Breakthrough Therapy That Uses Kids’ Own Immune Cells To Fight Leukemia Approved By FDA
“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. The price tag on the treatment is $475,000.
The Associated Press:
US Clears Breakthrough Gene Therapy For Childhood Leukemia
Opening a new era in cancer care, U.S. health officials on Wednesday approved a breakthrough treatment that genetically engineers patients' own blood cells into an army of assassins to seek and destroy childhood leukemia. The Food and Drug Administration called the approval historic, the first gene therapy to hit the U.S. market. Made from scratch for every patient, it's one of a wave of "living drugs" under development to fight additional blood cancers and other tumors, too. (Neergaard, 8/30)
The New York Times:
F.D.A. Approves First Gene-Altering Leukemia Treatment, Costing $475,000
The new therapy turns a patient’s cells into a “living drug,” and trains them to recognize and attack the disease. It is part of the rapidly growing field of immunotherapy that bolsters the immune system through drugs and other therapies and has, in some cases, led to long remissions and possibly even cures. The therapy, marketed as Kymriah and made by Novartis, was approved for children and young adults for an aggressive type of leukemia — B-cell acute lymphoblastic leukemia — that has resisted standard treatment or relapsed. The F.D.A. called the disease “devastating and deadly” and said the new treatment fills an “unmet need.” (Grady, 8/30)
The Washington Post:
FDA Clears First Gene-Altering Therapy — ‘A Living Drug’ — For Childhood Leukemia
“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. “New technologies such as gene and cell therapies hold out the potential to transform medicine” and cure intractable illnesses. He said companies are pursuing hundreds of experimental treatments involving gene therapy products. (McGinley and Johnson, 8/30)
NPR:
CAR-T Therapy For Leukemia Wins FDA Approval
The treatment, which is also called CTL019, produced remission within three months in 83 percent of 63 pediatric and young adult patients. The patients had failed to respond to standard treatments or had suffered relapses. Based on those results, an FDA advisory panel recommended the approval in July. (Stein, 8/30)