A new medical treatment is giving sight to blind patients and so much more. It's called gene therapy and, though costly, there's no doubt that it’s changing lives. Learn more about this new health care advancement from pharmacist Jay Weaver, AVP of Pharmacy Sales Operations at Blue Cross and Blue Shield of Texas (BCBSTX).
View Part 2 of Can changing your genes change your life?
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Blue Promise is a podcast and online video blog that aims to address complicated health issues with candid conversations from subject matter experts. New editions are published regularly and are hosted by Dr. Dan McCoy, President of Blue Cross and Blue Shield of Texas, and his co-host, Ross Blackstone, Director of Strategic Influence.
DAN:Can changing your genes change your life. A new medical treatment is giving sight to blind patients and so much more. It's called gene therapy and there's no doubt that it’s changing lives, but at what cost? Find out in this episode of Blue Promise
DAN:Thanks for joining us, I'm Dr. Dan McCoy and I'm the President of Blue Cross Blue Shield of Texas, I'm here with my co-host Ross Blackstone.
ROSS:Thank you Dr. McCall and we have a distinguished guest here with us, Jay Weaver is the Associate Vice President of Pharmacy Sales Solutions from BlueCross BlueShield of Texas. Thanks for being here.
ROSS:So, this is an interesting topic right. Gene therapy, we've heard a lot about it over here over the years it seems to be evolving and developing, Jay, just kind of start us off and tell us what is gene therapy?
JAY:Yeah. And I'll just like how fascinating this is that you know when I was in training years ago this was really just being thought of, it was a kind of a dream that we could actually change somebodies’ genetics to improve their health. So, you know when I think about something like gene therapy, I always think about all these terms that people use and it's easy to get lost in the terminology and jargon. So maybe we could do a quick analogy of what this is and kind of set up the conversation. So, if we think about our… people say your genome, what is your genome, your genome is really like the list of all the things all the instructions that make up us as an individual human, I think of the genome is kind of like a library right. It's a series of books and in each of that library. Each of those books are really are our chromosomes. So, everybody's having these chromosome tests these days is 23 and Me and other kinds of testing. If you think about those the chromosome is a list of a whole bunch of genetic information that makes us up and each of those chromosomes a book because there's multiple chapters in that book each of which tell us something about us or hair color eye color something about what makes us up how to make our insulin how to make our skin all of those things. So, within those as you get down to the chapters the chapters really define an area and within those chapters are pages and I think DNA is really like a page of information and sitting on that page are words like in a book. So, in the gene area there's different words and just like in a book if you rearrange those words differently it means a totally different thing. You could take the same 10 words and punctuation and rearrange them and mean something totally different. One time that means here at one time means skin things like that. So we think about gene therapy and what does gene therapy if we if we were to find out in the body that just like in a book if you took and had 10 copies hand written someone takes and copies them and copies them eventually there'd be an error somebody might transpose or switch the order of a word and it might make that sentence mean something very differently. So, as you think about that what is gene therapy, well gene therapy is going back in and we'll get into the ways that we do this but it's going back in and rearranging those words in the right order inside of our body. So, everybody does the right thing. In some cases, it could be that we don't make a protein that we need in our body like insulin or we might not make a certain protein in the lung. That's that that helps our lungs work correctly and by fixing that we don't have to take medicine for it our body sort of fixes itself.
DAN:So, let's just get something get out of the way right here at the beginning, so a lot of times, so people use the word gene therapy. It conjures up controversial ethical issues but in reality, we're not talking about like germ line therapy here where you're altering developing babies and embryos and fetuses like that. We're talking about people that have a disease that somehow the words were garbled in that chapter of the book and this is talking about going into rewriting that chapter just a little bit
JAY:Yeah. That's a great point to call out. I think that you know ethicists and all sorts of folks have a lot of interesting commentary around this. And what we're talking about today as you point out is that we'll use an example. I think the example many people like to use is a disease called cystic fibrosis. In that disease people lack the gene that is responsible for them to create a channel within their lungs and what happens is it's kind of like your car the secretions in the lungs get it's like if you didn't change your oil they get really thick and they can't clear things out of their lungs. And wouldn't it be great if we could just go in and put into that person a fixed copy that gene. So, they make the channel or the duct work if you will that makes their lungs were correctly and then it's like changing your oil and that you know kind of rejuvenate the lungs in a way that they can clear infections.
DAN:It's kind of a good analogy because I think back to those ethical considerations and in fact today the way we treat cystic fibrosis is a whole bunch of therapies that are just downstream from that gene. So, in real reality what you're talking about is just going upstream and changing the alternator in your car example there's not it's not really matter. It's not really a matter of doing anything different than medicine except it moves it really upstream with the source of the problem.
DAN:So how does it work. I mean if everybody's genome is unique to them how does scientists develop and develop a therapy that will fact that those few pages on my, in my book that's different from everybody else.
JAY:Yeah. This is the fascinating part and it'll setup probably some other conversations we'll have as we talk about this some more. But there was a great deal of public research done. It's called the Human Genome Project where we went inside of healthy people and said “What are all the genes that make us up” and we basically wrote the chapters in that book. So, we knew what was the right thing and we went and studied people who have certain disease and say what's different about them.
DAN:But let me just finish a question because I think this is really kind of an interesting nuance. You said a really key word you said. What's the right thing and clearly for some genes there is only one way to put those words together, right, right. But in reality, to the other kind of side of the coin is that there's some ways to tell a story with four or five different ways and still get to the same conclusion right. So that does make it a little complicated.
