September 29, 2021

Podcast - Chirping on Sickle Cell: A Discussion with bluebird bio and Dr. Anjulika Chawla, M.D., FAAP

The Eyes on Washington Podcast Series
"Chirping" on Sickle Cell: A Discussion with Bluebird Bio and Dr. Anjulika Chawla, M.D., FAAP

In honor of National Sickle Cell Awareness Month, Holland & Knight's Public Policy & Regulation Group is proud to partner with the Black Women's Health Imperative for a four-part podcast series on sickle cell disease. Sickle cell disease is a genetic disease that causes red blood cells, which are normally round, to become C-shaped like a sickle. It is a progressive and debilitating disease that can cause pain crises, organ damage and a shortened lifespan, and it disproportionately impacts communities of color, occurring in approximately one in every 365 Black or African American individuals in the United States. This series, co-hosted by Holland & Knight Senior Policy Advisor Shawna Watley and Black Women's Health Initiative Senior Project Manager Alanna Murrell, will offer discussions on sickle cell disease within the Black community, the latest trends and policy affecting those living with the disease and the new technologies, therapies and treatments that are being developed to target its underlying causes. We hope this series proves educational for listeners and sheds light on this important issue.

The first episode in this series features a discussion with Sonya Elling and Dr. Anjulika Chawla, M.D., FAAP, of bluebird bio, a Massachusetts-based company focused on developing gene and cell therapies for severe and rare genetic diseases including sickle cell. Dr. Chawla shares her experience working with patients who have sickle cell disease, from her first time encountering someone with sickle cell when she was in med school to her current work at bluebird, and emphasizes the need for greater education and awareness about the disease in both the medical and nonmedical communities. She also talks about the advancements in understanding and treatment of the disease, touches on the issue of long wait times at the ER for patients and explains new treatment and curative options to note.


Shawna Watley: So good morning, everyone. Thank you so much for joining us today. My name is Shawna Watley. I'm a senior policy advisor with Holland & Knight and a part of the Holland & Knight Public Policy & Regulatory Group. This is our podcast, our Eyes on Washington Podcast, and we're excited to be in partnership this morning with the Black Women's Health Imperative. So we welcome you to our first podcast in a four-part series on sickle cell disease. This discussion centers around sickle cell disease within the Black community, the latest trends and policy affecting those living with the disease, as well as new technologies, therapeutics and treatments that are being developed to target the underlying causes of sickle cell. Today, our guests include Dr. Anjulika Chawla, M.D., FAAP, who is a senior medical director of medical affairs in the U.S. SCD at bluebird bio, and Sonya Elling, senior director for Alliance Development and Government Affairs at bluebird bio. I am excited to introduce my co-host, Alanna Murrell, and she is the special project manager with Black Women's Health Imperative. So, Alanna, I'm going to kick it to you. Can you just share a little bit about the Black Women's Health Imperative and the work that you all are doing?

Alanna Murrell: Sure, thank you so much for the opportunity to co-host, and I'm happy to join. Black Women's Health Imperative is a national nonprofit organization dedicated to advancing health, equity and social justice for Black women and girls through policy, advocacy, education, research and leadership development. We identify the most pressing health issues that affect the nation's 22 million Black women and girls and invest in the best of the best strategies and organizations to accomplish those goals. So BWHI works to reduce the threat of sickle cell disease on Black women and other women of color. The devastating toll of sickle cell disease on African Americans requires urgent, evidence-based public health solutions to prevent and control this disease among communities of color. This serious, progressive and debilitating genetic disease can lead to organ damage and a shortened lifespan. Sickle cell disease impacts approximately 100,000 Americans. It occurs among one in every 365 Black or African American individuals born. Right now, we are working to raise awareness and discuss research-based solutions to alleviate the disproportionate burden of sickle cell disease on communities of color, and believe that we can do that with bluebird bio.

Shawna Watley: And so now I'd like to turn it over to Sonya. Sonya, can you share about the work that you all are doing?

