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Targeting IgA Nephropathy (Sponsored Content) · Masters in Business

• 0:00
  Speaker 1: Since you're a subscriber to this Bloomberg podcast, we thought
• 0:03
  Speaker 1: you'd be interested in a six episode sponsored podcast called
• 0:07
  Speaker 1: Targeting the Toughest Diseases produced by Vertex Pharmaceuticals and Bloomberg
• 0:12
  Speaker 1: Media Studios. It explores the innovative tools, methods, and unique
• 0:17
  Speaker 1: philosophy Vertex Pharmaceuticals is using to search for treatments for
• 0:21
  Speaker 1: some of humanity's most challenging diseases. Here's a recent episode.
• 0:30
  Speaker 2: Two year olds are a handful. They're full of energy
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  Speaker 2: and full of laughter. A lot of work for sure,
• 0:38
  Speaker 2: but also a source of joy.
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  Speaker 3: I definitely feel my calling in life was to be
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  Speaker 3: a mother, and I just feel so blessed to be
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  Speaker 3: able to have that opportunity to have a child. So
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  Speaker 3: it's been great.
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  Speaker 2: That's Rachel Benton, also known as Stetson's.
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  Speaker 3: Small Stetson is his name, Stetson Corey Benton. I've always
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  Speaker 3: liked unique names. I never knew of anyone named Stetson before.
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  Speaker 2: The thing about Stetson is, and he doesn't even know
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  Speaker 2: this yet, but he may have already saved his mom's life.
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  Speaker 2: A routine medical test that Rachel only took because she
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  Speaker 2: was pregnant showed protein in her urine That led to
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  Speaker 2: further tests, a biopsy, and eventually a phone call delivering
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  Speaker 2: a diagnosis. It was a.
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  Speaker 3: Pretty quick conversation. We know it is igiena fhropathy. It's
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  Speaker 3: a rare kidney disease. There is not a cure. We
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  Speaker 3: don't have a cause.
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  Speaker 2: The call was short, but for Rachel it changed everything. Hi.
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  Speaker 2: I'm Jordan Gospore, a member of the University of Southern
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  Speaker 2: California's Center for Health Journalism. This is Targeting the Toughest
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  Speaker 2: Diseases a podcast produced by Bloomberg Media Studios and Vertex Pharmaceuticals.
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  Speaker 2: In this series, we look at some of humanity's most
• 2:14
  Speaker 2: challenging diseases and how Vertex, a Boston based biotech company,
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  Speaker 2: is using innovative tools, methods, and a unique philosophy to
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  Speaker 2: search for treatments and cures. Today, we're looking at immunoglobulin
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  Speaker 2: A nephropathy, more commonly known as IgA nephropathy. It's a
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  Speaker 2: progressive chronic kidney disease that, for too long, was underreported
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  Speaker 2: and under researched. We'll talk to experts inside and outside
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  Speaker 2: of Vertex about the latest work being done in this field,
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  Speaker 2: But first back to Rachel's story. At the age of thirty,
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  Speaker 2: Rachel bent and was living a great life. She was
• 3:02
  Speaker 2: a licensed mortgage broker running her own agency. She owned
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  Speaker 2: her own house, and that fall she decided to throw
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  Speaker 2: a big Halloween.
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  Speaker 3: Party and he came with one of our mutual friends
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  Speaker 3: and we sort of rekindled from there.
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  Speaker 2: He was Tom, a guy she had dated on and
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  Speaker 2: off during her twenties.
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  Speaker 3: And then it was the end of November of twenty
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  Speaker 3: twenty that he asked me to marry him.
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  Speaker 2: A year later, they got married, and shortly after that,
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  Speaker 2: Rachel got pregnant. During her pregnancy, Rachel had regular medical checkups,
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  Speaker 2: including routine yearine tests.
