Companies Rush to Develop ‘Utterly Transformative’ Gene Therapies<br />In the next year or so, he said, that approach will also be studied in both children<br />and adults who have acute myeloid leukemia, which he described as a “tough disease.”<br />Researchers at the University of Texas MD Anderson Cancer Center in Houston are trying a completely different approach to engineering cells, one<br />that they hope might eventually yield an “off the shelf” treatment that would not have to be tailored to each individual patient and that might be less expensive.<br />“This has been utterly transformative in blood cancers,” said Dr. Stephan Grupp, director of the cancer immunotherapy program at the<br />Children’s Hospital of Philadelphia, a professor of pediatrics at the University of Pennsylvania and a leader of major studies.<br />The new leukemia treatment involves removing millions of white blood cells called T cells — often referred to as the soldiers of the immune system — from the patient’s bloodstream, genetically engineering them to recognize<br />and kill cancer, multiplying them and then infusing them back into the patient.<br />There will be many such studies, Dr. Grupp predicted, but, he said, “It’s early days.”<br />The T cells in the Novartis products, and in the earliest ones its competitors are developing, have been engineered to seek and destroy cells<br />that display on their surfaces a protein called CD19 — a characteristic of many leukemias and lymphomas.<br />Another target is being studied, and Dr. Grupp said the next step, which he called<br />“superimportant,” would be to attack two cellular targets in the same patient.
