Food and Drug Administration approved the first immunotherapy treatment in the United States.
Tisagenlecleucel, which will be marketed as Kymriah by Novartis Pharmaceuticals, is aimed at the treatment of children and young adults who have acute lymphoblastic leukemia (ALL).
FDA says tisagenlecleucel is a genetically modified T-cell immunotherapy. Each dose is a customized treatment that’s created by using an individual’s T-cells, which are a type of white blood cell that’s known as a lymphocyte. A patient’s T-cells are collected and sent to a manufacturing center, where they are modified to include a new gene that contains a specific protein that directs the T-cells to kill leukemia cells that have a specific antigen on their surface. After they’re modified, FDA says, the T-cells are infused back into the patient to kill the cancer cells. (Consumers Digest wrote about the arrival of immunotherapies in its report, “Medical Breakthroughs & New Frontiers 2015.”)
FDA says tisagenlecleucel is approved for patients up to age 25 and who have B-cell precursor ALL that resists treatment or has relapsed a second or later time. A clinical study of tisagenlecleucel among 63 patients showed an 83 percent remission rate within 3 months of the treatment, FDA says.
ALL is the most common childhood cancer in the United States, FDA says, and B-cell ALL is the most common form of the cancer. National Cancer Institute estimates that about 3,100 patients who are age 20 and under are diagnosed with ALL each year.
Unfortunately, FDA notes that treatment has the potential to cause severe side effects. It carries a boxed warning for cytokine release syndrome, which can cause high fever and flu-like symptoms, and neurological events. Both side effects can be life-threatening, FDA says. Other severe side effects include serious infections, low blood pressure, acute kidney injury and decreased oxygen. Most symptoms appear within 22 days after the patient is infused with the treatment.