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First gene therapy to treat childhood leukemia gets FDA approval

By Kristen Jordan Shamus, Detroit Free Press

The U.S. Food and Drug Administration approved on Wednesday the first-ever gene therapy to treat children and young adults with leukemia.

Called Kymriah, but better known as CAR T-cell treatment, the therapy is being hailed by doctors as revolutionary. It involves genetically modifying a patient’s own T-cells, which then can target and kill a form of acute lymphoblastic leukemia cells.

“This new treatment has the potential to change the face of cancer therapy for years to come, not just in childhood acute lymphoblastic leukemia but in other cancers in which a patient’s own T-cells can be collected, genetically modified and redirected to kill a patient’s tumor,” said Dr. Gregory Yanik, clinical director of the Pediatric Blood and Marrow Transplantation Program at the University of Michigan’s C.S. Mott Children’s Hospital. Mott was one of a few hospitals nationally to take part in clinical trials of the treatment.


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