Speaker 1: I'm Edward Adams of Bloomberg Media Studios. Since you're a
Speaker 1: subscriber to Bloomberg Prognosis, we thought you'd be interested in
Speaker 1: a new six episodes sponsored content podcast called Targeting the
Speaker 1: Toughest Diseases. It shows how the battle against some of
Speaker 1: humanity's most challenging diseases is happening at the intersection of
Speaker 1: business and medicine. The podcast explores how Vertex Pharmaceuticals, a
Speaker 1: Boston based biotech company, is using innovative tools, methods, and
Speaker 1: a unique philosophy to search for treatments and cures. Produced
Speaker 1: by Bloomberg Media Studios and sponsored by Vertex, the podcast's
Speaker 1: latest episode features nb a great Alonzo Mourning, recounting his
Speaker 1: fight against kidney disease and how scientists are working hard
Speaker 1: to provide hope for future generations of patients. You can
Speaker 1: subscribe today on Apple Podcasts, Spotify, or wherever you listen
Speaker 1: to your favorite podcasts. Here is the full episode. Miami
Speaker 1: Arena March. The Miami Heat are facing the Washington Bullets
Speaker 1: all night long. Alonso Morning of the Heat has been
Speaker 1: guarded by a giant of a man named George Morrisson
Speaker 1: at seven ft seven. Morrisson is one of the fiercest
Speaker 1: players in the league, the tallest player in NBA history,
Speaker 1: an impenetrable wall with a NonStop motor, but Alonso Morning
Speaker 1: is one of the greatest ever to step on the court.
Speaker 1: When the final buzzer goes, the Heat have won a
Speaker 1: hundred and twelve to ninety three, and Morning has scored
Speaker 1: an incredible fifty points. That game was a career high
Speaker 1: from Morning. He would go on to be a seven
Speaker 1: time All Star and a gold medalist in the two
Speaker 1: thousand Olympics. But little did he know his toughest opponent
Speaker 1: was to come. After returning from the Olympics in Sydney,
Speaker 1: Morning started into his usual offseason training, but he noticed
Speaker 1: something was wrong. I was experiencing lethargy, a demon in
Speaker 1: my legs, swelling in my lower extremities. Extremely tired, worn out.
Speaker 1: He thought it was jet lag, maybe the flu. He
Speaker 1: figured he'd just take a couple of days off then
Speaker 1: resume his training. His doctor had a different idea. He
Speaker 1: suggested they run some tests. I answered the phone next
Speaker 1: to the bit and he said, you know, I got
Speaker 1: your results back. He said that you've got this rare
Speaker 1: genetic disorder called focal segmental glamorio los carrosis, and I said, Doc,
Speaker 1: what is that? He said, It's a disease that cards
Speaker 1: the filters in the kidney. Vocal Segmental glamoryo los clerosis
Speaker 1: or f s GS caused a scar tissue to develop
Speaker 1: on the small parts of the kidneys that alter waste
Speaker 1: from the blood. I asked them three questions. I said,
Speaker 1: is there a cupidis He said no. I said, can
Speaker 1: I play basketball again? He said, I don't know? And
Speaker 1: then I said, well, am I gonna die? Hi? I'm
Speaker 1: Jordan's Gospel A. I'm a member of the University of
Speaker 1: Southern California's Center for Health Journalism. This is Targeting the
Speaker 1: Toughest Diseases a podcast produced by Bloomberg Media Studios and
Speaker 1: Vertex Pharmaceuticals. In this series, we look at some of
Speaker 1: humanity's most challenging diseases and how Vertex, a Boston based
Speaker 1: biotech company, is using innovative tools, methods, and a unique
Speaker 1: philosophy to search for treatments and cures. Today, we're looking
Speaker 1: at a po L one mediated kidney disease, a disease
Speaker 1: caused by a genetic abnormal one of African Americans carry
Speaker 1: this aftermality can cause a number of types of kidney diseases,
Speaker 1: one of which is f SGS, the condition Alonso Morning
Speaker 1: was diagnosed with. I thought to myself, not why me,
Speaker 1: but why right now? God? Why am I dealing with
Speaker 1: this right now of all times. I had just come
Speaker 1: back from the Sydney, Australia Olympics winning the gold medal,
Speaker 1: had just come off an amazing basketball season where I
Speaker 1: had First Team All NBA All Star Team. The list
Speaker 1: goes on, you know, all the accolades, and then all
Speaker 1: of a sudden, bam, this happens. Lots of young professional
Speaker 1: athletes view themselves as invincible. Alonso suddenly found out he wasn't.
