Speaker 1: I'm Edward Adams of Bloomberg Media Studios. Since you're a
Speaker 1: subscriber to Bloomberg BusinessWeek, we thought you'd be interested in
Speaker 1: a six episode sponsored content podcast called Targeting the Toughest Diseases.
Speaker 1: It shows how the battle against some of humanity's most
Speaker 1: challenging diseases is happening at the intersection of business and medicine.
Speaker 1: Here's a full episode featuring NBA great Alonzo Mourning recounting
Speaker 1: his fight against kidney disease.
Speaker 2: Miami Arena, March twenty ninth, nineteen ninety six. The Miami
Speaker 2: Heat are facing the Washington Bullets all night long. Alonzo
Speaker 2: Morning of the Heat has been guarded by a giant
Speaker 2: of a man named George Morrissan. At seven foot seven,
Speaker 2: Morrisson is one of the fiercest players in the league,
Speaker 2: the tallest player in NBA history, an impenetrable wall with
Speaker 2: a NonStop motor, but Alonzo Morning is one of the
Speaker 2: greatest ever to step on the court. When the final
Speaker 2: buzzer goes, the Heat have won one hundred and twelve
Speaker 2: to ninety three, and Morning has scored an incredible fifty points.
Speaker 2: That game was a career high for Morning. He would
Speaker 2: go on to be a seven time All Star and
Speaker 2: a gold medalist in the two thousand Olympics, But little
Speaker 2: did he know his toughest opponent was yet to come.
Speaker 2: After returning from the Olympics in Sydney, morning started into
Speaker 2: his usual off season training, but he noticed something was wrong.
Speaker 3: I was experiencing lethargy, edema in my legs, swelling and
Speaker 3: my lower extremities. Extremely tired, worn out.
Speaker 2: He thought it was jetlag, maybe the flu. He figured
Speaker 2: he'd just take a couple days off then resume his training.
Speaker 2: His doctor had a different idea. He suggested they run
Speaker 2: some tests.
Speaker 3: I answered the phone next to the bed and he said,
Speaker 3: you know, I got your results back. He said that
Speaker 3: you've got this rare genetic disorder called focal segmental glamary
Speaker 3: los carosis. And I said, Doc, what is that? He said,
Speaker 3: It's a disease that scars the filters in the kidney.
Speaker 2: Focal segmental glamory you losclerosis or FSGS causes scar tissue
Speaker 2: to develop on the small parts of the kidneys that
Speaker 2: filter waste from the blood.
Speaker 3: I asked them three questions. I said, is there a
Speaker 3: cure for this? He said, no, I said, can I
Speaker 3: play basketball again? He said, I don't know, And then
Speaker 3: I said, well, am I going to die?
Speaker 4: Hi?
Speaker 2: I'm Jordan Gospore. I'm a member of the University of
Speaker 2: Southern California's Center for Health Journalism. This is Targeting the
Speaker 2: Toughest Diseases a podcast produced by Bloomberg Media Studios and
Speaker 2: Vertex Pharmaceuticals. In this series, we look at some of
Speaker 2: humanity's most challenging diseases and how Vertex, a Boston based
Speaker 2: biotech company, is using innovative tools, methods, and a unique
Speaker 2: philosophy to search for treatments and cures. Today, we're looking
Speaker 2: at apol one mediated kidney disease, a disease caused by
Speaker 2: a genetic abnormality, one that thirteen percent of African Americans carry.
Speaker 2: This abnormality can cause a number of types of kidney diseases,
Speaker 2: one of which is FSGS, the condition alonzo morning was
Speaker 2: diagnosed with.
Speaker 3: I thought to myself, not why me, but why right now? God?
Speaker 3: Why am I dealing with dish right now? Of all times.
Speaker 3: I had just come back from the Sydney, Australia Olympics
Speaker 3: win in the gold medal. I had just come off
Speaker 3: from an amazing basketball season where I had first team
Speaker 3: All NBA All Star Team. The list goes on, you know,
Speaker 3: all the accolades, and then all of a sudden, bam.
Speaker 3: This happens.
Speaker 2: Lots of young professional athletes view themselves as invincible. Alonso
Speaker 2: suddenly found out he wasn't hung off the.
