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SCIENTIFIC LITERATURE · MED

Brain organoids in Parkinson's disease drug development: Human-specific models for translational discovery.

Drug Discov Today · 2026-07-17

DOI: 10.1016/j.drudis.2026.104742PMID: 42468620

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Research overview

The text below is the source article’s original abstract under a verified reuse license. cc by

Parkinson's disease (PD) poses a major unmet therapeutic challenge, with most drug candidates failing in clinical translation despite promising animal model data. Human induced pluripotent stem cell-derived midbrain organoids recapitulate key PD pathological hallmarks - including dopaminergic neuron loss, α-synuclein aggregation, and neuroinflammation - in a genetically defined, human-specific context. This review summarizes drug screening studies in midbrain organoids across genetic, toxin-based, and α-synuclein preformed fibril models. We highlight therapeutic interventions that rescue PD phenotypes and discuss the personalized medicine potential of patient-derived organoids. We also critically assess current limitations and outline how artificial intelligence integration and assembloid platforms are advancing organoid-based drug discovery towards regulatory acceptance.

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