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

For more than a year, Victoria Gray's life had been transformed. Gone were the sudden attacks of horrible pain that had tortured her all her life. 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 medication.

But one big question was: Would getting her blood cells genetically modified keep working, freeing her from the complications of sickle cell disease that had plagued her since she was a baby?

More than another year later, the answer appears to be: Yes.

"I'm doing great," Gray, now 36, said during a recent interview from her home in Forest, Miss. with NPR, which has had exclusive access to chronicle her experience for more than two years.

"I haven't any problems with sickle cell at all. I did get a cold about a week ago," she says with a nervous chuckle.

Victoria's 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 terrible attack.

"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?,'' she says. "But I did fine."

More than fine, actually. Much more, she says.

"This is major for me and my family," she says. "Two years without me being in the hospital? Wow. We just can't believe it. But we're so grateful."

She's doing so well for so long that she's officially no longer in the landmark study she volunteered for. That involved doctors taking cells out of her bone marrow, and editing a gene in the cells in their lab, using the revolutionary gene-editing technique known as CRISPR. CRISPR allows scientists to make very precise changes in DNA much more easily than ever before. Many think it will revolutionize medicine.

Doctors then infused billions of the modified cells back into Gray's body. The hope was the edited cells would produce a protein known as fetal hemoglobin, alleviating the symptoms of sickle cell.

And it appears to have worked, for Gray and other patients. Doctors have now treated at least 45 patients with sickle cell and a related condition known as beta thalassemia, and reported data indicating it's working for at least 22 of them.

"It really changes patients' lives," says Dr. Haydar Frangoul of the Sarah Cannon Research Institute in Nashville, Tenn., who heads the study. "You see the change almost immediately after patients leave the hospital, where they are feeling better and able to resume their lives normally without the horrible complications that can happen."

Vertex Pharmaceuticals in Boston, which is developing the treatment with CRISPR Therapeutics in Cambridge, hopes to seek Food and Drug Administration approval sometime in the next 18 to 24 months.

"This is very exciting for all of us," Frangoul says.

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 before, such as working full-time as a cashier at a Walmart, planning to go back to school, and playing with the family's new puppy, Rocky.

"I'm finally at a point now to where I feel normal, just doing mom things, housewife things," says Gray, who doctors will follow for 15 years to make sure the treatment keeps working and continues to be safe.

In the meantime, she enjoying "the simple things," she says, like spending time with her husband and children.

"I finally get to live a normal life and be happy. It's unbelievable," she says. "God did something great for me, and I'm happy about it."

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