Short Wave - A Revolutionary Experiment To Edit Human Genes

Episode Date: November 4, 2019

Victoria Gray has sickle cell disease, a painful and debilitating genetic condition that affects millions of people around the world. But an experimental gene-editing technique known as CRISPR could help her — and, if it does, change the way many genetic diseases are treated. Correspondent Rob Stein tells her story, an NPR-exclusive, and explains the science behind her treatment. Follow host Maddie Sofia on Twitter @maddie_sofia. Email the show at [e-mail]. See pcm.adswizz.com for information about our collection and use of personal data for sponsorship and to manage your podcast sponsorship preferences.NPR Privacy Policy

You're listening to Shortwave from NPR. Maddie Safaya here with NPR health correspondent Rob Stein. How's it going, Rob? Going great. I'm really excited to be on your cool new podcast, Maddie. Welcome to Shortwave, Rob. Thank you. So, Rob, today you're bringing us a story about a medical experiment that a lot of people in the scientific world are paying really close attention to.

Yeah, you know, it's arguably the hottest medical study in decades. And NPR got exclusive access to follow the very first patient. through this pioneering experiment. Can I just say how cool that is, Rob? Yeah, it took a lot of work, and we're really excited about it. Rob, you're very cool. Thanks. Well, shucks.

You know, the woman at the center of this experiment is Victoria Gray. Victoria's in room seven. I met Victoria over the summer at the Sarah Cannon Research Institute. It's in Nashville. Hello. I'm Rob Stein from NPR. Victoria Gray. Nice to meet y'all.

Victoria, she's 34. Her husband's in the National Guard. She's a stay-at-home mom. My twins, Jaden and Jada Dajja, they are 11. Jamarius is 12, and my stepdaughter Asia is 13. Wow, you've got your hands full. I do. It's the right time to get healed.

The right time to get healed. The right time to get healed. Right. Victoria, she has sickle cell disease, and it's a terrible genetic disease that's the most common among African Americans in the United States. Right, and the disease messes up your red blood cells. Exactly.

Instead of normal red blood cells, you know, the cells that carry oxygen in your body, sickle cell patients have hard, sticky, sickle-shaped red blood cells. They clog up the bloodstream and cause all kinds of awful health problems, including agonizing bouts of pain. Sometimes it feels like lightning strikes in my chest and real sharp pains all over. Sometimes I would be just bawled up and crying, not able to do anything for myself. That sounds just awful.

Yes, it is. You know, in those sickle cells, they've already damaged Victoria's heart, and they've made her really weak and prone to infections. And, you know, like a lot of people with sick of cell disease, Victoria, she had to quit work, she had to drop out of school. And she knows that many sickle cell patients, they don't make it past their 40s. It's scary, and it affected my oldest son, you know, because he's older, so he understands. So he started acting out in school.

And like his teacher told me, I believe Jamarious is acting out because he really believes you're going to die. Oh, Rob, this is breaking my heart a little bit. Yeah, it's a horrible situation. And not just for Victoria, but for literally millions of people around the world. But now there's hope because of the reason Victoria is in Nashville. She's the very first person in the United States to undergo treatment for a genetic, disease using CRISPR. And CRISPR is basically a way for doctors to edit the genes in human cells and in turn make Victoria's own cells into super cells. Do you remind me again why you called

them that? Because they're, you know, they go from the cells that make sickle cell to super cells, the cells that help me be better. And Maddie, if Victoria does get better, other people could too. So today on the show, Victoria Gray, the first U.S. patient to be treated with CRISPR for a genetic disease. It's a groundbreaking experiment that could fundamentally change how we treat all kinds of diseases. Okay, so Rob, we'll get to the science behind CRISPR in a second. First, you've been following Victoria Gray for months. Yeah. And you were with her recently for a really special moment.

Right. This was about a month ago. Victoria spent the summer in Nashville getting her treatment, getting billions of the genetically modified cells infused into her body and then being closely monitored. And I caught up with her when she was finally ready to go home. I'm excited, you know. I'm very excited about it. I know it's going to be emotional for me. I missed the hugs and the kisses and just everything.

It must be so excited. Yes, I ain't. Yeah, so, you know, it was three months of recovering from a grueling procedure, blood tests, exams, and really close monitoring by doctors. And, you know, Victoria, she realized she might have over-packed a little bit. Like, oh, my goodness, did I really bring all this? But before leaving, she had to stop by the TriStar Centennial Medical Center in Nashville one more time. So how have you been doing?

I'm doing good. Yeah, this is a big moment, friend. That's her doctor, Dr. Haider-Frangoull at the Sarah Cannon Research Institute. Are you excited about seeing the kids? Yes, I am. A big welcome sign for you in Mississippi? No, because it's a surprise. No one knows I'm coming.

You did not tell them? Oh, that's sweet. She didn't tell me she's coming home. Big surprise. Oh, wow. So they don't know you are coming? I'm just going to show up tomorrow. Like mom's home.

Yeah, so Victoria, she'll come back to Nashville once a month for checkups and blood tests. Okay, let's check you out. To see if those supercells are producing something called fetal hemoglobin. Fetal hemoglobin. Now, fetal hemoglobin. Did I just say it wrong? No. No, no, no, you're good.

Okay. It's just my favorite type of hemoglobin. Yes. You know what I mean? So, yeah, so fetal hemoglobin, it's a protein that fetus is making the womb, but babies stop producing shortly after they're born. Victoria's supercells, they were edited to make them start producing fetal hemoglobin again. And the idea is that will hopefully give her healthy red blood cells.