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  Speaker 3: We did a yearine test, a twenty four hour year
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  Speaker 3: in test, and that's where you have to take this
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  Speaker 3: big jug home and you have to peece straight into
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  Speaker 3: this jug every time for twenty four hours. And so
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  Speaker 3: I bring it to the doctor and they test it,
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  Speaker 3: and sure enough, they found protein in my earn.
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  Speaker 2: The doctor told her she wasn't facing any immediate risk,
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  Speaker 2: so they agreed to put that result aside, focus on
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  Speaker 2: having a healthy baby, and that they would investigate further
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  Speaker 2: once the baby was born Stetson. Corey Benton was born
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  Speaker 2: in May and as a new parent, Rachel's life instantly
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  Speaker 2: became a whirlwind.
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  Speaker 3: Honestly, I completely even forgot about the kidney stuff.
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  Speaker 2: But around the nine month mark she reached out to
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  Speaker 2: her doctor. She retested her urine, which confirmed she still
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  Speaker 2: had protein in her urine, so they decided to do
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  Speaker 2: a kidney biopsy. A biopsy is where a doctor uses
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  Speaker 2: a long needle to extract tiny pieces of tissue so
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  Speaker 2: they can do further tests. Rachel got the results over
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  Speaker 2: the phone.
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  Speaker 3: And he's like, you know, we did confirm some things.
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  Speaker 3: You have a rare kidney disease called IgA nephropathy or
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  Speaker 3: I GAN. So my fir question was, you know, what
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  Speaker 3: do I do now? How do I stop this? Is
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  Speaker 3: there a cure?
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  Speaker 2: You know?
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  Speaker 3: Just thinking of anything, like what's the next steps? What
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  Speaker 3: about medication? Blah blah blah.
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  Speaker 2: And he's like, well, the answer to almost all of
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  Speaker 2: her questions was we're not sure or it depends.
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  Speaker 3: I got off the phone and just cried. I think
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  Speaker 3: for probably two days straight. I just was overwhelmed with
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  Speaker 3: obviously the diagnosis and not knowing where this would take
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  Speaker 3: me in life. What the next You know, twenty four
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  Speaker 3: hours would look like a year or ten years. You know,
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  Speaker 3: how is this going to affect me in my day
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  Speaker 3: to day life and my son and my husband.
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  Speaker 2: She's adjusted her diet and takes medication to address her
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  Speaker 2: blood pressure and cholesterol, but the progress of the disease
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  Speaker 2: is variable. At one point, Rachel's kidney function dropped thirty
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  Speaker 2: points over two months. The doctor told her that at
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  Speaker 2: that rate, she'd be needing dialysis within a year. She's rebounded,
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  Speaker 2: but she doesn't know if that recovery will hold. She
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  Speaker 2: also deals with fatigue, not just new mom tiredness, but
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  Speaker 2: debilitating fatigue, where she'll sleep for twelve hours and still
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  Speaker 2: need a midday nap. In fact, Rachel says that for
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  Speaker 2: a couple of years before her diagnosis, she was already
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  Speaker 2: dealing with extreme tiredness, but never really considered it a
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  Speaker 2: medical condition or something that needed attention. In hindsight, she
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  Speaker 2: wishes she had gotten it checked out. Looking forward, her
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  Speaker 2: long term health and her ability to be the kind
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  Speaker 2: of mom she wants to be is uncertain.
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  Speaker 3: I have my days where I just feel very, very
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  Speaker 3: guilty being a other to him with a rare disease
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  Speaker 3: and not knowing in a year, ten years, however long,
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  Speaker 3: how I'm going to be and how active I get
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  Speaker 3: to be in his life. Every day. I just try
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  Speaker 3: to make the best of it though, and you know,
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  Speaker 3: whatever he wants to do, I put on a smile
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  Speaker 3: and try to push.
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  Speaker 2: Through immunoglobulin A nephropathy or IGAN. It's not a high
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  Speaker 2: profile disease. It's a pronic kidney disease that historically has
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  Speaker 2: not been well understood, but that's changing.