Sonya Elling: Thank you for having me, Shawna. It's my privilege to be here today and appreciate you including us. And it's been a privilege to be working with BWHI, an organization doing tremendous and incredibly important work fighting for health equity on behalf of Black women and girls. We're here, obviously, to talk about the work bluebird bio as well is doing around sickle cell disease, and I wanted to start first with those who may not be familiar with bluebird bio. We are a gene therapy company headquartered in Cambridge, Massachusetts, for developing gene and cell therapies for severe and rare genetic diseases, with the goal that people facing potentially fatal conditions but limited treatment options can live their lives fully. Beyond our labs, we're working to disrupt the healthcare system. What does that mean? We're trying to make sure that patients have access to treatments when they become available, and hopefully we want to help people better understand gene therapy. And we hope that it can become available to all those who might benefit from these therapies when they are hopefully approved by the FDA. "Bluebird" was selected because the term a "bluebird day" is a beautiful, sunny day after fresh snow. You have fresh snow, beautiful sunshine outside. What all of us at bluebird focus on is working together to get every patient possible their "bluebird day." So bluebird is meant to keep all of us in our labs and throughout the company focused, and in the community, focused on helping patients and getting as many people as possible their bluebird day. I am also excited to have my colleague, Dr. Anjulika Chawla — we call her Anju — to join us as well here today. She is, as you heard Shawna mention, is bluebird's senior medical director of medical affairs, where she focuses on sickle cell disease. But she's also a pediatric hematologist who continues to care for patients outside of her work at bluebird. And we'll let her talk about that a little bit. But I wanted to also make sure I took this time to talk about how I think she's the epitome of a superwoman. She's compassionate, she's wicked smart and Anju manages to maintain an amazing sense of common purpose while juggling so many responsibilities. Anju's going to chat with us today about her work as well as her work with her patients, or her kids, as she calls them, and help us better understand what might be on the horizon for treating sickle cell disease.

Introducing Dr. Anjulika Chawla and her Experience Treating Patients with Sickle Cell

Anjulika Chawla: Good morning, and thank you so much for the opportunity to come and speak today.

Sonya Elling: Absolutely. So I tried to touch a little bit on what you're doing, but if we could just take a moment for you to tell us what brought you into particularly treating, as well as now focusing on, patients of people with sickle cell disease.

Anjulika Chawla: I grew up actually in Vermont, which is not the world's most diverse place, but being somebody of Indian descent in a very un-diverse place, I learned how to speak up, advocate and as well as assimilate with what's going on around me. But it was my second year in med school that I was working in the emergency room and this gentleman of color comes in in rising pain. And it was one of the things of how do we figure out what's going on? And somebody said, "Oh, you might have sickle cell disease," which was the first time I'd ever heard about the term. Went looking up, trying to figure out what's going on and realize we know the molecular reasons why sickle cell disease happens, and yet we are doing so poorly in controlling that pain. This young gentleman who came in, he was in so much agony, and I couldn't help him quick enough. And then realizing that this is something that he dealt with in an unpredictable way. You know, he tried to keep a job but couldn't do it because he might have to leave work because something would trigger it. He was tired all the time. To be honest, I didn't get to know him all that well. I met him a couple of times in the emergency room, but it really triggered something in me about this super rare disease I'd never even heard before.

