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FDA OKs Second Gene Therapy for Cancer

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FDA OKs Second Gene Therapy for Cancer

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A second new personalized treatment for cancer has won approval from the Food and Drug Administration — a clear sign that such treatments will become more widely available for patients with no other options.

The treatment is called CAR-T and can have dramatic effects in some patients. But it is both grueling and expensive and will remain a last-ditch treatment for a few, specific cancer types.

The company that will market it, Gilead Sciences, has priced it at $373,000 and the FDA warns it can cause severe side effects.

The therapy is called Yescarta — the generic name is axicabtagene ciloleucel. It's described as gene therapy because the patient's own immune cells are removed and genetically engineered to better fight off the cancer.

It's a little different from similar approaches in which a cancer patient's immune cells are harvested and numbers boosted in the lab. And it's different from other gene therapy treatments for inherited diseases.

"Yescarta, a chimeric antigen receptor (CAR) T cell therapy, is the second gene therapy approved by the FDA and the first for certain types of non-Hodgkin lymphoma," the FDA said in a statement.

Related: New Gene Treatment for Cancer Offers Hope

"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," FDA Commissioner Dr. Scott Gottlieb said.

"This approval demonstrates the continued momentum of this promising new area of medicine and we're committed to supporting and helping expedite the development of these products."

In August, the FDA approved a different CAR-T treatment for B-cell acute lymphoblastic leukemia in patients under the age of 25 who have not been helped by other treatments.

"There will likely be thousands of lives saved in the next few years because of it."

The new treatment is approved for a different cancer — diffuse large B-cell lymphoma, which is the most common type of non-Hodgkin lymphoma. It's diagnosed in about 72,000 people each year, although just a fraction of these would be eligible for the new therapy.

"Yescarta is approved for use in adult patients with large B-cell lymphoma after at least two other kinds of treatment failed," the FDA said.

"There will likely be thousands of lives saved in the next few years because of it," said Dr. David Maloney, medical director of cellular immunotherapy at Fred Hutchinson Cancer Research Center.

"The FDA's ruling validates the revolution under way in the field of cellular immunotherapy, which enables us to engineer patients' own immune systems to eliminate cancer cells."

Related: FDA Advisers Say Yes to Experimental Cancer Therapy

The treatment carries big risks and the FDA has asked Gilead to take steps to watch for problems.

Patient's immune systems can release a flurry of compounds called cytokines that cause high fever —sometimes deadly — and flu-like symptoms and nerve damage. Half the patients treated suffered a severe side effect, the company said.

"Other side effects include serious infections, low blood cell counts and a weakened immune system," the FDA said.

"American taxpayers paid for the basic science behind this drug, and now we're asked to pay outrageous sums to get the treatment."

"The FDA is requiring that hospitals and their associated clinics that dispense Yescarta be specially certified. As part of that certification, staff involved in the prescribing, dispensing or administering of Yescarta are required to be trained to recognize and manage cytokine release syndrome and nervous system toxicities."

Plus, it's very expensive.

The treatment was originally developed at the National Cancer Institute, in the lab of Dr. Steven Rosenberg, who works on a variety of personalized cancer approaches.

The NCI licensed the treatment to Kite Pharma, and once it was shown to cause complete remission in half the patients who tried it, Gilead bought Kite.

Related: Cancer Moonshot Panel Says Focus on Immune Therapies

"American taxpayers paid for the basic science behind this drug, and now we're asked to pay outrageous sums to get the treatment," said David Mitchell, a cancer patient and the founder of Patients For Affordable Drugs.

"As a cancer patient, I look forward to the potential of CAR-T drugs. But drugs don't work if people can't afford them, and no American should pay $373,000 for a drug that taxpayers helped invent."

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