Published: Thu, August 31, 2017
Money | By Oscar Reynolds

FDA Approves First Gene Therapy in the United States

FDA Approves First Gene Therapy in the United States

Kymriah, a cell-based gene therapy known as a CAR-T drug, was approved to treat patients up to age 25 with what's called B-cell acute lymphoblastic leukemia.

But advocacy groups are afraid the historic new drug may turn out to be the most expensive ever sold in the U.S.

The US Food and Drug Administration approved a new leukemia treatment, which the agency considers the first gene therapy it has cleared to hit the market in the United States.

Kymriah is a genetically-modified autologous T-cell immunotherapy with each dose customized using the patient's own T-cells. "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". Novartis also considered the cost of bone-marrow transplants, which are now given to many leukemia patients whose cancer relapses.

Grupp is Emily Whitehead's doctor - she was the first patient to receive the cell therapy, and though she was as close to 48 hours from organ failure when first enrolled in the experimental treatment, Whitehead has now been cancer-free for five years. Researchers filter those cells from a patient's blood, reprogram them to harbor a "chimeric antigen receptor" that zeroes in on cancer, and grow hundreds of millions of copies.

In a key study of 63 advanced patients, 83 percent went into remission.

Kymriah treatment also has the possibility of serious side effects, including cytokine release syndrome (CRS), which consists of high fever and flu-like symptoms and can be life-threatening, and neurological events, which are also life-threatening.

It's a completely different way to harness the immune system than popular immunotherapy drugs called "checkpoint inhibitors" that treat a variety of cancers by helping the body's natural T cells better spot tumors.

At the time, American Cancer Society Deputy Chief Medical Officer Dr. Len Lichtenfeld said the real promise of these gene therapies is the potential to completely eradicate specific cancers both for the single patient as well as all their descendants. "This is a potentially curative therapy in patients whose leukemia is unresponsive to other treatments and represents the latest milestone in the shift away from chemotherapy toward precision medicine". For others, Kymriah serves as a “bridge to transplant, ” keeping them alive longer enough to undergo this therapy.

The approval of Novartis' CAR-T therapy comes just two days after Gilead Sciences Inc. announced an agreement to spend $11.9 billion and buy Kite Pharma Inc., which is developing its own CAR-T therapy scheduled for an FDA ruling by November 29.

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