From there to have the privilege of going down to Tulane in New Orleans, Louisiana, which is where I actually saw many patients with the disease, and then saw more pediatric patients. And I watched one of these kids who are generally happy, playful, wanting to do everything. And then, a change in the weather, something would happen — if they get an infection, they'd come in. And again, the pain is what's brought them forth trying to relieve it. And then I heard my first time dealing with acute chest syndrome, which is when somebody with sickle cell disease, again, can have a respiratory infection. It may have a pain crisis before it, and it triggers this reaction in the lungs that develops into this pneumonia that spreads all over the lungs within a matter of hours. Not days, not months, hours. And I watched this little guy, a 2-year-old, come in, and he went from, you know, having a little shortness of breath, requiring a little oxygen, to being in the ICU and intubated within 24 hours. We got him to turn around. But it was amazing how fast that happened and really wanted to work on how to make that better. And then I went to San Francisco, worked at UCSF as a fellow to get further training in both rheumatology and oncology for pediatrics. [That] was where I really realized, "This is for a lifetime." But also the realization as you grow older, the times of being sick become more and more frequent. And the only known accepted cure for sickle cell disease is a bone marrow transplant. You have to have organs that are healthy enough to undergo a transplant, and it's a high-risk procedure with many possibilities of things going wrong. Well, my thought was is that I really would like to see, "Well, can we do in the uterus? " We can make the diagnosis in the baby. The fetus shouldn't have an immune system, so maybe we could do this transplant in utero while the baby's in a safe, secure environment of the uterus. That was my dream. Unfortunately, guess what? Even a fetus has a little bit of an immune system. And one of the other things we learned is that you have to make space for the incoming marrow to take. You've got to get rid of some of the old marrow just to make space. And the only way to do that we know how, is chemotherapy or radiation, which, of course, is a really bad idea in a fetus. So from there I said, OK, well, I might take care of kids as they come. And at that point, with the new drugs on the horizon, hydroxyurea, which may actually work and help people with sickle cell disease. So that's when I came over to where I've been taking care of kids for, well, we won't say how long, but at least a couple decades. And so here was really this discussion of I've watched these kids grow. I've seen what the hopes and the dreams and aspirations are.

And what my goal always has been is to let everybody live the life that they choose, not determined by their disease.

So in coming to bluebird bio, it's really people behind this, really looking at this idea of not just trying a new medicine, but stopping the disease in its tracks by using something like gene therapy. I met a lot of other, as we say in New England, wicked smart people who do all kinds of crazy things to make this kind of dream a reality. The first to see if gene therapy can work. Second of all, to make it as safe as possible. And then, third, make it an option for anybody who's interested. And again, with FDA approval, hopefully we can prove both that it's efficacious and safe enough, that we can bring it out to everybody who could need it. And of course, this is where we need to raise awareness of what sickle cell disease is about, why is it worth it to consider something like gene therapy and then how do we work as a team to make it more accessible to everybody who wants it as an option.

Advancements in Sickle Cell Research and Treatment: Have We Made Progress?

Shawna Watley: Wow, that's so powerful, Dr. Anju, and thank you for the amazing work that you're doing. As you were speaking, I thought about when I first learned about sickle cell. In the '70s, this movie came out about a young man who had sickle cell. And it was so impactful for me because I always heard as an African American, "Oh, they have sickle cell trait," or, "You need to make sure you don't have sickle cell trait," but I didn't really understand what that meant and I didn't know how it impacted me or others who actually had sickle cell. So this movie was very eye-opening, and this young man at Vanderbilt, he ultimately passed away. So I guess my question is, you know, have we made the advances that we should have made in this 30-year time period? So if you could share a little bit about why you think there's barriers or whether, why there's been slow progress in really finding a cure or treatment.

Anjulika Chawla: Wow, that's a huge question.

We have made so much progress for sickle cell disease. The trouble is, is that the more we dig, the more we understand the problem's even bigger than we thought it was initially.

So that first step for newborn screening was first initiated in 1972 in New York State. We knew how to check somebody for sickle cell disease in 1972. And there was this, really this thought, about trying to bring this nationally. However, it wasn't until 2005 that 48 of the states actually started doing newborn screening for sickle cell disease. That's almost 30 years. But one of the things we were able to show in the meantime is by doing that newborn screening, by identifying which infants had sickle cell disease, we could start managing their disease right then and there instead of waiting for — one of the things that happens in the disease, the spleen dies off and doesn't protect you from blood infection, so the number one killer of young children with sickle cell disease was, in fact, blood infection killing them before their fifth year. And that might have been the presenting sign of sickle cell. So with this thing in place, they were being monitored. We were able to do education. And by the way, the biggest advancement we've made to date on sickle cell disease is education. All the other stuff is great and I'm glad we're doing it, but the number one step is education: talking with family, really thinking about this as something that lasts a lifetime and how to protect your child. And one of the glorious things I've been able to say with my transition through sickle cell disease is to be able to say, "You know what, you have sickle cell disease, but I expect your child to be able to do whatever they want to do. But we need to stay on top of this. We still have to manage it." As a mother of four, my children do make up when they have pain. But with somebody with sickle cell disease, you have to take it for what they're saying, because the pain comes from deep in the bone and there's no external way of telling it. So when your infant is screaming bloody murder at you, it might be a public tantrum, but it might be pain. Try some hydration, try some pain meds. Let's see if we can make this better. So that first part, education part, is that first step.

