美国国家公共电台 NPR After A Life Of Painful Sickle Cell Disease, A Patient Hopes Gene-Editing Can Help（在线收听）

STEVE INSKEEP, HOST:

OK. The first person to get treated in one of the most closely watched medical experiments in decades is now waiting - waiting to learn if the treatment is working. NPR health correspondent Rob Stein introduced us to Victoria Gray this summer. And now he continues his exclusive look at her story.

(SOUNDBITE OF KNOCKING)

ROB STEIN, BYLINE: Hi. Good to see you.

VICTORIA GRAY: Good to see you, too.

STEIN: I catch up with Victoria Gray in an apartment in Nashville near the hospital where she volunteered for the first attempt to use the revolutionary gene-editing technique called CRISPR to treat a genetic disease in the United States.

Thank you so much for seeing us and letting us come over. I really appreciate it.

GRAY: No problem.

STEIN: Victoria's packing. She's finally going home tomorrow to see her four kids in Mississippi for the first time in months.

GRAY: I'm excited, you know. I'm very excited about it. I know it's going to be emotional for me. I miss the hugs and the kisses and - just everything.

STEIN: You must be so excited.

GRAY: Yes, I am (laughter). Yes, I am.

(SOUNDBITE OF ZIPPER UNZIPPING)

STEIN: She unzips a suitcase and starts pulling clothes from the closet.

GRAY: Oh, my goodness. Did I really bring all of this?

STEIN: Victoria has sickle cell disease, a terrible genetic disorder that turns her red blood cells into deformed sickle-shaped cells that clog her bloodstream, ravage her body and torture her with agonizing bouts of pain.

GRAY: The pain is excruciating. It's like being in a car accident and having lightning in your chest. It's pain that makes a grown woman like me scream.

STEIN: Oh, my God. That just sounds terrible.

GRAY: Yeah. It's a overwhelming pain.

STEIN: Like many sickle cell patients, Victoria had to drop out of school, quit work, spend weeks in the hospital away from her kids, worry whether she'll live to see them grow up.

GRAY: It's horrible.

STEIN: You have to live with that every day.

GRAY: Yes - knowing that I could have a stroke or a heart attack at any time because I have these cells in me that are misshapen. Who wouldn't worry?

STEIN: But now Victoria has hope because doctors infused billions of her own cells back into her body this summer, cells Victoria calls her...

GRAY: Super cells.

STEIN: ...Because scientists altered a gene in the cells with CRISPR, which makes rewriting DNA really easy, to hopefully give Victoria normal, healthy red blood cells.

GRAY: Super cells. Yes. Yes. They got to be super, do great things in my body, you know, and to help me be better, you know, and help me have more time with my kids and my family.

(SOUNDBITE OF ZIPPER ZIPPING)

GRAY: That's one.

STEIN: First suitcase done?

GRAY: Yes (laughter).

STEIN: But before Victoria can finish packing and go home, she has to stop by the TriStar Centennial Medical Center in Nashville again.

HAYDAR FRANGOUL: So how have you been doing?

GRAY: I'm doing good.

FRANGOUL: So, yeah, this is a big moment, friend.

GRAY: Yes.

STEIN: Dr. Haydar Frangoul at the Sarah Cannon Research Institute treated Victoria.

FRANGOUL: Are you excited about seeing the kids?

GRAY: Yes, I am.

FRANGOUL: Are they going to have a big welcome sign for you in Mississippi?

GRAY: No because it's a surprise.

FRANGOUL: Oh, you didn't tell them?

GRAY: No one knows I'm coming.

FRANGOUL: You did not tell them?

GRAY: No.

FRANGOUL: Oh, wow. So they don't know you are coming?

GRAY: No. No. No. I'm just going to show up tomorrow, like, Momma's home (laughter).

FRANGOUL: That's pretty good. OK. Let's check you out.

GRAY: OK.

FRANGOUL: Take a deep breath for me.

STEIN: Dr. Frangoul says Victoria will come back once a month for checkups and blood tests to see if her super cells are producing something called fetal hemoglobin.

FRANGOUL: We are very hopeful that this will work for Victoria, but we don't know that yet.

STEIN: So Victoria will also keep detailed diaries about her health, how much pain she's having, how much pain medication she needs, blood transfusions.

FRANGOUL: Victoria 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.

STEIN: Dr. Frangoul will also watch over Victoria for years to see if the treatment is preventing organ damage, heart attacks, strokes and other complications that takes the lives of sickle cell patients and for any signs of any side effects from the treatment.

FRANGOUL: My hope and dream for Victoria is 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. I would love Victoria to be able to live a healthy, normal life. And she can see her - not only her children's graduation from high school but from colleges and getting married and having grandkids.

STEIN: Victoria hope so, too.

GRAY: Not just for me, but for other people, this would be mind-blowing. And, oh, my God, I can't imagine, you know, the lives that could be saved if this thing actually worked.

STEIN: That would be amazing.

GRAY: Yes, it would be.

(SOUNDBITE OF CURTAINS OPENING)

STEIN: Now it's time for the nurses to get some of Victoria's blood for the 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.

UNIDENTIFIED PERSON: All right. So how many days have you been here?

GRAY: I think we're in, like, day 80.

UNIDENTIFIED PERSON: Oh, I can't believe you're really leaving.

STEIN: As nurses Bonnie Carroll and Brooke Ryan fill one big vial after another, I realize I never asked Victoria something.

Is it weird to have genetically-modified cells in your body?

GRAY: No. I'm just genetically-modified now (laughter). I'm a GMO. Is that what they call it?

STEIN: Finally the nurses are done. And it's time to say goodbye for now.

UNIDENTIFIED PERSON: Good job. You did it.

GRAY: Thank y'all. Y'all going to make me cry.

(LAUGHTER)

UNIDENTIFIED PERSON: No (laughter). You're fine.

STEIN: But before I say goodbye to Victoria, I asked her about what comes next - this limbo period, waiting to find out if her super cells are working.

GRAY: Well, I'm spiritual, Rob. And it's something I prayed about. And just the way everything happened for me, I just feel like it was fate for me to be here. I just believe that, you know, God is doing this for me. You know, that's all I can say. So yes, pray (laughter).

STEIN: Rob Stein, NPR News, Nashville.

(SOUNDBITE OF ARMS AND SLEEPERS' "WHEN THE BODY")