Speaker 1: All of the phone and my face fell in my hands.
Speaker 1: I felt like I was gonna play until I was
Speaker 1: in my forties because I was in such great shape.
Speaker 1: Just airing it from somebody saying hey, you gotta stop
Speaker 1: playing it was humbling. He was deflating, you know, and
Speaker 1: I just was just sitting there just trying to figure out, Okay,
Speaker 1: how is this all going to materialize. We can't survive
Speaker 1: without our kidneys. They play a vital role. They help
Speaker 1: our bodies maintain just the right balance of electrolytes like potassium,
Speaker 1: They control blood pressure, the cleaner blood, and they even
Speaker 1: help maintain our hormone levels. Each kidney is made up
Speaker 1: of a million or so tiny filters called gloomy uli.
Speaker 1: They're like little coffee filters. The filtered liquid becomes urine
Speaker 1: and the protein left behind stays in our blood. But
Speaker 1: when the glomerulie become damaged, those proteins start leaking into
Speaker 1: the urine. The scary thing is it's estimated thirty seven
Speaker 1: million adults in the United States have kidney disease, and
Speaker 1: nine of them don't even know they have it. And
Speaker 1: in the case of a poe l one mediated kidney
Speaker 1: disease is the prime causal factor is invisible. It's genetic
Speaker 1: caused by mutations in the APO l one gene. Back
Speaker 1: in the early nineteen nineties, Dr David Friedman, a doctor,
Speaker 1: researcher and an associate professor with Harvard Medical School who
Speaker 1: currently works with Vertex on its clinical trials, first started
Speaker 1: to notice something unusual. He was seeing African American families
Speaker 1: where multiple members all had kidney disease. When there's an
Speaker 1: important inherited component of a disease, it tends to cluster
Speaker 1: in families. Understanding there was a genetic cause was just
Speaker 1: the beginning. Next, they had to find the exact gene.
Speaker 1: I think a real breakthrough in our understanding came in
Speaker 1: two thousand and eight when some teams at Johns Hopkins
Speaker 1: in the n i H were the first to find
Speaker 1: a location in the geno my chromosome twenty two where
Speaker 1: it became apparent that there was something strang on that
Speaker 1: was impacting kidney disease in people of African ancestry. Then
Speaker 1: Dr Friedman and his colleagues identified the specific mutations that
Speaker 1: led to this type of kidney disease. There were two
Speaker 1: important advances in technology which really helped us to pinpoint
Speaker 1: these two genetic variants in April one. The first was
Speaker 1: related to tools for identifying positive selection in the genome,
Speaker 1: and these these mathematical tools helped us envisioned the genome
Speaker 1: in a slightly different way. In the second major technological
Speaker 1: advance was a database of genetic variants in people of
Speaker 1: widely diverse ancestry, is called the Thousand Genomes Project. Up
Speaker 1: until that time, most of what we knew about genetic
Speaker 1: variation came from people of European ancestry, and this new,
Speaker 1: very powerful tool was a really equivalent of an encyclopedia
Speaker 1: of genetic variation around the world. From there, our understanding
Speaker 1: of apoel one has continued to increase, including why the
Speaker 1: risk variants in this gene only affect people of recent
Speaker 1: African descent, including African American, Latin X and Afro Caribbean communities.
Speaker 1: Thousands of years ago, a genetic mutation in the apoel
Speaker 1: one gene developed in sub Saharan Africa as a protective mechanism.
Speaker 1: We all have a gene that encodes for the april
Speaker 1: one protein, but the version which causes kidney disease contains
Speaker 1: some very slight changes in the instructions for building that
Speaker 1: protein that turn out to make it very effective for
Speaker 1: killing the trepanisomes that cause African sleeping sickness and humans.
