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Speaker 1: Most acquaintances that I have don't know I have sickle cell. Only my close friends know that I have sickle cell. It's something that I don't really like to share.

Speaker 2: When I come into the ER, I'm just like, going in here, they're going to think I'm addicted to pain medication. How do I convince them that I'm not?

Speaker 3: The pain is there. It's not something that you can ignore, but I have to ignore it just to get the day going.

Speaker 4: When my daughter was very young, I told her from the beginning, you're going to college, you're going to succeed. We got to figure out how to maneuver in this life. These are the cards that we have. We got to deal 'em. We got to play what we've been given.

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Kai Wright: It's Notes from America. I'm Kai Wright. Welcome to the show. The voices you just heard were gathered from individuals and families living with sickle cell disease by the Rutgers Cancer Institute of New Jersey. Sickle cell disease is a genetic blood disorder that causes excruciating acute and chronic pain among other health complications. Anyone can be born with it, but it is particularly prevalent among people of African descent. 1 in 13 Black Americans are born with the trait necessary to develop the disease. In an historic development, just this past Friday, the Food and Drug Administration approved two new genetic therapies for sickle cell.

One of them will be the first human use of the gene-editing tool called CRISPR. The scientist who developed that tool won the Nobel Prize for it a few years ago. There's some complicated science to understand in this news, but the take-home is we're witnessing a big step forward for a disease that has been a horror for so long in so many lives, at least 100,000 people in the US and millions around the world.

We're going to spend the show talking about sickle cell and what the new therapies mean for people and families living with it. I'm joined by Ashley Valentine. She's a researcher who spent years studying sickle cell both from health policy perspectives and in clinical trials. She also partnered with her brother, Marqus, to create the group Sick Cells, which is an advocacy and organizing group for people living with sickle cell. Ashley, thank you so much for joining us.

Ashley Valentine: Hi. Thank you for having me.

Kai Wright: Can you help us understand just the big picture of this news first off as someone who's spent so many years working on sickle cell? How do you explain what the FDA's approval of these two new therapies means?

Ashley Valentine: The FDA approval is really important. It's a big deal for the landscape of sickle cell disease. I actually was in the car with my mom going to pick up my car from the mechanic, and when we got the news, I cried first, and then she cried, and then we called my dad, and my dad was also emotional thinking about how many people this can help with sickle cell.

Why this is so important is we've been left without resources to care for people with sickle cell for almost the entirety since this disease has been discovered. In a short amount of time since really drug development has picked up in sickle cell in 2014, we have had some pretty major approvals. We had two drugs approved in 2019, one in 2017, and now we're here talking about gene therapies. Some see this as curative therapies, but therapies that are giving people their lives back away from sickle cell disease.

Kai Wright: You said some see these as curative therapies. Help me understand that piece of it. Do we consider this a cure?

Ashley Valentine: I think that the way we're talking about the gene therapies, I think it will evolve over time. The way I see it is you go from a state of sickle cell where you're having this chronic exclusionary pain, you're having bone damage, you're having the reality that without mitigating the disease, your organs will fail, and you will ultimately die in many cases, to not having that. Whether we call that a cure, or whether we call that mitigating the disease, I think the endpoint is what I'm really happy to see.

Kai Wright: Whichever way it goes, people's lives are going to get a lot better if they're able to get these therapies. Let's try to bite off the science a little bit just enough for people to understand what we're talking about. There are two gene therapies that have been approved. As I said, one of them uses the Nobel Prize-winning gene editing tool, CRISPR. Are you able, just in simple terms, to explain what this therapy is and how it works?

Ashley Valentine: I will do my best. Full disclosure, I am not a doctor, but I have sat through a lot of informational webinars and education meetings. My mom is a brilliant nurse that made us say words like acetaminophen instead of Tylenol. One is a gene therapy. That is the bluebird bio treatment. I don't know the name of it now, because they just got new names, and I haven't memorized them yet, but one is adding a gene.

It's a gene addition. Then there's the gene editing. That's the one you talked about. The CRISPR technology, that is actually going in and editing the gene, so it's almost cutting. If you want to think about it in visuals, the one gene therapy is almost like, they say an envelope, so it's putting into your body to change the genes. Then the other's like scissors where it's cutting the gene inside the body.

Kai Wright: Then just a little more of this to understand why those are a big deal for sickle cell itself. Maybe we also need to just level set on what sickle cell is. We understand what it does, it causes you to live in both chronic and acute pain, but what actually is sickle cell?

