Cell Design Labs, founded by University of California, San Francisco, synthetic biologist Wendell Lim, creates “programs” to install inside T cells, the killer cells of the immune system, giving them new abilities.
Known as “CAR-T,” the treatments are both revolutionary and hugely expensive. A single dose is priced at around $500,000 but often results in a cure. Gilead quickly paid $12 billion to acquire Kite Pharma, maker of one of those treatments.
The initial T cell treatments, however, work only with blood cancers.
The FDA calls the treatment, made by Novartis, the “first gene therapy” in the U.S. The therapy is designed to treat an often-lethal type of blood and bone marrow cancer that affects children and young adults. Known as a CAR-T therapy, the approach has shown remarkable results in patients. The one-time treatment will cost $475,000, but Novartis says there will be no charge if a patient doesn’t respond to the therapy within a month.
The therapy, which will be marketed as Kymriah, is a customized treatment that uses a patient’s own T cells, a type of immune cell. A patient’s T cells are extracted and cryogenically frozen so that they can be transported to Novartis’s manufacturing center in New Jersey. There, the cells are genetically altered to have a new gene that codes for a protein—called a chimeric antigen receptor, or CAR. This protein directs the T cells to target and kill leukemia cells with a specific antigen on their surface. The genetically modified cells are then infused back into the patient.
This is less than the $700,000 previously reported, but still a fortune.