On Wednesday, the Food and Drug Administration approved a revolutionary new type of cancer treatment that genetically modifies the body's own cells. The treatment, known as CAR-T therapy, is the first type of gene therapy approved in the U.S and will be used to attack one of the most common childhood cancers, acute lymphoblastic leukemia.
The treatment first requires researchers to sift T cells from a patient's blood. The T cells, key in producing the body's immune response, are then reprogrammed in the lab with the addition of a receptor, called the CAR (chimeric antigen receptor), that helps T cells better find and fight cancer cells. After the laboratory work, the modified cells are inserted back into the patient, where they get to work.
Ted Laetsch, a doctor at the University of Texas Southwestern Medical Center, said that while the long-term success of the treatment is still being studied, a "far higher percentage of patients go into remission with this therapy than anything else we've seen to date with relapsed leukemia." One study found that 83 percent of advanced patients went into remission after receiving the treatment.
"We're entering a new frontier in medical innovation with the ability to reprogram a patient's own cells to attack a deadly cancer," FDA Commissioner Scott Gottlieb said in a statement. "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."