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  Speaker 4: So I do clinics, so I see patients with gramariala disease.
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  Speaker 4: I run clinical trials, and I have my research work
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  Speaker 4: at the university and the laboratory.
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  Speaker 2: That's doctor Jonathan Barrett. He's based at the University of
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  Speaker 2: Leicester in England and as a world renowned leader in
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  Speaker 2: kidney research.
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  Speaker 4: So most of my career, no one was really interested
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  Speaker 4: in Nigeria property apart from a few people. Now it
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  Speaker 4: is you can't move at a kidney meeting without people
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  Speaker 4: talking about igenafropathy.
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  Speaker 2: One reason it was so under researched is that in
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  Speaker 2: the early stages, symptoms are often hard to detect.
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  Speaker 4: Normally, the patients I see have no idea why they're
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  Speaker 4: coming to see a kidney doctor.
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  Speaker 2: In many cases, it starts with an asymptomatic person having
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  Speaker 2: a urine test, like in Rachel's case, with a routine
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  Speaker 2: urine test during pregnancy.
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  Speaker 4: Perhaps you've joined a new gym, you get a physical,
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  Speaker 4: so you want to get a mortgage. You might have
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  Speaker 4: a job that requires you to have a physical and
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  Speaker 4: someone dips your urine and they don't wait a minute,
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  Speaker 4: there's blood and protein in here.
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  Speaker 2: Here's what we do know about the disease.
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  Speaker 4: So each kidney contains about a million filters and they
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  Speaker 4: are continually filtering the blood to generate the urine, and
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  Speaker 4: in igenafropathy, this protein called IgA starts sludging up those
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  Speaker 4: filters and stopping them from working properly. And if the
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  Speaker 4: filters don't work very well, they become leaky and blood
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  Speaker 4: and protein can appear in the urine.
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  Speaker 2: Over time, sometimes months, sometimes years, your kidney function declines.
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  Speaker 4: Some patients do really well with igenafropathy, and others end
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  Speaker 4: up on dialysis relatively quickly. And that's the bit we
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  Speaker 4: haven't quite worked out yet.
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  Speaker 2: In fact, we know the majority of adult patients with
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  Speaker 2: igaafhropathy will progress to instage kidney disease, which requires them
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  Speaker 2: to go on dialysis or get a transplant within twenty
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  Speaker 2: years of diagnosis. Doctor Barrett says there is evidence of
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  Speaker 2: a genetic component, but the connections aren't strong enough to
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  Speaker 2: do genetic screening right now. The only way to diagnose
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  Speaker 2: iganaphropathy is with the kidney biopsy.
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  Speaker 4: But what we think is that the major driver for
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  Speaker 4: this disease is the type of ida we normally produce
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  Speaker 4: in our gut and in our respiratory tract that normally
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  Speaker 4: gets into our sputum, into our tears, into our saliva.
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  Speaker 4: It's there to stop bugs entering the body, and it
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  Speaker 4: does a very very good job. But in igenic property,
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  Speaker 4: some of that ida actually gets into the bloodstream. When
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  Speaker 4: it's in the bloodstream, it's a bit out of place,
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  Speaker 4: and it tends to stick to itself, to stick to
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  Speaker 4: other proteins. There are antibodies in the circulation it sticks to,
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  Speaker 4: and that forms these large what we call immune complexes,
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  Speaker 4: which are essentially big aggregates of protein. If you've got
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  Speaker 4: big globs of protein, it's completely reasonable to think that
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  Speaker 4: those globs approtein are going to start clogging up the filter,
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  Speaker 4: which is exactly what happens, and when they clog up
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  Speaker 4: the filter, there's inflammation, they're scarring, and that's what eventually
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  Speaker 4: causes kidney failure.
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  Speaker 2: For people diagnosed with IgA nefhropathy. The biological effects are
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  Speaker 2: only part of the problem.
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  Speaker 4: The big challenge is actually the impact on mental wellness.
