WASHINGTON — Opening a new era in cancer care, the Food and Drug Administration on Wednesday approved the first treatment that genetically engineers patients’ own blood cells into an army of assassins to seek and destroy childhood leukemia.
The CAR-T cell treatment developed by Novartis Pharmaceuticals and the University of Pennsylvania is the first type of gene therapy to hit the U.S. market – and one in a powerful but expensive wave of custom-made “living drugs” being tested against blood cancers and some other tumors, too.
“This is a brand new way of treating cancer,” said Dr. Stephan Grupp of Children’s Hospital of Philadelphia, who treated the first child with CAR-T cell therapy – a girl who’d been near death but now is cancer-free for five years.
Novartis said it would charge $475,000 for the treatment, made from scratch for every patient. But, the company said there would be no charge if the patient didn’t show a response within a month.
CAR-T treatment uses gene therapy techniques not to fix disease-causing genes but to turbocharge T cells, immune system soldiers that cancer too often can evade. Researchers filter those cells from a patient’s blood, reprogram them to harbor a “chimeric antigen receptor” that zeroes in on cancer, and grow hundreds of millions of copies. Returned to the patient, the revved-up cells can continue multiplying.
“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.
This first use of CAR-T therapy is aimed at patients desperately ill with acute lymphoblastic leukemia that strikes more than 3,000 children and young adults in the U.S. each year. While most survive, about 15 percent relapse.
In a key study of 63 advanced patients, 83 percent went into remission. It’s not clear how long that benefit lasts.
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