From bench to clinic, are organoids ready for modeling drug delivery in brain disease?

Brain diseases remain a significant challenge in the field of modern medicine. The protective function of the blood-brain barrier (BBB) also complicates drug delivery for neurological disorders. However, under certain pathological conditions, the BBB is compromised during the progression of these pathophysiological processes. To integrate with clinical treatments, precise and personalized requirements exist for basic research. Three-dimensional (3D) in vitro systems, particularly organoids, serve as valuable models for preclinical disease research. Starting from the functionality and feasibility of organoids, this review explores their potential roles and applications in brain diseases. It also discusses advanced technologies to overcome in vitro modeling challenges and their applications in evaluating therapeutic strategies and drug delivery. Moreover, with the ability to predict and rapidly screen effective drugs, organoid modeling will be valuable in accelerating the clinical translation of new therapeutics.