Using organs-on-chips as models of drug efficacy

Organs-on-chips could be used to assess drug efficacy and support personalized medicine, write Adrian Roth and colleagues in a Perspective. “The key advantage [these systems] offer is the creation of more physiologically relevant human organ-like models that can potentially yield data on drug action that will better translate to humans than that from in vivo animal models or conventional cell systems,” write the authors. Traditional preclinical drug development generally relies on animal models and in vitro human cell culture. However, these approaches can poorly predict drug safety and efficacy in humans and contribute to failed clinical trials. Microphysiological systems (MPS) combine microsystems engineering with cell biology to create cell-culture models that recapitulate human physiology and biology and enable human multi-cell-type models systems; these systems better replicate complex tissue and organ functions than conventional cell culture. While MPS technologies have rapidly become powerful in vitro tools, their value for drug development is only just now becoming clear, write Roth and colleagues. “[Microphysiological system (MPS)] technologies may provide a way to better understand and address the main failures of clinical programs: lack of efficacy or unacceptable side effects that are not predicted in animals or simpler cell systems during early preclinical stages,” they write. Although MPS technologies are already being used in some preclinical drug safety studies, Roth et al. argue they could also be used to evaluate the efficacy and potential undesired effects of a drug before patients are exposed to it. What’s more, these systems could be particularly well-suited to developing personalized medicine approaches by seeding organs-on-chips with a patient’s own cells, allowing the most effective treatments to be identified. Finally, MPS technologies could enable rapid development and evaluation of new therapies, including vaccines, for emergent medical needs.