A breakthrough treatment that genetically engineers patients’ own blood cells into an army of assassins to destroy childhood leukemia has been approved by US officials, opening a new era in cancer care.
The Food and Drug Administration (FDA) called the approval historic, the first gene therapy to hit the US 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 tumours, too.
Novartis Pharmaceuticals set the price for its one-time infusion of so-called “CAR-T cells” at $600,000, but said there would be no charge for patients who didn’t show a response within a month.
Dr Grupp treated the first child with CAR-T cell therapy — a girl named Emily Whitehead who had been near death but has now been free of cancer for five years. “That’s enormously exciting,” he said.
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 harbour a “chimeric antigen receptor” or CAR that zeroes in on cancer, and grow hundreds of millions of copies. Returned to the patient, the revved-up cells can continue multiplying to fight disease for months or years.
It’s a completely different way to harness the immune system than popular immunotherapy drugs called “checkpoint inhibitors” that treat a variety of cancers by helping the body’s natural T cells better spot tumours. CAR-T cell therapy gives patients stronger T cells to do that job.
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