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.
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 CAR-T cell therapy — a girl who’d been near death but now is cancer-free for five years and counting. “That’s enormously exciting.”
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 to fight disease for months or years.
Most patients suffered side effects that can be grueling, even life-threatening. An immune overreaction called “cytokine release syndrome” can trigger high fevers, plummeting blood pressure and in severe cases organ damage, requiring special care to tamp down those symptoms without blocking the cancer attack. Also Wednesday, the FDA designated a treatment for those side effects.
Among concerns, sometimes leukemia can develop resistance, and sometimes patients worsen while waiting for their new cells, said Sekeres, who directs the Cleveland Clinic’s leukemia program and wasn’t involved with CAR-T testing.
For some patients, the new CAR-T therapy might replace bone marrow transplants that cost more than half a million dollars, noted Grupp, who led the Novartis study.
Initially, Novartis’ CAR-T version — to be sold under the brand name Kymriah (kihm-REYE’eh) — will be available only through certain medical centers specially trained to handle the sophisticated therapy and its side effects. Patients’ collected immune cells will be frozen and shipped to a Novartis factory in New Jersey that creates each dose, a process the company says should take about three weeks.
While this first use of CAR-T therapy only is aimed at a few hundred U.S. patients a year — relapsed ALL patients up to age 25 — it’s being tested as a treatment for thousands more. Kite Pharma’s similar CAR-T brand, developed by the National Cancer Institute, is expected to win approval later this year to treat aggressive lymphoma, and Juno Therapeutics and other companies are studying their own versions against blood cancers including multiple myeloma.