Gene editing saves a human life for the first time

For the first time in history, a life has been saved by gene editing. After all conventional treatments failed to provide positive results, 1-year-old Layla and her family believed the girl would die from leukemia. Her mother, Lisa, said:

%shareLinks-quote="We didn't want to give up on our daughter, though, so we asked the doctors to try anything." type="quote" author="Lisa" authordesc="" isquoteoftheday=%

With the permission of Layla's parents, doctors tested an experimental form of gene therapy on the 1-year-old by using genetically engineered immune cells from a donor. Within one month, all of the cancerous cells in Layla's bone marrow had been killed off.

While it's still too soon to say that the young girl is cured, she seems to be doing well. There are currently no signs of the cancer returning. Now, thanks to Layla's outcome, other patients are receiving the same treatment.

Layla was first diagnosed with acute lymphoblastic leukemia when she was 3 months old. She underwent chemotherapy followed by a bone marrow transplant, but neither treatment cured the young girl. Layla's parents Ashleigh and Lisa were told that nothing more could be done to save their child. It wasn't until the parents insisted that the doctors try something else that Waseem Qasim, the man developing a form of gene therapy to treat cancer, got involved.

Because Layla was too small and sick, she didn't have enough T-cells left to modify. However, Qasim's team had been developing "off-the-shelf" treatments in which donated T-cells are modified and given to patients. To prevent Layla's body rejecting and attacking the foreign T-cells, Qasim's team used gene editing to disable a gene in the donor cells that recognizes other cells as foreign.

When Qasim was contacted by Layla's doctors, he had only ever tested his engineered T-cells on mice. Layla's father Ashleigh said:

%shareLinks-quote="It was scary to think the treatment had never been used in a human before, but there was no doubt we wanted to try the treatment. She was sick and in lots of pain, so we had to do something. " type="quote" author="Ashleigh" authordesc="" isquoteoftheday=%

After three months, Layla received a second bone marrow transplant to restore her immune system. After the healthy immune cells recognized the foreign cells, the foreign cells were destroyed. Therefore, Layla no longer has genetically modified cells in her body. Qasim said:

%shareLinks-quote="It's incredibly encouraging. There are a whole bunch of other disorders we can now create fixes for." type="quote" author="Waseem Qasim" authordesc="" isquoteoftheday=%

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