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Home / Health / Doctors in the US use the CRISPR technique for the first time treatment of a genetic disorder: NPR

Doctors in the US use the CRISPR technique for the first time treatment of a genetic disorder: NPR



For the first time, physicians used the gene editing technology CRISPR to treat a genetic disorder in the United States. The patient with sickle cell disease talked to NPR about her treatment.



ARI SHAPIRO, HOST: [19659003] One of the most anticipated medical experiments has begun in Nashville. For the first time, scientists using gene editing technology CRISPR have attempted to treat a genetic disorder in the United States. NPR is the only news organization that has learned the identity of the first patient and talked to her. NPR health correspondent Rob Stein is here with more.

Hey, Rob.

ROB STONE, BYLINE: Hey, Ari.

SHAPIRO: Who is this woman and why is she treated experimentally? [1

9659003] STONE: Her name is Victoria Gray, she is 34 years old and lives with her husband and four children in Forest, Miss. And here's a bit about what she told me about when I met her recently at the Sarah Cannon Research Institute in Nashville.

VICTORIA GRAY: Well, I do not work. I am a mother who stays at home. But before I got too sick, I worked in the beauty department of Walgreens and went to school to become a nurse. But that was put on ice for health reasons.

SHAPIRO: Tell us about her illness. What is your condition?

STONE: Yes, so Victoria has a sickle cell disease and it's a terrible genetic disease that affects mostly African Americans in the United States. And instead of having normal red blood cells – you know, the cells that carry oxygen in your body – sickle cell patients have a hard, sticky – it's a crescent-shaped red blood cell that causes horrible bouts of excruciating pain and many can really cause serious health problems.

GRAY: Well, I have a high risk of stroke and a high risk of heart attack. And these things can happen to me in a moment. And my pain episodes can occur out of the blue. I can only laugh and in the next minute I cry in some of the worst pain you can ever imagine. It's a heavy burden, you know?

SHAPIRO: That sounds really tough. So how do doctors try to help people like Victoria Gray with CRISPR?

STONE: Right, that's the way it works. Doctors – They take cells from the bone marrow of sickle cell patients and use this CRISPR processing tool to process a gene in these cells to turn on the production of so-called fetal hemoglobin, which is normally only produced by fetuses, if they are in the womb and babies for a short time after they were born. And then they infuse billions of these genetically engineered cells back from the patient into their bodies to help treat their disease.

Dr. Haydar Frangoul, who is conducting the study in Nashville, explained a bit more about how this will work.

HAYDAR FRANGOUL: Here we are trying to introduce enough fetal hemoglobin into the red blood cell so that the red blood cells can become happy again, mushy and not sticky and hard, and can give off oxygen wherever it is supposed to.

SHAPIRO: So she's the first sickle cell patient to get this treatment. What does it contain? How was the process?

STEIN: Yes, parts of it were really difficult. She first had to undergo chemotherapy to remove her own bone marrow and make room for these CRISPR-edited cells. And then Dr. infused Frangoul her body with more than 2 billion of these geneditierten cells, and that was only a few weeks ago.

GRAY: They had the cells in a big syringe, and when they went in, my heart rate shot up very high. And that was a bit scary, a tough moment for me. And then I cried, but they were happy tears.

STEIN: How was that feeling?

GRAY: It was amazing. You know, it was kinda overwhelming, after all, what I had been through.

STONE: And Victoria – she calls her new genedited cells – she calls them her supercells.

Why do you call them supercells?

GRAY: Well, I have sickle cells, so it makes supercells when you just replace them with a better S (laugh).

SHAPIRO: Rob, it sounds like there's a lot of potential here. But what are the concerns?

STEIN: Yes, there are always concerns about a new experimental treatment. Is it safe? Will it work? And that's all really magnified with something so new. This is Laurie Zoloth. She is a bioethicist at the University of Chicago. And I talked to her about it.

LAURIE ZOLOTH: I am optimistic about the success of CRISPR. I just want it to be done carefully.

STEIN: Yeah, so they'll monitor you very closely to make sure the processed cells are safe and they do not cause any health problems themselves and then try to get any clues to see if they could work. And researchers plan to examine dozens of patients in medical centers in this country and in Canada in Europe. It could take months, and maybe even years, to know how well it works.

And I talked to Victoria about that. She says she knows the risks and that it's a very early study, but she can not help but hope that it helps her.

GRAY: I feel how everything has been, it was a kind of fate. And I feel special about being the first one to do it.

STONE: And we'll look at Victoria to see how it works.

SHAPIRO: This is Rob Stein from NPR with this exclusive story about CRISPR gene editing technology.

Thank you, Rob.

STEIN: Do you bet, Ari?

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