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Targeting Sickle Cell Disease (Sponsored Content) · Masters in Business

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Speaker 1: Since you're a subscriber to this Bloomberg podcast, we thought
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Speaker 1: you'd be interested in a six episode sponsored podcast called
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Speaker 1: Targeting the Toughest Diseases, produced by Vertex Pharmaceuticals and Bloomberg
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Speaker 1: Media Studios. It explores the innovative tools, methods, and unique
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Speaker 1: philosophy Vertex Pharmaceuticals is using to search for treatments for
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Speaker 1: some of humanity's most challenging diseases. Here's a recent episode,
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Speaker 1: and it's like.
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Speaker 2: If someone grabs your body and twists in the opposite
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Speaker 2: direction with each hand, it's like you can feel the
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Speaker 2: pressure in your bones and you can just feel every
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Speaker 2: part of your body is hurting.
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Speaker 3: Twenty years ago, during her sophomore year in college, Terry
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Speaker 3: Booker was hit with the worst pain of her life.
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Speaker 2: My legs were burning in then it just went up
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Speaker 2: throughout my whole entire body to the point where I
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Speaker 2: was unconscious and I was put on life support. I
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Speaker 2: wasn't able to breathe on my own. They told my
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Speaker 2: mom I had kidney failure, I had lung failure.
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Speaker 3: There was no clear path to treatment for her, just uncertainty.
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Speaker 2: They said, you just prayed because we don't know what's
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Speaker 2: going to happen to her.
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Speaker 3: What happened was she got better for a while, but
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Speaker 3: then the pain returned and she was back in the
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Speaker 3: emergency room. Her recurring visits to the er seeking relief
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Speaker 3: from the pain revealed a second problem, not a medical one,
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Speaker 3: a societal one. Terry is black, and as an African
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Speaker 3: American woman seeking pay meds, she was presumed to be
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Speaker 3: a drug addict and her medical issues were ignored.
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Speaker 2: It is infuriating because when you go into the er,
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Speaker 2: you're expecting to receive help. You're expecting to be treated
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Speaker 2: as a patient the need of something, not treated as
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Speaker 2: someone who is wanting to get their next high.
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Speaker 3: Hi. I'm Jordan Gospore. I'm a member of the University
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Speaker 3: of Southern California's Center for Health Journalism. This is Targeting
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Speaker 3: the Toughest Diseases a podcast produced by Bloomberg Media Studios
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Speaker 3: and Vertex Pharmaceuticals. In this series, we look at some
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Speaker 3: of humanity's most challenging diseases and how Vertex, a Boston
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Speaker 3: based biotech company, is using innovative tools, methods, and a
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Speaker 3: unique philosophy to search for treatments and cures. Today, we're
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Speaker 3: looking at. Sickle cell disease a blood disorder which can
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Speaker 3: cause organ failure, stroke, and even death. It's a disease
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Speaker 3: that affects roughly one hundred thousand Americans, including Terry Booker.
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Speaker 3: Sickle cell disease is not something you catch. It's a
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Speaker 3: genetic condition you're born with. Often babies are diagnosed before
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Speaker 3: they're even born. Terry was an exception. She didn't know
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Speaker 3: she had it until she was eleven.
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Speaker 2: They pricked my finger and I remember them putting it
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Speaker 2: under a microscope, and when they had it under a microscope,
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Speaker 2: all these people were rushing over and I remember this
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Speaker 2: so vividly, and they were like, oh, come here, come, Look.
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Speaker 3: Terry was in fifth grade and her mam had taken
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Speaker 3: her to the doctors because she had been complaining of pain.
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Speaker 2: And I'm like okay, and they say you see all
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Speaker 2: these little funny shaped cells and I'm like yeah. They're like, yeah,
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Speaker 2: that's sickle cell and I said okay, and I'm like,
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Speaker 2: well what does that mean.
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Speaker 3: Sickle cell disease, or sickle cell anemia, as it's often called,
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Speaker 3: affects red blood cells. Those are the cells that carry
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Speaker 3: oxygen to all the tissues in our bodies. Normally, our
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Speaker 3: red blood cells are soft and shaped like a doughnut,
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Speaker 3: so they can squeeze through even the smallest of blood vessels.
