Is Sickle Cell Anemia…Cured?
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Can you guys tell me a little bit about crick?

I think we could describe him as dynamic, very charismatic, compassionate, all the qualities I think that you would want in a kid. I think that can drink. We think that can drink in south .

deep cromer and her husband heth are describing their twelve year old son, kendra kendrick was born with sickle cell ima. A few weeks ago, mick league glide el lary talk to them about their experience.

I remember the day that we found out kindt was less than two weeks old, telling my husband felt like the weight of the world had fAllen on our shoulders because I knew what he looks like. He drop to his knees that .

bag .

and crab and IT devastated me.

Her son's body was attacking itself. People with sickle cell have a group of disorders that cause red blood cells to change and break down. They turn into these sharp half moon shapes.

Healthy cells die, and the sickle cells can block blood flow, causing excruciating pain cycle cell effects. Over twenty million people worldwide, one hundred thousand of them are in the us, and more than ninety percent of those hundred thousand are black. The disorder is genetic. And while deb and key had family members with sick cell, both thought they weren't Carriers.

I was actually raised um with a cousin who had sick cell disease and so I knew all too well some of the things that can go wrong with single l patients because I was very close to her SHE had been hospital as many, many times and so we were quite aware. And what we decided after losing our first baby was that there were no choices here that we would not on the on board with aboard a child. We wanted to Carry this baby to term and with only a twenty five percent chance with that we're gona take .

the chance. So my initial not knowing much of about to itself, but negativity about IT was that, uh, my song would not leave a long time. So I, you know, if you really doubt, ate to me and at that point time that my son was going have a long life of work hard .

ship kendric did struggle a lot, but this kid is a fighter .

and difficult. Last year he um missed one hundred days of school and twenty three, twenty four and he still somehow came out completing every assignment with three days.

Kendric body was frequently in crisis. He couldn't write his bike or play outside without the fear that suddenly his body would be react with pain. But then Keith in deb found out that the F, D, A had approved a type of gene therapy that could help and maybe even cure kendra.

We actually saw a click on sixty minutes, uh, maybe two years, part to the open therapy approval by F D. A. Um so we were in date by phone calls, mails, videos of that sixty minute clip.

That sixty minutes episode included doctor Francis Collins, director of the largest biomedical research agency in the world, using a very meaningful word.

I believe that this looks like a cure. I gotto be careful .

from every .

angle that I know how to seize this up. This looks like A Q so .

of course we wished and and drain that, that they'll be hopeful for kendra, but we thought that might be for multiple years uh, down the pike before somebody like, uh, before we can. Good opportunity that.

But that dream came much sooner than anyone expected. And last may, kendrick became the first person in the world to go through a commercially approved gene therapy for sick cell. Anima IT was a huge decision, one that had to be fast tracked because kander got so sick last january. And deb and key, they were terrified of all of the unknowns.

Coring over the documents, felt heavy. There's lots of talk of, you know what could happen the worst case scenario, of course, within disclosures, but they just seem really scary, frightening. A canter continues to remind us of his resilient does.

And when you see the word death on a disclosure phone that you have to sign off for your child, it's this harding. But you read that over and over, this the ultimately no in and that you set off on IT is definitely very scary.

Today on the show circle, ceLinda has been arranging people's lives for decades. now. There might be a cure, but who will get to use IT? And what does this mean for other types of gene era? Y, i'm shana roh.

Inflationary, you're listening to. What next? Tb, D, A show about technology, power and how the future will be determined? Stick around.

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after meeting ender's experience, we wanted to talk to someone who has been following the progression of treatments for cycle selenium. So we called up gene koala. She's a medical reporter for the new york times. She's been following the chrome family's journey for almost a year.

Canberra's family and cra, who, eager to talk about what was going on, they did. They wanted to make people aware of this disease. What IT does the families and the hope of having a cure between therapy.

the pain from cycle l cell was devastating for can deric. But the treatment had its own set of terrorist IT involves removing stem cells from the blood or marrow of the patient, modifying those stem cells and then putting them back in the patients body. And with that comes a lot of chemotherapy.

And for the first days, he doesn't real anything much. After a few days, somebody wheel occur with some frozen cells in a couple of bags, and they put the first, they put first bag into a little water bath, and they warm up up the body temperature, and then they infused IT into him. And then after the first bag, they put, they give the second bag in another one minute.

Then over, he's got the cells. He's just got a weight. Wait for the cells.

They just added, make their way to husband narrow, which is which we able do and take up residents there and start to divide and grow a whole new immune system. I was really surprised how bad and actually was. I mean, I knew that you had to have chema therapy. I knew that they're side effects, but I didn't realize quite how bad I was going to beat .

growing kenrick. A new immune system involved numerous blood transfusions and countless medications, all in a small room in a hospital.

So it's it's kindly endless. But after a few months, it's pretty much over. He can go back to school, finally has the ming system again, and he can live a Normal life.

As someone who has reported on medicine for a long time and reported extensively on sicle cell would IT like to have this new universe of gene therapies suddenly open, or at least a little bit open, to a population that has been suffering so much.

Its Sunny has ten nix feelings about IT because i'm so excited with people like kenda. Kk, he's never had a child who is never had a life. He couldn't do anything.

He never learned about a bicycle. Could paying crisis again, can go outside in the cold paying crisis. He couldn't do anything.

He was getting more and more withdrawn in himself, missing a lot of school. So is so exciting. Think some of these people can have a Normal life, but which really sad as you think.

I think about all the people who are sort of on a sidelined thicky. Well, you know, I want, I wanted tell I want a Normal life. I I can, I don't have access to this. So there is a lot of people who who can look on longingly and say, I really want IT and then you say, well, yeah, maybe ten or twenty people out of the twenty thousand who were ill, you could be eligible could get IT IT here. And so it's like they're dangling occurred front of you, but you can't have IT.

I wonder if you could talk to me about that a little bit because the therapy is commercially available to F, D, A approved. But what are the hurdles to to getting there you to actually getting the therapy?

Well, you have to find a medical center that has signed all the contracts with one of the two companies. And apparently this is a big, long negotiating thing. Then you have to have you have to qualify to as well.

And to qualify, you have to have a certain number of paying Prices the year you have to be, I think you have to be at least twelve years old, although they are now starting clinical try if children is Young gest buy. If you can believe, not sure there is an upper age come up. But after a while, when people have had sickle sell for a long time, there is a thought that they wouldn't be able to tolerate that he might thread. Their body is just too damaged.

Then of course, there's the monetary cost of all this .

medicate does pay in. I know maybe like half of the states, a lot of the states I haven't yet authors ted. So you have to if you're a medical patient, you have to be in a state that would authorize IT, then the the hospital has to get your your insurance company to be.

And one of the big issues for them, I say IT, takes a long time because these therapies are really, really expensive. The money for the actual team therapy, like the three point one million, is only part of IT because you also have to pay for the long hospital stay and all the other care we're getting. This is a lot of money.

Hospitals are reluctant to lay IT out in advance. So it's it's a big thing you have to give you go to the hospital, you have to they have to put you on their waiting list. They often decide according to who is the sickest, whose insurance has actually likely become too pretty quickly. So it's kinder was lucky, but a lot of people are not going to be lucky as he is.

Yeah I was wondering what do you think the next few years for this kind of treatment might look like as the scientific capacity experience to to do these gene at its to actually make this incredible change? Is IT gona be something that might be more widely available? I think that will .

be more widely available. But also, I think much Better things are going to come along that are cheaper, easier, more wifi available for everybody. There are a lot of companies interested now and they have a lot of good ideas. But I think we're a period now where there's a lot of hope, but a lot of access.

Do you think there is anything that you've seen in this processor that these companies or or medical centers have learned that translates to other diseases and conditions that are you based in some type of mutated gene?

You know, every time I open my emails is another gene therapy being tested, being approved. It's it's kind of incredibly and for many years, people touching therapy was just dead. IT wasn't going anywhere.

There was real disasters with IT. And companies are given up. And I just looked like I was never gonna work. The main problem now is, if you have a very rare disease, is getting a company, anybody interact in doing IT. So a lot of times the parents, they they start support organizations, support research.

They get everything going at with some researchers at the university ready to go, but you need a company to the clinical trials. And if it's a really rare disease, they may not they may have trouble finding anybody to do IT. So it's it's not that I can happen further diseases and IT is happening big time, but there's also diseases that the parents are really frustrate. They say in, we think we have something here, but we can take IT the next time.

Circle cell has long been a neglected disease. When lazy, talk to kendric's parents, one thing they want in people to understand is that the pain these people have is very real. Here's Keith and debt chroma again.

This because one person is active and well able to do certain things, and then they have. So l doesn't make the other person does that. The opportunity IT is serious that I just complaining about a little bit of pain and also for the medical community that please take you to someone who comes into the medical or E R E D and tells you that they have cell and they not just they just to seek for pain medications to get high, that they really need assistance.

I would agree with that is this heart name that the medical community sometimes discounts the pain that cycle patients sexually go through um and the fact that there are not many areas of the country where people have educated themselves on what sick cell actually looks like, what patients actually go through and so they often times do appear as drug seekers. So I think we would be remiss if we did not note that the cc cell community needing larger spotlight shined on the pain aspect um not just cured of therapy but also the care that IT takes to as a caregiver you know we we watch IT consistently but outside of our four walls, a lot of times people just don't have a clear understanding about what IT takes to care for a self patient.

Those people include medical profession onal. Here's gina again.

expect to one guy, he told me that he, he Carried a card with him. He said, here's my hematologist name. Here's how to contact him.

Every time he went to the hospital, he was, I think he was, he was an academic. He was like my system professor, and he he wore a suit when he went to the emergency room. So he didn't look like he was to somebody off the street.

And still he had problems even though he had this card. It's it's really difficult because for these families, it's not like every there aren't that many people coming into emergency room to the circle cell. And I think the emergency room doctors, who I assume busy, harried and see a lot of people coming in standing. I need I need some sort of a really an opi, just don't wanted believe that they just is easier to say no, not believe you them to take them seriously.

How much of this do you think is just based on racism?

Now IT is one of those things, sort of cans. Yeah, everybody thinks. And probably with good reason that, of course, that has to be.

But there is no way for me to answer that without this giving an opinion that's based on nothing, just based on the most people with sick sea or black. But that's a thought. That's a thought that that's why the disease was neglected .

for so long after the break. More from deb and keh about what's next for kendrick.

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season started sixty ninety nine for a limited time outback stake house no rules just right. We heard in great detail about all the pain and trauma associated with the gene therapy, and that's a lot for anyone, but it's especially a lot for a kid so lazy. Wanted to know how did Keith in deb as parents decide to try out this new treatment?

I've been following your family story, and it's very clear a kenric is a really smart kid and someone with a pretty amazing grasp of kind of all the variables in what he is going through. What kind of conversations do do you guys have with him when you realize this might be an option?

Well, we had, we had that. We had down as a family talked about this thing there been after that come along with in the process as well as the difficult is the things that may occur with having the gene .

therapy .

can to grass the idea this is, I don't know what IT takes as one six to go away .

one of the things that he was concerned with though is um what's gonna happen when I don't have to call sell anymore, right? This is marginal and its all or no and so how was that going to affect me? Um what are people gonna think and what's gonna happen to me? And so there were lots of conversations just about how i'll tell you.

I remembers seeing the sixty minutes epsom and showing IT to can drink and him saying, oh, i'm not gonna do that. That was his initial reaction because I won't have single tell anymore. And what would I do that now? And just having an opportunity to think about IT before he knew that I would be a possibility for him, he started to turn around because of all of the pain associated with what he was going through. But his initial reaction, just psychologically, I believe, was to say, what's gona happen to me after he didn't know any other .

life was a lot of wait. When you upe little boy.

he .

is. It's um we call him a little old man jokily. It's if you know you have to laugh sometimes to keep from crime because the way I see kendrick is having to have taken on so many adult situations in this of your lifespan that he's just mature way beyond his years. In a very short period of time, he has matured into a person that he could run circles around most high scholars. And entering into college.