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  Speaker 4: Because if you're a young person and I tell you
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  Speaker 4: you've got this disease, it's incurable. There's a risk it
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  Speaker 4: could cause kidney failure in the future, but I can't
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  Speaker 4: tell you when you might need to go on diallasis
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  Speaker 4: have you get your transplant, but I can't tell you when.
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  Speaker 4: That sense of uncertainty can be absolutely devastating for young people.
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  Speaker 4: You imagine developing your career, wanting to pay that big
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  Speaker 4: amount of money for a house, getting married, thinking about children,
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  Speaker 4: and you have this threat hanging over you of a
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  Speaker 4: disease that the doctors can't really predict.
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  Speaker 2: Addressing that fear and uncertainty requires showing patients there is
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  Speaker 2: a way forward, and that's what the team at Vertex
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  Speaker 2: is doing. Igienephropathy represents exactly the kind of medical problem
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  Speaker 2: Vertex is targeting. There's a long history of research to
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  Speaker 2: build on there's new technology available or Vertex thinks they
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  Speaker 2: can develop it. There's a large unmet need, and Vertex
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  Speaker 2: thinks it may be possible to tackle the underlying disease.
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  Speaker 5: Simply put, Verticy's goal is to help people suffering from
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  Speaker 5: serious diseases, and we do that by investing in the
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  Speaker 5: scientific innovation to create what we believe could be truly
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  Speaker 5: transformative medicines for people with these diseases.
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  Speaker 2: That's Mark Bunnage. He's the head of research at Vertex
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  Speaker 2: Pharmaceuticals and leads research on the company's pipeline of serious diseases.
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  Speaker 5: Now, we focus on cause of human biology and on
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  Speaker 5: human validated targets, and then we ask the question what's
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  Speaker 5: the right approach to take, and we are completely metalitiagnostic.
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  Speaker 5: We'll use whatever is the right tool in the toolbox
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  Speaker 5: if you like, to address their biology and then to
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  Speaker 5: come up with a transformative therapy.
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  Speaker 2: Earlier, doctor Barrett explained how igianophropathy occurs. It's a build
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  Speaker 2: up of what he calls big globs of protein that
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  Speaker 2: deposit and cause injury to the filters in your kidneys.
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  Speaker 2: But how can we stop that from happening. To answer that,
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  Speaker 2: I sat down with doctor Maniche Maski. He's a board
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  Speaker 2: certified nephrologist helping lead Vertex research into IgA nephropathy. Doctor
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  Speaker 2: Maski explained that B cells, an important part of our
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  Speaker 2: immune system to fight off infections, can lose control and
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  Speaker 2: produce antibodies that hurt our own organs, in this case,
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  Speaker 2: the kidneys. Can you tell us a little bit about
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  Speaker 2: where the field is going and what's being done for
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  Speaker 2: people living with IgA nephropathy.
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  Speaker 6: Absolutely, we now understand that loss of B cell control
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  Speaker 6: is what leads to the downstream mechanisms resulting in kidney
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  Speaker 6: injury and kidney function loss. So lots of research has
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  Speaker 6: been performed to try to understand what is influencing the
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  Speaker 6: B cell to lose control. It turns out that two
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  Speaker 6: proteins called Bath and april are key drivers.
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  Speaker 2: Okay, so B cells are a type of white blood
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  Speaker 2: cell that helps power our immune system. Can you tell
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  Speaker 2: us a little bit more about what Bath and April
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  Speaker 2: are and how they're involved in B cell control and
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  Speaker 2: how all that relates to iganephropathy.
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  Speaker 6: So, first, Jordan, you're absolutely right that B cells, when
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  Speaker 6: they're doing what they're supposed to do, protect us from infections.
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  Speaker 6: They make cand of bodies that help us neutralize various pathogens.
