes a modular platform designed to physiologically integrate multiple human tissue engineered organ models though vascular connections that mimic how organs are connected to one another in the human body. The connected tissue engineered models are individually matured prior to integration to provide the high biological fidelity needed to accurately recapitulate organ level responses of health and disease. The use of human induced pluripotent stem cells enables the use of patient-specific cells and gene-edited cells, as well as provides a platform to further precision medicine initiatives. This award reflects NSF's statutory mission and has been deemed worthy of support through evaluation using the Foundation's intellectual merit and broader impacts review criteria.