Speaker 1: I'm Edward Adams of Bloomberg Media Studios. Since you're a
Speaker 1: subscriber to Bloomberg Business Week, we thought you'd be interested
Speaker 1: in a new six episode sponsored content podcast called Targeting
Speaker 1: the Toughest Diseases. It shows how the battle against some
Speaker 1: of humanity's most challenging diseases is happening at the intersection
Speaker 1: of business and medicine. The podcast explores how Vertex Pharmaceuticals,
Speaker 1: a Boston based biotech company, is using innovative tools, methods,
Speaker 1: and a unique philosophy to search for treatments and cures.
Speaker 1: Produced by Bloomberg Media Studios and sponsored by Vertex, the
Speaker 1: podcast's latest episode features NBA 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's the full EPISO showed 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 yet 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 dima and
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 scars
Speaker 1: the filters in the kidney. Focal Segmental glamoryo losclerosis, or
Speaker 1: f s GS caused a scar tissue to develop on
Speaker 1: the small parts of the kidneys that filter waste in
Speaker 1: the blood. I asked them three questions. I said, is
Speaker 1: there a cure forness? He said no. I said, can
Speaker 1: I play basketball again? He said, I don't know? And
Speaker 1: then I said, WHOA 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 APO l one mediated kidney disease, a disease caused
Speaker 1: by a genetic abnormality. One of African Americans carry this
Speaker 1: oftermality 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 from somebody saying hey, you gotta stop playing.
Speaker 1: It was humbling and 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 gloomeryulie 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: ninety percent of them don't even know they have it.
Speaker 1: And in the case of a po l one mediated
Speaker 1: kidney disease, the prime causal factor is invisible. It's genetic
Speaker 1: caused re mutations in the a po l one gene.
Speaker 1: Back in the early nineteen nineties, Dr David Friedman, a
Speaker 1: doctor researcher and an associate professor with Harvard Medical School
Speaker 1: who currently works with Vertex on its clinical trials, first
Speaker 1: started to notice something unusual. He was seeing African American
Speaker 1: families where multiple members all had kidney disease. When there's
Speaker 1: an important inherited component of a disease, it tends to
Speaker 1: cluster in families. Understanding there was a genetic cause was
Speaker 1: just 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 strong that was
Speaker 1: impacting kidney disease in people of African ancestry. Then Dr
Speaker 1: Friedman and his colleagues identified the specific mutations that led
Speaker 1: to this type of kidney disease. There were two important
Speaker 1: advances in technology which really helped us to pinpoint these
Speaker 1: two genetic variants in April one. The first was related
Speaker 1: to tools for identifying positive selection in the genome, and
Speaker 1: these these mathematical tools helped us envisioned the genome in
Speaker 1: a slightly different way. In the second major technological advance
Speaker 1: was a database of genetic variants in people of widely
Speaker 1: diverse ancestry, is called the Thousand Genomes project. Up until
Speaker 1: that time, most of what we knew about genetic variation
Speaker 1: came from people of European ancestry, and this new, very
Speaker 1: powerful tool was a really equivalent of an cyclopedia of
Speaker 1: genetic variation around the world. From there, our understanding of
Speaker 1: apoel one has continued to increase, including why the risk
Speaker 1: variants in this gene only affect people of recent African descent,
Speaker 1: including African American, Latin X and Afro Caribbean communities. Thousands
Speaker 1: of years ago, a genetic mutation in the apoel one
Speaker 1: 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 april one. It's very very effective
Speaker 1: for killing japanizomes and preventing African sleeping sickness, which is
Speaker 1: the same version of the protein which makes the kidneys
Speaker 1: of people sick who have this genetic variant. We now
Speaker 1: understand that having one a poel one risk variant could
Speaker 1: protect you from many forms of sleeping sickness. But if
Speaker 1: you were unlucky enough to inherit to one from your
Speaker 1: mother and one from your father, your chances of getting
Speaker 1: kidney disease goes up tenfold dore once your kidneys start
Speaker 1: to fail, there really are only a few ways to
Speaker 1: stay alive. One is dialysis. It replaces the function of
Speaker 1: the kidney. Dr Janice Lee studies kidney disorders at Emory
Speaker 1: University School of Medicine in Atlanta, Georgia. Kidneys excrete waste products,
Speaker 1: and they get rid of excess fluid from our bodies,
Speaker 1: so that's what the dialysis machine does. Two needles are
Speaker 1: inserted into a patient's arm. One draws blood out and
Speaker 1: sends it through the machine to be cleaned. The other
Speaker 1: needle returns the clean blood back into the patient, which
Speaker 1: is pretty much exactly the way our kidneys work, except
Speaker 1: 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: they're really getting their blood clans just three days a
Speaker 1: week for a few hours. And so patients can feel
Speaker 1: washed out when they get off of dialysis because they've
Speaker 1: had all this fluid from two days worth of not
Speaker 1: excreting any waste products or fluid. There's no cure available
Speaker 1: for chronic kidney disease. The medications currently on the market
Speaker 1: focus on making sure a person's kidneys don't deteriorate further.
