A one-year-old girl, who once had ‘incurable’ leukemia, is now cancer-free thanks to a British hospital’s use of gene-edited, “designer” immune cells to treat the deadly disease.
Layla was diagnosed with Infant Acute Lymphoblastic Leukemia when she was just 14 weeks old. Following the diagnosis, the child was admitted to London’s Great Ormond Street Hospital intensive care unit. Doctors described Layla’s cancer as “one of the most aggressive forms of the disease we have ever seen.”
The child received several rounds of chemotherapy and was given a bone marrow transplant to replace damaged blood cells. Despite the transplant, the cancer returned about seven weeks later.
Not wanting to give up, doctors told the family of an experimental treatment, which had only been tested on lab mice, was being developed in the hospital. The type of treatment could be used to treat the aggressive form of cancer Layla had.
“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, Layla’s father, Ashleigh Richards, said.”
The experimental treatment uses “molecular scissors” to modify the T-cells from a healthy donor. The hospital said the treatment added new genes to the healthy donor cells. Specific genes were altered to allow the healthy T-cells to become “invisible” to powerful anti-leukemia medication that would normally kill the cells. The altered cells were then reprogrammed to only target and kill the drug resistant leukemia.
Hospital staff, working in conjunction with a biotech company, had to obtain special permission to use the untested treatment on Layla.
“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?’ said Waseem Qasim, the hospital immunologist, said in a statement. “The treatment was highly experimental and we had to get special permissions, but she appeared ideally suited for this type of approach.”
After the cells were delivered through an intravenous line, Layla spent several months in isolation to allow her immune system to recover. The hospital said the child also received a second bone marrow transplant to replace her entire blood and immune system once doctors were confident the cancer cells were gone.
“We have only used this treatment on one very strong little girl, and we have to be cautious about claiming that this will be a suitable treatment option for all children,” Qasim said. “But, this is a landmark in the use of new gene engineering technology and the effects for this child have been staggering.
“If replicated, it could represent a huge step forward in treating leukemia and other cancers.”