Leukemia Treatment Breakthrough

After several rounds of chemotherapy and a bone marrow transplant, doctors at the Great Ormond Street Hospital (GOSH) explained that there were no treatment options left for Layla, and offered palliative care.“We didn’t want to accept palliative care,” said Lisa, in a press release. “So we asked the doctors to try anything for our daughter, even if it hadn’t been tried before.”
Recent developments had been made in the lab regarding a treatment where immune cells (T-cells) are programmed through gene therapy to identify and kill cancerous cells. Oftentimes, though, leukemia patients do not have enough health T-cells to collect in the first place, meaning doctors must use modified T-cells from donors. A team at GOSH and the UCL ICH, along with scientists at the University College London and biotech company Cellectis, had been developing a bank of donor T-cells (called UCART19 cells) to be used in the final stage of testing before clinical trials could begin. When Qasim heard of Layla’s case, it was these cells that came to mind.
“The approach was looking incredibly successful in laboratory studies, and so when I heard there were no options left for treating this child’s disease, I thought, ‘Why don’t we use the new UCART19 cells?’” Qasim explained. “The treatment was highly experimental and we had to get special permissions, but she appeared ideally suited for this type of approach.”
After some time, doctors were confident the leukemia cells were gone. They gave Layla a bone marrow transplant to replace her entire blood and immune system, which had been wiped out from the treatments. She is now recovering at home, though she’ll return to GOSH for regular checkups on her bone marrow cells and blood count.
Though Layla’s case was a huge success, doctors warn about making assumptions about the treatment.