Journal Article

DKFZ-2025-00245

http://join2-wiki.gsi.de/foswiki/pub/Main/Artwork/join2_logo100x88.png Laboratory-Engineered Glioblastoma Organoid Culture and Drug Screening.

Wang, C. (First author)DKFZ* ; Stöffler, N. (First author)DKFZ* ; Liu, H. (Last author)DKFZ*

2025
JoVE Cambridge, MA

This record in other databases:

Please use a persistent id in citations: doi:10.3791/67593

Abstract: Glioblastoma (GBM) is described as a group of highly malignant primary brain tumors and stands as one of the most lethal malignancies. The genetic and cellular characteristics of GBM have been a focal point of ongoing research, revealing that it is a group of heterogeneous diseases with variations in RNA expression, DNA methylation, or cellular composition. Despite the wealth of molecular data available, the lack of transferable pre-clinic models has limited the application of this information to disease classification rather than treatment stratification. Transferring the patients' genetic information into clinical benefits and bridging the gap between detailed descriptions of GBM, genotype-phenotype associations, and treatment advancements remain significant challenges. In this context, we present an advanced human GBM organoid model, the Laboratory Engineered Glioblastoma Organoid (LEGO), and illustrate its use in studying the genotype-phenotype dependencies and screening potential drugs for GBM. Utilizing this model, we have identified lipid metabolism dysregulation as a critical milestone in GBM progression and discovered that the microsomal triglyceride transfer protein inhibitor Lomitapide shows promise as a potential treatment for GBM.

Keyword(s): Glioblastoma: genetics (MeSH) ; Glioblastoma: pathology (MeSH) ; Humans (MeSH) ; Organoids: metabolism (MeSH) ; Organoids: drug effects (MeSH) ; Brain Neoplasms: genetics (MeSH) ; Brain Neoplasms: pathology (MeSH) ; Drug Screening Assays, Antitumor: methods (MeSH) ; Drug Evaluation, Preclinical: methods (MeSH)

Note: #EA:A240#LA:A240#

---

Contributing Institute(s):

1. A240 Molekulare Neurogenetik (A240)

Research Program(s):

1. 311 - Zellbiologie und Tumorbiologie (POF4-311) (POF4-311)

Appears in the scientific report 2025

---

Database coverage:
; BIOSIS Previews ; Biological Abstracts ; Clarivate Analytics Master Journal List ; Essential Science Indicators ; IF < 5 ; JCR ; PubMed Central ; SCOPUS ; Science Citation Index Expanded ; Web of Science Core Collection

---