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iPS Cells & Organoids - Sci-Fi vs Reality

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Transcript

David Panchision: Having this new tool is really a breakthrough. It's a game-changer in the ability to study disease and identify new treatments for disease.  If you could take any cell in the body and turn it into this immature stem cell-like state, if you could do it for animals you could also do it for humans -- and if you could do it for humans, you could do it for patients.  And automatically, you have the ability to study the cellular and the molecular basis of a person's disorder.  And that's exactly what's happened over the last 6 or 7 years. Human induced pluripotent stem cells, which is what we call them now, iPS cells, then you can use these cells as a disease-in-a-dish, in order to identify new treatments. And you can determine whether they might have adverse effects in the dish -- even before they go into patients.  People have been able to generate these cells from people with schizophrenia, people with autism, people with bipolar disorder.  And its really opened up a whole new world of investigation to us.  There are other ways in which people have been able to harness an amazing capacity of these cells to self-organize into structures that look like the organs that are in your own body. Recently there have been studies that have used these cells to develop what are called organoids.  And that sounds very science fictiony and Frankenstein-like.  But what they are is they are structures that the cells make all by themselves -- with a little bit of coaxing from the scientists -- but they look like miniature brains.  And they have the organization of the brain.  They have the cell types that are very similar to the cell types in the brain, in the right organization. What that does is that allows you to look whether the organization of these structures differs between cells from normal non-disease subjects versus patients.  And that's important for diseases like schizophrenia, because the evidence suggests that these are developmental disorders.  And to be able to recapitulate -- to show the developmental process all over again -- in a dish is an incredibly powerful tool. And allows you to discover whether there are mechanisms that aren't functioning properly in these developing structures from patients.  And it allows us to look, in very fine detail, about what the exact defects are in disorders like autism spectrum disorders, schizophrenia, bipolar disorder.  Because all of these disorders fundamentally are disorders of communication between these cells -- not just individually, but in circuits. Because, fundamentally, these disorders are circuit disorders. The question is whether you can actually recapitulate a predictive model of diseases using these simplistic cultures.  Where this technology will have the most relevance is not so much a doctor taking a cell or tissue from a patient and turning it into cells -- because those technologies, or those procedures, will take too long. You wouldn't be able to necessarily do a test on an individual patient using their own cells. What you can do is understand the rules of the game. Are there certain patients with say, a certain set of genes, a certain variant of genes, which would allow them to respond in a certain way to particular drugs, while other patients who might have the same disease may not respond in exactly the same way.  So once you understand fully the genetics of particular disorders, then you can use these cells to develop a whole set of drugs that are based on whether they have a certain set of gene variants versus another set of gene variants, then we come to a world of precision medicine. It's not personalized medicine, in which a drug is tailored to any individual -- but it's a world in which you can say "because you show these certain set of genes, you're more likely to respond to drug X rather than drug Y." And you can develop diagnostic tests based on that.