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CRISPR For Sickle Cell Disease Shows Promise In Early Test


To a totally different story now and some exciting news from the world of science. Doctors are reporting the first evidence that genetically edited human cells could safely treat sickle cell disease. That is a common and devastating genetic blood disorder.

NPR health correspondent Rob Stein has had exclusive access to the first patient treated in this experiment here in the U.S. And he's here now with an update. Hi, Rob.

ROB STEIN, BYLINE: Hi. How you doing?

KELLY: I am well. Thank you. So this patient being treated for sickle cell - her name is Victoria. What else do we know?

STEIN: That's right. It's Victoria Gray. And we've been following her through this really closely watched medical experiment, which is testing the revolutionary gene-editing technique called CRISPR to try to treat genetic disorders for the first time. And we finally have the first hints that it might be working for sickle cell.

Now, just to remind people, Victoria - she's 34. She lives in Forest, Miss. She was born with sickle cell, and that turns her red blood cells - the cells that carry oxygen - into deformed sickle-shaped cells that cause excruciating attacks of pain and all kinds of really terrible health problems. And that usually cuts the lives of sickle (inaudible) patients short.

KELLY: OK. So to the news that gene-editing might be helping - I mean, how's it working? How does it help?

STEIN: Yeah. So the idea here is that scientists - they took cells out of Victoria Gray's bone marrow. They edited a gene in the cells to make the cells produce a protein - a protein called fetal hemoglobin. Then they infused billions of these edited cells back into Victoria's body over the summer in the hopes that these cells will make enough fetal hemoglobin to turn Victoria's deformed red blood cells into healthy red blood cells. And scientists are reporting the first signs. It appears like it is working.

KELLY: The first sign. So what exactly are they reporting is happening?

STEIN: Yeah. So I was actually in the exam room at the TriStar Centennial Medical Center in Nashville recently when Victoria met with her doctor - his name is Haydar Frangoul - to find out the latest about whether or not the cells were working. We can listen to a little bit of that.

HAYDAR FRANGOUL: So how are you doing, Victoria?

VICTORIA GRAY: I'm doing good, Dr. Frangoul.

FRANGOUL: You're doing all right? Life is good?

GRAY: Yes.

FRANGOUL: OK. So I'm terribly excited about your blood work today.

GRAY: Great.

KELLY: Great, so terribly excited - but again, what exactly happened?


KELLY: What do these test results show?

STEIN: OK. Now you want to know actually what they show. They show that these genetically modified cells are producing fetal hemoglobin, that protein that doctors hope will help Victoria. In fact, the edited cells look like they're already pumping out more than twice as much of this fetal hemoglobin as they thought they needed to treat sickle cell disease. So that's a first hint that this might be working. And so far, it seems like it's safe.

GRAY: Oh, my goodness.

FRANGOUL: So - no, but I'm very, very proud of you. You are doing really well. Everything looks wonderful, OK?


KELLY: Wow. What a moment to witness there, Rob.

STEIN: Yeah.

KELLY: Were you able to ask Victoria what - how she's feeling, what she thinks about all this?

STEIN: Oh, yeah, for sure. And you know, she's trying to be cautious 'cause she knows this is really early days. But she thinks it might already be helping her. She hasn't had any of those awful attacks of pain that sickle cell can cause since she got the cells. She hasn't needed any blood transfusions. And she's even been able to start to do things she's never been able to do before. Like, she got to go watch her son play football for the first time.

GRAY: It's amazing, you know, to have a chance at a different type of life.

STEIN: And can you believe it?

GRAY: It's a miracle, you know? When you pray and ask God for something for so long, all you have left is hope. And so, yes, I can believe it.

STEIN: And you know, the same treatment may also be starting to help another patient with a related blood disorder known as beta thalassemia.

Now, you know, as I said, the researchers - they're being very careful here to make it clear that this is just the very early sign, very first step. They still don't know the answers to some of the, you know, most important questions. Is the fetal hemoglobin really improving Victoria's health? Will the cells keep working? Will it help her live longer? And will it be safe in the long term?

Here's Dr. Frangoul again from the Sarah Cannon Research Institute.

FRANGOUL: For Victoria, I'm very encouraged just to see her today, how happy she is. I mean, this, to me, means a lot. She is enjoying her family, and she hasn't made any trips to the emergency room or the hospital - is a huge achievement for us.

STEIN: Yeah, it seems life-changing for her, really.

FRANGOUL: Yes, it definitely does, although it isn't too early to celebrate. So we are observing her very carefully to see how she does.

STEIN: So, you know, Dr. Frangoul and other researchers will follow Victoria for - and dozens of other patients, really, for many months and years, really, to see if the edited cells are actually making them healthier, helping them live longer and are safe over the long term.

KELLY: So many questions about whether this will all be practical and affordable going forward...

STEIN: Absolutely.

KELLY: ...But exciting news.

NPR's Rob Stein, thank you.

STEIN: Oh, sure thing.

(SOUNDBITE OF RAJSTAR'S "CANDLELIGHT") Transcript provided by NPR, Copyright NPR.

Rob Stein is a correspondent and senior editor on NPR's science desk.
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