JAY:Very much and think about just you know us sitting around this table. We have different hair colors and eye colors. Well you know we're all we're all humans and we all have hair and eyes but we just we look at
DAN:But there are some critical genes you just can't misspell the words
JAY:That is correct and that's I think what we're speaking to in these kinds of therapies are those that are we know have a very big impact on health in and cost and quality of life and ultimately longevity of life.
DAN:So back to the way these work and let me kind of throw you some examples and you respond to me. There's a couple of broad categories right. You could theoretically take of a correct or normal gene and you could put it inside of a virus. Right. Because that's the way that we transmit these today to other people as the viruses put it in a virus and give it to people and it would just insert that Gene right into all the cells in your body and fix it right. That phase one.
JAY:That's one of the major.
DAN:And then there's some evolving technology where you could take the correct gene and you could actually put it in a different kind of virus and actually go in and cut out the bad gene and put the good gene in.
DAN:Now this sounds a little sci fi-ish, right.
JAY:Kind of is right. I mean which is fascinating
DAN:It's pretty fascinating. But you can start to see that there's a lot of opportunity to correct some pretty bad diseases. Cystic fibrosis is one, muscular dystrophy would be another, a lot of down the road but today in medicine are we really just kind of talking about the future or is the future starting to be here.
JAY:Yeah that's a great question. Some of the future is now, in fact, a lot of the research that was going on in this space has been going on since the late 1980s and evolving because it asis you can think about as we'd bring new technologies to market often takes decades to understand how to make them safe, how to make them effective, what are the right ways to bring in in fact what you described earlier that that taking a virus and transmitting it into the body was one of the key things we had to work out is how do we how do we make the virus very safe for the person. So, it's you know can do its job but we don't actually react to the virus. You think about just even a common cold we get a cold from a virus and it makes your nose run and makes you feel bad for about a day or two. We certainly don't want those kinds of events. So how do we engineer the delivery, so, we don't actually hurt the person but can bring that. So we've got to make sure that as we look at these new technologies and you know all technologies can have side effects even just an injection giving you an injection of the arm of a very old drug could cause an infection from getting the injection right when you're entering the body. We want to make sure the right person gets the therapy so that we're good stewards of people's health.
DAN:And I think the other thing to kind of keep in mind to and kind of keep me level headed here but a lot of drugs like for high cholesterol and diabetes they're so downstream that they really are reviews of the book you just talked about. I mean they're way downstream. You could you could create a molecule a small molecule drug and it could treat thousands of people just that one molecule of the drug but a lot of times on these genetic diseases they're very targeted to select people that have that level of jumbled code meaning that they're very specific. Much smaller groups of people that are involved with the disease which I think would correct me if I'm wrong lead to them being extraordinarily expensive.
DAN:That's unique to individual patients right.
JAY:Yeah. And I'm going to I'm going to take that a little bit further. I'll come back to that which is really the targets for these today because we're in the early goings of this sort of therapy it takes a very specific target meaning there's multiple reasons that people might get diabetes. We notice that it's a multi genetic sort of disease and there's also lifestyle factors. It would be very hard to correct something like that today with the therapy because we'd have to go and fix maybe ten or twelve genes in somebody's body. So, what we've started with our genes that we know there's a very specific thing to go in. To your point earlier
DAN:Like a switch.
JAY:Yeah right. And so, there's only a handful of diseases that are that specific that we can fix one gene and actually change the person's life. And that's really what we're talking late here. So, to your point it's a very small set of patients and you know to do all that drug development work and to bring something to market over that time for such a small portion of patients is it's going to be costly. Right. It's gonna be a rare occurrence whereas the diabetes drug that you mentioned earlier is leverage over a lot larger group of people. Right and you could bring the cost down.
DAN:And I think the safety part of it. Correct me if I'm wrong to hear but a lot of these therapies are thought to be at least hope, to be, maybe that's a better word one and done right. So, you might get a therapy and you're one an done and it makes a lot of sense right because you're giving someone and DNA and theoretically it's getting put in all their cells. I'm not going to say it's going to be durable forever but regardless that's what the thought processes and correct me if I'm also wrong you're giving it in a virus that you're probably going to get immunity to so you may only get one shot at kind of getting it right. So, it does raise the stakes of asking the question OK. Is it the appropriate drug for therapy because I might… it's going to a potentially make an unalterable change in my DNA and two it might limit me or at least create some roadblocks to getting a further therapy down the road? Is that fair?
JAY:Yeah that that that's a fair point. And to that point what we try to do is make sure to take all of the preponderance of evidence medical evidence and expert opinion and all the different inputs that we can do to make the very best decision around who should who should be candidate for that. Given this early going so we do that in the form of medical policy and authorizations and things like that to really make sure that we can help providers who are, you know also very new at this to partner and find the right combination of the person and the therapy that we hope to get the best outcome from these.
DAN:The scientists in the world are thinking that there's a lot of potential and not just in the traditional genetic diseases that have the switches but also things like cancer where people have tumors in the body and obviously there's a gene that's just wrong in the cancer. But theoretically if you put a fixed gene in that tumor you could turn it off.
JAY:In fact I think like two thirds of the research right now that's being funded through NIH grants and other things is actually in this space to your point cancer.
DAN:Lots to look forward in this particular area and we'll be back to cover that in just a minute. Thanks for joining us for this edition of Blue Promise.
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