Brain Organoid Platform for Parkinson’s Disease Drug Discovery

Background

With the global rise in life expectancy, neurodegenerative diseases, such as Parkinson’s disease, have become an increasing health burden. Conventional two-dimensional cell cultures fall short in mimicking the brain’s natural environment, limiting their effectiveness in drug discovery.

In contrast, brain organoids offer a breakthrough as three-dimensional, multi-cell type tissue cultures derived from stem cells. These organoids closely replicate the structure and function of human brain regions in vitro, providing a more accurate platform for evaluating potential drug efficacy.

Earlier organoid platforms faced significant challenges, particularly in their complex cultivation requirements. OIST scientists have pioneered an innovative brain organoid platform that addresses these challenges, enhancing its commercial viability and setting a new standard in organoid technology. This advancement not only holds promise for Parkinson’s disease drug discovery but also paves the way for more effective treatments for other neurodegenerative disorders.

Technology Overview

OIST scientists have developed an innovative protocol to generate 3D human brain organoids that demonstrate the progressive aggregation of alpha-synuclein and the formation of Lewy body-like inclusions. This human-specific Parkinson’s Disease (PD) model utilizes midbrain-specific organoids derived from human pluripotent stem cells. These cells carry PD-linked genetic mutations introduced through CRISPR-Cas9-mediated genome engineering.

Remarkably, these organoids exhibit strong pathophysiological signatures, including neuronal loss. This in vitro PD modeling system offers a promising tool to demonstrate PD-specific phenotypes. It provides a versatile platform for understanding the pathophysiological mechanisms of PD. Consequently, the 3D organoid model with PD-associated genetic mutations fulfills the need for a consistent and user-friendly drug screening platform.

Benefits

• Advanced 3D human disease model
• Easy to use protocol
• High reproducibility
• Reliable drug testing platform

Applications

• Drug Discovery for Neurodegeneration
• Regenerative Medicine
• Disease Pathology