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  Speaker 6: When B cells lose control, they can set off a
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  Speaker 6: cascade of events that result in processes that harm the body,
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  Speaker 6: so called autoimmune diseases. Bath and April are two similar
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  Speaker 6: but distinct proteins that control the life cycle of the
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  Speaker 6: B cell. So bath more in the early stage of
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  Speaker 6: development and maturation of the B cell and april more
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  Speaker 6: in the later stage of the B cell development all
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  Speaker 6: the way through to becoming an antibody secreting cell. Bath
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  Speaker 6: in april really seem to be two of the most
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  Speaker 6: critical proteins driving this transition from something that would protect
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  Speaker 6: us to something that would actually make antibodies that will
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  Speaker 6: harm our native tissues.
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  Speaker 2: This is a complex scenario. What's the innovative thinking that
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  Speaker 2: Vertex is considering here.
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  Speaker 6: We know that the levels of bath in april are
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  Speaker 6: actually elevated in people with ig and ephropathy, and that
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  Speaker 6: these levels correlate with measures of kidney function and damage.
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  Speaker 6: So through that we believe both of these drivers are
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  Speaker 6: involved in uncontrolled B cells. Not Bath alone and not
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  Speaker 6: april alone. There's a need to cast the widest net
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  Speaker 6: to catch the main actors involved in the pathogenesis of
• 15:52
  Speaker 6: IgA and nephropathy. What we hope to see with this
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  Speaker 6: approach is the potential to restore immune balance for our
• 15:59
  Speaker 6: patients and give them the best chance at remission in
• 16:04
  Speaker 6: the protin area, which is the protein spilling in the
• 16:07
  Speaker 6: urine he maturia, which is the spilling of blood in
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  Speaker 6: the urine with associated stabilization of kidney function.
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  Speaker 2: For Rachel Benton, living with uncertainty has become a constant burden.
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  Speaker 3: It really scares me. For my son, I feel like
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  Speaker 3: I let him down. I guess, you know, it's not
• 16:30
  Speaker 3: fair for him to have a mom that is going
• 16:33
  Speaker 3: to be sick for the rest of our life. It
• 16:35
  Speaker 3: might not be bad today, but we don't know about tomorrow,
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  Speaker 3: you know. And I just I feel guilt in that
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  Speaker 3: aspect of him just not having a healthy mom. Same
• 16:46
  Speaker 3: for my husband, for him to have a wife that
• 16:49
  Speaker 3: just doesn't healthy and it breaks my heart for them really.
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  Speaker 3: But I try to stay positive and take care of
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  Speaker 3: myself as much as I can, but it definitely does
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  Speaker 3: play a huge role on your mental health for sure.
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  Speaker 2: Day to day.
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  Speaker 3: Some days I can be like, you know what, I
• 17:07
  Speaker 3: am a warrior I got this. Everything's great. In other
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  Speaker 3: days it's like I am not okay, and I feel
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  Speaker 3: like I could just crawl under a rock and stay there.
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  Speaker 2: Rachel, like a lot of people living with IgA nephropathy,
• 17:21
  Speaker 2: has become an advocate, encouraging others to get checked.
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  Speaker 3: All of my friends because of this, I'm like, please
• 17:28
  Speaker 3: get your yearlys done, but also ask for that urine test.
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  Speaker 3: You know, if you see any sign of protein in
• 17:34
  Speaker 3: your urine, just double check it. Why not?
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  Speaker 2: Exactly why not? Kidney health is not top of mind
• 17:43
  Speaker 2: for most people, but it's worth talking to your doctor about.
• 17:47
  Speaker 2: You can also go to Vertex's website vrtx dot com
• 17:51
  Speaker 2: for more information on Vertex's commitment to IgA nephropathy and
• 17:55
  Speaker 2: other serious kidney diseases. This is Targeting the Toughest Diseases
• 18:03
  Speaker 2: a podcast from Bloomberg Media Studios and Vertex Pharmaceuticals. If
• 18:07
  Speaker 2: you like what you hear, subscribe and leave us a review.
• 18:11
  Speaker 2: I'm Jordan Gospore. Thanks for listening.