Bringing Gene Therapy to the Table | Dr. Jennifer Adair | TEDxNashvilleSalon

I want to start with a poll how many of you here have heard of gene therapy before raising him okay keep your hands up for a second and if you haven't raised your hand yet you probably should but how many of you with your hands up have been treated with or know someone else who's been treated with gene therapy pretty much every hand just went down okay regardless of how you answered those two questions I want you to look at this list of diseases on the screen behind me please raise your hand if you or someone you know has been diagnosed with any of these diseases would it surprise you to know that this is a list of diseases which gene therapy is currently being used to treat and successfully so why don't more people have access to gene therapy because it's complicated and expensive I've been doing gene therapy for nine years in October and I'm gonna tell you two stories the first patient I ever gave gene therapy to and another patient who changed my mindset about how we need to be thinking about how to make treatments available to everyone this is Charlie the first patient that I gave gene therapy to in 2009 Charlie was in his early 50s living with his wife and his daughter in Homer Alaska practicing as a psychiatrist doing what most of us would do on the fourth of July celebrating with friends and family on a lake about midway through the day Charlie came in from a little boat ride and his wife noticed that a speech was slurring she thought at first have you been drinking but he hadn't in fact it was something more serious than that a trip to the ER and head scan showed that Charlie had Stage four brain cancer glioblastoma multiforme II a few months before Charlie's diagnosis in 2009 this disease killed senator Ted Kennedy two weeks ago Senator John McCain was most in fact about a hundred and ninety-two thousand people around the world will be diagnosed with this disease in the next year and seventeen thousand of them will lose their battle with it it's a highly aggressive form of cancer most patients including Charlie only have a 50% chance of surviving one year and that's with intensive therapy that includes surgery chemotherapy radiation more chemo therapy Charlie felt like he needed to try something more and he took the chance on a clinical trial and it happened to be our clinical trial for gene therapy to treat his disease now in 2009 this map is showing you the number of centers around the world who were doing some form of gene therapy now I told you Charlie lived in Alaska so it may seem like he came to Seattle because it was the closest to him but in fact in 2009 we were the only Center doing gene therapy for glioblastoma while each of these centers was capable they were all studying different diseases different clinical trials why did Charlie even have to come to Seattle well the type of gene therapy we were giving him was called blood cell gene therapy we have to first bring him into the clinic take blood cells out of him then we have to move those to a clean room facility because all of the manipulation we're gonna do is gonna happen outside of the body this is an example of what our clean room facility in Seattle looks like controlled airflow highly skilled staff equipment monitoring regulation of every material and reagent that comes in and out multi-million dollar infrastructure once we have the cells there maybe we don't want all the blood cells maybe we only need T cells or maybe we only need stem cells so now we need to separate those out that takes more complex technology and people who know how to use it after that we have to get the DNA into those cells that we want to target this is a snapshot of what part of that process looks like it can take anywhere from 2 to 14 days depending on the cell and the type of the DNA that you're trying to get in you see 3 people in this photo one person who's reading directions line by line one person who's actually performing those instructions one at a time and a third person who's observing to make sure that they all get done correctly and in the right order again multi-million dollar infrastructure after all that manipulation we have to make sure those cells are ready to go back into the patient and that they're safe that we haven't introduced a contaminant that they're functioning the way that they should be and then and only then can they go back to the hospital and get infused into the patient that is of course unless your patient shows up wearing a Michigan shirt when he knows that you are a rival of hi-oh State University fan so I told him I would only let him take this picture if I could cover it up not only to Charlie have to come to Seattle for two weeks to receive this gene therapy had to stay for another two weeks to be monitored after that and then he had to come back to Seattle every three to four weeks for nine months for additional treatment and research so was all that bang worth its buck well in addition to Charlie we cheated treated ten more patients and we changed the survival from less than 50% at one year to 100% at one year Charlie got six and a half years enough time to see his daughter graduate from that horrid University Michigan the ability to go back to work full-time until his retirement but he didn't know that in 2009 I didn't know that in 2009 none of us could have predicted that that would be the outcome so I want to ask you a question you don't need to raise your hand this time if you'd been given a diagnosis with a prognosis of 11 months would you be willing to leave your community your family your friends your co-workers and take a chance on something like this nine out of those eleven months he had to be back and forth in Seattle so when I treated Charlie along with the team at Fred hutch in 2009 this is what I was thinking about thank goodness he was willing to take this chance what if we could move this therapy to where he is how can we move gene therapy to where the 200,000 people that are gonna be diagnosed this year are going to be that was the first time I thought about how can we simplify this think about how to get more clinics doing this that same year I was going to be confronted with a problem in gene therapy of much larger scale in proportion and it was all started by this man Timothy Ray Brown he's also known as the Berlin patient or the only man on the planet right now who's been cured of an HIV infection for more than 