The other advancement that has come about is hydroxyurea, which again, it's one of those things people ask me, "Well, it's a chemotherapy drug, why are we using this for sickle cell disease?" Well, it's because it's a chemotherapy drug. It really does something amazing. It causes stress on the bone marrow where those red blood cells are made and forces those red blood cells, that instead of making all the sickle, it makes a small percentage of fetal hemoglobin. It switches back to that baby hemoglobin, which has that ability to interact with the sickle hemoglobin. So it doesn't polymerase, it doesn't cause syphilis. Now, it's not 100 percent effective. It really helps a lot, but it's not 100 percent effective. So what we're seeing now — and over the years what I've seen — is that kids taking the hydroxyurea, we don't see them in the hospital anymore. Makes my day. We used to. When I was training, we'd have any time on our service, four to five kids that were in with sickle cell disease, in the hospital in pain. Now, at most, we have one or two. Still happening, there's still pain, we haven't stopped it all, but it's so much better than it was before. But what we're seeing as they grow into adults is the hydroxyurea doesn't help us much. And I don't know if it's because of the hydrea. I don't know [if it's] because there's slow progression of the underlying disease that hydrea can't compensate for. I don't know the reason for it, but it's one of those things that I think we need to continue to do better. And this is where you bring to date, what are the advances that are happening? So I do think the next step really is on these new medications that they're coming out with. They do different kinds of things. They help stop polymerization, they stabilize the hemoglobin, they make the blood vessels more slippery so the cells can slide through. Great ideas. And each one of these eases suffering a little bit more, which is amazing. And we need to keep having that.

So then you had asked me, as far as, you know, with these advances, well, isn't sickle cell disease taken care of? Why is this such an issue? The biggest issue and the biggest barrier I see at this point in time is care for adults with sickle cell disease. When I started practice, the expectation is that they would die in their late teens and early 20s. That expectation doesn't hold anymore. Now we have folks that, again, the average lifespan of somebody with sickle cell disease, the more severe form, is 47. Ain't good enough, by the way, but we've made a lot of progress. So I think for me, the thought I want to get across is we have made a lot of progress, but there's a whole lot more to do. And this is where voices like this become very important. The need for management, comprehensive care and doctors who specialize in this, who want to take care of this over the long term, is really low. And where these folks have to go, if they don't have good access to hematologists — which about 50 percent of the sickle cell population does not, just because of their geographic location, which is not OK — that's something that I'm hoping that this group will kind of think about and address a little bit more with one of the things that we've tried in the last decade or so to see if we can inform and teach our internal medicine colleagues, our emergency room physician colleagues, to say if you can't get access to a hematologist, that somebody at least has some information on how to take care of you. But the truth of the matter is, sickle cell disease is still a rare disease. For an ER physician or for an internal medicine doc to have enough understanding of sickle cell disease, how it affects the body, how to prepare for the future, it's really hard to do for one or two patients in your practice or a patient that you may see once every month. It's not, it's not enough, they've got many other things on the plate. So that next step is, is then, OK, we need to figure out how to get patients to a hematologist, or to somebody — it doesn't have to be a doctor even, it could be a nurse practitioner — or a provider who really can think about the person as a whole and over a life span. So I think that's where the next step really lays. And then once we get people involved and get into this comprehensive care model, that's when we can start having discussions about what can we do to cure, what are the kind of options that are out there and what kind of options may be coming that may help your life immensely.

