The FDA Just Approved a Second Revolutionary Cancer-Killing Gene Therapy
Gilead Sciences’ gene therapy that uses a patient’s own genetically modified immune cells to treat certain types of non-Hodgkin lymphoma was approved by the FDA on Wednesday, Oct. 18.
The treatment, Yescarta, is a form of CAR-T therapy, where some of a patient’s white blood cells are taken out of the body and re-engineered to target and kill cancerous cells only in the body. Once this has been completed they are placed back into the patient, and the T-cells get to work and begin combatting against the cancer. CART-cells are built with proteins where they seek out cancer protein cells in the body that they destroy.
FDA Commissioner Scott Gottlieb, himself a cancer survivor, called Yescarta “another milestone in the development of a whole new scientific paradigm.”
The treatment was approved after a six-month trial of around 100 patients with an aggressive form of diffuse large B-cell lymphoma, a cancer that affects immune cells.
In the recent trial, Yescarta cured 36% of patients completely, and in 82% of patients, their tumours shrivelled down to less than half their original size.
This comes just two months after the FDA approved the first iteration of CAR-T therapy for a form of childhood leukaemia. Novartis’ Kymriah has much higher success rates in clinical trials, curing 83% of patients.
Yescarta also comes at a hefty price: one dose of your own genetically modified cells costs $373,000. Kymriah is even more expensive, costing $475,000 for treatment.
“We’re entering a new frontier in medical innovation with the ability to reprogram a patient’s own cells to attack a deadly cancer,” Scott Gottlieb, MD., FDA Commissioner said in a statement when Kymriah was approved.
“New technologies such as gene and cell therapies hold out the potential to transform medicine and create an inflection point in our ability to treat and even cure many intractable illnesses. At the FDA, we’re committed to helping expedite the development and review of ground-breaking treatments that have the potential to be life-saving.”
However, these therapies, and similar ones in the pipeline raise difficult challenges for policy-makers. “Safety, access and cost-we have to figure all that out if everyone is going to benefit,” said Karen Maschke in a paper published by the Hastings Center.