10 years now Timothy didn't get gene therapy what he got was a blood cell therapy from a person whose DNA made their blood cells resistant to HIV infection while that was a one-in-a-million treatment for Timothy it paved the way for gene therapy to become a treatment option for HIV positive persons around the world and affect the same type of outcome that would mean not having to take antiretroviral drug drugs every day for the rest of their lives in 2013 dr. Hans Peter King showed here along with the American Foundation for AIDS research the Timothy Ray Brown Foundation and the National Institute of Health received a grant along with other colleagues that Fred it's part of a large collaboratory to study how we could make gene therapy to treat HIV a reality as part of this funding source we in Seattle get to host an HIV cure meeting once a year it's actually going to be in the next few weeks and that meeting not only includes researchers who are working on how to make gene therapy for HIV a possibility it also includes persons living with HIV community advocate members like Josh and others that was in 2014 the first time I saw this map this is a map of HIV prevalence in the in the world darker blue means more HIV positive persons now between 2009 and 2013 the number of clinics capable of doing gene therapy definitely increased but you can see that there's still a very large problem with distribution and we're not talking about 200,000 people a year we're talking about more than 36 million people living with HIV right now another 2.1 million every year 150,000 of which will be children who primarily live in sub-saharan Africa so same meeting sitting here thinking about all of this stuff prominent researcher comes up on the stage talking about gene therapy and how we're making it a possibility person living with HIV sitting in the audience raises their hand and says the hope is amazing how do you envision getting this complex therapy to the millions of patients who need it and the researchers response was well first we have to show it can work and then we'll figure out how to get it there what would you want a treatment that had been developed someplace else on people who didn't share your genetics didn't share your culture didn't share your environment didn't share your lifestyle we know that those things all impact how treatment outcomes are effective so I'm sitting there going I don't think that's okay there has to be a way for us to do this now and if none of these people are gonna stand up and do it then I guess I'll try so I thought about this process all this complex infrastructure and technology that's required well obviously if we could just put the DNA in a syringe and take that around the world and inject it into a person that would be great and I can tell you that we're working on that for some of those diseases on that list we're gonna get there sooner than others but for glioblastoma and HIV not so quickly the reason is that the blood cell we need to hit with our DNA is a stem cell that sits in the bone marrow and they're very few so we don't yet have the technology to inject DNA and have it get to enough of those very few cells in the marrow and not get to a bunch of other cells that we don't want it to go to so we still need to do this outside of the body okay so I know what we mean I have all these things right we got to be able to process the blood product we've got to be able to get to the cells we want we need to be able to keep those cells happy outside the body for a period of time we need to be able to add the DNA in a specific order and sequence so I just started looking what technologies are already out there there has to be some things that I can sort of piecemeal together to make this go right so maybe put it into a box that we can just ship around the world in coincidentally I found one device it's manufactured by a German company it couldn't do gene therapy but it had all the components that we needed actually already in one unit so we bought one we said okay let's see if we can reconfigure this to do what we need to do in the order we need to do it sometimes that took hands-on effort I like to tell people this bag hanger only comes standard on devices purchased in Hawaii in the Caribbean I was just the only one tall enough to reach the time other times we had to blend technologies really complex alignment to get what we needed until we found that someone else also already manufactured that and we can just incorporate it into our system it took two years and more than 20 healthy people donating their blood products to us to practice on as if they were gene therapy patients before we came to this gene therapy in a box on a portable table meets all the same criteria that the multi-million dollar infrastructure cleanroom needs can go anywhere that comes with pre-programmed interfaces in theory all the things that you would need to add the DNA to add the gene can be things that you could get from a pharmacy provided in a kit it's a proof-of-concept hasn't left Fred hutch yet we're working on that part getting the word out there is part of that battle we think that now there's no excuse why we can't be treating close to 60 million people on the planet or at least overcoming those last barriers to getting that much gene therapy out into the world we also think that the more people have access to this type of treatment the more diseases we're going to discover could be benefited by this treatment approach that means that we can treat patients like Charlie patients like Timothy patients like everyone in this room wherever they exist in their own communities so if you think that you might be a candidate for gene therapy talk to your doctor about it they're the ones who are gonna have to let us in the door if you're a healthy person consider donating blood or other tissues to research so that more scientists like me can develop new approaches like this I want to say thank you especially to all of the patients that we treated in Seattle and around the world with gene therapy because they did take a big risk they did step outside their communities to make this possible I thank all of you for your attention you

Channel: TEDx Talks

Views: 45,522

Keywords: TEDxTalks, English, Technology, AIDS, Big problems, Biology, Biotech, Body, Cancer, Disease, Genetics, Global issues, Health, HIV, Medicine, Science, Women

Id: DKsdl23aPdQ

Length: 14min 23sec (863 seconds)

Published: Tue Sep 26 2017