Ashley Valentine: Sickle cell is a blood disorder. It's a genetic blood disorder. That's something I want to say first and foremost. We talk a lot about the race and the ethnic makeup of the people who impact sickle cell, but fundamentally, it's a hemoglobinopathy. It's a disease of the hemoglobin, your blood. What happens is a healthy person or someone that has healthy hemoglobin, our bodies produce whole red blood cells and the red blood cells carry oxygen throughout the entire system. If you think about what happens when you have circulation loss, or you block circulation somewhere, that's what the sickle cells do to the body.

Someone with sickle cell, their body isn't producing those round, healthy blood cells that bring oxygen to the various parts of the body. Their body is producing these stiff, rigid blood cells that don't carry oxygen, and they die really fast. What that looks like the way it manifests in the disease is that you have basically loss of circulation to major organs. We know that if you don't have circulation to a specific organ, it dies.

We talk about the pain of sickle cell, but strokes are common in sickle cell. Seizures, chronic leg ulcers. Many people don't have spleens. We have heart failure, we have damage to the eyes, joint replacements all over. Sickle cell is a very, very severe disease. It's just until the disease starts to break down the body, it's invisible. This pain is one of the hallmark complications of sickle cell. It is this excruciating pain. When you see someone go through a pain crisis, it's the most gut-wrenching thing ever. The pain is usually the indicator that something in their body is losing oxygen or is blocking the circulation.

Kai Wright: You do want to point out that while race is a really important part of this, we have to first talk about the disease itself. Help me understand that distinction. This has been very important for African Americans, for people of African descent.

Ashley Valentine: What happens with sickle cell disease is the way it's taught first is off of race and ethnicity. It's taught that it's this disease that only Black people can get, and that's not true. Thinking about how people get sickle cell first, to help us understand what's going on here, it is evolutionary response to malaria. Basically anywhere in the world where there's malaria, there's also sickle cell.

Kai Wright: Got it.

Ashley Valentine: India has the highest rates of sickle cell. India and Nigeria have the highest rates of sickle cell globally. What it looks like in the United States is that it disproportionately impacts Black people, but there's also people here who have Mediterranean background, who are Hispanic that has sickle cell disease, but in the US it does disproportionately impact Black people.

Kai Wright: That is really eye-opening for me. Thank you for that, Ashley, because I didn't really understand that that was the why, that this goes back to malaria. As we start to head towards a break, and we're going to come back to all of this in more detail later in the show, but I also want to just talk about one more therapy. Because the genetic therapies, as you said, they're not the first to emerge. Some people will recall a big story, not that long ago about the FDA approving a bone marrow treatment that people considered, is that going to be a cure. Can you explain that? Just the distinction from what we're talking about now?

Ashley Valentine: Yes, but high-level, I'm not a doctor.

Kai Wright: Understood.

Ashley Valentine: Bone marrow transplant requires a match. The bone marrow is where the sickle cells are made, and so that is being transplanted so that it's not producing sickle cells or it's producing sickle cell trait. There's many differences. One of the distinctions I can draw here is that the gene therapies don't require donors and the bone marrow transplants do require donors. When you're talking about populations, like the Black population or some of the Hispanic populations, there's less likely to have donors and donor banks. That's one of the major differences where we could see there being a benefit in the gene therapies here in sickle cell.

Kai Wright: They're less likely to have donors and donor base, can you just unpack that for a second so we know what you're talking about?

Ashley Valentine: Yes. This, I think, is a health equity piece. If we think of a blood donor, because this is something that many of us do, you'll see that there's always a need for Black blood donors or donors from different ethnicity groups, because you're more likely to be able to accept blood. You have to match a certain blood type in order to receive blood. People with sickle cell oftentimes will receive blood transfusions. If they don't have the right match for blood, they can produce antibodies against the blood. You don't want that. You're more likely to have a match for blood donors if you're coming from the same ethnic or racial group.

That's why there's this call for an increased need for Black blood donors or certain other ethnic groups. The same also is applicable to bone marrow donors or bone marrow transplants. You can have full sibling matches, for instance, or you can try to go to a donor bank to get a match for the donor. We see less of that. Maybe in the future, when the way that we do bone marrow donation is more equitable, maybe we'll see that there's more matches for some of these minority populations. Right now, that's not the case.

Kai Wright: The take home being that one of the big differences here now with the gene editing or with the gene therapy is that it is potentially available to a lot more to people as a consequence of not needing those donors. We're going to have to take a break. I'm talking with Ashley Valentine, a sickle cell researcher and advocate, about the groundbreaking gene therapies that the FDA has just approved for this painful disease. Coming up, I recently connected with a young Congolese American woman. Just this summer, she underwent the bone marrow transplant we were just talking about. She's going to tell us her story of living with sickle cell for 21 years, and what that treatment does and does not mean for her now. Stay with us.