Speaker 3: Phone and my face fell in my hands. I felt
Speaker 3: like I was gonna play till I was in my
Speaker 3: forties because I was in such great shape. Just airing
Speaker 3: it from somebody saying hey, you got to stop playing.
Speaker 3: It was humbling and it was deflating. Yeah, and I
Speaker 3: just was just sitting there just trying to figure out, Okay,
Speaker 3: how is this all going to materialize?
Speaker 2: We can't survive without our kidneys. They played a vital role.
Speaker 2: They help our bodies maintain just the right balance of
Speaker 2: electrolytes like potassium, They control blood pressure, they cleaner blood,
Speaker 2: and they even help maintain our hormone levels. Each kidney
Speaker 2: is made up of a million or so tiny filters
Speaker 2: called gloomerulie. They're like little coffee filters. The filtered liquid
Speaker 2: becomes urine and the protein left behind stays in our blood.
Speaker 2: But when the glomerulaie become damaged, those proteins start leaking
Speaker 2: into the urine. The scary thing is it's estimated thirty
Speaker 2: seven million adults in the United States have kidney disease,
Speaker 2: and ninety percent of them don't even know they have it.
Speaker 2: And in the case of apol one mediated kidney disease,
Speaker 2: the prime causal factor is invisible. It's genetic caused by
Speaker 2: mutations in the apoel one gene. Back in the early
Speaker 2: nineteen nineties, doctor David Friedman, a doctor researcher and an
Speaker 2: associate professor with Harvard Medical School who currently works with
Speaker 2: Vertex on its clinical trials, first started to notice something unusual.
Speaker 2: He was seeing African American families where multiple members all
Speaker 2: had kidney disease.
Speaker 4: When there's an important inherited component of a disay disease,
Speaker 4: it tends to cluster in families.
Speaker 2: Understanding there was a genetic cause was just the beginning. Next,
Speaker 2: they had to find the exact gene.
Speaker 4: I think a real breakthrough in our understanding came in
Speaker 4: two thousand and eight when some teams at Johns Hopkins
Speaker 4: and the NIH were the first to find a location
Speaker 4: in the genome. My chromosome twenty two, where it became
Speaker 4: apparent that there was something strong that was impacting kidney
Speaker 4: disease in people of African ancestry.
Speaker 2: Then in twenty ten, doctor Friedman and his colleagues identified
Speaker 2: the specific mutations that led to this type of kidney disease.
Speaker 4: There were two important advances in technology which really helped
Speaker 4: us to pinpoint these two genetic variants in April one.
Speaker 4: The first was related to tools for identifying positive selection
Speaker 4: in the genome, and these mathematical tools helped us in
Speaker 4: vision the genome in a slightly different way. And the
Speaker 4: second major technological advance was a database of genetic variants
Speaker 4: in people of widely diverse ancestries called the Thousand Genomes Project.
Speaker 4: Up until that time, most of what we knew about
Speaker 4: genetic variation came from people of European ancestry, and this new,
Speaker 4: very powerful tool was a really equivalent of an encyclopedia
Speaker 4: of genetic variation around the world.
Speaker 2: From there, our understanding of apol one has continued to increase,
Speaker 2: including why the risk variants in this gene only affect
Speaker 2: people of recent African descent, including African American, LATINX and
Speaker 2: Afro Caribbean communities. Thousands of years ago, a genetic mutation
Speaker 2: in the apol one gene developed in Sub Saharan Africa
Speaker 2: as a protective mechanism.
Speaker 4: We all have a gene that encodes for or the
Speaker 4: april one protein, but the version which causes kidney disease
Speaker 4: contains some very slight changes in the instructions for building
Speaker 4: that protein that turn out to make it very effective
Speaker 4: for killing the trapanosomes that cause African sleeping sickness in humans.
Speaker 2: Sleeping sickness is a disease spread by the bite of
Speaker 2: an infected fly, and it can cause death within a
Speaker 2: matter of weeks. But this genetic mutation stops it in
Speaker 2: its tracks.
Speaker 4: Because it was so effective, it spread very quickly in
Speaker 4: the population. But it's these proteins with a slightly different
Speaker 4: version of april one. It's very very effective for killing
Speaker 4: trypanosomes and preventing African sleeping sickness, which is the same
Speaker 4: version of the protein which makes the kidneys of people
Speaker 4: sick who have this genetic variant.
