First ever cell-based gene therapy approved by FDA

This is pretty neat. This is not a pure diabetes news – but it has a strong impact. Yesterday, the FDA approved the first ever cell-based gene therapy treatment in the US, in this case for a form of juvenile leukemia (acute lymphoblastic leukemia, in children and young adults up to 25):

“The FDA approved Kymriah, which scientists refer to as a “living drug” because it involves using genetically modified immune cells from patients to attack their cancer. […] The treatment involves removing immune system cells known as T cells from each patient and genetically modifying the cells in the laboratory to attack and kill leukemia cells. The genetically modified cells are then infused back into patients. It’s also known as CAR-T cell therapy.”

The reason while I think this is pretty cool: this time, the application is not diabetes – but an upcoming one could be: genetically modified immune cell therapy is now a weapon against design. Pretty darn great!

I feel like I am living in my own future :slight_smile:

On second thought, I now realize that we all are, from the second after we were born and on.


This opens the door to many future cell-based gene therapy, so definitely good news.

The therapy has proven in a small trial to be very helpul >80% success rate on patients who failed the first line treatment. But the price is a little eye watering, and it is as always a bit galling that the US taxpayers spent a bunch in the R&D of it, and don’t seem to get any benefit to this spend, economically speaking.

Selected quotes form the article:

Novartis, which developed the drug, says the one-time treatment will cost $475,000 for patients who respond.

“Let’s remember, American taxpayers invested over $200 million in CAR-T’s discovery.”