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Welcome back, it's Notes from America, I'm Kai Wright. This past Friday, the FDA approved the groundbreaking gene therapies that could essentially cure people of sickle cell disease, which is a painful blood disorder that has plagued millions of people around the world for so long. Previously, the only real therapy available to people living with sickle cell was a bone marrow transplant. I recently spoke with someone who's been through that treatment. Magaly Ghonda is 21 years old, and her whole life has been shaped by sickle cell disease. From the time she was born, Magaly has dealt with unimaginable pain. This past August, she was finally able to undergo that bone marrow transplant.

I connected with her to hear her reflections on a life with sickle cell and what having a cure, as it's called, means to her. Warning, Magaly is very straightforward about the mental health challenges that she has faced as a young person living with this disease. Our conversation does include discussion of suicide. If you are having suicidal thoughts, help is available for the national Suicide Crisis Lifeline. You can speak to someone today by calling or texting 988. I caught up with Magaly while she was in the hospital for one of many follow-up treatments for her transplant. Hospitals have sadly been a common part of her life.

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Do you mind just describing what you're there for today, what's going on for you today?

Magaly Ghonda: Yes. I'm in the hospital right now because I was having a pain crisis and I was throwing up quite a bit. Yesterday, I threw up nine times. They wanted to monitor me and make sure the new medications I'm on aren't the ones causing it.

Kai Wright: You've lived with this for 21 years, and it's been days like today for much of your life. Is that the way to understand it, that you're always in and out of care?

Magaly Ghonda: Yes, absolutely. I've had 200 or 300? I need to ask my dad, but I've had over 100 admissions.

Kai Wright: Wow. In 21 years.

Magaly Ghonda: Yes. It's a lot. Really, it's a life-threatening disease. It's been hard. It's depressing when majority of your life is being in the hospital. When it comes to sickle cell, it's feeling pain. Think of a migraine, and a really bad migraine and imagine that, anywhere on your body 10 times, that's what sickle cell feels like. Now, due to the sickle cell, I have gotten something called avascular necrosis. It's a loss of blood supply to the bones. I have it in both of my hips and both of my shoulders. My hips are the ones that are really deteriorating, but I'm hoping it'll get better now that I'm doing what the doctors are saying.

Kai Wright: You have received some treatment for this. We're talking today about a new gene editing therapy that could treat sickle cell, but the summer you had a different treatment, a bone marrow transplant. First off, just in layman's terms, can you describe what that treatment was?

Magaly Ghonda: Basically, you do radiation, then chemotherapy. Then they take whoever your donor is, they take their blood, mix it some other medications, and they put it in. What it does is it gets rid of your cells so that you have healthy ones, and you don't have to have sickle cell anymore. I'm going to admit, it's not easy, because you're in the hospital for months. Because I've been in the hospital since July.

Kai Wright: With this treatment.

Magaly Ghonda: Yes.

Kai Wright: You said part of this bone marrow treatment, it was, you said, "So I don't have sickle cell anymore." Does that mean that you're cured from it?

Magaly Ghonda: Yes, I'm cured from it. Since it's already done damage to my body, because my nerves are messed up, they said I will still have some chronic pains, but it's better now because I don't have to worry about being in the ICU because I had a bad crisis and stuff like that.

Kai Wright: This treatment which, this is, again, the thing that predated the gene therapy we're talking about that's in the news now, it did in fact get rid of the sickle cell for you, but because you've lived with it for 21 years, the damage to your body is what you now have to live with.

Magaly Ghonda: Exactly.

Kai Wright: How common was this treatment, the bone marrow therapy, up until now? Is this something that a lot of people with sickle cell were able to do, or was it experimental?

Magaly Ghonda: It wasn't experimental. It's a choice. I had said no, when I was first offered it for years, because I was afraid. You choose if you want to do it, but usually they'll recommend it for people that always get extreme pains like me. Some hospitals will only take you if you're a certain age. Depending on how old you are, you can do it. Most people don't do that stuff because they're afraid. The majority of people with sickle cell I know, they don't want to do it because they're like, what's the point if I'm still going to have pain? I'm very blunt about what actually goes on. I don't tell them like, yes, I don't have any pain anymore. I'm not going to tell them that because I know that's not true.

Kai Wright: It helps, but it doesn't make everything go away.

Magaly Ghonda: Exactly.

Kai Wright: How did your parents first realize you have sickle cell? It's something you're born with, but it's not like you can communicate that pain as an infant. How did they find out that you have the disease?

