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Application of human 3D brain organoids in regenerative medicine and drug testing.

Description:

 

Human 3D brain organoids for regenerative medicine and drug testing

 

Technology Overview

 

Current drug discovery strategies often utilize cell lines and animal models for

compound development, drug screening, efficacy testing, and establishing dose-responses. However, such model systems are often limited, particularly for brain disorders and cancers. Traditional cell culture systems can unintentionally alter physiological properties of cells given the artificial 2D environment, while animal models are very costly and do not fully recapitulate human disorders or diseases, resulting in less efficient and expensive drug discovery. An improved high throughput model system is necessary that maintains fidelity to in vivo human physiological properties yet can be

easily manipulated and tested.

 

Dr. Song and colleagues have established human 3D brain organoid systems that closely resemble human development and have already been applied for modeling several brain disorders. Using a highly optimized protocol consisting of human stem cells, tissues or cancer cells and miniaturized spinning bioreactors, 3D brain organoids can be synthesized that recapitulate in vivo properties, contain more functional and mature cells, and have increased yield, stability and survivability. These brain organoids have been utilized for understanding the underlying mechanism of the Zika virus, screening neuroprotective compounds, modeling glioblastoma and identifying personalized therapeutics.

 

 

The maintenance of cellular, mechanical and topographical cues in the 3D

microenvironment enables enhanced in vitro modeling of brain cancer, and

neuropsychiatric and neurodegenerative diseases, to improve and accelerate

the efficiency, safety, discovery and cost of drug screening for brain cancer and neuropsychiatric and neurodegenerative diseases.

 

Overview of human 3D brain organoid generation and use in drug screening. 1) Using human-derived cells (e.g., human stem cells, tissue, cancer cells) 2) 3D brain organoids are developed that recapitulate in vivo physiological properties. Applications for brain organoids include 3) evaluating brain development and disease modeling; 4) use in cell therapy; and 5) high throughput drug screening, where ultimately drug hits and validation will 6) lead to the development of novel pharmacotherapies.

 

ADVANTAGES

 

• Improves efficiency and reduces costs of modeling brain disorders and cancers.

• Improves testing of personalized therapeutics and drug response.

• Maintains 3D brain-like cytoarchitecture that recapitulates and retains physiological properties compared to 2D cell lines.

 

 

APPLICATION

 

• Test beds for drug screening and genomic manipulation

• Modeling and understanding brain disease pathogenesis

 

 

STAGE OF DEVELOPMENT

 

• Reliable generation using miniaturized spinning bioreactors

• Applied use for Zika virus (mechanism & screening of therapeutic compounds), and glioblastoma (screening personalized therapeutics)

 

 

REFERENCE MEDIA

 

• Qian et al. Cell, 2016, 165 (5) -1238

• Qian et al. Nat Protoc, 2018 13(3) – 565

• Vissers et al. Adv Drug Deliv Rev, 2019

 

DESIRED PARTNERSHIPS

 

Sponsored Research Agreements

 

 DOWNLOAD PDF

 


Patent Information:
For Information, Contact:
Neal Lemon
Associate Director, PSOM Licensing Group
University of Pennsylvania
nlemon@upenn.edu
Inventors:
Hongjun Song
Keywords: