Targeting the Toughest Diseases (Sponsored Content)

a unique philosophy to search for treatments and cures. Produced by Bloomberg Media Studios and sponsored by Vertex, the podcast's latest episode features nb a great Alonzo Mourning, recounting his fight against kidney disease and how scientists are working hard to provide hope for future generations of patients. You can subscribe today on Apple Podcasts, Spotify, or wherever you listen to your favorite podcasts. Here is the full episode. Miami

Arena March. The Miami Heat are facing the Washington Bullets all night long. Alonso Morning of the Heat has been guarded by a giant of a man named George Morrisson at seven ft seven. Morrisson is one of the fiercest players in the league, the tallest player in NBA history, an impenetrable wall with a NonStop motor, but Alonso Morning is one of the greatest ever to step on the court. When the final buzzer goes, the Heat have won a hundred and twelve to ninety three, and Morning has scored

an incredible fifty points. That game was a career high from Morning. He would go on to be a seven time All Star and a gold medalist in the two thousand Olympics. But little did he know his toughest opponent was to come. After returning from the Olympics in Sydney, Morning started into his usual offseason training, but he noticed something was wrong. I was experiencing lethargy, a demon in my legs, swelling in my lower extremities. Extremely tired, worn out.

He thought it was jet lag, maybe the flu. He figured he'd just take a couple of days off then resume his training. His doctor had a different idea. He suggested they run some tests. I answered the phone next to the bit and he said, you know, I got your results back. He said that you've got this rare genetic disorder called focal segmental glamorio los carrosis, and I said, Doc, what is that? He said, It's a disease that cards

the filters in the kidney. Vocal Segmental glamoryo los clerosis or f s GS caused a scar tissue to develop on the small parts of the kidneys that alter waste from the blood. I asked them three questions. I said, is there a cupidis He said no. I said, can I play basketball again? He said, I don't know? And then I said, well, am I gonna die? Hi? I'm Jordan's Gospel A. I'm a member of the University of

Southern California's Center for Health Journalism. This is Targeting the Toughest Diseases a podcast produced by Bloomberg Media Studios and Vertex Pharmaceuticals. In this series, we look at some of humanity's most challenging diseases and how Vertex, a Boston based biotech company, is using innovative tools, methods, and a unique

philosophy to search for treatments and cures. Today, we're looking at a po L one mediated kidney disease, a disease caused by a genetic abnormal one of African Americans carry this aftermality can cause a number of types of kidney diseases, one of which is f SGS, the condition Alonso Morning was diagnosed with. I thought to myself, not why me, but why right now? God? Why am I dealing with

this right now of all times. I had just come back from the Sydney, Australia Olympics winning the gold medal, had just come off an amazing basketball season where I had First Team All NBA All Star Team. The list goes on, you know, all the accolades, and then all of a sudden, bam, this happens. Lots of young professional athletes view themselves as invincible. Alonso suddenly found out he wasn't. All of the phone and my face fell in my hands.

I felt like I was gonna play until I was in my forties because I was in such great shape. Just airing it from somebody saying hey, you gotta stop playing it was humbling. He was deflating, you know, and I just was just sitting there just trying to figure out, Okay, how is this all going to materialize. We can't survive

without our kidneys. They play a vital role. They help our bodies maintain just the right balance of electrolytes like potassium, They control blood pressure, the cleaner blood, and they even help maintain our hormone levels. Each kidney is made up of a million or so tiny filters called gloomy uli. They're like little coffee filters. The filtered liquid becomes urine and the protein left behind stays in our blood. But when the glomerulie become damaged, those proteins start leaking into

the urine. The scary thing is it's estimated thirty seven million adults in the United States have kidney disease, and nine of them don't even know they have it. And in the case of a poe l one mediated kidney disease is the prime causal factor is invisible. It's genetic

caused by mutations in the APO l one gene. Back in the early nineteen nineties, Dr David Friedman, a doctor, researcher and an associate professor with Harvard Medical School who currently works with Vertex on its clinical trials, first started to notice something unusual. He was seeing African American families where multiple members all had kidney disease. When there's an important inherited component of a disease, it tends to cluster

in families. Understanding there was a genetic cause was just the beginning. Next, they had to find the exact gene. I think a real breakthrough in our understanding came in two thousand and eight when some teams at Johns Hopkins in the n i H were the first to find a location in the geno my chromosome twenty two where it became apparent that there was something strang on that

was impacting kidney disease in people of African ancestry. Then Dr Friedman and his colleagues identified the specific mutations that led to this type of kidney disease. There were two important advances in technology which really helped us to pinpoint these two genetic variants in April one. The first was related to tools for identifying positive selection in the genome, and these these mathematical tools helped us envisioned the genome

in a slightly different way. In the second major technological advance was a database of genetic variants in people of widely diverse ancestry, is called the Thousand Genomes Project. Up until that time, most of what we knew about genetic variation came from people of European ancestry, and this new, very powerful tool was a really equivalent of an encyclopedia

of genetic variation around the world. From there, our understanding of apoel one has continued to increase, including why the risk variants in this gene only affect people of recent African descent, including African American, Latin X and Afro Caribbean communities. Thousands of years ago, a genetic mutation in the apoel one gene developed in sub Saharan Africa as a protective mechanism.

We all have a gene that encodes for the april one protein, but the version which causes kidney disease contains some very slight changes in the instructions for building that protein that turn out to make it very effective for killing the trepanisomes that cause African sleeping sickness and humans. Sleeping sickness is a disease spread by the bite of an infected fly, and it can cause death within a matter of weeks. But this genetic mutation stops it in

its tracks. Because it was so effective. It's spread very quickly in the population, but it's these proteins with a slightly different version of eight pl one. It's very very effective for killing japanizomes and preventing African sleeping sickness, which is the same version of the protein which makes the kidneys of people sick who have this genetic variant. We now understand that having one apoel one risk variant could

protect you from many forms of sleeping sickness. But if you were unlucky enough to inherit too, one from your mother and one from your father, your chances of getting kidney disease goes up tenfold fire Once your kidneys start to fail. There really are only a few ways to stay a Lot five. One is dialysis. It replaces the function of the kidney. Dr Janice Lee studies kidney disorders

at Emory University School of Medicine in Atlanta, Georgia. Kidneys excrete waste products, and they get rid of excess fluid from our bodies, so that's what the dialysis machine does. Two needles are inserted into a patient's arm. One draws blood out and sends it through the machine to be cleaned. The other needle returns the clean blood back into the patient, which is pretty much exactly the way our kidneys work. Except while your kidneys work slowly. Twenty four hours a day,

seven days a week. Dialysis means sitting in a chair plugged into a machine for two to three hours at a time, three times a week. It works, but it's

not ideal if you really think about it. We go to the bathroom excrete our urine two or three four times or more a day, so when patients are on dialysis, there getting their blood clans just three days a week for a few hours, and so patients can feel washed out when they get off of dialysis because they've had all this fluid from two days worth of not excreting any waste products or fluid. There's no cure available for chronic kidney disease. The medications currently on the market focus

on making sure a person's kidneys don't deteriorate further. The current options include medications that suppress the immune system, diuretics, ace inhibitors or a r B medications to control blood pressure, or lower urine protein anticoagulants to prevent blood clots. And then there's the option Alonso Morning had done for his f s GS a kidney transplant. That approach requires you to be fortunate enough to find a suitable donor. On average,

it also only last ten to twelve years. Vertex Pharmaceuticals is a company that is researching a poll one mediated kidney disease and other tough diseases where there's a huge unmet need. They are targeting conditions where the human biology is understood, the technology already exists, or Vertex thinks that can develop it, and where Vertex has an approach they think maybe transformative. They have several programs in their investigational research,

including a poll one mediated kidney disease. Dr Ogo ed Buna leads clinical development for the team researching kidney disease at Vertex. As we speak here in the US, there are probably more than a hundred thousand people waiting for a kidney, and not everyone is fortunate to be able to get one. Many people will die waiting for a kidney. The staggering number of people living with and dying from kidney disease is one of the main reasons why Dr

ed Buna joined Vertexas efforts. It was really heartbreaking, you know, at the beginning of the year, I'd have a whole host of patients on theiralysis and at the end of the year one or two out of every three would have you know, costa. That was just too depressing for me. Why did Vertex choose to focus on a pole one

versus other types of kidney disease? This is actually one of the most difficult kidney diseases that have lagged a minority population or underserved population for so long, and for the longest time, we have attributed this to either bad diet or lack of exercise or nutrition. But we do know now that there is a genetic basis for a

lot of this disparity. And I think in typical Vertex fashion, we go after diseases that have a serious and met need, and in addition to that, we go after diseases where the underlying cause is well understood and for which we apply, you know, the best science available to try and address it.

What can we look forward to. Part of the reason why I'm actually so excited about what we're doing here had Vertex, is because we have found small molecule therapies that are investigational to the underlying cause of what I've described as april one media to kidney disease. We have evaluated these potential therapies in experimental settings and first in human studies, as well as a preliminary proof of concept study in patients. Developing a potential small molecule therapy is

no easy feat. Dr ed Buna says scientists a Vertex have gone through hundreds of thousands of candidate molecules and their kidney disease research. Nothing good or great comes easy. There in lies the promise and the excitement well the research continues. Dr ed Bunah says he and his team at Vertex will do everything they can to raise awareness for the disease. That includes educating on preventative measures like genotyping. You can help determine whether a patient carries the A

L one genetic variant. So, in addition to supporting the clinical community and patients in increasing the rates of diagnosis of kidney disease, we also want an increase in the awareness and of genotyping so that precise diagnosis can be made so that the right therapy come brought to the patient. Alons of Morning is one of those great examples of people that went through the sign symptoms, the worries, the challenges of stage advanced kidney disease, got a transplant and

he got back on his feet. Alonso Morning underwent a kidney transplant three years after his initial diagnosis. So the recovery process was grueling and it was extremely painful at times, and it was difficult. But if you think about anything in life worth having, is very difficult to get it. And I was trying to get my help back, and I was trying to get back on the court. So I was trying to get back to some sense of normalcy. But it challenged me tremendously, and it really truly challenged me.

Morning talks about his struggle to get and then adapt to living with a new kidney, knowing full well that he had a lot of advantages that was in optimal condition, excellent health, you know. But that was the benefit of my recovery so fast, because the doctors told me, like, hey, you know, if you wasn't this high performance athlete, then you probably wouldn't have bounced back as fast after the transplant, and bounced back he did. Just three years after his transplant.

Alonso Morning would go on and win a championship with the Miami Heat in two thousand six, and today he's partnering with Vertex as a spokesperson using his profile and his experience to advocate and educate. You know, a big part of it is to try to inspire and provide hope and encouragement for the those who are going through

the same ordeal. And Morning says he's hoping Vertex will be able to help kidney patients by raising awareness of the disease, encouraging people to visit their doctor, and continuing to search for a potential treatment. So if this can happen, then all of what I've gone through is so much more of a worthwhile because maybe to help save other people's lives. This is targeting the toughest diseases. A podcast from Bloomberg Media Studios and Vertex Pharmaceuticals. If you like

what you hear, subscribe and leave us a review. I'm Jordan's Gospel. Thanks for listening.