Magaly Ghonda: When I was three months old, I was crying and I was holding my leg. Since I was a newborn, newborn babies are crying, but they don't cry and hold a certain part. For me, I was holding my leg, crying. That's when the doctor suspected I had sickle cell. He did a test, and it turned out I did. That's how my parents found out I had it. It was a surprise, because my mom thought she didn't have the trait. Then my dad knew he had the trait, but he thought my mom didn't have it since her mother told her they don't have sickle cell in their family. It turned out that her father did have it, but she didn't know. When they had me, I ended up having the sickle cell trait. They were surprised and they were stressed out.

Kai Wright: This is also how your mother found out, your illness is how your mother found out she had the sickle cell trait.

Magaly Ghonda: Yes. Exactly.

Kai Wright: That had to be tough.

Magaly Ghonda: Absolutely. Because you feel like it's your own slot for the rest of your life sometimes.

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Kai Wright: What about yourself? You grew up with sickle cell. How did you manage that? Did you talk to your friends about it?

Magaly Ghonda: I never said anything. Nobody knew I had sickle cell. The majority of my friends just found out this year because I-- even my friends from middle school.

Kai Wright: Wow.

Magaly Ghonda: I was really good at hiding it. Some people did just ask me oh where were you? Why weren't you here? They get nosy. They ask a lot of questions, but I just said I had the flu or I just made something up that is common.

Kai Wright: Why did you feel like you had to hide it?

Magaly Ghonda: I felt embarrassed. It just felt embarrassing because everybody is like they can do stuff, they're normal, but then you can't do half of the stuff that everyone does in their whole life. I couldn't play in the snow. I had to quit gymnastics because it was giving me too many crises.

Kai Wright: When did you first realize just how much it would affect your life?

Magaly Ghonda: Middle school, because when I was in middle school I missed, what, 60 days out of the school year because I was in the hospital so much. Then that's when I was thinking, how am I supposed to live if I can't take care of myself when I'm in a crisis? How am I going to get a job? How am I going to keep my job if I'm always quitting because of a crisis?

Kai Wright: How did that make you feel thinking, about those kinds of challenges in your future?

Magaly Ghonda: I was suicidal to be honest with you. I was suicidal. I did try to kill myself at that time and because I was just like there's no point of this. I was thinking just in my head, I was like I can't do anything. Most people don't hire you anyway when you have a disability. They're supposed to but most people don't, and so it's a lot of discrimination as well.

Kai Wright: I want to linger on that bit for a minute. I know it's a difficult thing to talk about, but as a middle school student thinking I'd rather end my life than live like this, how did you get out of that hole? How did you find a way out of that?

Magaly Ghonda: Honestly I just ignored it, but my depression got really bad last year and then I tried to kill myself again. My family was there and they helped me out a lot so I'm very grateful for them. It was sad. In my head I was just thinking, first of all most men don't want to marry a woman that's sick. I was thinking it's going to affect me financially, it's going to affect my relationships and social, and all that stuff if I can't do anything. I've been here since July, I've missed birthdays, hangouts, stuff like that, weddings, so it becomes very stressful. I'm on medication. I got a psychiatrist after that attempt and they put me on an antidepressant. That's what's been helping me.

Kai Wright: I am glad to hear it.

Magaly Ghonda: My dad as well.

Kai Wright: Your dad as well. How is your dad helping you?

Magaly Ghonda: He's always, always there for me. My dad's a businessman. He has a business in Congo. He goes there a lot for his business and then once he comes, if I just say you need to come back, he will come back. Since I was younger, he's always been like that. He's been with me my whole life. He's very supportive. He's very kind and he's patient. My dad is my best friend, so I'm very grateful that he was there for me.

Kai Wright: I am so happy that you have that support.

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Your bone marrow transplant, just to talk about that for a second. Your bone marrow transplant was dependent on a family match for a donor?

Magaly Ghonda: Yes.

Kai Wright: Who was your donor and when did you find out there was a match?

Magaly Ghonda: My donor was my brother. He was a half match. A half match is someone that their cells match yours. It's not a full match but it matches it a little bit. They used his blood and put it inside of me. Now I have his blood inside of me and my sickle cell is gone, and so I'm really thankful for what he did. He heals me, because without his blood I would still be screaming in pain all the time.

Kai Wright: That is certainly a blessing. I am so happy that he was able to help. How did you discover this treatment was an option for you?

Magaly Ghonda: My doctor recommended it for me. I was scared so I said no for years, but then this year I changed my mind.

Kai Wright: When you say you were scared to do it, explain that. Why were you scared?

Magaly Ghonda: Because it's not as like easy thing. A lot of people die during surgery. I wasn't into the hospital but another hospital I went to four people passed away from BMT.

Kai Wright: From the treatment itself, four people had died that you knew about?

Magaly Ghonda: Yes. I was originally going to do my transplant at that hospital, but because those people had died, they took everybody off the list to try and figure out what's going on and what's causing it.

