Blue Promise: Can Changing Your Genes Change Your Life? (Part 1)


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 (Music) 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. McCoy and we have a distinguished guest here with us, Jay Weaver is the Associate Vice President of Pharmacy Sales Solutions
from Blue Cross and Blue Shield of Texas. Thanks for being here. JAY: Thank you. ROSS: Okay, 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 hair 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 work 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. JAY: Absolutely. ROSS: 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 effect 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 ask you 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. JAY: That’s correct 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 as is 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. 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. JAY: Yeah. That’s unique… DAN: 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 here, 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 is 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, okay is it the appropriate drug for therapy because
I might… it’s going to A. potentially make an unalterable change in my DNA and 2. 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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