Shawna Watley: Thank you so much. As I was thinking about your answer, one of the things that we're working on here in Washington, D.C., is making sure we have more people of color coming through the pipeline that are medical doctors and who can actually be in communities that sickle cell impacts. So that's something we're definitely working on. Sonya, I don't know if you want to share a little bit of some of the legislative work that we're doing and working with some of the members of Congress to try to help educate and just build off of the work that you all are doing at bluebird bio.

Lengthy ER Wait Times and How to Fix Them

Sonya Elling: Sure. And I did want to also ask this, you've mentioned throughout this conversation, Anju, about ER, and it's my understanding, exactly as you pointed out, too many individuals with sickle cell don't necessarily have a designated hematologist. And so they do end up having to go to the ER for their treatment. And there's been a survey and some studies done this year that show there's a considerable lengthy wait for these individuals. I think it's fair to say implicit bias is occurring. And they're saying now, the most recent study I read said that wait times go from one and a half hours to 10 hours for an individual to actually receive treatment when they come into an ER in a crisis. There was an NIH study for 1973, which is so disheartening to know that there was a study that long ago that spoke to the importance of individuals being treated within the first 30 minutes of coming to an ER in regards to helping alleviate their pain, and yet here we are now. That it's been that long since this NIH study talking about how imperative it was that people have their pain addressed within 30 minutes of seeking care and that we're now in a situation where some people are reporting 10 hours. They're sitting waiting in the ER, so any thoughts you have to help proffer more education and regarding implicit bias, as well as partnering with some organizations that are on the forefront of this, and how can we start helping make an impact to address this?

Anjulika Chawla: Like I said, the more we learn about sickle cell disease, the larger we realize the problem is. I think about that article in 1973, and as far as what was required to get into an emergency room, get treated and get out is so much less than what it is today. I have to get a little personal here. I just ended up spending Monday night in the emergency room with my son who's getting wheeled out for appendicitis. Called my doctor who said, "You're right, he needs to be evaluated. Go to the emergency room, I'll call in. They'll move you along quickly, because if this is appendicitis, it's clearly a medical emergency." The line — and this is, again, the state of life with COVID in the hospital these days — the line to get into the ED, the door, was all the way out to the parking lot. It was a 30-minute wait to get to the door of the emergency room, not because of any sense of the emergency room, by the way, and not any fault of me. We all are trying to do the right thing, but there is a huge demand for the ER and trying to get your kids treated. And I am certain every child there needed to be there. The second thing that happened as we got in, they were such nice people. They were like here, you know, let me get you a wheelchair. He should be sitting down. He'd been standing for the last 30 minutes as we tried to get to the door, which was great, but then it was, what's your date of birth? What do you, you know, what's your insurance? What are all these various pieces of information? And I was like, "I was just told to come urgently to the ER. I am not prepared for all these questions." And I'm a physician mother. I should know better. I was flustered a bit, but we went through that and then we were sent back to the waiting room. And while we were waiting, we could see them rolling a stretcher from an ambulance to go in, and I was like, "Somebody else was even sicker than my son. Oh my God, we're going to have to wait longer." And then from there, we get checked into the nurse to give him some pain meds. They check in with the doctor, then they finally bring us back. It was about an hour, hour and a half in. Once we went in again, everything was very pleasant, very nice. Really did their job well, got his blood drawn, got all these things done, the doctor had to come in and evaluate. But by the time I actually got my question across to the physician and for the physician to put the order in, from the moment I hit the door, two and a half hours.

Now, take somebody with sickle cell disease who's — especially if you're a young adult, you know that when you go to the emergency room, you're giving up your sense of dignity. You're giving up any control because they're going to dictate what meds you take, when you take it, what you do, all that kind of stuff. You're giving up all that control. So you usually try to manage your own pain, usually the studies show for two or three days before heading into the ED. So when you go to ED writhing in pain, this bone pain where, one of my kids who's now a lawyer, lovely lady, but she described it to me as somebody taking a hot, dull knife, stabbing it in my bones and pulling up, waiting about a minute and then doing it again and again. But dealing with this kind of pain and then having to wait 30 minutes to get to the door, wait another 30 minutes while you get to the nurse, wait another hour to two hours before you can actually get pain relief? Oh my God, you know?

