CRISPR gene-editing treated her sickle cell disease and she's still thriving : Shots - Health News A young Mississippi woman is thriving two years after getting treated for sickle cell disease with the revolutionary gene-editing technique known as CRISPR.

First sickle cell patient treated with CRISPR gene-editing still thriving

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SARAH MCCAMMON, HOST:

Two years ago, we met Victoria Gray. She's a pioneer in the new age of genetic medicine. Gray became the first person with sickle cell disease to be treated with the revolutionary gene-editing technique called CRISPR. NPR health correspondent Rob Stein has had exclusive access to follow Gray's story, and he brings us this update.

ROB STEIN, BYLINE: Last time I spoke with Victoria Gray, the news couldn't be better. Gone were the sudden attacks of horrible pain. Gone was the devastating fatigue that had left her helpless to care for herself or her kids. Gone were the nightmarish nights in the emergency room getting blood transfusions and powerful pain meds. But it had been more than a year since we last spoke, so I was holding my breath when I called her just before Christmas that she was still OK. After all, one big question was, would getting her blood cells genetically modified keep working?

So how are you?

VICTORIA GRAY: I'm doing great.

STEIN: You are?

GRAY: Yes, I am.

STEIN: Oh, that's great. So, well, what's going on?

GRAY: Well, it's close to the holidays, so right now just been Christmas shopping for the kids and family.

STEIN: And her health - any signs of sickle cell, the genetic blood disorder that had plagued her all her life?

GRAY: Oh, my health been doing great. You know, I haven't had any problems with sickle cell at all. I did get a cold about a week ago, but other than that, everything is fine.

STEIN: Victoria is still so traumatized by a life of sickle cell that just getting a cold still terrifies her. A simple cold had been one of many things that could trigger a pain attack.

GRAY: Like having PTSD, you know, I was scared. Like, OK, is this going to make me sick? Am I going to end up in the hospital from this? So you get the worry when things come up that you know usually triggers a sickle cell crisis, you know, the weather change. But I did fine, so I'm good.

STEIN: More than good, actually - way more.

GRAY: This is major for me and my family. You know, two years without me being in the hospital - wow. We just can't believe it. But we're so grateful.

STEIN: She's been doing so well for so long that she's officially no longer in the original study she volunteered for. Doctors took cells out of her bone marrow, edited a gene in their lab, and infused the modified cells back into her body to give her healthy red blood cells. And it's not just Victoria, who's now 36 and lives in Forest, Miss. Doctors have now treated at least 45 patients with sickle cell and a related condition known as beta thalassemia. Dr. Haydar Frangoul at the Sarah Cannon Research Institute in Nashville runs the study.

HAYDAR FRANGOUL: It really changes patients' lives. You see the change almost immediately after patients leave the hospital, when they are feeling better and able to resume their life normally without the horrible complications that can happen from sickle cell disease.

STEIN: It's gone so well, the companies developing the treatment hope to seek Food and Drug Administration approval sometime in the next 18 to 24 months.

FRANGOUL: This is very exciting for all of us.

STEIN: Like millions of other families, it still hasn't been an easy year for Victoria. She lost an aunt and a cousin to COVID-19, and she's still hunkering down with her family to protect themselves. But she's been doing so well she's been doing things she never thought possible, like work full time as a cashier at a Walmart, start thinking about going back to school, play with the family's new puppy, Rocky.

GRAY: It's been great. I'm finally at a point now to where I feel normal just doing mom things, housewife things. It's the simple things, like running around the yard with my dog and taking a bath on my own, taking a shower, finishing laundry in one day.

STEIN: Wow, that must be amazing.

GRAY: Yes, it is. I finally get to live a normal life and be happy. It's unbelievable, but God did something great for me, and I'm happy about it.

STEIN: Doctors will continue to follow Victoria and other volunteers in the research for 15 years to make sure the revolutionary treatment she received keeps working safely. Rob Stein, NPR News.

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