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Longevity Briefs: The Lab-grown Mini-organs Transforming Drug Discovery

Posted on 23 May 2023

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Longevity briefs provides a short summary of novel research in biology, medicine, or biotechnology that caught the attention of our researchers in Oxford, due to its potential to improve our health, wellbeing, and longevity.

Why is this research important: Have you ever wondered how scientists can test new drugs and treatments for human diseases without using actual humans? One of the most promising methods is to use organoids.

Organoids are miniature 3D structures, created in a laboratory, that mimic the features and functions of real organs. They are often created from human stem cells, which have the ability to turn into any type of cell in the body. By using these lab grown ‘mini-organs’, researchers can study in greater detail how diseases develop and progress, how different individuals respond to drugs, and how to design personalised therapies.

A cross-section of a brain organoid. Source: Madeline A Lancaster/IMBA/EPA

Many human diseases are complex and involve multiple cell types, tissues, and organs. For example, heart disease mainly affects the cardiovascular system; Alzheimer’s disease affects the brain and nervous system; and COVID-19 affects the respiratory systems.

Traditional models for studying these diseases, such as animal models or 2D cell cultures, have limitations and cannot fully capture human physiology and pathology. Organoids offer a more realistic and relevant model that can better reflect the diversity and complexity of human diseases. On top of this, organoids can also be derived from patients’ own cells, allowing researchers to investigate the genetic and environmental factors that influence disease susceptibility and drug response.

Process of organoid generation and drug evaluation

What did the researchers do: Researchers at the Weill Cornell Medicine in New York City reviewed the current progress and future directions in using hPSC-derived organoids for drug discovery and genetic screening.

The review focused on three main types of organoids: endoderm derivatives (such as lung, liver, intestine, and pancreas), mesoderm derivatives (such as heart, kidney, and skeletal muscle), and ectoderm derivatives (such as neural and retinal tissues). The researchers described the protocols for generating different types of organoids, and the phenotypes and molecular pathways that they recapitulate. They also discussed some examples of how organoids have been used to identify novel drugs and targets for a variety of different diseases.

Strategy for deriving differing organoids types

Key takeaway(s) from this research:

Using breakthrough techniques like CRISPR-Cas9, scientists can now study diseases linked to our genes more effectively. This helps them understand complex diseases better and faster.

Technological advancements are bridging the gap between organoids and primary tissues. Organoids and organ systems can now be assembled to more closely simulate human organs, complete with finely tuned and integrated cell-signaling networks.

Due to the expression of key receptors, organoids are becoming essential in studying how our bodies react to viruses. They play a crucial role in testing potential treatments, enhancing our response to diseases like COVID-19.

Organoids have immense potential, but they also have limitations. For instance, they don’t fully mature like actual organs and can be slightly different from each other. These factors are important when using organoids for drug testing.

The study points out new technologies, such as organ-on-a-chip and iPSC banks, that are transforming how we use organoids. These advancements are accelerating progress in the field, making organoids an even more powerful tool in discovering new drugs.

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    1. Human pluripotent-stem-cell-derived organoids for drug discovery and evaluation. J. Vandana et al. 2023. Cell, Stem Cell, volume 30, issue 5, p571-591, may 04, 2023. doi:
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