Kai Wright: Now I understand why you were scared. It's quite a dangerous treatment. It carries a lot of risk. Now that you've done it, I know that you still have a lot of challenges from the recovery and from the damage that sickle cell has done to your body over the years. Emotionally, do you feel any relief?

Magaly Ghonda: I do because now I'm thinking of all the stuff that I could not do and now I can do it, so that makes me more excited about life now.

Kai Wright: What are you excited to do?

Magaly Ghonda: Muay Thai and boxing

Kai Wright: Muay Thai and boxing, okay.

Magaly Ghonda: It's a good way to vent your anger and it's fun anyways. It's fun. I'm a really active person. Oh, I skateboard too.

Kai Wright: Doing those things before would just be too painful and too dangerous.

Magaly Ghonda: Yes. It'll cause a crisis. The doctors, they weren't really too happy that I was a skater, because they just like, you have sickle cell, you're doing too much.

Kai Wright: Let me just tell you though, that is remarkable to me-

Magaly Ghonda: Thank you.

Kai Wright: -that you were doing all of that. I can't do all of that and I don't have pain problems. It is really remarkable.

Magaly Ghonda: I learned how to skateboard during quarantine because I was bored and I have a skateboard and I never used it, but now I'm addicted to skating.

Kai Wright: That's wonderful.

Magaly Ghonda: Yes.

Kai Wright: That's wonderful.

Magaly Ghonda: Thank you.

Kai Wright: I thank you, Magaly for sharing all of this, and from a hospital no less. It's a tough story but one I think a lot of people can benefit from hearing.

Magaly Ghonda: I think so too. Thank you.

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Kai Wright: That was Magaly Ghonda. This past August she received a bone marrow transplant to cure her of her sickle cell disease itself. Though as she said, she still lives with the intense complications it has caused in her body. Once again, if you are having suicidal thoughts, help is available through the national Suicide Crisis Lifeline. You can speak to someone today by calling or texting 988.

I'm now joined once more by Ashley Valentine, a sickle cell researcher and advocate, and co-founder of the advocacy group, Sick Cells. Ashley, Magaly covered a lot but I do want to start with the mental and emotional health challenges she described. How common is that? I gather that there are uniquely high rates of suicidal ideation among people living with sickle cell.

Ashley Valentine: What I will say first is I am so proud of many of the sickle cell disease advocates. It's just this bravery and this resilience that you find in the sickle cell space that I think is unsung. I know earlier we talked about how the people who discovered CRISPR received Nobel Prizes, but I also think that the patients who enrolled in the trials, their families, the providers that presented these clinical trials to these patients, I think the sickle cell disease community has a really strong hand in getting these treatments to market.

Something like the CRISPR technology, that's going to benefit society, and it got here because of people like Magaly who were brave enough to share their story and to share their story honestly, and then had that family support to keep them here.

Kai Wright: Before we take a break just so while we're right on this topic, are there resources available for people struggling with mental health who have sickle cell? Are there resources specifically for that community you could point people to?

Ashley Valentine: I would say specifically for the sickle cell disease community, it starts back with our healthcare providers. I think having candid conversations with their healthcare providers are really important. Then in terms of the social support, there's many sickle cell community-based organizations. In your state, you can find local organizations, and on the Sick Cells website, there's also a little map that has local community-based organizations.

While I point people there so you have the medical interventions with your care teams, but then also at the community level, it helps to have that peer-to-peer support. You can talk to someone who's going through some of this with you, to know that you're not alone. Sickle cell can be isolating, so it really does take a village to make us feel empowered and whole.

Kai Wright: Any pain problem, anyone who's lived with it or lived with someone with it knows how isolating it can be, so I can only imagine. The website for Sick Cells is sickcells.org I assume.

Ashley Valentine: Yes, absolutely.

Kai Wright: Sickcells.org, if you're looking for those resources. We need to take a break. Listeners, if you or someone in your life is touched by sickle cell disease, do you have a question about the new treatments or about dealing with the disease? Generally, Ashley Valentine has been a researcher, an advocate, and a family member to someone living with the disease. She may have particular help for caretakers and other family members, or people living with sickle cell disease. You can call us or you can text us with your questions about sickle cell, how you're living with it, or where you can find resources for it. I'll talk more with Ashley and take your calls after a break. Stay with us.

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Rahima: Hi, everyone. My name is Rahima and I help produce the show. I want to remind you that if you have questions or comments, we'd love to hear from you. Here's how. First, you can email us. The address is notes@wnyc.org. Second, you can send us a voice message. Go to notesfromamerica.org and click on the green button that says start recording. Finally, you can reach us on Twitter and Instagram. The handle for both is NoteswithKai. However you want to reach us, we'd love to hear from you and maybe use your message on the show. All right, thanks. Talk to you soon.

