Food and Drug Administration approved a new form of cell-based gene therapy for adults with certain types of large B-cell lymphoma, marking only the second time a gene therapy has been approved for the disease. In CAR-T therapy for hematologic cancer, a patient's own T cells are engineered to seek and destroy cancer cells. These are then sent to a facility in El Segunda, California - which Kite opened in June a year ago - where they are stimulated to proliferate and transduced with a retroviral vector to introduce the auto sequence into the patient's T cells before being propagated in cell culture bags.
Axicabtagene ciloleucel (Yescarta) has been approved to treat adult patients with diffuse large B-cell lymphoma who have not responded to or who have relapsed after at least 2 other kinds of treatment.
"The acquisition of Kite establishes Gilead as a leader in cellular therapy and provides a foundation from which to drive continued innovation for people with advanced cancers", GILD CEO, John F. Milligan, Ph.D., said in a statement.
Yescarta is the second CAR-T treatment approved by the FDA. To date, 100 patients have reportedly been treated with Yescarta. These can include life-endangering neurologic toxicities - with symptoms of headache, limb numbness, loss of memory, vision, and/or intellect - and cytokine release syndrome, when a storm of immune proteins called cytokines are released into a patient's circulatory system. It's the second time the FDA has approved a gene therapy for use in the USA following a procedure meant to treat leukemia earlier this year.
The market capitalization of Gilead is now reeling at 104.65B based on its aggregate value over its current share price and the total amount of outstanding stocks. Patients must be informed of these risks before receiving the therapy. "In just several decades, gene therapy has gone from being a promising concept to a practical solution to deadly and largely untreatable forms of cancer", said FDA Commissioner Scott Gottlieb, MD, in a statement announcing the approval.
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Michaela Almgren, a clinical assistant professor at University of South Carolina College of Pharmacy, pointed out this will not be available in just any facility due to the many challenges surrounding extraction of a patient's T-cells, adjustment of genes, and reengineering the cells in sufficient quantity.
"We believe this is only the beginning for CAR-T therapies", said Arie Belldegrun, MD, FACS, the founder of the Gilead subsidiary, Kite, that won the approval. Almgren was not involved in development of the new therapy.
Similar CAR-T therapies are being developed by Juno Therapeutics Inc., Bluebird Bio Inc., and others.
Gilead said about 7,500 lymphoma patients each year in the United States would be eligible for CAR-T therapy.
Almgren said the approval of Yescarta is "certainly very exciting".