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Is Patient Organoid Testing the Future of Prescription Medicine?
In the Netherlands, the Dutch health minister and biotech company Vertex shut down negotiations in May to make the company's new prescription drug available to the public. The negotiations didn't go well because the price is way too high.
However, prescription drug prices are a problem all over the world with many companies. This is why a coalition of biologists, insurers and patient groups are proposing organoid testing to find out how well new drugs work for specific patients.
Organoid Characteristics and How Scientists Make Them
Organoids are clusters of cells that are smaller than sesame seeds and resemble organs in many ways. Scientists have made these miniature organs to resemble hearts, livers, kidneys, mammary glands, pancreases, salivary glands and stomachs.
However, they don't have all of the features that they need to grow and function like real organs, such as a system of blood vessels. Despite this, they're far advanced from the 2D cell cultures that scientists were growing in labs. The new technology lets researchers see how infections and genetic mutations hinder organ function.
Scientists make organoids from stem cells. Some of them use pluripotent stem cells, which are isolated from human embryos or reprogrammed from mature cells. In theory, they can be turned into any cell type. Others use adult stem cells that they isolate from the tissues that line the organs and help them heal after injuries. The structure is simpler than pluripotent stem cells, but the organoids can show how genetic differences alter organ functions or how the body responds to certain drugs.
Researchers grow the stem cells under precise conditions that transform them into numerous cell types that cooperate and self-organize. During the first weeks and months, the organoids portray how organs grow, allowing scientists to detect defects during the process. Cincinnati Children's Hospital Medical Center's Developmental Biologist James Wells describes it as being able to watch a birth defect unfold in a dish.
Using Organoid Testing to Determine the Effectiveness of New Prescription Drugs
Researchers have successfully used organoids to learn how the Zika virus affects developing brains. Now, they believe that they can use organoids personalized for patients to determine whether or not new drugs will work for them. Hubrecht Institute's Stem Cell Biologist Hans Clevers describes organoids as patient avatars, allowing them to see which patients will benefit the most from certain drugs. In the cases where this technology is being used in trials, several insurers have started covering expensive prescriptions.
If scientists can move forward with using organoids to test the effectiveness of all new drugs in patients, it could have a substantial effect on the drug business. The technology may be used around the world to decide who receives the newest pharmaceuticals, and it could make treatments more affordable.
However, it's still unknown how well organoids mimic real organs because they don't have all of the elements of real organs. Also, it's hard for scientists to create large, consistent clusters of organoids to standardize disease and drug testing.
The Potential for Transplanting Organoids to Replace Tissues
Beyond using organoids to test drug effectiveness, scientists say that there's potential to use them as transplants to repair or regrow organs. They've been able to successfully graft colon and liver organoids in mice. However, they need to be able to make larger organoids for this to be successful in humans. Wells estimates that it could to take 10 more years before it's possible to even try it.