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Kai Wright: It's Notes from America. I'm Kai Wright. We are talking about the FDA's approval this past Friday of two groundbreaking gene therapies that could essentially cure people of sickle cell disease. Sickle cell is a painful blood disorder that has plagued millions of people around the world for so long with very little treatment available. I'm joined by Ashley Valentine, a sickle cell researcher and advocate, and founder of the group, Sick Cells, which advocates and organizes with people living with sickle cell.

We can take your calls if you have a question about sickle cell or about the new treatments available for it. 844-745-TALK. That's 844-745-8255. I'm particularly interested in hearing from you if you or someone in your life is touched by the disease. Maybe you have a question about the new treatments. Maybe you have a question about dealing with the disease generally. We'll see if Ashley can offer any insight.

You can call or text us. 844-745-TALK. That's 844-745-8255. Ashley, we heard Magaly's story before the break. She talked about how family stepped up for her. Her father has been an essential support. Her brother was her donor for the bone marrow transplant she received. Your family has been deeply affected by sickle cell disease as well. Your late brother, Marqus, was born with it. He was five years older than you and so you got to witness the impact of this disease firsthand. Can you tell us more about how it shapes a family?

Ashley Valentine: Magaly touched on a lot of points that we see as common threads in sickle cell and sickle cell families. Much like Magaly's family, my mom was screened before having children. She knew she had sickle cell trait. My dad was also screened before having children and he was told he did not have sickle cell trait. There's three of us. My oldest brother and myself, we don't have sickle cell trait or disease. It wasn't until my mom was at the very end of her pregnancy with Marqus that my dad had to have oral mouth surgery and they said, "By the way, you have sickle cell trait," and Marqus was born with sickle cell disease.

This idea that families with sickle cell trait and many of them don't know, and today and they're still having children with sickle cell disease because they don't know. That family guilt is something that you all hold for the course of our lives. I think outside of that part many sickle cell families that we work with have really strong love and support for their people with sickle cell disease. Many of us who are blessed enough, we have that one caregiver. In my case, it was my mom.

She's a nurse, and so she handpicked Marqus's care team to make sure that he was taken care of the best way he could be. Then my dad was always present too to pick up where my mom couldn't. I think that the families that we work with at sickle cell, it's very clear, these are the cards that we're dealt with and so we will make it work. We will learn how to navigate these systems, and as best as we can we'll have a good time doing it.

Kai Wright: I'm also curious about just the emotional work of that caretaking. I know you and your brother were incredibly close. You founded this organization together. Did you consider yourself a caretaker for him at any point in his life?

Ashley Valentine: I didn't. That goes back to our parents. My parents made sickle cell so much fun. Again, you'll see this, I was often the princess of the playroom. When Marqus would go into crisis, and he was in and out of crisis all the time, it wasn't necessarily the broader family that took care of us. Because much like the story we heard before, we didn't really share what was going on because of that stigma attached to it. My parents tried to make our life as normal as possible. If Marqus was hospitalized during Thanksgiving, for instance, we would have a Thanksgiving party at the hospital.

Or my parents would split it up and my mom would have to go to the hospital with Marqus, and my dad would take me to whatever activities. Or we had some really important neighbors at the end of the block that stepped in and were basically surrogate parents. Because if both parents were busy, then I would be in the car with the neighbor, and they were making sure to get us to and from the activities. It wasn't until I was in high school, nearly in college that the thought that, oh wow, this could actually take Marqus's life early even crossed my mind.

Kai Wright: Wow. Just to hit that very quickly. You said it wasn't until high school till it even crossed your mind. Then when it did, what did you all have to confront together in that moment?

Ashley Valentine: The way sickle cell progresses is that there's different stages. We see that during transition. When peds leave the pediatric setting and they move to the adult setting, that's where we see the highest spike of mortality. That's where the social consequences of being a disorder that disproportionately impacts Black and brown people really kicks in. In a pediatric setting, you have that full care team, you're cute, people are kind to you. When you get to that adult setting, it's the cold reality that now you're a big person in that skin, and so people are treating you for being a big person in that skin and not having a blood disorder.

The way that played out in my family is Marqus was moved out of pediatrics into adult care without a seamless transition. In that time, he was taken off of blood transfusions, which is a therapy for sickle cell, and put on hydroxyurea, which he didn't really respond well to. He developed seizures and the repeat seizures made him very, very sick. He'd have full seizures and he would be unconscious for a full minute seizing. My mom could not get anyone to believe her or help us. That's where we get to the part where she took it upon herself to basically interview her own care team for Marqus and put him into a new hospital.

