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.
From FDA Approves Groundbreaking Gene Therapy for Cancer on MIT Technology Review:
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.