Originally Published August 30, 2017
Today, the U.S. Food and Drug Administration (FDA) issued what it has called a “historic action,” making the first gene therapy available in the United States. The FDA approved tisagenlecleucel (Kymriah) for certain pediatric and young adult patients with a form of acute lymphoblastic leukemia (ALL).
“We’re entering a new frontier in medical innovation with the ability to reprogram a patient’s own cells to attack a deadly cancer,” said FDA Commissioner Scott Gottlieb, MD. “New technologies such as gene and cell therapies hold out the potential to transform medicine and create an inflection point in our ability to treat and even cure many intractable illnesses.”
Tisagenlecleucel, a cell-based gene therapy, is approved in the United States for the treatment of patients up to 25 years of age with B-cell precursor ALL that is refractory or in second or later relapse.
Tisagenlecleucel is a genetically modified autologous T-cell immunotherapy. Each dose of tisagenlecleucel is a customized treatment created using an individual patient’s own T cells. The patient’s T cells are collected and sent to a manufacturing center, where they are genetically modified to include a new gene that contains a specific protein (a chimeric antigen receptor, or CAR) that directs the T cells to target and kill leukemia cells that have a specific antigen (CD19) on the surface. Once the cells are modified, they are infused back into the patient to kill the cancer cells.
