Published: Thu, October 19, 2017
Medicine | By Brett Sutton

FDA Approves Radical New Gene-Altering Cancer Treatment

FDA Approves Radical New Gene-Altering Cancer Treatment

The US Food and Drug Administration has approved a second gene therapy for cancer, the first to target non-Hodgkin lymphomas. That drug, Kymriah, developed by Novartis, sells for $475,000. "That policy will also clarify how we will apply our expedited programs to breakthrough products that use CAR-T cells and other gene therapies".

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The new drug's maker, Kite Pharma, an industry leader in the emerging field of cell therapy founded by Israeli scientist Dr. Arieh Belldegrun, was acquired in late August by Gilead Sciences for $11.9 billion. Each treatment is customized using a patient's own immune cells to fight cancer cells. Each Yescarta dose is created by collecting and genetically modifying a patient's T-cells, a type of white blood cell, to include a new gene that targets and kills lymphoma cells. Patients who relapse after multiple rounds of chemo or a stem-cell transplant "rarely achieve complete remission", he said. That's why these immunotherapy treatments are called "living drugs".

"This is the beginning of a new era of cancer therapy", oncologist Dr. Armin Ghobadi, of Washington University in St. Louis, said in a university news release. "Immunotherapy is dramatically changing the way we approach blood cancer treatment". After the cells are modified, they are infused back into the patient, where they patrol for years.

Diffuse large B-cell lymphoma is the most common type of the disease in adults, with some 72,000 new patients diagnosed annually in the U.S., according to the American Cancer Society, and some 20,000 will die from it this year.


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. "Now, thanks to this new therapy many patients are in remission for months". Yescarta will be manufactured in Kite's state-of-the-art commercial manufacturing facility in El Segundo, California.

Although Yescarta didn't work for all 101 patients assessed in the pivotal trial, the trial results "show the potential for a cure", Chang said. The FDA warns that axicabtagene ciloleucel has a boxed warning for cytokine release syndrome, which causes a high fever, flu symptoms, and neurological toxicities. The FDA is requiring Kite to do a long-term safety study and train hospitals to quickly spot and handle those reactions. The tumors in 72 percent of the test subjects shrunk and even disappeared completely in 51 percent of the subjects.

While it is billed as a one-time treatment, because the patients' cancer is so far advanced, it returns in some. While this therapy may be groundbreaking, it comes with potentially lethal side effects.

The FDA is now considering an unrelated gene therapy to treat an inherited form of blindness. Spark Therapeutics' treatment for a rare form of blindness could be approved within months. "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".

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