His provider up until the day he died was the most outstanding provider that I've ever met, and I think one of the best humans that I've ever met because he listened to my mom. He didn't see a Black woman. He didn't see this lady that thinks that she's bossy. He saw that she was a colleague, she was smart, she was talking about science and clinical settings. He took what she said for face value and treated Marqus as if he would treat any of his other oncology patients. It was that transition moment where I really saw the ugliness of what society sees sometimes when they see people with sickle cell. We were able to move through that and find a care team that would make the rest of his life, the rest of our life just really great.

Kai Wright: Here's to mom. Good job, mom. [laughs]

Ashley Valentine: Shout out to mom.

Kai Wright: Shout out to mom. Let's go to a caller I want to start with somebody that Ashley introduced us to actually. Tristan Lee is in Williamsport, Pennsylvania. He's a patient advocate who has been living with sickle cell himself since 1983. Tristan, I gather you wanted to bring up the cost of these new treatments, yes?

Tristan: Yes. My question is, because as Ashley was saying, since sickle cell predominantly affects the Black and brown community, and a lot of us have Medicare and Medicaid, so that $2 million price tag is maybe a deterrent. It's like everybody wants the cure and wants to definitely go forward with it. At the same time, I just feel like that $2 million price tag could maybe be a turnoff to some people. How could we actually tackle that to where maybe it's more affordable, or maybe we find more funding in that, and resources in that sense? That's my main question.

Kai Wright: Spectacular question, Tristan. Ashley, so first off, explain the $2 million price tag par. Then secondly, to Tristan's point, what does that mean for access to it?

Ashley Valentine: That's right. Also, hi, Tristan.

Tristan: Hey, Ashley. [laughs]

Ashley Valentine: The $2 million price tag, I guess sticker shock, that's called the list price. In most cases, the list price doesn't make it to the patient. Out of that price, the insurance is going to be paying for a lot of that. Hospitals are going to have a portion of that cost as well, and then the patient hopefully will get very little of that cost. I understand why the price tag of $2.2 million in sickle cell is jarring. Some of this is, we have to look at the historical context for sickle cell. This disease was discovered in 1910.

The first disease modifiers to ever be approved for sickle cell was hydroxyurea and that was a generic drug, and it cost $50 a month. You go from a $50 a month drug to the next set of drugs being $80 to $100,000 a year at the list price, to the next set of therapies being $2.2 million. You can see how that may appear unreasonable, but the reality is it's not. Let's look at what sickle cell costs. I pulled some stats here. On the system, it's estimated that sickle cell costs $2.9 billion annually to take care of people with sickle cell.

That's the hospitalization cost, that's the cost of medications, and people still die young. We also know that on the family level, it costs about $10,000 a year to care for someone with sickle cell disease. Then the annual cost for that person is about $30,000 a year in health care expenditures. The $2.2 million is that one price cost, one-time cost. After that, we're assuming that you don't have sickle cell disease anymore, it makes up for itself in the long run if you're talking about health economics. If we look at other--

Kai Wright: Thinking of the individual patient, though, because I just don't want people to lose sight in the middle of this, we're talking about $2.2 million numbers. Your point is that that's the public investment in it and it's worth it when you consider the public investment. What does it mean for the individual patient who might have to face that?

Ashley Valentine: I wouldn't say that's public investment in it. I would say that's going to be the cost that the insurance companies will be negotiating with the manufacturers and then looking at hospitals. From our perspective, it should be and will be covered by insurance. That's something that we do at Sick Cells, is that we work with commercial payers. That's the Blue Cross Blue Shields, the Aetnas.

Then we also work with government payers, so that would be CMS. That's Medicaid and Medicare, to help them understand those pieces that I was laying out. For the actual patient, the patient won't be spending $2.2 million for a therapy because that's the list price. That cost and that price will be made up in other areas. Then you'll have these insurances paying for that.

Kai Wright: Let's go to Terri in Philadelphia. Terri, welcome to the show.

Terri: Hi, thank you.

Kai Wright: Thanks for calling. Did you have a question for Ashley?

Terri: I have a comment, essentially. Ashley is doing amazing work at Sick Cells, and I had the pleasure of meeting and knowing the Valentine family, including Marqus.

Kai Wright: Oh, wow.

Terri: I just wanted to say, because of this press and these new drugs, hopefully someone with sickle cell who isn't connected to the larger group can become connected. Because when I met Marqus and others at the FDA in 2012, it changed my life. Being with other sickle cell patients, I have sickle cell myself, SC, and knowing that what you're going through is normal, it makes you feel so different about you having sickle cell.

