New child cancer drug 'a historic breakthrough' - at €400,000 per dose
US regulators have approved a breakthrough treatment that genetically engineers patients' own blood cells into an army of assassins to seek and destroy childhood leukaemia.
The Food and Drug Administration (FDA) said the approval on Wednesday was historic, the first gene therapy to hit the US market. Made from scratch for every patient with a charge of €400,000 for a single dose, it's one of a wave of "living drugs" under development to fight other blood cancers and tumours.
Swiss company Novartis, which created the drug and employs 450 people in its Irish unit, said there would be no charge for patients who didn't show a response within a month of the one-off infusion of so-called "CAR-T cells".
"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 near death but has now been 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 turbocharge T cells, immune system soldiers that cancer too often can evade.
Researchers filter those cells from a patient's blood, reprogramme 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.
"We're entering a new frontier in medical innovation with the ability to reprogramme a patient's own cells to attack a deadly cancer," said FDA commissioner Scott Gottlieb.
The first CAR-T version, developed by Novartis and the University of Pennsylvania, is approved for use by several hundred patients a year who are desperately ill with acute lymphoblastic leukaemia (ALL).
In a study of 63 advanced patients, 83pc went into remission soon after receiving the CAR-T cells. Importantly, it's not clear how long that benefit lasts: some patients did relapse months later. The others are being tracked to see how they fare long term.
"A far higher percentage of patients go into remission with this therapy than anything else we've seen to date with relapsed leukaemia," said Dr Ted Laetsch of the University of Texas Southwestern Medical Centre, one of the study sites.
Most patients suffered side-effects that can be gruelling, even life-threatening, including high fevers, plummeting blood pressure and, in severe cases, organ damage.
The FDA designated a treatment for those side-effects on Wednesday.