<i>[light music]</i> <i>HELEN: Sometimes it just comes out of nowhere.</i> <i>Pain would start in my arms,</i> <i>then it’d go to my legs and it’ll start in my back.</i> <i>I describe it as needles poking me all the time.</i> <i>My sweet 16, my friends that were there,</i> <i>they didn’t know I was in pain.</i> <i>They wanted me to, like, dance, have fun</i> <i>and all of that.</i> <i>I’ve gotten used to, like, showing that</i> <i>oh, I don’t have pain.</i> <i>I’ll just act like if I’m normal.</i> <i>♪ ♪</i> <i>I never thought there was gonna be a cure.</i> <i>I always thought I was gonna</i> <i>stay with sickle cell, like, forever.</i> <i>This trial, no other teenager have come through it yet.</i> I’m gonna be the first one. <i>[dramatic music]</i> <i>♪ ♪</i> [kisses] <i>HAYLEE: This is our family.</i> <i>FATHER: We’re like the “Obandos Reality Show.”</i> - Ahh! [laughs] HELEN: Haylee, that’s gonna break it. Haylee! <i>That’s me, the big sister Haylee.</i> <i>That’s Helen. You just saw her sweet 16.</i> <i>That’s our brother, Ryan,</i> <i>our mom, our dad, and our abuelito.</i> <i>Here’s how to look at it:</i> <i>I’m telling you this story,</i> <i>- but it’s about her. </i>- Oh, no! <i>HAYLEE: Why am I telling it to you?</i> I’m the fun one. [laughs] <i>And Helen—</i> <i>RYAN: Helen—she’s quiet, sometimes shy,</i> and she sometimes gets annoying, too. - I call her my little old lady. - She corrects me a whole lot. - She is so mature for her age. <i>HAYLEE: What does she think about herself?</i> - Myself, I feel like I’m very shy, not talkative. I love a lot of people. I’m very kind. Hopeful. <i>[light somber music]</i> <i>HAYLEE: My sister is my best friend.</i> <i>We were both born with a genetic blood disorder</i> <i>called sickle cell,</i> <i>but Helen was always way sicker.</i> - [coughing] <i>I’m telling you this story</i> <i>because something big is going to happen in our family.</i> <i>Helen’s about to go through</i> <i>a breakthrough experimental gene therapy.</i> [laughter] <i>What happens to her could have implications</i> <i>for thousands or even millions of people around the world.</i> <i>Scientist are studying my sister</i> <i>to see what happens when they rewrite her DNA.</i> [beep] <i>HELEN: My relationship to blood</i> <i>is like everywhere in my body</i> <i>as, like, something you can control.</i> I don’t think about dying. But my mom, she really usually gets scared all the time. - A lot of people don’t even know what sickle cell is. <i>SHEILA: Like, you and I would have normal blood cells.</i> <i>A nice, round shape</i> <i>where they can go through your body</i> <i>and through your veins normally.</i> <i>Theirs sickle.</i> <i>They—sort of like banana moon shapes.</i> <i>HELEN: Say it’s a car crash outside,</i> <i>and they block the whole highway,</i> <i>so nothing could get through.</i> <i>My red blood cell gets, like, clotted in one area</i> and that’s the area that starts to hurt. - They can be happy little girls one minute. Seconds later, they’re screaming, “I’m hurting.” <i>When you don’t have oxygen going throughout your body,</i> <i>little by little, sickle cell deteriorates the body,</i> starting off with the bones and their organs. And, you know, vision. Um—they’re basically dying slowly. <i>- My name is Sedika Gazey,</i> <i>and I have sickle cell.</i> <i>- My name’s Braxton Hubbard.</i> <i>I’m 26, and I have sickle cell.</i> <i>- My name is Andre. I’m 14,</i> <i>and I have sickle cell disease.