CRISPR For Sickle Cell Disease Shows Promise In Early Test : Shots - Health News Researchers edited the DNA in bone marrow cells taken from a Mississippi woman with sickle cell disease to produce a treatment that could alleviate the excruciating effects of her inherited illness.

Gene-Edited 'Supercells' Make Progress In Fight Against Sickle Cell Disease

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Scientists are reporting the first evidence that one of the most exciting medical experiments in decades appears to be working. Billions of genetically modified cells from a patient with sickle cell disease have started doing what doctors had hoped. NPR health correspondent Rob Stein has been following this patient's journey, which could have implications for millions of patients around the world. Today Rob continues his exclusive coverage.

ROB STEIN, BYLINE: It's a foggy morning, and I'm waiting to meet Victoria Gray in a parking garage in downtown Nashville. Hi, Victoria.


STEIN: How are you doing? So good to see you.

GRAY: Yeah, it's good to see y'all, too.


STEIN: Victoria just drove back to Nashville after spending some time at home with her family in Forest, Miss., for the first time in months.

GRAY: It's been a while.

STEIN: Yeah, it's been a while. So it's an exciting day for you, huh?

GRAY: Yes, it is.

STEIN: How are you feeling?

GRAY: I feel good. I'm hoping to get some good news today.

STEIN: Good news from her doctor about whether the billions of genetically modified cells might - just might - be helping her fight sickle cell disease, which has tormented her all her life.

Are you nervous at all?

GRAY: No, I'm not. No.


GRAY: Uh-uh - just about the needles (laughter).

STEIN: Victoria will have to get a ton of blood tests today.

UNIDENTIFIED PERSON: TriStar Medical Group...

STEIN: She checks in at the Tricentennial (ph) Medical Center in Nashville.


GRAY: Yes, Victoria Gray for Dr. Frangoul.

UNIDENTIFIED PERSON: Could you go ahead and sign in for me?


STEIN: As we're waiting, I notice Victoria's wearing a black sweatshirt with big letters spelling out warrior.

I like your sweatshirt.

GRAY: Thank you. Yeah. You know, they call sickle cell patient warriors. And I saw this shirt at Walmart, so I had to get it (laughter).

STEIN: I did not know they called sickle cell patients warriors.

GRAY: Yes. Because it's a constant battle, you have to be to deal with it.

STEIN: Sickle cell is a cruel genetic disorder that deforms red blood cells into defective sickle-shaped cells that jam up her bloodstream, wreak havoc in her body and cause sudden attacks of what some people say is the worst pain imaginable.

GRAY: I had moments where I was standing, laughing, talking with friends. And then the next thing you know, my husband had to carry me into the emergency room because I couldn't use my legs because they were hurting so bad. And when you can't help yourself, it's just one of those feelings that just makes you want to give up. You know?

STEIN: That just sounds unbelievably awful.

GRAY: Yes, it is.

UNIDENTIFIED PERSON: You can go on back now.

STEIN: Victoria finds an empty chair, sits down and stretches out her arm for the nurses.

UNIDENTIFIED NURSE: All right. So you're going to feel a stick on three. OK? One, two, three - stick. All right. So you should be good.

STEIN: As the nurses fill one big tube after another, I asked Victoria about her homecoming a few weeks earlier, when she got to see her four kids for the first time since she spent the summer in Nashville getting treated. It was a grueling procedure.

GRAY: My oldest son, when he did his double take and realized I was in the car, he took off running. And he just grabbed me and held onto me. Yeah. And the twins saw me from the inside the house. They knew it was Mama.

STEIN: That must have been amazing.

GRAY: Yes, it's emotional for me, you know, 'cause I love them so much. I did this for them. Yeah. So it's worth it.

STEIN: The nurses are finally done after filling 16 vials.

UNIDENTIFIED NURSE: OK. All finished. You did great.

GRAY: Thank you.

STEIN: Victoria finally heads over to see her doctor.

HAYDAR FRANGOUL: How are you? How are you? You're doing all right?

STEIN: Dr. Haydar Frangoul is with the Sarah Cannon Research Institute.

FRANGOUL: OK, great. Let's check you out. Take a deep breath for me.

GRAY: (Exhaling).

FRANGOUL: Deep breath.

GRAY: (Exhaling).

FRANGOUL: Good job. Well, you look amazing, Victoria.

GRAY: Thank you.

FRANGOUL: You look really good.

STEIN: After the exam, Dr. Frangoul finally shows Victoria what she's been waiting for - the results of some of her blood tests.

FRANGOUL: So look at this. This is very exciting.

GRAY: Yes.

FRANGOUL: Are you excited about it?

GRAY: Yes, I am.

FRANGOUL: OK. I am super excited about your results today.

STEIN: That's because it seems to be working. Victoria's genetically modified cells have started doing what they're supposed to be doing.

FRANGOUL: It looks like there are signs that you are starting to make fetal hemoglobin, which is very exciting for us.

STEIN: Scientists used the powerful gene editing technique CRISPR to edit the DNA in Victoria's cells to make them produce a protein called fetal hemoglobin. The hope is that would turn Victoria's deformed red blood cells into normal, healthy ones. And so far, nearly half the hemoglobin in her blood is fetal hemoglobin. And it's still rising. But Dr. Frangoul knows he has to be cautious.

FRANGOUL: It is still too early to celebrate. I just want to make sure this is something we watch very carefully every visit and see how things are going.

STEIN: The treatments seem safe so far, and the edited cells look like they may already be helping Victoria. She hasn't needed any blood transfusions since she got the cells, hasn't had any of those awful attacks of pain.

FRANGOUL: And you haven't been in the hospital since I last saw you, correct?

GRAY: No, it's...

FRANGOUL: That's good. Excellent. Perfect.

STEIN: And so is that because of the procedure - because of the cells?

FRANGOUL: Well, we are hoping it is. Again, it is too early to figure it out, but this is extremely encouraging. We are very happy that she didn't have any ER visits. She hasn't had any hospital stays since we've done the procedure. So this is really good.

STEIN: And is that unusual? Would you have expected to usually have a pain episode in this period?

GRAY: Of course. Like, with the time that passed by, I would at least have something. You know?

STEIN: Wow. That sounds amazing.

GRAY: Yeah, it is for me. It's special, especially coming up on the holidays because sometimes I would be in the hospital on Christmas. And so I'm looking forward to a whole new life for all of us.

STEIN: Doctors will have to follow Victoria for years and study more patients to answer the big questions - are the cells really helping patients live healthier lives? Will they keep working? Will they keep working safely? And will they actually help patients live longer?

FRANGOUL: This would be life-changing, not only for Victoria but for many sickle cell patients, because if this is determined to be safe and effective, I think it can be transformative for patients with sickle cell disease.

STEIN: Victoria calls her edited cells her super cells.

So your super cells seem to be working.

GRAY: Yeah, they seem to be super after all (laughter). Yes.

STEIN: Victoria's already doing things she could never do before, like go to one of her son's football games for the first time.

GRAY: You know, the simple things in life - I don't really want anything extravagant. I just want a simple life with my family - you know, people that I love and people they love me - and just live. You know?

STEIN: Rob Stein, NPR News, Nashville.

GRAY: This could be the beginning of something special (laughter).


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