Reliability of high-quantity human brain organoids for modeling microcephaly, glioma invasion and drug screening

Nat Commun. 2024 Dec 19;15(1):10703. doi: 10.1038/s41467-024-55226-6.

Abstract

Brain organoids offer unprecedented insights into brain development and disease modeling and hold promise for drug screening. Significant hindrances, however, are morphological and cellular heterogeneity, inter-organoid size differences, cellular stress, and poor reproducibility. Here, we describe a method that reproducibly generates thousands of organoids across multiple hiPSC lines. These High Quantity brain organoids (Hi-Q brain organoids) exhibit reproducible cytoarchitecture, cell diversity, and functionality, are free from ectopically active cellular stress pathways, and allow cryopreservation and re-culturing. Patient-derived Hi-Q brain organoids recapitulate distinct forms of developmental defects: primary microcephaly due to a mutation in CDK5RAP2 and progeria-associated defects of Cockayne syndrome. Hi-Q brain organoids displayed a reproducible invasion pattern for a given patient-derived glioma cell line. This enabled a medium-throughput drug screen to identify Selumetinib and Fulvestrant, as inhibitors of glioma invasion in vivo. Thus, the Hi-Q approach can easily be adapted to reliably harness brain organoids' application for personalized neurogenetic disease modeling and drug discovery.

MeSH terms

  • Animals
  • Benzimidazoles / pharmacology
  • Brain Neoplasms / pathology
  • Brain* / drug effects
  • Brain* / pathology
  • Cell Line, Tumor
  • Drug Evaluation, Preclinical* / methods
  • Glioma* / drug therapy
  • Glioma* / genetics
  • Glioma* / pathology
  • Humans
  • Mice
  • Microcephaly* / genetics
  • Microcephaly* / pathology
  • Neoplasm Invasiveness
  • Organoids* / drug effects
  • Organoids* / pathology
  • Reproducibility of Results

Substances

  • Benzimidazoles