Speaker 2: We now understand that having one apo l one risk
Speaker 2: variant could protect you from many forms of sleeping sickness,
Speaker 2: but if you were unlucky enough to inherit too, one
Speaker 2: from your mother and one from your father. Your chances
Speaker 2: of getting kidney disease goes up tenfold, Doctor Fire, Doctor j.
Speaker 2: Once your kidneys start to fail, there really are only
Speaker 2: a few ways to stay alive. One is dialysis. It
Speaker 2: replaces the function of the kidney. Doctor Janice Lee studies
Speaker 2: kidney disorders at Emory University School of Medicine in Atlanta, Georgia.
Speaker 5: Kidneys excrete waste products, and they get rid of excess
Speaker 5: fluid from our bodies. So that's what the dialysis machine does.
Speaker 2: Two needles are inserted into a patient's arm. One draws
Speaker 2: blood out and sends it through the machine to be cleaned.
Speaker 2: The other needle returns the clean blood back into the patient,
Speaker 2: which is pretty much exactly the way kidney's work, except
Speaker 2: while your kidney's work slowly. Twenty four hours a day,
Speaker 2: seven days a week. Dialysis means sitting in a chair
Speaker 2: plugged into a machine for two to three hours at
Speaker 2: a time, three times a week. It works, but it's
Speaker 2: not ideal if.
Speaker 5: You really think about it. We go to the bathroom
Speaker 5: excrete our urine two or three four times or more
Speaker 5: a day. So when patients are in dialysis, they're really
Speaker 5: getting their blood cleansed just three days a week for
Speaker 5: a few hours, and so patients can feel washed out
Speaker 5: when they get off of dialysis because they've had all
Speaker 5: this fluid from two days worth of not excreting any
Speaker 5: waste products are fluid.
Speaker 2: There's no cure available for chronic kidney disease. The medications
Speaker 2: currently on the market focus on making sure a person's
Speaker 2: kidneys don't deteriorate further. The current options include medications that
Speaker 2: suppress the immune system, diuretics, ace inhibitors or ARB medications
Speaker 2: to control blood pressure, or lower urine protein anticoagulants to
Speaker 2: prevent blood clots. And then there's the option Alonzo Morning
Speaker 2: had done for his FSGS, a kidney transplant. That approach
Speaker 2: requires you to be fortunate enough to find a suitable donor.
Speaker 2: On average, it also only lasts ten to twelve years.
Speaker 2: Vertex Pharmaceuticals is a company that is researching apol one
Speaker 2: mediated kidney disease and other tough diseases where there's a
Speaker 2: huge unmet need. They are targeting conditions where the human
Speaker 2: biology is understood, the technology already exists, or Vertex thinks
Speaker 2: it can develop it and where Vertex has an approach
Speaker 2: they think maybe transformative. They have several programs in their
Speaker 2: investigational research, including apol one mediated kidney disease. Doctor ogo
Speaker 2: Egbunna leads clinical development for the team researching kidney disease
Speaker 2: at Vertex.
Speaker 6: As we speak here in the US, there probably more
Speaker 6: than one hundred thousand people waiting for a kidney, and
Speaker 6: not everyone is fortunate to be able to get one.
Speaker 6: Many people will die waiting for a kidney.
Speaker 2: The staggering number of people living with and dying from
Speaker 2: kidney disease is one of the main reasons why doctor
Speaker 2: ed Buna joined Vertex's efforts.
Speaker 6: It was really heartbreaking, you know, at the beginning of
Speaker 6: the year, I'd have a whole host of patients on
Speaker 6: dialysis and at the end of the year, one or
Speaker 6: two out of every three would have, you know, passed on.
Speaker 6: That was just too depressing for me.
Speaker 2: Why did Vertex choose to focus on a pall one
Speaker 2: versus other types of kidney disease.
Speaker 6: This is actually one of the most difficult kidney diseases
Speaker 6: that have lagued a minority population and underserved population for
Speaker 6: so long and for the longest time we have attributed
Speaker 6: this to either a bad diet or lack of ex
Speaker 6: size or nutrition, but we do know now that there
Speaker 6: is a genetic basis for a lot of this disparity.
