FDA Committee Okays Breakthrough Leukemia Treatment
A Food and Drug Administration (FDA) advisory committee unanimously recommended approval of a new "living drug" approach for children and young adults with a common type of leukemia. If approved by the FDA, this treatment will be the first gene therapy okayed by the agency. It’s considered to be one of the most important developments in cancer research in decades.
Called CAR-T cell immunotherapy, the breakthrough treatment approach uses genetically engineered cells from a patient's own immune system to attack cancer cells. It involves taking cells from the patient's body, modifying the genes, and infusing the modified cells back into the patient.
CAR-T cell immunotherapy was developed to treat children and young adults ages 3 to 25 with B-cell acute lymphoblastic leukemia—the most common childhood cancer in the United States—who have relapsed after undergoing standard treatment. Although research results have been promising, resulting in remission in 83 percent of the 88 patients who have received CAR-T so far, some concerns about safety and long-term complications remain.