Experimental CRISPR Treatment For Sickle Cell Disease Appears Effective : Shots - Health News Since receiving a landmark treatment with the gene-editing tool CRISPR, a sickle cell patient has the strength to care for herself and her children — while navigating the pandemic.

A Year In, 1st Patient To Get Gene Editing For Sickle Cell Disease Is Thriving

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As the country struggles through a deadly pandemic and all the protests happening across the country, a young woman has been sheltering at home in Mississippi with her children like millions of others. But she is not like any other American. Victoria Gray is the first person with a genetic disorder to get treated in the United States with a revolutionary gene-editing technique. NPR health correspondent Rob Stein introduced us to Victoria last year when he got exclusive access to tell her story. Today, we hear how Victoria has been faring.

ROB STEIN, BYLINE: The last time I saw Victoria, her doctors were just finding the first hints her experimental treatment may have started working. But the big question was, was it really working? And was it really making her healthier? Today, it's been almost a year since she got treated. And the news couldn't be better. Almost all the complications of the terrible genetic disease she was born with have essentially disappeared.

VICTORIA GRAY: It's wonderful, you know? It's the change that I've been waiting on my whole life.

STEIN: Victoria's 34. She was born with sickle cell disease, which turns red blood cells, the cells that carry oxygen, into deformed sickle-shaped cells that get jammed inside blood vessels causing horrible attacks of excruciating pain.

GRAY: Before the treatment, it would be so bad it would be crippling.

STEIN: Victoria knew many sickle cell patients die young from the damage the disease wreaks on their bodies. So she volunteered to let scientists infuse billions of her own bone marrow cells that had been modified with a gene-editing technique called CRISPR back into her body to, hopefully, give her healthy red blood cells. And Victoria can hardly believe that it looks like it actually worked.

GRAY: It's a very big deal, Rob. It's a very big deal for me because now I feel like I can just live, you know, and plan for the future.

STEIN: Victoria hasn't had any of those terrible pain attacks since she got her new cells, hasn't needed any blood transfusions, gone off almost all the powerful pain drugs she's needed to get by.

GRAY: I haven't had to be in the hospital or go to any emergency rooms. So I've been able to stay at home with my kids and do more. It's a huge change.

STEIN: A huge change that, in some ways, came just in time. Right after she got home to Mississippi from the hospital where she got treated in Nashville, the National Guard deployed her husband to Washington. And then the pandemic hit. Victoria was suddenly home alone with three of her kids.

GRAY: For one thing I did have to do was become a teacher. So that was hard, trying to navigate education for the kids while they were out and working with the teachers to make sure they didn't get behind and focus in on staying safe.

STEIN: Her great aunt died from COVID-19 early on, so did her pastor. Friends at her church have been getting sick. And even though her immune system is still weak, Victoria has to keep going to buy the family groceries. And then George Floyd was killed by police in Minnesota. At times, Victoria says it feels overwhelming.

GRAY: I feel like everything happened so fast. Like, I was trying to recover. And my husband had to leave. Then the pandemic hit. Then the death of George Floyd and the protests and rioting. It's a lot. So it hasn't been easy.

STEIN: And maybe the hardest part has been trying to help her children understand all this.

GRAY: Having to explain to my kids, you know, about safety just because of their skin color, that's hard. That's been one of the hardest things for me. It's heartbreaking for me. And - I'm sorry, Rob. And it's hard to explain to them. And sometimes you can't find the right words.

STEIN: But if she hadn't had the treatment, Victoria doesn't know how she'd be coping. She would have been too weak to care for her kids, probably ended up in the hospital when hospitals were never scarier.

GRAY: And since my treatment, I've been able to do everything for myself, everything for my kids. So it's been joy, not only for me, but for the people around me that's in my life.

STEIN: Now, Victoria is just one patient. And she'll have to be followed for years to make sure the treatment continues to be safe and keeps helping her. But the fact that the edited cells have now been working so well for so long has made her doctors far more confident that her gene-edited cells are really doing what they'd hoped and will keep working for Victoria and other patients.

GRAY: It's hard to put into words, you know, the joy that I feel and being grateful for a change this big. It's been amazing.

STEIN: Victoria's now hopeful for things she thought she'd never get to see.

GRAY: High school graduations, college graduations, weddings, grandkids - I thought I wouldn't see none of that. I'm sorry. And now I feel like I'll be there to help my daughters pick out their wedding dresses. And we'll be able to take family vacations and that they will have their mom, you know, every step of the way.

STEIN: And Victoria hopes her story will give other people hope, too, not just with sickle cell, but for many diseases.

GRAY: We need this right now more than ever, you know? It's a blessing to be able to know that you can one day be cured from something that you thought you could possibly have for the rest of your life and that there was no hope. It gave me hope when I was losing it. So I feel joy, you know, knowing that there is hope.

STEIN: Rob Stein, NPR News.


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