A Food and Drug Administration advisory committee unanimously recommended this week that the agency approve this “living drug” approach for children and young adults who are fighting a common form of leukemia. The treatment consists in taking cells from a patient’s body, modify the genes, and then introducing those modified cells back into the person who has cancer. In a study on humans who had not any other chance, at 25 sites in 11 countries administered the treatment to 88 patients aged 3 to 23. It produced remissions in 83 percent of patients, which is a very high rate of positive response.
Battle of immune cell (in red) against a leukemia cell
The treatment is part of one of the most important developments in cancer research in decades. There are some concerns about its safety over the long term and about its cost but the advisory committee considered this as less important. “This is the most exciting thing I’ve seen in my lifetime,” said Dr. Timothy Cripe, an oncologist at the Nationwide Children’s Hospital in Columbus. The new treatment is known as CAR-T cell immunotherapy and the drug used is known as CTL019 or tisagenlecleucel. It should replace standard treatment for B cell acute lymphoblastic leukemia, which is the most common childhood cancer in the United States.Several family members of children who benefited from the treatment in its experimental phase have made emotional appeals to the committee to recommend approval.