Lab-grown organoids help researchers model human disease, test experimental drugs and advance precision medicine
What are organoids?
Organoids are self-organizing three-dimensional (3D) structures grown from stem cells that mimic the cellular structure, heterogeneity and function of actual human organs. They are a model of aspects of an organ or tissue, not a complete miniature organ. Because of their biological specificity, they offer advantages over traditional, two-dimensional in vitro research techniques.
How are organoids grown?
Organoids can be derived from two types of stem cells: pluripotent stem cells (PSCs) or adult tissue-derived somatic stem cells.
- PSCs are stem cells capable of developing into many different cell types in the body. Organoids derived from PSCs can recreate aspects of the developmental process through which tissues and organs form.
- Tissue-derived somatic stem cells are found in developed tissues that help maintain and repair those tissues. Organoids derived from adult tissue stem cells model tissue repair rather than early development. This approach generally works with tissues that have some natural capacity to regenerate.
What are organoids used for?
Model diseases: Organoids' cellular organization, genetic profiles and function allow scientists to observe how pathologies develop and evolve - such as breast cancer, fatty liver disease, glaucoma, ulcerative colitis and Alzheimer's.
Personalized and regenerative medicine: By deriving organoids from a patient's own cells, researchers can rigorously test targeted treatments on the patient's specific tissue model, including "tumor avatars" of rare, hard-to-treat cancers. They also allow scientists to study methods to repair and regenerate damaged human tissues.
Drug discovery and testing: Before medications advance to clinical trials, scientists can test how the experimental therapies interact with human tissue. Patient-grown tumor organoids, for example, provide a three-dimensional matrix that mimics the tumor's microenvironment, which are the cells, molecules and structures surrounding a tumor that can influence how it grows and responds to treatment. Scientists can screen drug candidates to see which treatments are most effective against the biological activity of the specific tumor.
Developmental insights: Organoids help scientists see how organs form, grow and interact in real time with complex biological systems, such as the microbiome.
Gender differences in disease: Many diseases, such as heart valve disease, affect men and women differently. Organoids can incorporate age and sex characteristics to better mimic real patients and provide gender-based insights.
What are the limitations of organoids?
Organoids may not fully reproduce an organ's vasculature, immune system, physical environment or interactions with other organs. They do not replace the need to establish drug safety and effectiveness in people through appropriately designed clinical trials.
Organoid-related research at CU Anschutz:
- How CU Anschutz is Bringing Precision Medicine to Patients With Rare Cancers
- CU Ophthalmology Researcher Receives Award to Further Potentially Sight-Saving Glaucoma Research
- CU Cancer Center's Newest Rising Star Focuses on 'Opposite Ends of the Breast Cancer Spectrum'
- Shared Resources Pilot Grant Opens New Frontiers in 3D Cell and Tissue Printing
- CellSight Contributes Light-Sensitive Retinal Organoids and RPE Cells to New AMD Study
- Ophthalmology Leader Sets Sights on Revolutionizing Vision Care
- Cracking the Code for Once 'Incurable' Diseases
Key points:
- Organoids are 3D structures grown from stem cells that reproduce some characteristics and functions of human tissues and organs.
- Researchers can grow organoids from pluripotent stem cells or stem cells derived from adult tissues.
- Scientists use organoids to study disease development, human development and interactions between cells.
- Patient-derived organoids can help researchers test how specific tissues or tumors respond to experimental treatments.
- Organoids offer valuable research models, but do not reproduce every feature of a complete human organ.