US Discovers Breakthrough Gene Therapy for Childhood Leukemia
US health officials approved a breakthrough treatment that can genetically engineer patients’ own blood cells into an army that destroys childhood leukemia
The FDA named this approval historic, the first gene therapy to hit the US market. The special thing about it is that it is made from scratch for every patient.
Novartis Pharmaceuticals set the price for one-time infusion of the “CAR-T cells” at $475,000, but mentioned that there will be no charge for patients who don’t show response within a month.
“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.”
CAR-T treatment uses gene therapy techniques to turbocharge T cells. Researchers filter them 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. When returned to the patient, the cells can continue multiplying to fight disease for months, and even years.
It is a completely different method to harness the immune system than common drugs called “checkpoint inhibitors” that treat a variety of cancers by simply helping the T cells better spot tumors.
“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.