</i> - I didn’t know that there was gonna be a lot of people with sickle cell. I thought it was just the— it was just only me. Sickle cell hurts pretty badly. - It’s hard to describe. - A sickle cell pain crisis feels like your bones are being crushed. - Feels like having broken glass running through your veins. - As if someone is grabbing one of your organs and squeezing it with nails. You would prefer death over living— going through it. That’s the type of pain. <i>HAYLEE: My mom is the family expert on sickle cell,</i> <i>so she’s going to tell you more about it.</i> <i>SHEILA: About 100,000 people in the U.S. have sickle cell.</i> - Hey, everyone, this is Shirell, the sickle cell diva. <i>SHEILA: And millions of people around the world do, too.</i> <i>Most have ancestors from the African continent.</i> - It’s important to figure out what your genotype is— <i>SHEILA: Some have families from Latin America, like us.</i> <i>People also have it in the Arabian Peninsula,</i> <i>India, and the Mediterranean.</i> <i>About one in 500 African Americans have it.</i> <i>It’s the most common inherited blood disorder in the U.S.</i> <i>But attention and funding for research</i> <i>hasn’t matched the scale of the problem.</i> - I feel like sickle cell’s not important. - I’m gonna be straightforward with you. I think it’s like that because it’s a predominantly black illness. - You have to sometimes live almost... like you don’t have a care in the world. [laughing] - It just changes the trajectory on everything you do. <i>STUART: We don’t treat the primary problem they have.</i> <i>We treat pain crises, we treat stroke.</i> It’s almost like having a fire in a building and you’re putting the fire out on the second and third floor, but the fire began in the basement. <i>SHEILA: This is Dr. Orkin.</i> <i>He’s been studying blood for over 30 years.</i> <i>STUART: We’d like to do something which gets at</i> <i>the sort of root of the problem.</i> <i>SHEILA: What’s causing Helen’s problem</i> <i>is her hemoglobin,</i> <i>the stuff inside her red blood cells.</i> <i>Sickle cell makes it stick together.</i> Here we go. Aww, there’s Helen. <i>When she was a baby, it wasn’t sticky.</i> <i>That’s because there are two types of hemoglobin:</i> <i>fetal and adult.</i> <i>The fetal kind doesn’t sickle.</i> <i>If Dr. Orkin could figure out how to flip the switch</i> <i>in Helen’s body and tell it to make</i> <i>fetal hemoglobin again, forever,</i> <i>she could be cured.</i> <i>For years, they’ve struggled to do it.</i> <i>STUART: We didn’t have a clue what to look for, really.</i> <i>SHELIA: Then in 2008,</i> <i>bang, it happened. [alarm blaring]</i> <i>One of Dr. Orkin’s students found the piece of DNA</i> <i>that can help flip the switch.</i> <i>So they tried it on some mice...</i> <i>and it worked.</i> <i>STUART: And that’s the basis</i> <i>for a current gene therapy trial.</i> <i>It’s the exact experiment.</i> <i>SHEILA: Then Boston Children’s Hospital</i> <i>tried it on three adults.</i> <i>It’s still early,</i> <i>but it looks like it’s working on them, too.</i> - It’s quite remarkable. - [singing in Spanish] NURSE: Helen? - [singing continues] - Yeah. TECHNICIAN: And one more big, deep breath. <i>SHEILA: But they really need to try it on a kid,</i> <i>like Helen.</i> <i>The older a person gets, the more permanent damage</i> <i>sickle cell does to the body.</i> <i>It made me feel that she was gonna die,</i> <i>and she had no time.</i> - [speaking Spanish] - Haylee, look out, baby— <i>HAYLEE: Okay, remember I told you</i> <i>Helen and I were both born with sickle cell?</i> - Ow! <i>HAYLEE: I don’t have it anymore,</i> <i>and this is why.</i> <i>Last year, Helen was really sick in the hospital.</i> <i>I mean, like, bad.</i> <i>But then... </i>- It was actually a doctor who approached me and asked me if I was interested in testing my kids for a bone marrow transplant. <i>HAYLEE: If we found Helen a bone marrow donor,</i> <i>she might be cured.