US clears first ‘living drug’ for tough childhood leukemia
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 on the U.S. market, and one in a powerful but expensive wave of custom-made “living drugs” being tested against blood cancers and other tumors, too. The FDA called the approval historic.
“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 CART cell therapy, a girl near death but who 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.