Insights into the application of brain organoids

Despite the significant advances in using animal models in medical research, some preclinical studies from those models failed to be translated into clinical practice successfully. This could be due to subtle differences in the brain structure and cellular composition across species which limit its application.

Therefore, brain organoids recapitulating the characteristics of the human brain have tremendous potential in the field of biological research and medical applications.

This article gives an insight into areas where brain organoids can contribute (1,2,3,4):

  1. Studying brain development: Brain organoids provide a tool to study the complex processes of human brain development. Researchers can gain insights into abnormalities associated with neurodevelopmental disorders by observing the formation of neural structures, neuronal migration, and the development of different cell types.
  2. Neurological Disorders: Brain organoids can be used to model and investigate various neurological disorders, including schizophrenia, autism spectrum disorders, epilepsy, and neurodegenerative diseases such as Parkinson’s and Alzheimer’s. By studying the development of brain organoids derived from patients’ cells, researchers can gain insights into disease mechanisms and potentially develop new treatment strategies.
  3. Drug discovery and screening: Brain organoids can be employed to test the different compounds and assess their effects on neuronal development to analyze the efficacy and safety of potential drugs targeting neurological disorders. It can help to identify promising drug candidates reducing reliance on animal models. Further improvement in patient care can be implemented using brain organoids derived from patient-specific cells to tailor precise treatment plans.
  4. Neurotoxic and environmental effects: By exposing organoids to specific compounds or toxins, brain organoids can be used to assess the neurotoxicity of chemicals and environmental factors. Researchers can evaluate the impact of such harmful environmental factors on brain development and function, helping to identify potential risks and inform regulatory decisions.

Continued research and technological advancements in this area are crucial to unlocking their full potential.


  1. Sun, N., Meng, X., Liu, Y. et al. Applications of brain organoids in neurodevelopment and neurological diseases. J Biomed Sci 28, 30 (2021).
  2. Qian X, Song H, Ming GL. Brain organoids: advances, applications and challenges. Development. 2019 Apr 16;146(8):dev166074. doi: 10.1242/dev.166074. PMID: 30992274; PMCID: PMC6503989.
  3. Shou Y, Liang F, Xu S, Li X. The Application of Brain Organoids: From Neuronal Development to Neurological Diseases. Front Cell Dev Biol. 2020 Oct 22;8:579659. doi: 10.3389/fcell.2020.579659. PMID: 33195219; PMCID: PMC7642488.
  4. Chen HI, Song H, Ming GL. Applications of Human Brain Organoids to Clinical Problems. Dev Dyn. 2019 Jan;248(1):53-64. doi: 10.1002/dvdy.24662. Epub 2018 Oct 2. PMID: 30091290; PMCID: PMC6312736.