The approval of the first gene therapy marks what likely will be considered the next new major era of drug development in the U.S., according to experts.
The FDA approved the first chimeric antigen receptor T cell (CAR-T) therapy, Kymriah (tisagenlecleucel)—formerly CTL019—for certain pediatric and young adult patients with a form of acute lymphoblastic leukemia (ALL), ushering in a new approach for cancer treatment and other serious and life-threatening diseases. It is approved 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.
According to the American Cancer Society, ALL is a cancer that starts from the early version of white blood cells called lymphocytesin, the bone marrow (the soft inner part of the bones, where new blood cells are made). The disease progresses quickly and is the most common childhood cancer in the U.S. The National Cancer Institute estimates that approximately 3,100 patients aged 20 and younger are diagnosed with ALL each year.
Fighting from within
Kymriah is a novel immunocellular therapy and a one-time treatment that uses a patient's own T cells to fight cancer, according to the manufacturer, Novartis. It is the first therapy based on gene transfer approved by the FDA.
Each dose of Kymriah is customized, using an individual patient’s own T-cells, a type of white blood cell known as a lymphocyte, according to the FDA. 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 that directs the T-cells to target and kill leukemia cells that have a specific antigen on the surface. Once the cells are modified, they are infused back into the patient to kill the cancer cells.
“Personalized therapies like this, that harness the power one’s own immune system—the killing power of the T-cell in particular—represent enormous potential in treating a wide array of cancer types at various stages of progression,” says Managed Healthcare Executive Editorial Advisor David Calabrese, senior vice president, chief pharmacy officer at OptumRx.
“While extremely encouraging and exciting, this excitement must also be properly tapered by an appreciation of the fact that we still have a substantial amount of challenges to overcome and questions to answer in ascertaining the true clinical value of these therapies, particularly in solid tumors, and overcoming the magnitude of toxicity and the unprecedented costs that come along with therapies of this nature,” Calabrese says.