As the first patient to receive an experimental treatment that relied on the gene-editing technique CRISPR continues to do well 17 months later, more patients seem to be benefiting, too.



Doctors are now more confident than ever that an experimental gene editing treatment is working. This raises new hopes for millions of people. For the past year and a half now, NPR health correspondent Rob Stein has been following the story of Victoria Gray. She lives in Forest, Miss. She was born with a blood disorder called sickle cell disease, and she is the first patient to get this groundbreaking treatment in the United States. Rob has an update.

ROB STEIN, BYLINE: All her life, Victoria Gray dreamed of flying, but she'd always been afraid to get on a plane because she was so sick.

VICTORIA GRAY: It was one of the things that I was waiting to get a chance to do (laughter).

STEIN: So despite the pandemic, Victoria got on a plane for the first time this summer to go see her husband, who's been deployed with the National Guard in Washington, D.C., for months.

GRAY: It was exciting. I had a window seat, and I got to look out the window and see the clouds and everything.

STEIN: She wore a mask the whole time to protect herself from the coronavirus, kept her distance from other people at the airport and arrived happily in Washington, even though she's afraid of heights.

GRAY: I didn't hyperventilate like I thought I would (laughter).

STEIN: It's just one of the many new things that Victoria's been able to do since we last spoke back in the spring.

GRAY: It's amazing. You know, it's better than I could have imagined, feel like I can do what I want now (laughter).

STEIN: Victoria was born with sickle cell disease. It's a devastating genetic disorder that turns red blood cells into deformed sickle-shaped cells that cause sudden attacks of torturous pain and all kinds of other awful, debilitating, often eventually fatal complications. Victoria, who's 35, worried she may never live to see her kids grow up.

GRAY: Not being able to feed myself, not being able to take baths or comb my hair, just totally dependent on someone else to care for me. It was just - it was horrible. It was terrible.

STEIN: So she volunteered last year to let doctors use the gene-editing technique called CRISPR to edit a gene in her bone marrow cells to try to give her normal, healthy red blood cells. And more than a year and a half later, all of the terrible complications of her disease are gone - no more awful pain, no more transfusions, terrifying emergencies in the hospital.

GRAY: Oh (laughter), this is really a life-changer for me. I can't even describe it, Rob. It's magnificent.

STEIN: And now, it looks like Victoria is no fluke. This CRISPR gene-editing treatment seems to be safe and working for at least two other people with sickle cell, as well as seven more people with a similar blood disorder called beta thalassemia. The results are so encouraging that nine others have now also been treated in the United States, Europe and Canada with more coming.

HAYDAR FRANGOUL: It is a big deal because we were able to prove that we can edit human cells, and we can infuse them safely into patients. And it totally changed their life.

STEIN: Dr. Haydar Frangoul at the Sarah Cannon Research Institute in Nashville is running the study Victoria's in.

FRANGOUL: It is opening the door for us to show that this therapy can not only be used in sickle cell and thalassemia, but potentially can be used in other disorders. We are terribly excited.

STEIN: Now, it hasn't been an easy year and a half for Victoria, far from it. Like millions of other Americans, she's been stuck at home struggling to keep her kids safe, helping them do school on their computers. But because she's feeling so much better today than even six months ago, she's been able to handle it at all, even with her husband away, and gotten to do other things she's never done before, like watch her oldest son's football games, see her daughter cheerleading.

GRAY: And taking care of my kids and being there for them - you know, I was - I would take care of them the best that I could. And I would try to do things. But before they would ask me to do something for them, they like, Mama, how are you feeling today? Mama, are you sick? And they don't do that anymore, you know?

STEIN: Now, doctors will have to follow Victoria and many other patients for years to be sure the treatment with CRISPR is safe and that it keeps working. Victoria hopes it will.

GRAY: Oh (laughter), this is really the greatest (ph) life-changer for me, and I pray that it be the same for others. It was my light of hope.

STEIN: She's looking forward to learning to swim, traveling more when the pandemic finally ends and watching her children grow up without worrying about their mother dying. But for now, she's content, hunkering down, playing board games with her family and getting ready to spend Christmas with her kids instead of alone in some hospital.

Rob Stein, NPR News. Transcript provided by NPR, Copyright NPR.