It's important to connect with other sickle cell patients because you don't feel as isolated as you all were talking about before. It's not just about all learning and learning about what's going on, and learning about the new drugs, but it's also learning and connecting with people who literally know what you're going through.

Kai Wright: Thank you so much for that, Terri. Ashley, on this point of connecting with other folks that have it in, and it's so resonant to me to know that it's not just you, you're not crazy.

Ashley Valentine: That's exactly right. This is where I was saying the peer-to-peer is really important. The reason why it's important to find each other with sickle cell is because we face a lot. If we're not believed in healthcare systems, if we're not believed in school, if you're not believed by your immediate family or your wider family about living with sickle cell, or they just don't know about it, you sometimes are saying, am I being dramatic?

Am I the only one? Why am I the only one like this? Then you start meeting other people from the community and you say, oh, there's all of us like this. You start to learn about new treatments. You start to know what worked for them, what worked for you. You're going to find that in communities who have to rely on each other to navigate these systems that we have to work within.

Kai Wright: Thank you, Terri, for sharing that. Let's go to Johnny in Lamar, Texas. Johnny, welcome to the show.

Johnny: Hi. I was really dismayed and shocked to hear that the medical professionals can treat an adult or teenager way different than a cute little child. I know it's human nature, but that's for another day. My question has to do with the long-term damages from sickle cell. Are all the damages to the body permanent or can some of them be remediated? In particular, I'm thinking about the latest research to do with reconnecting nerve tissue. Has anyone in the sickle cell research community reached out to those scientists about what they know and how they can help?

Kai Wright: Thank you for that, Johnny. What about that, Ashley, just on the idea of repairing the damage?

Ashley Valentine: A lot of the damage caused by sickle cells is permanent. This is why disease modifiers are so important because you want to try to slow down the disease. If you think about some of these therapies, gene therapies, bone marrow transplants, you want to try to do that before the disease really takes hold of the body. Magaly had talked about avascular necrosis. That is bone death. Once a bone dies, you have to replace it. It doesn't grow back. I know there is a lot of research looking at things like reconnecting nerve tissues and the sorts. Maybe in the future that's something that will be a viable option. Once damage is done by sickle cell, it's usually permanent.

Kai Wright: Johnny also mentioned something, he came back to being appalled, and I agree, the idea that healthcare professionals would treat the adult differently than the child with this disease. You've made a few references to just how people are treated. In general, what are the forces that are leading people to be mistreated? What ways are they being mistreated by the healthcare system? Is this because of the fact that this is so often people of color or people of African descent?

Ashley Valentine: Let me try to do this in 60 seconds.

Kai Wright: Please.

Ashley Valentine: My background is sociology. I think what I haven't done is explain the idea of stigma or prejudice. Sickle cell gets a double whammy. There's the disease itself is stigmatized. When the medical professional sometimes hear the word sickle cell, they have all these preconceived notions because accurate education about the actual pathology of the disease is not taught. If we think about me and you, if we don't know about something, or if you don't know what's in the dark room, or if you don't know what's behind the wall, it's up to your imagination to fill what you don't know.

For sickle cell, if you're not taught the manifestation of the disease, the pathology of the disease, then what's left is space for you to fill those unknowns with any type of preconceived notion that you've learned from your colleagues or any one or two experiences that you've learned in the field. That's why you see that shift, and all that goes into stigma. That's how you see the differences when you're in pediatrics and when you're in the adult settings. Does that make sense?

Kai Wright: It does. We have to wrap up in these last 30 seconds. Your brother's dream was that the next generation of people with sickle cell would have it easier than he did. Do you think that is coming to fruition?

Ashley Valentine: I do. I really think so. Marqus's greatest dream was to get the story out there. When we started Sick Cells, when you googled sickle cell disease, it was just some skinny dark people in hospital beds, and that's really what it was. Now when you google sickle cell, you see stories and major news outlets, you see beautiful portraits of families, you see people showing their scars. I really, really am hopeful that in the next 5 to 10 years we're going to see sickle cell in a completely different light, and people are going to be living a lot longer, and a lot happier and healthier.

Kai Wright: That's wonderful. Ashley Valentine is the co-founder of the nonprofit, Sick Cells. Thanks so much for being with us, Ashley. I learned a lot tonight.

Ashley Valentine: Thank you for having me.

Kai Wright: Notes from America is a production of WNYC Studios. Follow us wherever you get your podcast and on Instagram @noteswithkai. This week's show was produced by Regina de Heer. Our team also includes Karen Frillman, Suzanne Gaber, Rahima Nasa, Lindsay Foster Thomas, Jared Paul, and Milton Ruiz. Andre Robert Lee is our executive producer. I'm Kai Wright. Thanks for hanging out.

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