"We've never seen anything like this before and I believe this therapy may become the new standard of care for this patient population", Dr Stephan Grupp of Children's Hospital of Philadelphia said in a statement. "As a breakthrough immunocellular therapy for children and young adults who desperately need new options, Kymriah truly embodies our mission to discover new ways to improve patient outcomes and the way cancer is treated".
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.
This first use of CAR-T therapy is aimed at patients desperately ill with acute lymphoblastic leukaemia, which strikes more than 3,000 children and young adults in the USA each year.
The National Cancer Institute estimates 3,100 patients are diagnosed each year with ALL, which can come in several forms. Patients may be at increased risk for infections for a prolonged period of time after infusion as the treatment may also destroy normal B cells.
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.More news: Oil prices down as USA reels from Harvey
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"We're entering a new frontier in medical innovation with the ability to reprogram a patient's own cells to attack a deadly cancer", FDA Commissioner Scott Gottlieb said in a news release following the approval. It's these patients for whom Kymriah is intended. They are then reintroduced back into the patient to do battle against the tumor.
The drug has shown promising remission and survival rates in clinical trials, the FDA said. In addition, clinicians involved in the prescribing, dispensing, and administering of the therapy will need training to recognize these side effects. In a trial of Kymriah, 83 percent of 63 patients with hard-to-treat disease were in remission within three months, according to Novartis. The worst is cytokine release syndrome, a common immunotherapy complication that causes potentially life-threatening fever and flu-like symptoms. Kymriah's maker, Novartis, hasn't provided a price for the drug.
Novartis had received a trifecta of designations that helped speed approval: the FDA gave fast-track, breakthrough therapy, and priority review status to tisagenlecleucel's application.
Manufacturing and delivery are more complex in CAR-T therapies than for a typical drug. "It's not a one-shot deal".