And what's scary about pain crisis, which most of our ER staff really still don't understand, is that particularly as you get older, a pain crisis can lead to death. It is actually one of the leading causes of death in that 20- to 30-year-old period. This is truly a need to be in the emergency room. This is not just somebody says, 'Hey, I got something hurt in here.'

So what do we need to do to help fix the ER? First, to realize we need to walk in each other's shoes. As a hematologist, I need to walk in the ER doc's shoes. He only sees one or two patients with sickle cell disease. I need to, you know, the triage person, who probably, you know, maybe got mentioned once what sickle cell disease was, but he's seeing 30 patients a day checking everybody and doing everything. Why are you going to remember exactly what's happening with sickle? So what are the things we can do? The first is not to lay blame, not to lay blame on ER and not to lay blame on the patient. We need to have a conversation that's open and honest and have a discussion of how to do it better. And to do this at a policy level, that's what I would like to see. Make that a requirement that conversation happens. That I think is one big step that we could do. And again, to have that conversation before everybody's sick. Do this when they're well, not after the mother complains of the treatment that they got in the emergency room.

The Need for Education: Training, Policy and Communication     

Sonya Elling: So we've been wondering and talking about with BWHI, we've seen HHS had in its budget training to help disparities and maternal mortality and wondering how we could build on that to improve the quality of care for people with sickle cell and have it be required as well for sickle cell. And I interrupted you to ask, are you thinking that could be sufficient, or [do] you think also having there being training to make sure there is some type of ongoing training to make sure people remember all the way back to nursing school or med school what sickle cell is, if that is not obviously their area of focus, and particularly if they now are practicing in the ER and they've been in there for maybe a decade or two and don't encounter these individuals as often?

Anjulika Chawla: So I think training is important. Now, considering I've spent 20 years reteaching our ER staff, reteaching my residents, reteaching our fellows, reteaching the attendings to the point where they don't necessarily remember what to do, but they know to call me, that has been the big step. Again it's the conversation that matters versus the actual topic. So I think, yes, I do think training is important. Don't get me wrong, we still need that. But it's only short-lasted. It doesn't last past a year, as much as I would love for it to last longer than that. Think about it. When we teach our patients about how to manage this disease, we ask them to come in every six months to make sure they continue to understand it. Because as we grow, as we develop, as we deal with real life, you forget. And as I get older, I forget more, but that's another story. So with that idea of is there a way to build in, not that a specific training happens one time. And this is what I worry about with some of these initiatives. It's all gung-ho. In 1973 we were all gung-ho. And here we are again, thankfully, that we're all gung-ho. But how do we make that last, how do we make that linger? And the only way to do that is to require it, because what is needed is going to change. In my dream world, I would love to see that all of these folks are sickle cell survivors. So they get cured of their disease, but they may still have issues from what damage the disease has already caused. So their providers need to have that conversation of understanding what sickle cell disease was, so that training is going to have to change. The way we talk about it. So that ability to have that conversation, just to see you walk in each other's shoes for a little bit, see what's needed. Because I think, first of all, I think it would reduce bias, implicit or explicit, for that matter. I think that idea of building trust, which goes both ways, not just the patients of the care system and the care system of the patients, it's got to go both. So I think that's where we need to have that conversation. This is where I don't know where policy can play in this. You guys are the smart ones on this. But this is where I think we kind of need to go. And, again, the idea that sickle cell disease is a genetic disease. It lasts the whole lifetime, it doesn't disappear. And that there may be some really good times when people feel just fine and can do anything anybody else can, and they should be able to do that. And if they're in the emergency room and if they happen to be on their phone trying to look up sickle cell disease or trying to figure something out, that the patient is not ignoring the care team. And if the care team is looking it up, the patient shouldn't assume that they're ignorant. What can I say? I really think it's all about communication as always.

