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Gene Editing Reverses Aggressive Leukemia In Baby

Update Date: Nov 09, 2015 10:18 AM EST

Layla Richards, a baby hit by leukemia, became the first person in the world to benefit from a gene editing technique. Currently, she is in remission, Reuters  reported.

At first, she was diagnosed with infant acute lymphoblastic leukemia (ALL), which her doctors called "one of the most aggressive forms of the disease" they had ever diagnosed.

Layla was immediately subjected to chemotherapy and treated with a bone marrow transplant to replenish her blood cells.

But the doctors at Great Ormond Street Hospital (GOSH) soon had no treatment options and suggested that she could benefit from palliative care.

"Doctors don't want to say that there's nothing we can do and offer palliative care, but sometimes that's the only option," Layla's mom, Lisa, said in a news release. "We didn't want to accept palliative care and give up on our daughter though so we asked the doctors to try anything."

Hence, they suggested an experimental treatment, involving the use of T-cells that were modified using molecular "scissors" named Talen. The T-cells were altered and were made to "hide" from the leukemia drug in Layla's body so that they would not be killed, and secondly would fight against leukemia.

This was developed by experts from GOSH, University College London (UCL) and Cellectis, a biotech company.

A few weeks later, she was shown to be improving, and her cancer is now in remission.

"As this was the first time that the treatment had been used, we didn't know if or when it would work and so we were over the moon when it did. Her leukemia was so aggressive that such a response is almost a miracle," Dr. Paul Veys, Layla's lead doctor and director of bone marrow transplant at GOSH said.

Waseem Qasim, a professor of cell and gene therapy at UCL and consultant immunologist at GOSH, however explained that it cannot be concluded that the therapy would help all children.

"But, this is a landmark in the use of new gene engineering technology and the effects for this child have been staggering," Qasim said. "If replicated, it could represent a huge step forward in treating leukemia and other cancers."

The results and details of Layla's case will be presented at the annual meeting of the American Society of Hematology in Orlando.

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