I think obviously the hardest thing being a parent is to see your child suffer. And I guess I just wonder if you will feel comfortable talking about the process of watching kendra be sick, but then also knowing that the gene therapy would require some suffering to get .

through liftings pace. Danger and a lot of what the disease has offer there are kendricks you know limbs were starting to fail ham and he had already had gone through um a procedure to repair his hicp. And um but the a vast run across is that was in his hit had started to spread and so I was in his back and in his shoulder and IT was scraping.

And so for us and what we know is that once IT starts to spread to your organs, cycle can really, really be very damaging and restrict the blood flow and often cause strokes and bring damage and death. And we actually, through working just in the sick cell community, have met parents who have lost their children very Young. And so for us, IT was a matter of just trying to determine what the best course of action would be for arson. IT might not be the best course of action for everyone, but for us. And all of the things that he was going through, we could see gray skies ahead.

What was the day like I believe IT was october twenty first when kenric left the hospital.

Hello, emotional. IT was emotional. There have been lots of tearful moments over the past weeks, and i've felt just filled with so much joy. But also.

anxiety is there because he .

was fragile and he had been through a lot. His skin is burn to almost beyond recognition. He felt weak.

And even though they were feeding him with the, you know, with with lipids and T P N, and he looks, you know like he. Was strong in that sense. There were so many signs that he had really been trial by fire. And so there was some trepidation about being able to care for him but there was so much joy about you making .

IT through IT. How does he feel now?

Um this is good moment, his bad moment. He's up and down. He step those up quite often um he has some energy, people of energy at tomes when he's run run out that Larry but just this is up and down and ping up and down the floor.

Just happy to you be move around as other times you can get up the bed or doesn't want to get you out. The back doesn't kill grain. Yes, paying, but he always describes as my circle still pay. And so I think that's a different form form as well. Not to realize that is not no lower in paying as well as for a single sale.

Have you all talked about the future, whether that you know the next few weeks or or even years.

we've talked about the need to arian win ourselves and not really knowing what that looks like you now um for me for sure, just as a mom caring for a child who has become increasingly ill, I am very, very thankful for you know having a reale estate career but IT has been diminished so much over the past few years that you know i'm thinking, what else is there? What else can I do or do I stay in this? And I absolutely love IT and then there's kendra has friends that are saying, hey, join the track team or or you know what he he's like, maybe I can run with daddy.

I need to work out. I need to get some ways like things that we don't recognize on. And I believe all of us are just excited about what the future hope, the travel.

yes.

the planning we get, the plan.

We've missed a lot of opportunities because of his sickness. We are planning to go somewhere, then he gets sick. So, uh, maybe go out on the town one, you know, just us as a day can go out because this is not feeling well, or in this as end of visuals to do something that we make like that girl, because he said, so we don't want love one partner along to deal with the h issues of the circle story. He had to go to the hospital, is that we going to be fun to find something different about ourselves. And that worried about kinda s .

health has kendric had the mental space to think about our now what he wants to be when he grows up?

absolutely.

So he one plan is he .

was to be a good medicines. Uh, so that's, I guess, to give back to for all the things he's gone to world. So IT was actually go to high school graduate early. So you go to college early, so that way can get to medical school early, so you can get to become a geneticist, so you can go on about this is his practice.

Deb and key, thank you so much for your time and for being open with your family story. I really, really appreciate you, and I wish you all the best.

Thank you.

Thank you so much.

Deb and Keith romer are the parents of kendra romer, the first person to receive a commercially approved gene therapy treatment for circle ceLinda. Earlier, we spoke with gene kata. She's a medical reporter for the new york times, and that is IT for our show today.

What next? T B T is produced by evan cambell, patric fort and myself shao. oh.

Our show is edited by page osburn. Alesha gomera is vice president of audio for slate. T vt is part of the larger what next family. And hey, you know what? Now, more than ever, we'd really love your support to keep doing thoughtful, independent journalism.

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