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Speaker 3: But sickle cell disease changes them. Specifically, it causes the
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Speaker 3: hemoglobin proteins inside the red blood cells to change their structure.
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Speaker 3: Instead of being donut shaped, they become curved like a
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Speaker 3: cresset moon, or, as the name implies, like a sickle.
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Speaker 3: These odd shaped cells are also hard and sticky, which
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Speaker 3: means sometimes they can't flow smoothly through blood vessels, and
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Speaker 3: when that happens, they start piling up like cars on
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Speaker 3: a busy highway, making it really hard, if not impossible,
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Speaker 3: for oxygen to reach where it needs to go. It's
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Speaker 3: that lack of oxygen to tissues that causes the stabbing pain.
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Speaker 3: That's the medical side of the disease, but as Terry
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Speaker 3: mentioned earlier, there's also a societal component.
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Speaker 4: To address circle cell disease, we also have to address
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Speaker 4: the fundamental issues that relate to systemic racism and how
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Speaker 4: it impacts on assets to care.
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Speaker 3: That's doctor Isaac Odame. He studies the ways racism and
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Speaker 3: medical care intersect when it comes to sickle cell disease.
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Speaker 3: He's the medical director of the Global Sickle Cell Disease
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Speaker 3: Network at the Center for Global Child Health. Doctor Odame
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Speaker 3: grew up in West Africa.
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Speaker 4: Psyco cell disease was something that I was very familiar with.
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Speaker 4: Not only did I have this some family members who
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Speaker 4: had children with psycosol disease, but some classmates and schoolmates
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Speaker 4: who also lived with the condition. So I was exposed
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Speaker 4: very early to the ravages of this disease.
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Speaker 3: The first documented case of psycle cell anemia in America
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Speaker 3: was in nineteen ten, just over a century ago, but
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Speaker 3: its history dates back thousands of years.
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Speaker 4: Sucer cell disease has been known for centuries in parts
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Speaker 4: of Africa, and if you go into the oral tradition,
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Speaker 4: they did have descriptions of the disease which typified the
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Speaker 4: chronic and intercurrent as creating pain associative of the disease.
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Speaker 3: Oddly enough, the mutation that changes the cell shapes was
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Speaker 3: once an evolutionary advantage protecting against malaria.
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Speaker 4: It predominantly affects people of African descent because malaria is
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Speaker 4: very endemic in Africa. It occurs in people of Indian descent,
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Speaker 4: Mediterranean descent, and parts of the Middle East Arabian Peninsula,
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Speaker 4: and when you look at the common link between all
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Speaker 4: these areas is malaria anddemicity. So it was a survival
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Speaker 4: advantage it provided against malaria, and the percentages of people
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Speaker 4: with traits in the population will rise over time.
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Speaker 3: Having sickle cell trait is actually quite common in African populations.
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Speaker 3: It evolved over time because it prevents the development and
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Speaker 3: spreading of malaria. That historical context explains why the majority
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Speaker 3: of sickle cell patients in the United States are African Americans.
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Speaker 3: That means sickle cell care provides a clear example of
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Speaker 3: the racial inequities and disparities in the medical system. That
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Speaker 3: includes access to care and quality of care, both of
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Speaker 3: which are lower for African Americans. Doctor Odami is hopeful
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Speaker 3: that sickle cell treatments in development will not just treat
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Speaker 3: the disease, but will address those other complex issues as well.
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Speaker 4: I think more and more the patients are becoming more empowered,
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Speaker 4: and I think they call themselves warriors, which is the
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Speaker 4: appropriate term. They're not only dealing with the ravages of
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Speaker 4: the disease, but they're also having to fight a system
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Speaker 4: that doesn't recognize their needs appropriately, and so the warriors
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Speaker 4: are beginning to speak out aloud. The system is beginning
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Speaker 4: to respond.
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Speaker 3: Vertex Pharmaceuticals is a leader in that response.