</i> <i>But the odds were against her.</i> <i>The hospital would only do it</i> <i>if the donor was a sibling who didn’t have sickle cell</i> <i>and who matched exactly.</i> <i>If the match wasn’t perfect, it would be too risky.</i> <i>You could even die.</i> - They sent swabs home for our DNA. <i>HAYLEE: And the news came back.</i> <i>Drum roll. </i>SHEILA: Where’s Ryan? WOMAN: Behind you. <i>HAYLEE: Our little brother,</i> <i>our hero, Ryan, is a match.</i> <i>But not for Helen.</i> <i>For me.</i> <i>RYAN: It was upsetting</i> ‘cause I wanted to help both of my sisters, then they could be, like, cured. SHEILA: Ryan? Ryan. Say hi. <i>HAYLEE: I got Ryan’s bone marrow.</i> <i>I got better.</i> <i>But it wasn’t supposed to be me.</i> <i>Helen was always the sicker one.</i> [cheering] SHEILA: It’s her rebirth. <i>HAYLEE: My parents even tried to have another baby</i> <i>to be Helen’s donor.</i> <i>My mom did IVF four times.</i> <i>Not even a single embryo was the right match.</i> <i>SHEILA: I felt scared</i> <i>and sometimes useless, too.</i> <i>[light music]</i> <i>♪ ♪</i> - You big, bro. [chatter] <i>HAYLEE: And then we got the news.</i> <i>Helen was approved to be the first teen in this trial.</i> <i>She won’t need a bone marrow donor.</i> <i>They’ll use the cells that are already in her body</i> <i>to help her.</i> <i>SHEILA: I think things work out</i> <i>in a miraculous way, if you can call it that.</i> <i>HAYLEE: We’re all just praying it’ll work.</i> - If I woke up and I was cured, no more pain, I think the first thing I would do is just... Man, I don’t think I’ve ever thought about that. - Ooh, if I was cured, I would go outside with no coat on. [both laughing] - I would travel. I would be traveling everywhere. - Graduate college. [sighs] - Jumping out of a plane. - No. - [laughs] - I don’t know. I actually don’t know. <i>[light music]</i> <i>HELEN: When I’m cured, I’m gonna be happier</i> <i>and I’m gonna be, like, more out—like, outgoing.</i> SHEILA: Hurry up. - Am I leaving it? <i>♪ ♪</i> <i>ABUELITO: [speaking Spanish]</i> <i>♪ ♪</i> - Ew! - I need another hug— - Nah. You’re sweaty. Mami! Ew! - [Ryan laughing] <i>♪ ♪</i> <i>HELEN: A lot of my friends don’t know I have sickle cell.</i> <i>Nobody knows that I’m going through this process yet.</i> <i>I haven’t told anybody.</i> <i>♪ ♪</i> <i>[upbeat percussive music]</i> <i>♪ ♪</i> [laughs] <i>HAYLEE: To get her body ready</i> <i>for gene therapy, Helen needs chemotherapy.</i> - Yeah, I started the chemo today. <i>HAYLEE: That would make room for her new stem cells,</i> <i>but it’s very dangerous.</i> <i>It also means she has no protection</i> <i>until her immune system reboots.</i> - So far, I’m doing good. <i>HAYLEE: My parents have to go to work,</i> <i>so I’m going to be the one</i> <i>staying with her at the hospital.</i> <i>HELEN: I literally told my mom, I was like,</i> <i>we can have parties in the hospital now.</i> - I’m telling you, I don’t remember nothing. SHEILA: Girls, behave. I love you. Good night. HAYLEE: Bye, Mami. <i>All right, love you. SHEILA: Good night.</i> <i>I love you, too.</i> <i>HAYLEE: While Helen waits to get her new cells,</i> <i>I’m gonna hand off the story over to my mom again</i> <i>to explain what happens to them.</i> <i>[upbeat jazzy music]</i> <i>SHEILA: To get Helen’s stem cells ready for gene therapy,</i> <i>She took a drug</i> <i>that forced them into her bloodstream.</i> <i>A machine then separated just the stem cells.</i> <i>Those are the cells that we’re going to be rewriting.</i> <i>DAVID: We’re gonna reeducate the blood cells</i> and make them think that they’re still in the fetus. <i>SHEILA: To do that, scientists use a virus.