I came across this article and figured I'd pass it along. Pretty amazing results! Price tag is a bit steep though...
FDA Approves Groundbreaking Gene Therapy for Cancer
A revolutionary cancer therapy that uses genetically engineered immune cells has been approved by the U.S. Food and Drug Administration, ushering in a new era of cancer treatment. 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.
“We’re entering a new frontier in medical innovation with the ability to reprogram a patient’s own cells to attack a deadly cancer,” said FDA commissioner Scott Gottlieb in a statement. 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. In a clinical trial of 63 children and young adults with a type of acute lymphoblastic leukemia, 83 percent of patients that received the CAR-T therapy had their cancers go into remission within three months. At six months, 89 percent of patients who received the therapy were still living, and at 12 months, 79 percent had survived.
David Mitchell, founder of an advocacy group called Patients for Affordable Drugs, said in a statement that the $475,000 cost is “excessive” and claims the federal government spent $200 million in early research on CAR-T therapy before Novartis purchased rights to the treatment. The group recently met with the company to appeal for a “fair” price for its therapy. Previous estimates predicted a price tag between $600,000 to $750,000.
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