FDA Approves First Gene Therapy For Leukemia

Aug 30, 2017
Originally published on August 30, 2017 1:56 pm

The Food and Drug Administration on Wednesday announced what the agency calls a "historic action" — the first approval of a cell-based gene therapy in the United States.

The FDA approved Kymriah, which scientists refer to as a "living drug" because it involves using genetically modified immune cells from patients to attack their cancer.

The drug was approved to treat children and young adults up to age 25 suffering from a form of acute lymphoblastic leukemia who do not respond to standard treatment or have suffered relapses.

The disease is a cancer of blood and bone marrow that is the most common childhood cancer in the United States. About 3,100 patients who are 20 and younger are diagnosed with ALL each year.

"We're entering a new frontier in medical innovation with the ability to reprogram a patient's own cells to attack a deadly cancer," FDA Commissioner Scott Gottlieb said in a written statement.

"New technologies such as gene and cell therapies hold out the potential to transform medicine and create an inflection point in our ability to treat and even cure many intractable illnesses," Gottlieb said.

The treatment involves removing immune system cells known as T cells from each patient and genetically modifying the cells in the laboratory to attack and kill leukemia cells. The genetically modified cells are then infused back into patients. It's also known as CAR-T cell therapy.

"Kymriah is a first-of-its-kind treatment approach that fills an important unmet need for children and young adults with this serious disease," said Peter Marks, director of the FDA's Center for Biologics Evaluation and Research.

"Not only does Kymriah provide these patients with a new treatment option where very limited options existed, but a treatment option that has shown promising remission and survival rates in clinical trials," Marks said in the FDA statement.

The treatment, which is also called CTL019, produced remission within three months in 83 percent of 63 pediatric and young adult patients. The patients had failed to respond to standard treatments or had suffered relapses. Based on those results, an FDA advisory panel recommended the approval in July.

The treatment does carry risks, however, including a dangerous overreaction by the immune system known as cytokine-release syndrome. As a result, the FDA is requiring strong warnings.

In addition, the treatment will be initially available only at 32 hospitals and clinics that have been specially trained in administering the therapy.

Novartis, which developed the drug, says the one-time treatment will cost $475,000 for patients who respond. People who do not respond within a month would not be charged, and the company said it is taking additional steps to make sure everyone who needs the drug can afford it

But some patient advocates criticized the cost nevertheless.

"While Novartis' decision to set a price at $475,000 per treatment may be seen by some as restraint, we believe it is excessive," says David Mitchell, founder and president of Patients For Affordable Drugs. "Let's remember, American taxpayers invested over $200 million in CAR-T's discovery."

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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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