CAR-T Therapy For Leukemia Wins FDA Approval : Shots - Health News The process modifies patients' immune cells to attack their own cancer cells. It was approved to treat acute lymphoblastic leukemia in children and young adults — the most common childhood cancer.
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FDA Approves First Gene Therapy For Leukemia

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FDA Approves First Gene Therapy For Leukemia

FDA Approves First Gene Therapy For Leukemia

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The Food and Drug Administration today announced an action that it's calling historic. The FDA says it's approved the first form of gene therapy in the United States. NPR health correspondent Rob Stein reports.

ROB STEIN, BYLINE: Scientists have been trying to use genetic engineering to cure people for decades.

SCOTT GOTTLIEB: This was always held out as a way to alter the course of many vexing diseases and maybe even deliver the ability to cure some deadly disorders.

STEIN: But FDA Commissioner Scott Gottlieb says it's been really hard. Promising approaches fizzled. Some patients even died in early experiments.

GOTTLIEB: Today is an important milestone in a long journey we've been on to transform clinical medicine by using modern advances in genomics. FDA is announcing the approval of the first gene therapy product in the United States.

STEIN: The product is called Kymriah. But some scientists call it a living drug. That's because it's made from a patient's own immune system cells. The FDA's Peter Marks says scientists removed cells known as T cells and genetically modify them in the lab to turn them into something called CAR-T cells.

GOTTLIEB: When the CAR-T cells are given back to the patient, the new genetic material that has been introduced directs the T cells to bind to and kill the cancer cells.

STEIN: Kind of like how a laser-guided missiles zero into attack their targets. And so far, it looks like it works really well. Kymriah produced remissions in 83 percent of patients suffering from acute lymphoblastic leukemia, the most common form of childhood cancer in the United States. So the FDA approved this new living drug for kids and young adults who either don't respond to standard treatments or suffer relapses.

Now, this treatment can be dangerous. It can trigger the immune system to overreact, causing sometimes life-threatening complications. So the FDA is only letting doctors who have gone through special training at 32 hospitals or clinics around the country to use it. And it's expensive - really expensive - $475,000. But Novartis, the company that makes it, is defending the price. Bruno Narasimhan of Novartis says the company will only charge patients who actually get better within a month.

BRUNO NARASIMHAN: In other words, there is no charge if the patient doesn't respond in this timeframe.

STEIN: And the company says it will do other things to make sure everyone who needs the treatment gets it. In the meantime, the FDA says genetically modified immune cells are also showing promise for many other diseases. Rob Stein, NPR News.


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