New Treatments and Potential Cures on the Horizon

Sonya Elling: So, Anju, I've heard you talk about gene therapy and new treatments on the horizon, and I've also heard you reference potential cures or curative intent treatments and was wondering if you could help us better understand that, and particularly help us that don't have medical degrees like you, so we can understand and get excited about some of the stuff we keep seeing and hearing about in the media.

Anjulika Chawla: Yeah, what is the cure is an incredible question. So it's easy to think about what a cure is for an infection, right? The infection goes away. But how do you cure a genetic disease? So, it depends on how you define a cure. So if you go for the strictest definition, it's to not have the gene in the first place, which is not something that as humans we can do or I think is the right thing to do, is to mess with somebody's germline before they've even come out or got even to the uterus, at the cellular level, to say, "OK, we will now switch this one gene to take out sickle." First of all, it's not feasible at this time. And then the other thing is that you may change other things in the person that you don't know. So then what does it mean when we say that an allogeneic — so a stem cell transplant where bone marrow comes from somebody else — as that being a cure and that's considered an accepted cure? Well, what you're doing there is you're taking the cells that make sickle hemoglobin out of the body, and you're putting cells in that don't make sickle hemoglobin and just make normal hemoglobin. But one of the things that it doesn't do, those are only the stem cells. So in your ovaries or in your testes, you're still going to be making cells that always have SS, if you happen to be somebody who has SS disease, and you're going to pass on the sickle gene down to your infant, even if you're cured of the disease. So is that really a cure? And the other limitation of transplant is any damage that's happened from sickle cell disease to the point of the transplant is there. It's not that you reverse it, but with transplant, the hope is that you can actually stop any progression. In older adults with sickle cell disease, they get pulmonary hypertension where the blood flow to the lungs is very, very difficult and it strains the heart. Well, if the heart gets strained to the point where it scars, it's still going to be a weak heart. So even if you cure the sickle cell disease, the act of sickling that's happening, that heart still has to strain, and that strain may not go away. That may last for a lifetime. So, then next step is when you talk about gene therapy, what happens there is, we're taking the person's own stem cells, so blood stem cells, put them in the lab, modify them and then put those stem cells back in the person. So the thing about those stem cells is they all have to have transform. Right? Because, does anything do that 100 percent of the time? And they all need to make either reduced amounts of sickle down to sickle traits, or they have to have, make it, a hemoglobin that actually interacts with the sickle so you don't get polymerization. And again, it only works at the point that you use it. It's not going to work for any damage that happened beforehand. It's just for slowing or stopping sickling at the other side. Then the final question is, what's enough? Do you really need that cure all the way down to the gene from that infant that hasn't even been born yet? Or is it so good that you don't have pain? You do this in a kid or a young adult that then can grow and do what they need to do without so much [pain] and organ damage that they actually live full lives, full, extensive lives. And that brings up, if a near cure is a functional cure — and this is some of the terms that you may hear out there that people are throwing around — is that enough? And the answer to that is, we're going to find out, but we really don't have the answer quite yet. We've got some ideas, and there's people, thankfully, that want to come with us on this journey, with their patients, with sickle cell disease, who say, "My disease is so bad right now, anything is going to be better than what's in my future." I'm so happy that you're joining us and taking that big leap to think about what else could be done and to see what a cure could be and if, what is a near cure, and is that good enough?

Shawna Watley: I'm just thrilled that we're bringing light and helping to shed light on this important issue. You know, over the summer, I don't know if you were surprised, but I certainly was, when The New York Times did the four-part series on sickle cell. It just blew me away. And exactly, it's just an exciting time. And I'm just happy that we're able to work with you all and bluebird bio and Black Women's Health Imperative to be a part of the education process and figure out how we can develop policy here in Washington that's going to be helpful to the efforts that you're doing on a daily basis. In closing, I hope you all have a bluebird day, and I just thank you for participating in this very important discussion. And we look forward to continue to be partners with you all. And please don't ever hesitate to reach out if Holland & Knight can be supportive of what you're doing.

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