Landmark gene therapy approved
In a major milestone in the fight against cancer, the Food and Drug Administration (FDA) last week approved a new treatment that genetically alters patients’ immune cells to make them seek out and destroy childhood leukemia. The landmark decision marks the first time gene therapy involving “living drugs” has been cleared for use in the U.S., reports The New York Times. It came after a study in which 83_percent of critically ill patien ts went into remission within three months of having the treatment. Marketed as Kymriah, the gene therapy was approved for children and adults up to age 25 with B-cell acute lymphoblastic leukemia, the most common childhood cancer in the U.S. It will be available to the 20_percent of patients who either don’t respond to standard treatment or relapse after initial therapy. Only about 600 people across the country will currently be eligible, but the one-time treatment is already being tested for a range of other diseases, including non-Hodgkin’s lymphoma and multiple myeloma. “We’re entering a new frontier in medical innovation,” says FDA Commissioner Scott Gottlieb. “New technologies such as gene and cell therapies hold out the potential to transform medicine.” Kymriah will cost $475,000— less than a bone marrow transplant, which can also cure some forms of leukemia.