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Baby Beats Aggressive Leukemia Thanks to Experimental Treatment

The Short of It

A baby diagnosed with an aggressive form of leukemia has been given a new lease on life, thanks to an experimental new gene editing treatment that had previously only been performed on mice.

The Lowdown

No parent wants to volunteer their child for an untested treatment, but Ashleigh and Lisa Richards were desperate. At 14 weeks old, their daughter, Layla, was diagnosed with a particularly aggressive form of acute lymphoblastic leukemia (ALL). Powerful doses of chemotherapy, bone marrow transplants and another experimental treatment did little to tame the deadly cancer. Just before Layla's first birthday, talk turned from fighting the disease to palliative, end-of-life care.

Heartbroken, the Richards told their doctors at Great Ormond Street Hospital for Children in London that they were willing to try anything to save their daughter. As luck would have it, researchers at the hospital were working on a new gene-editing treatment called UCART19, which adds new genes to healthy donor T-cells, or immune cells, that are essential to beating leukemia. The catch? So far, the procedure had only been tested on mice, and only one vial of treatment was left.

Ashleigh and Lisa signed on, and final permission was granted after an emergency ethics committee meeting at the hospital. Still, that didn't mean it was smooth sailing for the family.

"It was scary to think that the treatment had never been used in a human before, but even with the risks, there was no doubt that we wanted to try the treatment," Ashleigh said in a press release. "She was sick and in lots of pain, so we had to do something. Doctors explained that even if we could try the treatment, there was no guarantee that it would work, but we prayed it would."

In UCART19, doctors use molecular tools to slice specific genes in such a way that the T-cells, or immune cells, become invisible to the leukemia drug that normally kills them. In addition, the cells are rejiggered so they only target and fight against leukemia cells. Those changed healthy cells are then delivered to the patient intravenously, a process that takes about 10 minutes.

Afterward, Layla was isolated for several months to prevent any infections from attacking her weakened immune system. Meanwhile, five weeks in, doctors confirmed the cancer cells were gone. Ashleigh got the good news first and immediately called Lisa.

"I thought it was bad news," she said, "but then he said 'it's worked,' and I just cried happy tears."

Once she was declared cancer-free, Layla received a bone marrow transplant to replace her entire blood and immune system and was sent home. Doctors still monitor her progress during regular check-ups, and she may need to take medicine to maintain her health. Though the possibility of a relapse is possible, the Richards family is staying cautiously optimistic.

"I consider ourselves lucky that we were in the right place at the right time to get a vial of these cells," Lisa said. "We always said that we had to try new things as we didn't want to be saying 'what if?'"

The Upshot

A cure for cancer? Well, not so fast. While Layla's doctors declared victory here, they're quick to point out that such a risky treatment may not be right for all kids. They're also still keeping a close eye on how their patient is progressing. Still, at this point, I think it's safe to say we can celebrate the success of one little girl and her very gutsy parents. I can't imagine it was easy for them to sign up their 1-year-old for something so risky, but the gamble paid off—big time.

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