In August of 2017, the Food and Drug Administration made history by approving the first gene therapy in the United States. The treatment, known as CAR-T immunotherapy, may help young patients suffering from acute lymphoblastic leukemia (ALL), a bone marrow and blood cancer.
“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, M.D. “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. At the FDA, we’re committed to helping expedite the development and review of groundbreaking treatments that have the potential to be life-saving.”
The treatment uses a patient’s own T cells to target and attack cancerous cells (a T cell is a type of white blood cell that identifies and triggers responses to invading pathogens). The T cells are collected from the patient’s body and altered in a lab to include new genes that are capable of identifying cancerous cells. Six months later, 30% of patients treated were in complete response, and 74% of those remained in remission after they were treated. This treatment has huge implications for the future of gene therapies; scientists may now be able to create a targeted approach to formerly incurable diseases.