For the first time ever, experimental gene editing technology has been used to treat a baby with a fatal condition. Just don’t mess with the embryos. This episode was produced by Victoria Chamberlin, edited by Miranda Kennedy, fact checked by Avishay Artsy, engineered by Patrick Boyd and Andrea Kristinsdottir and hosted by Sean Rameswaram. Listen to Today, Explained ad-free by becoming a Vox Member: vox.com/members. Transcript at vox.com/today-explained-podcast. Baby KJ as he prepares to leave the hospital. Photo courtesy of Children’s Hospital of Philadelphia. Help us plan for the future of Today, Explained by filling out a brief survey: voxmedia.com/survey. Thank you! Learn more about your ad choices. Visit podcastchoices.com/adchoices
Chapter 1: What is the story of baby KJ?
It's a big week for baby KJ. After spending nearly his entire first year of life in the Children's Hospital of Philadelphia, he is going home. Baby KJ is not like your average baby. He was diagnosed with a rare genetic disease shortly after birth, something that roughly one in a million babies have. But baby KJ got a genetic treatment for it that no baby has ever had. And it worked.
He's had quite a nice little growth spurt. I like to think it's really helped him grow some nice chubby cheeks.
Man, the day he walks into like school with a book bag on and we like let him go at the door, like, I might have to take the day off that day.
The miracle of baby KJ coming up on Today Explained.
KJ, buddy, what you doing down there? What are you doing? KJ!
Hey, everyone. It's Nilay Patel, editor-in-chief of The Verge and host of Decoder, my show about big ideas and other problems. We have a special exclusive episode for you that we're really excited about. It's an interview with Google CEO Sundar Pichai. I sat down with Sundar during the Google I.O.
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There are fewer than 1,000 billionaires in the U.S. Why do they matter so much?
The solution is not to talk about wealth as a target. It's to talk about unfairness and corruption and self-dealing.
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Chapter 2: What rare condition does baby KJ have?
They're told that he's quite healthy and we're going to put him in the NICU for now, but, you know, he'll be back with you very soon. And then basically within 48 hours, a nurse pulls Kyle aside, the father, and pulls up KJ's arm and drops it down. And instead of flopping, as you would expect a baby's arm or anyone's arm, really, it's to do, it kind of shutters down.
And what they find is that his ammonia levels are in the thousands, when it should be, you know, like 10 or 20. And this is very dangerous.
This toxin, ammonia, builds up in your blood and then eventually will build up in your brain. If that went on unchecked for a day to two days, the patient would be at very high risk of death.
One of the doctors came to us and said, we think we know what's wrong. Your son is very sick. But the best place in the world for your child to be when he's very sick is next door.
So they rushed KJ across the street, basically, to the Children's Hospital of Philadelphia from UPenn Hospital. And they immediately put him on medicines to bring down that ammonia, put him on a strict diet. And they sequenced his genome. And they say, okay, what exactly is the issue here? And they find that he has a mutation in this one gene.
But what they realize is that this is actually a mutation that might be editable, that we might be able to make a gene editing treatment for.
We either have to get a liver transplant or give him this medicine that's never been given to anybody before, right? I mean, what an impossible decision to make. I just think that we felt like this was the best possible scenario for a life that... At one point, we didn't know if he would be able to have.
For the last couple years, they had been basically preparing for a baby like KJ. Because there had been all these advancements in gene editing over the last decade. Many of your listeners probably have heard vaguely of CRISPR. There's one drug already approved for sickle cell. There's more in the works.
But the advancements had come to the point where you could make these really fine-tuned changes in DNA. And that... both creates some opportunities and some challenges. And the opportunity is there you can like treat as, you know, happened with KJ ultimately.
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Chapter 3: How did gene editing help baby KJ?
You can look back at, like, publications and how people talked in the 80s or 90s when they kind of thought they were on the threshold of being able to, you know, replace genes and do things like this, and they weren't, and it didn't work. And now you hear you have a baby like KJ, and we don't yet know what his life will hold.
We don't yet know exactly how well this works, but, like, the early signs are very promising, and that is... It is incredible, and it comes out of all of this gritty, biochemical, unsexy work and all this funding over all these years that have now produced this.
Fairly soon, if all goes well, all six of us will all be able to be at home, sit on the couch, watch a movie. We're planning for him to come home.
Are other people getting in line? I mean, so this is the big question. And it's sort of the more pessimistic, like the easy thing to do, sort of like if you're skeptical, is to look at this and be like, amazing, exciting. Love that this happened for KJ. Researchers pulled off something incredible. Not repeatable. You can't do it again. It's not going to work. And they have a really valid point.