Speaker 1: Sleeping sickness is a disease spread by the bite of
Speaker 1: an infected fly, and it can cause death within a
Speaker 1: matter of weeks. But this genetic mutation stops it in
Speaker 1: its tracks. Because it was so effective. It's spread very
Speaker 1: quickly in the population, but it's these proteins with a
Speaker 1: slightly different version of eight pl one. It's very very
Speaker 1: effective for killing japanizomes and preventing African sleeping sickness, which
Speaker 1: is the same version of the protein which makes the
Speaker 1: kidneys of people sick who have this genetic variant. We
Speaker 1: now understand that having one apoel one risk variant could
Speaker 1: protect you from many forms of sleeping sickness. But if
Speaker 1: you were unlucky enough to inherit too, one from your
Speaker 1: mother and one from your father, your chances of getting
Speaker 1: kidney disease goes up tenfold fire Once your kidneys start
Speaker 1: to fail. There really are only a few ways to
Speaker 1: stay a Lot five. One is dialysis. It replaces the
Speaker 1: function of the kidney. Dr Janice Lee studies kidney disorders
Speaker 1: at Emory University School of Medicine in Atlanta, Georgia. Kidneys
Speaker 1: excrete waste products, and they get rid of excess fluid
Speaker 1: from our bodies, so that's what the dialysis machine does.
Speaker 1: Two needles are inserted into a patient's arm. One draws
Speaker 1: blood out and sends it through the machine to be cleaned.
Speaker 1: The other needle returns the clean blood back into the patient,
Speaker 1: which is pretty much exactly the way our kidneys work.
Speaker 1: Except while your kidneys work slowly. Twenty four hours a day,
Speaker 1: seven days a week. Dialysis means sitting in a chair
Speaker 1: plugged into a machine for two to three hours at
Speaker 1: a time, three times a week. It works, but it's
Speaker 1: not ideal if you really think about it. We go
Speaker 1: to the bathroom excrete our urine two or three four
Speaker 1: times or more a day, so when patients are on dialysis,
Speaker 1: there getting their blood clans just three days a week
Speaker 1: for a few hours, and so patients can feel washed
Speaker 1: out when they get off of dialysis because they've had
Speaker 1: all this fluid from two days worth of not excreting
Speaker 1: any waste products or fluid. There's no cure available for
Speaker 1: chronic kidney disease. The medications currently on the market focus
Speaker 1: on making sure a person's kidneys don't deteriorate further. The
Speaker 1: current options include medications that suppress the immune system, diuretics,
Speaker 1: ace inhibitors or a r B medications to control blood pressure,
Speaker 1: or lower urine protein anticoagulants to prevent blood clots. And
Speaker 1: then there's the option Alonso Morning had done for his
Speaker 1: f s GS a kidney transplant. That approach requires you
Speaker 1: to be fortunate enough to find a suitable donor. On average,
Speaker 1: it also only last ten to twelve years. Vertex Pharmaceuticals
Speaker 1: is a company that is researching a poll one mediated
Speaker 1: kidney disease and other tough diseases where there's a huge
Speaker 1: unmet need. They are targeting conditions where the human biology
Speaker 1: is understood, the technology already exists, or Vertex thinks that
Speaker 1: can develop it, and where Vertex has an approach they
Speaker 1: think maybe transformative. They have several programs in their investigational research,
Speaker 1: including a poll one mediated kidney disease. Dr Ogo ed
Speaker 1: Buna leads clinical development for the team researching kidney disease
Speaker 1: at Vertex. As we speak here in the US, there
Speaker 1: are probably more than a hundred thousand people waiting for
Speaker 1: a kidney, and not everyone is fortunate to be able
Speaker 1: to get one. Many people will die waiting for a kidney.
Speaker 1: The staggering number of people living with and dying from
Speaker 1: kidney disease is one of the main reasons why Dr
Speaker 1: ed Buna joined Vertexas efforts. It was really heartbreaking, you know,
Speaker 1: at the beginning of the year, I'd have a whole
Speaker 1: host of patients on theiralysis and at the end of
Speaker 1: the year one or two out of every three would
Speaker 1: have you know, costa. That was just too depressing for me.