We are very hopeful that this will work for Victoria, but we don't know that yet. She is continuously monitored after she leaves Nashville today. So doctors have asked. So doctors have asked Victoria to keep detailed diaries about her health, you know, how much pain she's having, how much pain medications she needs, how many blood transfusions she needs. So she, in my mind, is a pioneer in this. We are very excited.

This is a big moment for Victoria and for this pivotal trial. Because if we can show that this therapy is safe and effective, it can potentially change the lives of many patients over years. Okay, Rob, I think now is a great time to try and explain what CRISPR actually is. Okay. So the first thing is, scientists didn't just, like, make this up. They stole it. They stole it from, you're not going to believe this, from microbes, like bacteria. Right, because it's one of the ways some bacteria defend themselves from viruses,

kind of like their own version of an immune system. Because just like humans, bacteria can be infected by viruses. And after those viruses attack, little bits of the viral DNA get saved, in the bacterial cells. So they can kind of remember that virus later. Right. So the next time the virus attacks those cells, the bacteria are like, uh-oh, I know this DNA, bad, bad, bad. And the bacteria literally slices up the DNA of the virus destroying it.

Scientists figured out how to harness this power and pretty much adapt it to zero in on any part of the genetic code and make very precise changes. It's a little more complicated than this, but basically cutting out the DNA they don't want, and in some cases, swapping in DNA that they do want. Exactly. Some researchers refer to this as molecular scissors, little scissors go snip-tipped in the DNA. And it's enormously powerful. It literally enables scientists to rewrite the genetic code.

But that's raised some concerns, too, right? Absolutely. CRISPR has generated a lot of controversy. And mostly, that's about scientists trying to use CRISPR to make changes in DNA in ways that can be passed down for generations, like editing genes and humans. human sperm, human eggs, or human embryos. Right. And there's that scientist in China who did that. Exactly. He created the world's first genetically modified babies, twin girls, and that conjured up all kinds of scary, brave new world scenarios about using genetic engineering to create some kind of

super race or something. But the reasons doctors and scientists are really excited about CRISPR technology is his potential to treat a lot of diseases, like sickle cell. But there are some safety concerns, right? Absolutely. You know, whenever scientists try to do that. try something new and something as powerful as this, it always raises fears that something could go wrong. You know, the early days of gene therapy were scarred by major setbacks, such as the case of Jesse Gelsinger. He was a teenager who died after suffering an adverse reaction to an experimental gene therapy. It's a big concern about CRISPR is that the editing could go off target, could go awry and cause unintended changes in the DNA that could cause health problems on their own. And that's why

Dr. Frank Gould will be watching Victoria really carefully for years. My hope and dream for Victoria is for me to allow her to spend quality time with her family, go to the park with them, play with them without having to struggle with pain and weakness. And she can see her not only her children's graduation from high school, but from colleges and getting married and having grandkids. And Victoria, you know, she hopes so too. and not just for herself, but she realizes that this could have implications far beyond her own life. Oh, my God, I can't imagine, you know, the lives that could be saved if this thing actually works.

That would be amazing. Yes, it would be. So what's amazing about Victoria is that she's the first, but scientists hope not the last. And back in Nashville, I watched as some nurses drew Victoria's blood for this study she's in. it's being conducted by two biotech companies and will eventually involve dozens of patients in the United States, Canada, and Europe. All right, so how many days have you been here? I think we're in like day 80. Oh, I can't believe you're really leaving.

And as these nurses, you know, Bonnie Carroll and Brooke Ryan fill one big vile after another, I realized I never asked Victoria something. Is it weird to have genetically modified cells in your body? No, I'm just genetically modified now. I'm a GMO, is that what they call it? She's hilarious. Yeah, Victoria, she's great. And it was really sweet to see her say goodbye to these nurses who've been taking care of her now for months. Good job. You did it.

Thank you, y'all. Y'all going to make me cry. So how long until we know if it's working? So Dr. Frangle is monitoring Victoria, first of all, to make sure there aren't any immediate side effects from the treatment itself. Sure. Yeah. And he's also testing her to see if her superiors. supercells are making fetal hemoglobin, but it'll take months to see if any fetal hemoglobin in her cells may

actually be making her healthier and years to see if that translates into a longer life for Victoria. And it'll keep watching her for many, many more years to make sure there are no long-term side effects from the treatment itself. And one of the last things I asked Victoria was about this waiting game, this limbo period that she's in right now. Well, I'm spiritual. I'm something and I prayed about, and just the way everything happens for me, I just feel like it was fate for me to be here. So I just believe that God is doing this for me. So there is a lot of ethical and regulatory stuff that will have to come along with CRISPR. Yeah, absolutely.

Big questions that should be asked and answered about who gets to use it, who has access to it, and how much it's going to cost. Absolutely. But it is kind of amazing to think about, like, if this treatment's successful, there could be a time in the future where these kinds of debilitating genetic diseases can just be edited out. Yeah, yeah. I mean, that's the great hope behind all this. And it's, you know, it's a really exciting time in science, not only because of CRISPR, but because of a whole new generation of gene editing techniques that are coming along that could be even more powerful than CRISPR. And they could enable scientists to re-engineer the DNA in all kinds of living things.

All right, Rob Stein, health correspondent on the NPR Science Desk. Thank you so much. Oh, sure, Maddie. No problem. And I also want to thank Victoria for really opening up and letting us follow her along through this amazing journey. I'm Mattis Sophia. Thanks for listening to Shortwave from NPR. We'll see you tomorrow.