Speaker 6: And I think in typical Vertex fashion, we go after
Speaker 6: diseases that have a serious unmet need, and in addition
Speaker 6: to that, we go after diseases where the underlying cause
Speaker 6: is well understood and for which we apply the best
Speaker 6: science available to try and address it.
Speaker 2: What can we look forward to.
Speaker 6: Part of the reason why I'm actually so excited about
Speaker 6: what we're doing here at Vertex is because we have
Speaker 6: found small molecule therapies that are investigational to the underlying
Speaker 6: cause of what I've described as equal one mediated kidney disease.
Speaker 6: We have evaluated these potential therapies in experimental settings and
Speaker 6: first in human studies as well as a preliminary proof
Speaker 6: of concept study in patients.
Speaker 2: Developing a potential small molecule therapy is no easy feat,
Speaker 2: doctor ed Buna, says scientists a Vertex have gone through
Speaker 2: hundreds of thousands of candidate molecules in their kidney disease research.
Speaker 6: Nothing good or great comes easy. Therein lies the promise
Speaker 6: and the excitement While.
Speaker 2: Their research continues. Doctor Egbunna says he and his team
Speaker 2: at Vertex will do everything they can to raise awareness
Speaker 2: for the disease. That includes educating on preventative measures like genotyping,
Speaker 2: which can help determine whether a patient carries the apol
Speaker 2: one genetic variant.
Speaker 6: So, in addition to supporting the clinical community on patients
Speaker 6: in increasing the rates of diagnosis of kidney disease, we
Speaker 6: also want an increase in the awareness and of genotyping
Speaker 6: so that precise diagnosis can be made so that the
Speaker 6: right therapy can be brought to the patient. Dollons of
Speaker 6: Money is one of those great examples of people that
Speaker 6: went through the science, symptoms, the worries, the challenges of
Speaker 6: n stage advanced kidney disease. I stunding and he got
Speaker 6: back on his feet.
Speaker 2: Alonzo Morning underwent a kidney transplant three years after his
Speaker 2: initial diagnosis.
Speaker 3: So the recovery process was grueling and it was extremely
Speaker 3: painful at times, and it was difficult. But if you
Speaker 3: think about anything in life worth having, it's very difficult
Speaker 3: to get it. And I was trying to get my
Speaker 3: help back in. I was trying to get back on
Speaker 3: the court, So I was trying to get back to
Speaker 3: some sense of normalcy, but it challenged me tremendously, and
Speaker 3: it really truly challenged me.
Speaker 2: Morning talks about his struggle to get and then adapt
Speaker 2: to living with a new kidney, knowing full well that
Speaker 2: he had a lot of advantages.
Speaker 3: That was in optimal condition, excellent health, you know, but
Speaker 3: that was the benefit of my recovery so fast, because
Speaker 3: the doctors told me, like, hey, you know, if you
Speaker 3: wasn't this high performance athlete, then you probably wouldn't have
Speaker 3: bounced back as fast after the transplant.
Speaker 2: And bounce back he did. Just three years after his transplant.
Speaker 2: Alonso Morning would go on and win a championship with
Speaker 2: the Miami Heat in two thousand and six, and today
Speaker 2: he's partnering with Vertex as a spokesperson, using his profile
Speaker 2: and his experience to advocate and educate.
Speaker 3: You know. A big part of it is to try
Speaker 3: to inspire and provide hope and encouragement for those who
Speaker 3: are going through the same ordeal.
Speaker 2: And Morning says he's hoping Vertex will be able to
Speaker 2: help kidney patients by raising awareness of the disease, encouraging
Speaker 2: people to visit their doctor and continuing to search for
Speaker 2: a potential treatment.
Speaker 3: So if this can happen, then all of what I've
Speaker 3: gone through is so much more of a worthwhile because
Speaker 3: I'm able to help save other people's lives.
Speaker 2: This is Targeting the Toughest diseases. A podcast from Bloomberg
Speaker 2: Media Studios and Vertex Pharmaceuticals. If you like what you hear,
Speaker 2: subscribe and leave us a review. I'm Jordan Gospore. Thanks
Speaker 2: for listening.