Speaker 1: Why did Vertex choose to focus on a pole one
Speaker 1: versus other types of kidney disease? This is actually one
Speaker 1: of the most difficult kidney diseases that have lagged a
Speaker 1: minority population or underserved population for so long, and for
Speaker 1: the longest time, we have attributed this to either bad
Speaker 1: diet or lack of exercise or nutrition. But we do
Speaker 1: know now that there is a genetic basis for a
Speaker 1: lot of this disparity. And I think in typical Vertex fashion,
Speaker 1: we go after diseases that have a serious and met need,
Speaker 1: and in addition to that, we go after diseases where
Speaker 1: the underlying cause is well understood and for which we apply,
Speaker 1: you know, the best science available to try and address it.
Speaker 1: What can we look forward to. Part of the reason
Speaker 1: why I'm actually so excited about what we're doing here
Speaker 1: had Vertex, is because we have found small molecule therapies
Speaker 1: that are investigational to the underlying cause of what I've
Speaker 1: described as april one media to kidney disease. We have
Speaker 1: evaluated these potential therapies in experimental settings and first in
Speaker 1: human studies, as well as a preliminary proof of concept
Speaker 1: study in patients. Developing a potential small molecule therapy is
Speaker 1: no easy feat. Dr ed Buna says scientists a Vertex
Speaker 1: have gone through hundreds of thousands of candidate molecules and
Speaker 1: their kidney disease research. Nothing good or great comes easy.
Speaker 1: There in lies the promise and the excitement well the
Speaker 1: research continues. Dr ed Bunah says he and his team
Speaker 1: at Vertex will do everything they can to raise awareness
Speaker 1: for the disease. That includes educating on preventative measures like genotyping.
Speaker 1: You can help determine whether a patient carries the A
Speaker 1: L one genetic variant. So, in addition to supporting the
Speaker 1: clinical community and patients in increasing the rates of diagnosis
Speaker 1: of kidney disease, we also want an increase in the
Speaker 1: awareness and of genotyping so that precise diagnosis can be
Speaker 1: made so that the right therapy come brought to the patient.
Speaker 1: Alons of Morning is one of those great examples of
Speaker 1: people that went through the sign symptoms, the worries, the
Speaker 1: challenges of stage advanced kidney disease, got a transplant and
Speaker 1: he got back on his feet. Alonso Morning underwent a
Speaker 1: kidney transplant three years after his initial diagnosis. So the
Speaker 1: recovery process was grueling and it was extremely painful at times,
Speaker 1: and it was difficult. But if you think about anything
Speaker 1: in life worth having, is very difficult to get it.
Speaker 1: And I was trying to get my help back, and
Speaker 1: I was trying to get back on the court. So
Speaker 1: I was trying to get back to some sense of normalcy.
Speaker 1: But it challenged me tremendously, and it really truly challenged me.
Speaker 1: Morning talks about his struggle to get and then adapt
Speaker 1: to living with a new kidney, knowing full well that
Speaker 1: he had a lot of advantages that was in optimal condition,
Speaker 1: excellent health, you know. But that was the benefit of
Speaker 1: my recovery so fast, because the doctors told me, like, hey,
Speaker 1: you know, if you wasn't this high performance athlete, then
Speaker 1: you probably wouldn't have bounced back as fast after the transplant,
Speaker 1: and bounced back he did. Just three years after his transplant.
Speaker 1: Alonso Morning would go on and win a championship with
Speaker 1: the Miami Heat in two thousand six, and today he's
Speaker 1: partnering with Vertex as a spokesperson using his profile and
Speaker 1: his experience to advocate and educate. You know, a big
Speaker 1: part of it is to try to inspire and provide
Speaker 1: hope and encouragement for the those who are going through
Speaker 1: the same ordeal. And Morning says he's hoping Vertex will
Speaker 1: be able to help kidney patients by raising awareness of
Speaker 1: the disease, encouraging people to visit their doctor, and continuing
Speaker 1: to search for a potential treatment. So if this can happen,
Speaker 1: then all of what I've gone through is so much
Speaker 1: more of a worthwhile because maybe to help save other
Speaker 1: people's lives. This is targeting the toughest diseases. A podcast
Speaker 1: from Bloomberg Media Studios and Vertex Pharmaceuticals. If you like
Speaker 1: what you hear, subscribe and leave us a review. I'm
Speaker 1: Jordan's Gospel. Thanks for listening.