Opening a new era in cancer care, the Food and Drug Administration on Wednesday approved the first treatment that genetically engineers patients' own blood cells into an army of assassins to seek and destroy childhood leukemia. The CAR-T cell treatment developed by Novartis and the University of Pennsylvania is the first type of gene therapy to hit the U.S. market — and one in a powerful but expensive wave of custom-made "living drugs" being tested against blood cancers and some other tumors, too. "This is a brand new way of treating cancer," said Dr. Stephan Grupp of Children's Hospital of Philadelphia, who treated the first child with CAR-T cell therapy — a girl who'd been near death but now is cancer-free for five years and counting. Made from scratch for every patient, it's one of a wave of "living drugs" under development to fight additional blood cancers and other tumors, too. "We´re entering a new frontier in medical innovation with the ability to reprogram a patient´s own cells to attack a deadly cancer," U.S. Food and Drug Administration Commissioner Scott Gottlieb said in a statement. The FDA approved Kymriah for use in pediatric ALL patients and is expected to approve a CAR T-cell therapy for treatment of DLBCL and ALL in adults being developed by Kite Pharma Inc.

Sam Tinaglia, 18, of Park Ridge was one of the first 100 children in the country to participate in clinical trials of CAR T-cell therapy for acute lymphoblastic leukemia. In a clinical trial of 63 children and young adults with a type of acute lymphoblastic leukemia, 83 percent of patients that received the CAR-T therapy had their cancers go into remission within three months.
The Food and Drug Administration has approved the first treatment that genetically engineers patients' own blood cells to destroy acute lymphoblastic leukemia. The approval makes Novartis the proprietor of the first gene therapy to hit the U.S. market.