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Speaker 5: For it to actually help people's lives, it has to
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Speaker 5: work for them and it has to work for society.
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Speaker 3: That's doctor David Altcheler, the chief scientific officer at Vertex.
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Speaker 3: They have a unique approach to choosing the diseases they target.
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Speaker 5: Vertex has decided to focus on a set of diseases
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Speaker 5: where we see great unmet need, where we see the
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Speaker 5: human biology is clear, and we feel that we have
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Speaker 5: the technology, the insights, the wherewithal to make a difference.
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Speaker 3: When it comes to Vertex's sickle cell program, Doctor William
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Speaker 3: Hobbs is in charge of clinical development.
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Speaker 6: You know, I started in medicine being interested in hematology
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Speaker 6: and sickle cell disease in particular, mainly from a research standpoint,
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Speaker 6: and that was because disease has long been considered a
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Speaker 6: disease where the underlying cause of disease could be targeted,
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Speaker 6: if innovative approaches could be identified, and so I really
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Speaker 6: came at it from this scientific point of view, and
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Speaker 6: then along the way I started to meet sickle cell
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Speaker 6: disease patients and their families coordinate their care and realized
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Speaker 6: there was really a lot more to it than a
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Speaker 6: just a scientific question. And early on in my academic
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Speaker 6: career as a physician scientist, I started an adult sickle
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Speaker 6: cell disease treatment center, and I still remember this. One
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Speaker 6: of the first patients that I started seeing was a
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Speaker 6: woman who had long standing and severe disease, and at
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Speaker 6: her first clinic visit with me, as she walked in,
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Speaker 6: sat down and said, Hi, doctor Hobbs, I've just got
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Speaker 6: to know one thing, and that is if I ever
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Speaker 6: need you, if I'm in an emergency room or a hospital,
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Speaker 6: it's two in the morning, it's a night, it's a weekend,
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Speaker 6: I just need to know that you're there on the
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Speaker 6: other end of the phone twenty four hours a day,
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Speaker 6: seven days a week, whenever I need you, because if
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Speaker 6: you're not, then there's no reason for me to be here.
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Speaker 6: And it was that awareness that I realized at that
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Speaker 6: point that if you're going to take care of patients
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Speaker 6: living with sickle cell disease, that you really need to
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Speaker 6: be all in.
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Speaker 3: That's a big ask.
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Speaker 6: It was a big ass but you know, it's actually
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Speaker 6: a fair ask. And I think if you look at
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Speaker 6: the history of how sickle cell disease patients have generally
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Speaker 6: been treated by the medical system. I think sickle cell
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Speaker 6: patients aren't asking for anything unique or above and beyond
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Speaker 6: or special. They're really asking for equitable care and what
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Speaker 6: we would offer to anyone else with the chronic severe disease.
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Speaker 3: So what treatment options are available for patients with sickle
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Speaker 3: cell disease?
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Speaker 6: Yeah, you know, the causal biology of sickle cell disease
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Speaker 6: has been known for many years, but for a long time,
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Speaker 6: the only therapies available were able to reduce, but not
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Speaker 6: really eliminate complications of disease in some but not all patients.
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Speaker 6: And we've come a long way in the last few years,
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Speaker 6: but there's still a lot of work yet to be done.
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Speaker 6: And I often think about the sickle cell disease patients
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Speaker 6: that I've cared for over the years, knowing that they
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Speaker 6: were in desperate need of new treatment options, and so,
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Speaker 6: you know, we continue to look at multiple angles and
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Speaker 6: innovative options with the goal of treating the underlying cause
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Speaker 6: of the disease.
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Speaker 3: I'm curious, from Vertex's point of view, why has Vertex
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Speaker 3: decided to invest so heavily in R and D specifically
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Speaker 3: with sickle cell.
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Speaker 6: For the most part, sickle cell disease has been largely
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Speaker 6: ignored by the pharmaceutical industry for a long time, and
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Speaker 6: I think there's always historically been a perception that it's
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Speaker 6: a very difficult disease to go into from a commercial
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Speaker 6: and marketing standpoint. However, if one has an approach that's
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Speaker 6: based on solid biology and reckon is the unmet need
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Speaker 6: that exists in sickle cell disease, where there's millions of
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Speaker 6: patients waiting worldwide, it becomes very compelling to do it.