</i> <i>They put the instructions to flip the hemoglobin switch</i> <i>inside the virus</i> <i>so it can carry it into her cells</i> <i>and drop it off, like a delivery truck.</i> <i>This delivery truck actually comes from HIV.</i> <i>HIV is apparently really good at carrying DNA.</i> <i>They figured out how to use it</i> <i>so there’s no way that it can be transmitted.</i> <i>Once the instructions to flip the hemoglobin switch,</i> <i>it does its work on Helen’s stem cells.</i> <i>They put them in this giant, weird-looking freezer.</i> <i>♪ ♪</i> [music playing on doll] - Hi! [Sheila laughs] <i>HAYLEE: In this cooler is a tiny bag</i> <i>of millions of my sister’s new and improved stem cells.</i> <i>They look like nothing special,</i> <i>but they can change a lot of people’s futures.</i> - This one’s going all the way to her. It’s clamped. SHEILA: Ryan, what are you doing? Come on. Get up. NURSE: Yeah, great. Looks good. SHEILA: Looks good. - Here, this one’s start. This is you. - Ready? - You got it? - Mm-hmm. - Go. - Whoo-hoo! - You did it. - Helen, wake up. SHEILA: [laughs] - Nice job. - Good Job. NURSE: Good job. - Yeah, good job. NURSE: Those are her cells. SHEILA: The foggy stuff is her cells? NURSE: Well, yeah. <i>♪ ♪</i> <i>HAYLEE: Things are about to get so, so hard for Helen.</i> <i>The effects of the chemo haven’t kicked in yet,</i> <i>but they will soon.</i> - From today on, you’re gonna feel a little bit more weak. You know that, right? So once everything starts running through your whole body, like, the good stuff, then you start feeling better again. But it’s a process. - Still sleepy? - Lay down. Why don’t you lay down? - You can sleep, Helen. - Helen, lay down. <i>♪ ♪</i> <i>HAYLEE: This is it.</i> <i>The stem cells are in her body.</i> <i>If they do what doctors are hoping,</i> <i>they’ll reproduce until her blood is filled</i> <i>with fetal hemoglobin.</i> <i>All we can do now is keep her super safe from infections.</i> <i>And wait, and wait...</i> <i>and wait.</i> <i>[light music]</i> - Discrimination that people with sickle cell face: a lot. - You’re not feeling this. It’s all mental. It’s all in your head. - I get called drug seeker a lot. - Asking my doctor for meds, I feel horrible. - My 14-year-old needs morphine. - When I was in the hospital, they called the police. - Do we look like drug seekers? - They held me down and, like, ripped all my tubes out. They thought I was out there to get high. - We know how African Americans are treated in this country. I feel some type of way about it, but... um, I’m not surprised. - Right now, I’m driving to work. I try calling them every— maybe every hour, sometimes every 30 minutes to see what they’re doing. They get aggravated, especially Helen. She goes, “Dad, what do you want? You just called.” We’ll go on and be on, whatever they’re at, whatever they need. My blood. <i>♪ ♪</i> <i>HAYLEE: So Helen got her cells,</i> <i>she did her chemotherapy.</i> <i>She’s really tired.</i> <i>[chatter on TV]</i> [razor buzzing] - Helen, this is for you, baby. [upbeat music playing] [all speaking Spanish] SHEILA: How you feeling? Are you in pain? This procedure’s not all peaches and cream, right? <i>♪ ♪</i> <i>[somber music]</i> <i>♪ ♪</i> - Losing my hair, and, like, not being able to talk ‘cause my throat is really, really hurting. <i>HAYLEE: It feels like long days</i> <i>because we’re not doing nothing.</i> <i>So we’re just waiting until we leave the hospital.</i> There’s food right here, look. You want mac and cheese? Well, you have to eat something. <i>[light music]</i> <i>♪ ♪</i> <i>HELEN: I just wanna get through the process.