Promises and Challenges of Organoid-Guided Precision Medicine

TY - JOUR

T1 - Promises and Challenges of Organoid-Guided Precision Medicine

AU - Bose, Shree

AU - Clevers, Hans

AU - Shen, Xiling

PY - 2021/9/10

Y1 - 2021/9/10

N2 - Organoids are self-organizing, expanding 3D cultures derived from stem cells. Using tissue derived from patients, these miniaturized models recapitulate various aspects of patient physiology and disease phenotypes including genetic profiles and drug sensitivities. As such, patient-derived organoid (PDO) platforms provide an unprecedented opportunity for improving preclinical drug discovery, clinical trial validation, and ultimately patient care. This article reviews the evolution and scope of organoid technology, highlights recent encouraging results using PDOs as potential patient "avatars" to predict drug response and outcomes, and discusses critical parameters for widespread clinical adoption. These include improvements in assay speed, reproducibility, standardization, and automation which are necessary to realize the translational potential of PDOs as clinical tools. The multiple entry points where PDOs may contribute valuable insights in drug discovery and lessen the risks associated with clinical trials are also discussed.

AB - Organoids are self-organizing, expanding 3D cultures derived from stem cells. Using tissue derived from patients, these miniaturized models recapitulate various aspects of patient physiology and disease phenotypes including genetic profiles and drug sensitivities. As such, patient-derived organoid (PDO) platforms provide an unprecedented opportunity for improving preclinical drug discovery, clinical trial validation, and ultimately patient care. This article reviews the evolution and scope of organoid technology, highlights recent encouraging results using PDOs as potential patient "avatars" to predict drug response and outcomes, and discusses critical parameters for widespread clinical adoption. These include improvements in assay speed, reproducibility, standardization, and automation which are necessary to realize the translational potential of PDOs as clinical tools. The multiple entry points where PDOs may contribute valuable insights in drug discovery and lessen the risks associated with clinical trials are also discussed.

U2 - 10.1016/j.medj.2021.08.005

DO - 10.1016/j.medj.2021.08.005

M3 - Article

C2 - 34617071

SN - 2666-6340

VL - 2

SP - 1011

EP - 1026

JO - Med (New York, N.Y.)

JF - Med (New York, N.Y.)

IS - 9

ER -