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Speaker 6: And so this is really about patients. It's understanding the
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Speaker 6: medical need, it's understanding the causal biology and then innovating
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Speaker 6: on treatment approaches, which is what we ad Vertex do.
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Speaker 6: And you could say that it's this is in our DNA.
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Speaker 3: Sickle cell isn't a new disease. There's been a lot
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Speaker 3: of work done and a lot of innovation. What is
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Speaker 3: VerTech bringing to this that's new.
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Speaker 6: Our aim is to investigate multiple different types of approaches
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Speaker 6: that can give us the best opportunities to hopefully treat
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Speaker 6: the causal biology of sickle cell disease for as many
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Speaker 6: patients as possible. Whenever you do something like that, there
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Speaker 6: are a number of unknown questions that have to be answered,
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Speaker 6: both scientific and medical, and we're learning to understand what
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Speaker 6: all of those are. And I think what we're also
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Speaker 6: understanding is that for diseases like sickle cell disease, where
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Speaker 6: there's such a huge need for treatment options, that serial
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Speaker 6: innovation is required. And I think this is a focus
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Speaker 6: for us to continue to innovate, because, as we all know,
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Speaker 6: technology doesn't stand still, and so neither should we.
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Speaker 3: That's the approach philosophically, but what does it actually look
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Speaker 3: like when you put it into practice.
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Speaker 6: We've learned a lot from natural history studies and a
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Speaker 6: lot of other data about how we hope to be
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Speaker 6: able to target causal biology with the aim of treating
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Speaker 6: the effects of sickle cell disease. So for Vertex, it
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Speaker 6: really begins with a deep scientific insight into causal biology
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Speaker 6: and then serially innovating using all available tools and modalities
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Speaker 6: to identify potential treatment options. And so it's essentially identifying
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Speaker 6: the right tool for the job. And one of the
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Speaker 6: things that's unique about Vertex and the approach is just
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Speaker 6: that it's so completely focused on the patient and what
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Speaker 6: the potential treatment outcomes could be. And so the focuses
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Speaker 6: really have we identified and cracked the right science that
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Speaker 6: can translate into something that could be meaningful for patients.
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Speaker 6: So it's the goal is a high bar, and we
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Speaker 6: do that relentlessly. We do it very aggressively, and we
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Speaker 6: constantly strive to out innovate even ourselves within our own
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Speaker 6: programs here, and that's how we do science and develop medicines.
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Speaker 3: The research, the testing, and the potential approvals are moving
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Speaker 3: forward as quickly as possible, and in the meantime, Terry
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Speaker 3: Booker is just trying to live her best life.
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Speaker 2: I exercise, I hang out with my kat Lewis, who
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Speaker 2: I love and who he always knows when it's something
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Speaker 2: wrong because he'll stay under me a little bit longer
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Speaker 2: or a little bit more. And I cook often. I
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Speaker 2: cook most of my meals. And when I say I
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Speaker 2: cook every day, I cook every day. And I pray.
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Speaker 3: What she's praying for ultimately is a cure. Terry says,
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Speaker 3: until that happens, she'll continue to keep the faith.
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Speaker 2: My faith has grown so much through this process because
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Speaker 2: I feel like as a human, I feel like it's
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Speaker 2: no way I could be going through these things. I
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Speaker 2: should be in a whole crying somewhere.
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Speaker 7: So our pain is real and we cope and we
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Speaker 7: do what we have to do.
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Speaker 2: To try to live a quote unquote normal life.
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Speaker 7: But our pain is real. And when people understand that,
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Speaker 7: I feel like you can understand a sickle cell patient.
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Speaker 3: This is targeting the toughest diseases. A podcast from Bloomberg
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Speaker 3: Media Studios and Vertex Pharmaceuticals. If you like what you hear,
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Speaker 3: subscribe and leave us a review. I'm Jordan Gospore. Thanks
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Speaker 3: for listening.