</i> <i>♪ ♪</i> - My brother Brandon had sickle cell. His heart couldn’t take it, and it stopped on him at age 27. I’m 26. You don’t even know if you really have a future, so you don’t really plan out a life. - Emotionally, it feels... like it’s the end of the world. - It makes you stay distant. You don’t wanna get attached. <i>PATIENT: You’re sitting there and you’re like,</i> this is always gonna be my life. What’s the point of it? <i>♪ ♪</i> - Gene therapy—I’m so hopeful, but how much does it cost? Will it be like “Hunger Games“? [laughs] You know? Do the rich get cured, or is it for everyone? - It’s not very much to hold on to right now. As much as you wanna believe in everything that’s coming, you just need to see it at this point. <i>♪ ♪</i> SHEILA: We have to go. HAYLEE: Wait, so we’re leaving right now? SHEILA: Why does it matter if we leave right now, in two minutes, or in two hours? HAYLEE: No, I’m not leaving in two hours because I’m tired of this place. Ma, look, I got a pineapple one, too. SHEILA: A what? <i>HAYLEE: It’s way too early</i> <i>to tell if the gene therapy worked yet,</i> <i>but Helen is finally strong enough to bring home.</i> NURSE: We’re ready? SHEILA: We’re so ready. NURSE: Perfect. Get excited, on the move. SHEILA: Whoo-hoo! [“This Girl is on Fire” playing on speaker] ♪ ♪ [all cheering] [cheering, clapping] ALICIA: ♪ This girl is on fire ♪ [cheering] <i>SHEILA: Haylee chose “Girl on Fire”</i> <i>for Helen to walk out of the hospital.</i> <i>“Girl on Fire” is like liberty,</i> <i>I can do this, I’ve done it.</i> <i>It’s a new beginning. It’s a new start.</i> [chatter on TV] - When I was leaving the hospital... I don’t know—I was listening to the lyrics, and it’s like... They said, “You’re on top of the world.” [inhales deeply] - Did it make you feel sad? That’s why? Why? - [whimpers] [Helen crying] <i>SHEILA: I remember her telling me</i> <i>that she had, like, a fear</i> <i>that I just did all this, and it doesn’t work.</i> I—I know her pretty well. I know that’s what’s going through her head. “Did I just do all this for nothing?” - Say, “No more sickle cell,” baby, okay? No more pain, no more nothing. The red one is the sickle cell, remember? SHEILA: Good-bye. <i>[light upbeat music]</i> <i>♪ ♪</i> <i>ERICA: Helen is now three months</i> <i>after her gene therapy.</i> <i>We’re seeing more fetal hemoglobin</i> <i>than she was making before, which was the goal.</i> <i>Between the fetal hemoglobin and sickle hemoglobin,</i> <i>it’s like half and half, which is awesome.</i> Because before, she only had a couple percent of fetal hemoglobin. SHEILA: Yeah. That’s awesome, look! ERICA: Yeah. SHEILA: Helen! ERICA: Anything hurting? - Mm-hmm. - ERICA: Any pain in your arms, head, chest, hips? - Nope. ERICA: Nowhere. Hooray. [laughter] SHEILA: She’s doing good. - She’s kind of a pioneer. <i>HELEN: I was scared. I didn’t wanna do it</i> <i>‘cause I didn’t wanna be, like,</i> <i>the first person to go through it.</i> <i>But then I kinda got over—</i> <i>I was like, I’m gonna be better.</i> <i>I’m gonna have a better life.</i> <i>HAYLEE: So, here we are.</i> <i>I’ve watched my sister do things</i> <i>I never thought she’ll do.</i> [upbeat traditional music playing] <i>♪ ♪</i> <i>SHEILA: She’s thriving. FATHER: She’s free.</i> No more suffering. That’s an extremely happy feeling. <i>HAYLEE: Her doctors are being cautious,</i> <i>but it looks like the treatment is working.</i> - Do it! <i>HAYLEE: Boston Children’s Hospital</i> <i>will study my sister for the next 15 years</i> <i>to find out. </i>BYRON: Blow, blow, blow! <i>HAYLEE: I always knew Helen was special.</i> <i>I mean, she’s related to me, so obviously.</i> <i>But flowing through her veins right now</i> <i>could be the key to helping</i> <i>lots of sick people get better.</i> <i>My little sister’s blood is a pretty big deal.</i> <i>♪ ♪</i>