Toddler’s ‘Incurable’ Cancer Cured With New Gene-Editing Treatment

At 14 weeks old, a little girl named Layla was diagnosed with Infant Acute Lymphoblastic Leukemia (ALL) and rushed to Great Ormond Street Hospital (GOSH) in London. The very aggressive cancer returned even after chemotherapy and a bone marrow transplant. Shortly after Layla’s first birthday, her parents were told nothing more could be done but to give their daughter palliative care, which is a fancy term for, “Keep your family member as comfortable as possible in their final days.” Layla’s mother, Lisa, said, “We didn’t want to accept palliative care and so we asked the doctors to try anything for our daughter, even if it hadn’t been tried before.”

Waseem Qasim of the University College London heard of Layla’s case and suggested something that indeed hadn’t been tried before in humans. It’s called UCART19 cells, a modified form of T-cells (immune cells). The treatment is similar to one which already exists, where a patient’s own T-cells are gene edited to attack cancer cells. For patients like Layla, however, there aren’t enough T-cells to collect and engineer. This is where the new UCART19 cells come in. They’re made from donor T-cells, and — until Layla — they had only been tested on mice.

Making matters even more touch and go, there was only one vial of the treatment ready, and the hospital had to convene an emergency ethics committee meeting to discuss if they could even go ahead with treatment. In the end, the parents consented and the single vial of treatment was given through Layla’s Hickman Line.

[Layla’s father] Ashleigh says: “We thought that the little bit of liquid in the syringe was nothing, and asked ‘what is that going to do when bags and bags of chemo haven’t worked.’ The nurse said this was about quality and not quantity though.” (Via)

And quality it was. Just weeks later, Layla appeared to be cancer free and was given another bone marrow transplant. She’s now recovering at home with her family, and receives regular checkups to confirm she’s still cancer free. Professor Qasim says “we have to be cautious about claiming that this will be a suitable treatment option for all children,” but it could be a huge step forward if the results can be replicated in other patients. Full clinical trails will start early next year.

(Via Great Ormond Street Hospital and Ars Technica)