Gene Editing Treats Leukemia

One-year-old Layla Richards has remained cancer-free months after receiving an experimental gene editing therapy.

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Layla Richards and familyGREAT ORMOND STREET HOSPITALIn June a group of London clinicians obtained special permission to treat Layla Richards, a one-year-old infant diagnosed with leukemia, with an experimental gene editing therapy that had not yet seen clinical trials. The therapy eliminated the diseased cells, and so far Layla remains cancer-free. The scientists will present their case study at the American Society of Hematology’s 2015 meeting during a poster session (December 5).

The treatment represents the first time that gene editing therapy has been used to such a life-saving degree. Layla was diagnosed with acute lymphoblastic leukemia at three months old and didn’t respond to traditional chemotherapy regimens or a bone marrow transplant. “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,” Layla’s mother Lisa Foley said in a press release. The medical team contacted Waseem Qasim, an immunologist at University College London (UCL) who was working on an experimental therapy.

Current therapies in development genetically engineer a patient’s own T cells to target the cancer. But in some cases—like Layla’s—the cancer doesn’t leave enough T cells undamaged to engineer. ...

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