US regulators have approved Kymriah, the first cancer drug that uses a patient’s own cells to fight cancer, but the price tag of $475,000 has sparked debate. The drug, made by Novartis, treats acute lymphocytic leukemia, the most common childhood cancer in the US.
Kymriah is a one-time intravenous treatment that involves engineering a patient’s T-cells to attack cancer. It is intended for children and young adults who do not respond to standard treatment, which typically pushes 85% of children into remission for five years or longer.
Dr Stephan Grupp of Children’s Hospital of Philadelphia, who led the Novartis study, described the drug as “enormously exciting”. He treated the first patient, a girl near death who is now cancer-free five years later. However, critics argue the price is excessive, with David Mitchell of Patients for Affordable Drugs calling it “astronomical”.
A British study suggested the upper bound for such a drug could be $649,000. Novartis CEO Joseph Jimenez said the price balances patient access with return on investment. The company is collaborating with Medicare on a plan where the government pays only if patients respond within the first month.
The FDA approval followed a study of 63 patients, where 83% went into remission, though some relapsed months later. Side effects include cytokine release syndrome and neurological toxicities. Patients must travel to one of 32 US sites for cell collection.
