Novartis’ CAR-T gene therapy, the first approved by FDA, to cost $475,000, by Emma Court.

Novartis AG’s CAR-T cell therapy was approved by the Food and Drug Administration on Wednesday, making it the first gene therapy to be available in the U.S. …

Novartis’ … Kymriah was approved for young people up to age 25 with a form of acute lymphoblastic leukemia (ALL). …

CAR-T, or chimeric antigen receptor T-cell therapy, uses a patient’s immune T-cells and re-engineers them to better fight cancer.

As such, each dose of Kymriah is customized to the individual patient’s T-cells through genetic modification.

Commenter C Taxpayer:

I was following the news on the clinical trials of this one. Basically, they took a HIV virus, modified it so that it (A) can’t replicate, (B) doesn’t do anything HIV normally does, (C) modifies the white blood cells to attack specific antigens (those on the leukemia cells), and (D) modifies them to replicate in the blood stream. They pull out blood, expose the white blood cells in that blood to this virus, then inject it back in the patient.

The effectiveness of this therapy is insane. Nearly complete remission rates in the terminal patients they did their phase 1 trials on. The main problem is basically you are turning your entire immune system against the cancer cells in your body. If you have extensive cancer, your body goes from ignoring this to all out war. So, you can actually die from the severe immune response your body has. (although they mitigate this risk with drugs) They called this part of the therapy “shake and bake” because you run a massive fever, shake, and you basically feel like dog doodoo for a few weeks. …

Good news though, if this therapy works, they can use it to target ANY condition that has specific antigens which can be zero’d in on. (so virtually all cancers can be cured with it, but each variant needs to be researched and approved one at a time, so this could take decades as there are 100’s of variants of cancer).

hat-tip Matthew