Speaker 1: The 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 has researched ing a poll one
Speaker 1: mediated kidney disease and other tough diseases where there's a
Speaker 1: huge unmet need. They are targeting conditions where the human
Speaker 1: biology is understood, the technology already exists, or Vertex thinks
Speaker 1: that can develop it, and where Vertex has an approach
Speaker 1: they think maybe transformative. They have several programs in their
Speaker 1: investigational research, including a poll one mediated kidney disease. Dr
Speaker 1: Ogo ed Buna leads clinical development for the team researching
Speaker 1: kidney disease at Vertex. As we speak here in the US,
Speaker 1: there are probably more than a hundred thousand people waiting
Speaker 1: for a kidney, and not everyone is fortunate to be
Speaker 1: able to get one. Many people will die waiting for
Speaker 1: a kidney. The staggering number of people living with and
Speaker 1: dying from kidney disease is one of the main reasons
Speaker 1: why Dr ed Buna joined Vertexas efforts. It was really heartbreaking,
Speaker 1: you know, at the beginning of the year, I'd have
Speaker 1: a whole host of patients on theiralysis and at the
Speaker 1: end of the year, one or two of every three
Speaker 1: would have you know, costa. That was just too depressing
Speaker 1: for me. Why did Vertex choose to focus on a
Speaker 1: pole one versus other types of kidney disease? This is
Speaker 1: actually one of the most difficult kidney diseases that have
Speaker 1: lagged a minority population and underserved population for so long,
Speaker 1: and for the longest time we have attributed this to
Speaker 1: either bad diet or lack of exercise or nutrition. But
Speaker 1: we do know now that there is a genetic basis
Speaker 1: for a lot of this disparity. And I think in
Speaker 1: typical Vertex fashion, we go after diseases that have a
Speaker 1: serious and met need, and in addition to that, we
Speaker 1: go after diseases where the underlying cause is well understood
Speaker 1: and for which we apply, you know, the best science
Speaker 1: available to try and address it. What can we look
Speaker 1: forward to. Part of the reason why I'm actually so
Speaker 1: excited about what we're doing here at Vertex is because
Speaker 1: we have found small molecule therapies that are investigational to
Speaker 1: the underlying cause of what I've described as april and
Speaker 1: one media to kidney disease. We have evaluated these potential
Speaker 1: therapies in experimental settings and first in human studies as
Speaker 1: well as a preliminary proof of concept study in patients.
Speaker 1: Developing a potential small molecule therapy is no easy feat.
Speaker 1: Dr ed Buna says scientists a Vertex have gone through
Speaker 1: hundreds of thousands of candidate molecules and their kidney disease research.
Speaker 1: Nothing good or great comes easy. Therein lies the promise
Speaker 1: and the excitement well the research continues. Dr ed Bunah
Speaker 1: says he and his team at Vertex will do everything
Speaker 1: they can to raise awareness for the disease. That includes
Speaker 1: educating on preventative measures like genotyping, which can help the
Speaker 1: ternaman whether a patient carries the A L one genetic variant. So,
Speaker 1: in addition to supporting the clinical community and patients in
Speaker 1: increasing the rates of diagnosis of kidney disease, we also
Speaker 1: want an increase in the awareness and of genotyping so
Speaker 1: that precise diagnosis can be made so that the right
Speaker 1: therapy can be brought to the patient. Alonso Morning is
Speaker 1: one of those great examples of people that went through
Speaker 1: the science symptoms, the worries, the challenges of stage advanced
Speaker 1: kidney disease, got a transplant, and he got back on
Speaker 1: his feet. Alonso Morning underwent a kidney transplant three years
Speaker 1: after his initial diagnosis. So the recovery process was grueling
Speaker 1: and it was extremely painful at times, and it was difficult.
Speaker 1: But if you think about anything in life worth having,
Speaker 1: is very difficult to get it. And I was trying
Speaker 1: to get my help back, and I was trying to
Speaker 1: get back on the court, So I was trying to
Speaker 1: get back to some sense of normalcy. But it challenged
Speaker 1: me tremendously, and it really truly challenged me. Morning talks
Speaker 1: about his struggle to get and then adapt to living
Speaker 1: with a new kidney, knowing full well that he had
Speaker 1: a lot of advantages that was in optimal condition, excellent health,
Speaker 1: you know. But that was the benefit of my recovery
Speaker 1: so fast, because the doctors told me, like, hey, you know,
Speaker 1: if you wasn't his high performance athlete, then you probably
Speaker 1: wouldn't have bounced back as fast after the transplant, and
Speaker 1: bounce back he did just three years after his transplant
Speaker 1: alongso 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 those who are going through the
Speaker 1: same ordeal. And Morning says he's hoping Vertex will be
Speaker 1: able to help kidney patients by raising awareness of the disease,
Speaker 1: encouraging people to visit their doctor, and continuing to search
Speaker 1: for a potential treatment. So if this can happen, then
Speaker 1: all of what I've gone through is so much more
Speaker 1: of a worthwhile because maybe to help save other people's lives.
Speaker 1: This is targeting the toughest diseases. A podcast from Bloomberg
Speaker 1: Media Studios and Vertex Pharmaceuticals. If you like what you hear,
Speaker 1: subscribe and leave us a review. I'm Jordan's Gospel. Thanks
Speaker 1: for listening.