On August 30, 2017, the U.S. Food and Drug Administration (FDA) issued what the agency calls a “historic action” by approving the first gene therapy treatment available in the United States.
The new treatment known as Kymriah (tisagenlecleucel) was approved to treat children and young adults up to age 25 suffering from B-cell acute lymphoblastic leukemia (ALL). Kymriah works by removing immune cells called T-cells from a patient. The T-cells are then sent to a laboratory where they are genetically modified to include a gene for a protein called a “chimeric antigen receptor” (CAR), that essentially reprograms a patient’s own cells to attack leukemia cells. The modified T-cells are then infused back into the patient. The treatment has proved to be extremely effective, with an 83% remission rate in clinical trials. Because Kymriah is a complex procedure that works by having the T-cells modified specifically for every patient, there is some concern over the availability and cost of such treatment. Currently, Kymriah has the hefty price tag of $475,000.
Regardless, this new form of gene therapy is no doubt a major accomplishment for the medical field, and it leaves excitement for future medical innovations and treatments. In fact, the FDA is already looking to approve gene therapy for lymphoma later this year.
“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,” Scott Gottlieb, FDA Commissioner, said. “At the FDA, we’re committed to helping expedite the development and review of groundbreaking treatments that have the potential to be life-saving.”