The Food and Drug Administration on Wednesday announced what the agency calls a "historic action" — the first approval of a cell-based gene therapy in the United States.
The FDA approved Kymriah,
which scientists refer to as a "living drug" because it involves using
genetically modified immune cells from patients to attack their cancer.
The drug was approved to treat children and young adults up to age 25 suffering from a form of acute lymphoblastic leukemia who do not respond to standard treatment or have suffered relapses.
disease is a cancer of blood and bone marrow that is the most common
childhood cancer in the United States. About 3,100 patients who are 20
and younger are diagnosed with ALL each year.