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