Revolutionary gene therapy approved
US officials have approved the first treatment that genetically engineers patients’ own blood cells to seek and destroy a childhood leukaemia.
The CAR-T cell treatment developed by Novartis and the University of Pennsylvania is the first type of gene therapy to hit the US market — and one in a powerful but expensive wave of custom-made “living drugs” being tested against blood cancers and some other tumours.
The Food and Drug Administration 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 had been close to death, but now is cancer-free for five years and counting. That’s enormously exciting.”
CAR-T treatment uses gene therapy techniques not to fix disease-causing genes, but to “turbo-charge” T cells, immune system “soldiers” that cancer can often evade. Researchers filter the cells from a patient’s blood, reprogramme them with a “chimeric antigen receptor” that targets cancer, and grow hundreds of millions of copies.