The researchers involved here won't say how much it costs. It definitely costs in the millions of dollars. There's no one pulling up that money to make that at scale for thousands of infants or even hundreds of infants or even dozens of infants. This was supported in part by the NIH, who helped do some of the manufacturing for the monkey studies, if I recall correctly.
And the NIH is currently facing massive funding cuts. And so where is the money, where is the process going to come to do this at scale? Also, I should add, like, you can only kind of—you can only do this— Basically with a couple different types of conditions. You can do it with conditions that affect the liver.
You can do it, it's more complicated, but you can do it with conditions that affect the blood. Pretty much anything else, researchers are not yet at a place where they can reliably do this kind of gene editing work. And so for now, this won't be a one-off, but there won't be that many patients who benefit from it in the next few years, probably not. Yeah.
Is there anyone who's saying, hold the line? Maybe this isn't the best idea? Is there anyone pushing back?
There's someone who's like, no, they should not have treated KJ. But there are, there will be, and there have to be discussions around what is the best use of our resources? Should we be doing these N of 1 cases versus trying to find ways of treating large swaths of KJ?
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Chapter 4: What was the process of developing KJ's treatment?
Whether you're an aspiring comedian or just curious about the money side of making people laugh, this episode is packed with insights you won't want to miss. Listen wherever you get your podcasts or watch on youtube.com slash yourrichbff.
Good job.
Scientists used CRISPR technology to edit the genes of baby KJ to save his life. But let us not forget that we have also, as a human race, used CRISPR to edit the genes of an embryo.
A scientist in China in 2018 manipulated embryos that were then taken from the lab, put into a woman in the hope of establishing a pregnancy. And it turns out that at least two pregnancies were established.
Two beautiful little Chinese girls named Lulu and Nana came crying into the world as healthy as any other babies a few weeks ago.
One couple gave birth to twins in 2018, and then a third child was born in 2019. Those children are born of genetically modified embryos, which means that they are in fact the first genome-edited children, and they get referred to as the CRISPR babies. During IVF, a technology called CRISPR was used on embryos, disabling a particular gene that allows HIV to enter a cell.
CRISPR is the technology, just an acronym that stands for Clustered Regularly Interspaced Short Palindromic Repeats. And you can appreciate why nobody would want to say that out loud. And so they are our CRISPR babies.
Their genes were reportedly manipulated to see if we could create humans who could be resistant to HIV, smallpox, and cholera. That seems good, right? But there was a lot of debate. We invited bioethicist Francoise Baylis to tell us why.
In a way, to tell the story accurately, one needs to appreciate and know that the initial response, which was largely on social media in China following this announcement, was really quite positive.
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Chapter 5: What challenges exist for gene editing treatments?
And then once it is in a woman's body and is continuing to develop, we have all kinds of other technologies we use to scrutinize the development and to see whether or not we think we're going to have a healthy birth. And so it's in this context that we now start imagining Well, if this isn't on the right track, what do we do?
Well, could we identify embryos that have a problem before we actually transfer them? And then you'd only transfer the healthy embryos. And we make that transition in the 1990s.
So we go from being able to look at the embryo, thinking about transferring the embryo in a healthy context, to getting to a point where we can take the embryo and look at it and make a judgment about its quality and then decide to transfer or not. And now getting to the stage where we're thinking, well, while we're looking at this thing in the lab, why don't we just tinker with it?
And there's some risks to doing that tinkering.
Well, there's always risks to doing tinkering because the important thing about research is it's a step into the unknown. We don't actually know what we're going to do, what we're going to find. And that's true whether we're talking about patients enrolled in a clinical trial or whether we're talking about biological material in a laboratory.
So I think we need to appreciate that when we don't know, we don't know the good things or the potentially harmful things. And I think that's the context in which many people are worried about manipulating the human embryo, because if you've made a mistake, that mistake will be visited upon generation after generation after generation.
You're going in, you're cutting the DNA, and you could just mess things up.
Is anyone regulating this process? I mean, thinking about what's going on with the, I don't know, scientific community in the United States right now, funding is being pulled, regulations are being revoked. What's going on in the rest of the world when it comes especially to gene editing?
If you were to look at it globally, I would say to you about half of the world's countries explicitly prohibit this kind of research. No country explicitly permits this kind of research. And there are a number of countries where they haven't bothered to write anything about this research. And just to put that in perspective, I mean, you can think about small islands.
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