Abstract

The NIH Microphysiological Systems Program: In Vitro Tools for Drug Development and Precision Medicine

Danilo Tagle, Associate Director for Special Initiatives, National Institutes of Health

Approximately 30% of drugs have failed in human clinical trials due to adverse reactions despite promising pre-clinical studies, and another 60% fail due to lack of efficacy. One of the major causes in the high attrition rate is the poor predictive value of current preclinical models used in drug development despite promising pre-clinical studies in 2-D cell culture and animal models. The NIH Microphysiological Systems (Tissue Chips) program led by NCATS is developing alternative approaches and tools for more reliable readouts of toxicity and efficacy during drug development. Tissue chips are bioengineered microphysiological systems utilizing human primary or stem cells seeded on biomaterials manufactured with chip technology and microfluidics that mimic tissue cytoarchitecture and functional units of human organs.  These platforms can be a useful tool for predictive toxicology and efficacy assessments of candidate therapeutics. Effective partnerships with stakeholders, such as regulatory agencies, pharmaceutical companies, patient groups, and other government agencies is key to widespread adoption of this emerging technology. Tissue chips can also contribute to studies in precision medicine, environment exposures, reproduction and development, infectious diseases, microbiome and countermeasures agents.