Brain Organoids in Parkinson’s Disease Drug Development: Human-Specific Models for Translational Discovery

Organoids are three-dimensional, stem-cell–derived cultures that mimic the structure and function of human tissues, providing improved physiological relevance over traditional 2D cell cultures and animal models. Their ability to replicate patient-specific biology makes them valuable for drug discovery, toxicity testing, and personalized medicine, enabling the prediction of individual responses to therapies. In the context of Parkinson’s disease (PD), midbrain organoids recapitulate key neurodegenerative phenotypes, including dopaminergic neuron loss, α-synuclein aggregation, metabolic dysfunction, and neuroinflammation, and have been successfully used to test various therapeutic strategies. Because PD is highly heterogeneous and current treatments remain largely symptomatic, there is an urgent need for improved preclinical models to overcome translational failures. Organoids offer a powerful solution to accelerate the development of effective disease-modifying treatments.