Standard drug development processes have always relied on animal trials, particularly rodents, for validation of drug efficacy and safety before entering a human body. One of the biggest challenges facing life sciences researchers is the mere fact that mice are simply not humans. The use of animal models has clear limitations to drug development due to their significant differences from human beings. This is one crucial reason why clinical trials to this day still have high failure rates and the costs of drug discovery are skyrocketing. We urgently need to find new and better ways to test drugs that can more accurately predict and model diseases.
The advent of 3D cell organoids might present one piece of the solution and is an area we are paying close attention to. Organoids are basically cultured tiny versions of human organs. It is a type of cell culture that allows cells to grow in a complex, three-dimensional structure that more closely mimics the composition and architecture of cells in the human body. In contrast to traditional 2D cell cultures, which grow cells on a flat surface, organoids can more realistically resemble the structure and function of real organs. Derived from stem cells, they have the ability to self-regenerate and self-organize, and differentiate into all major cell types. New innovative techniques such as microfabrication and 3D printing are also expanding the possibilities and scale of what can be achieved with this new technology.
There is a wide range of organoids that can be created, corresponding to all the different types of tissues and organs in the human body. So far, scientists have already been able to develop organoids that resemble the brain, kidney, lung, intestine, stomach, and liver. These can then be used to model diseases including cancer, developmental disorders, and genetic diseases. Organoids are therefore a promising tool that will allow us to gain new insights into how tissues form and grow, observe the behavior and progression of cancer cells, and study the interactions between drugs and these miniature organs in order to test the efficacy of drug candidates. This technology will be particularly important for complex and uniquely human tissues and diseases, such as neurodevelopmental or neuropsychiatric diseases like autism spectrum disorder and schizophrenia. Organoids derived from patients with autism have already given us an inside look into genetic abnormalities that affect cell growth. Researchers have also used organoids to study how the Zika virus causes microcephaly during early embryonic development by disrupting the normal differentiation of neuron-producing cells.
Organoids are not only useful in helping us understand the early stages of diseases, but also have tremendous potential as therapeutic tools. One application involves attempts to transform or transplant cells developed from organoids for treat diseases. For example, researchers are finding ways to convert intestinal epithelial cells into insulin-producing beta cells, which they tested using intestinal organoids. This could be potentially game-changing for the treatment of diabetes. It may even be possible to create organoids from a patient's own cells (iPS cells, or Induced Pluripotent Stem Cells). This can be extremely useful to the study of rare diseases, where there is a low patient population and short supply of trial data, making progress in the field slow and limited. The ability to generate almost limitless amount of tissue or cells from individual patients will enable patient-specific treatment plans and open new doors for personalized medicine. Finally, by enabling the growth of cells at three dimensions in high density in a much more time- and cost-efficient manner, organoids can accelerate and enhance the entire drug discovery process for specific diseases and expand the surface area of therapeutic targets possible to be discovered and designed. By unlocking unforseen insights into human biology and pathology, organoids present endless possibilities in the coming years and we are just seeing the first innings of it.