ips cells

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-likestate, if you could do it for animals you could also do it for humans -- and if youcould do it for humans, you could do it for patients. and automatically, you have theability to study the cellular and the molecular basis of a person's disorder. and that's exactlywhat's happened over the last 6 or 7 years. human induced pluripotent stem cells, whichis 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 adverseeffects in the dish -- even before they go

into patients. people have been able to generatethese 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 waysin which people have been able to harness an amazing capacity of these cells to self-organizeinto structures that look like the organs that are in your own body. recently therehave been studies that have used these cells to develop what are called organoids. andthat sounds very science fictiony and frankenstein-like. but what they are is they are structures thatthe 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. theyhave the cell types that are very similar

to the cell types in the brain, in the rightorganization. what that does is that allows you to look whether the organization of thesestructures differs between cells from normal non-disease subjects versus patients. andthat's important for diseases like schizophrenia, because the evidence suggests that these aredevelopmental disorders. and to be able to recapitulate -- to show the developmentalprocess all over again -- in a dish is an incredibly powerful tool. and allows you todiscover whether there are mechanisms that aren't functioning properly in these developingstructures from patients. and it allows us to look, in very fine detail, about what theexact defects are in disorders like autism spectrum disorders, schizophrenia, bipolardisorder. because all of these disorders fundamentally

are disorders of communication between thesecells -- not just individually, but in circuits. because, fundamentally, these disorders arecircuit disorders. the question is whether you can actually recapitulate a predictivemodel of diseases using these simplistic cultures. where this technology will have the most relevanceis not so much a doctor taking a cell or tissue from a patient and turning it into cells -- becausethose technologies, or those procedures, will take too long. you wouldn't be able to necessarilydo a test on an individual patient using their own cells. what you can do is understand therules of the game. are there certain patients with say, a certain set of genes, a certainvariant 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 sameway. so once you understand fully the genetics of particular disorders, then you can usethese cells to develop a whole set of drugs that are based on whether they have a certainset of gene variants versus another set of gene variants, then we come to a world ofprecision medicine. it's not personalized medicine, in which a drug is tailored to anyindividual -- but it's a world in which you can say "because you show these certain setof genes, you're more likely to respond to drug x rather than drug y." and you can developdiagnostic tests based on that.