The U.S. Food and Drug Administration today approved Yescarta (axicabtagene ciloleucel), a cell-based gene therapy, to treat adult patients with certain types of large B-cell lymphoma who have not responded to or who have relapsed after at least two other kinds of treatment. Yescarta, a chimeric antigen receptor (CAR) T cell therapy, is the second gene therapy approved by the FDA and the first for certain types of non-Hodgkin lymphoma (NHL). FDA Commissioner Scott Gottlieb, himself a cancer survivor, called Yescarta "another milestone in the development of a whole new scientific paradigm." Yescarta is approved for use in adult patients with large B-cell lymphoma after at least two other kinds of treatment failed, including DLBCL, primary mediastinal large B-cell lymphoma, high grade B-cell lymphoma and DLBCL arising from follicular lymphoma. What actually worked for the 55-year-old photographer was a radical treatment in which his own white blood cells were genetically re-engineered to kill his cancer. More impressively, 51% went into complete remission, meaning their cancer was undetectable. The severe fever and neurological side effects led the FDA to give the treatment a boxed warning, its most severe. That doesn't include the costs of dealing Yescarta's side effects, which include life-threatening fevers triggered by the immune response the therapy creates that can mean weeks in the hospital, or other drugs patients must receive.
– The Food and Drug Administration has approved a second "living drug" to fight cancer—a personalized treatment that alters a patient's own cells to turn them into cancer-killers. The new "CAR T-cell therapy" has been approved for non-Hodkins lymphoma patients with few other treatment options, a group that could number around 3,500 per year, the Washington Post reports. But the innovative therapy won't come cheap: Forbes reports that the cost will be $373,000 per patient, not including the cost of treating side effects that could include dangerous fevers. A similar treatment approved for leukemia in July costs $475,000 per patient. Doctors believe the new treatment, produced by Kite Pharma under the brand name Yescarta, could save thousands of lives, the New York Times reports. In the study that led to FDA approval, 54% of 101 patients went into complete remission, and 39% of the 101 were still in remission an average of 8.7 months later. "Today marks another milestone in the development of a whole new scientific paradigm for the treatment of serious diseases," FDA Commissioner Scott Gottlieb said in a statement. "In just several decades, gene therapy has gone from being a promising concept to a practical solution to deadly and largely untreatable forms of cancer." (The fight against breast cancer is also making progress.)