FDA Approves First-of-Its-Kind Cancer Treatment, New Frontier

FDA Announcement

The FDA has for the first time approved a treatment that uses a patient’s own genetically modified cells to attack cancer. The agency calls this “a new frontier” in medicine. FDA Commissioner Scott Gottlieb, MD, called the approval of the therapy, named Kymriah, a “new frontier in medical innovation.”

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FDA Details

Todays’ FDA approval allows a process known as CAR T-cell therapy to fight B-cell acute lymphoblastic leukemia. Unfortunately, this childhood cancer is almost always fatal. Therefore, most importantly, it opens up a new approach to fighting cancer by harnessing the body’s immune system. This has been a long-sought goal of medical researchers.

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B-cell acute lymphoblastic leukemia attacks the blood cells that make antibodies, which help your body fight off disease. Current treatment are chemotherapy, radiation, or transplants of bone marrow.  However, in some cases, treatment fails and the cancer comes back. Hence, when that happens, the odds of survival drop to 1 in 10.

The new treatment starts with doctors extracting disease-fighting white blood cells, known as T cells, from a patient’s blood. The cells are then frozen and genetically re-engineered to attack a specific protein on the cancerous B cells. Finally, T-cells are re-infused into the patients’ body where they hunt and destroy cancer cells. And, as a result,  because these cells are taken from the patient’s own body, there is no need for anti-rejection drugs.

FDA: Test Trial Results Very Promising

In three trials involving about 150 people, the remission rates were 69%, 83%, and 95%. A total of 17 patients died after receiving the treatment; 14 of them from the disease and three from infections, according to documents the Novartis company filed with the FDA.

Dangerous Side Effects Remain a Concern

The therapy can have dangerous side effects, mainly a condition known as cytokine release syndrome. That happens when T cells release a lot of a chemical messenger into the bloodstream. This affects the vascular system, causing high fevers and sharp drops in blood pressure. More than 60% of patients in clinical trials had side effects due to cytokine release, Novartis reported, but none of those reactions were fatal.

Because of the side effects, it won’t be available everywhere. Hospitals and clinics will have to be specially certified to administer the treatment.  Doctors and other staff will also have get additional training before they can prescribe it.

Other side effects included anemia, nausea, diarrhea, and headaches.

Big Possibilities and a Big Price Tag

Another concern is the price tag associated with the therapy: The process is reported to cost as much as $475,000.

In a press release, the Center for Medicare and Medicaid Services (CMS) announced that it was exploring “innovative payment modes and arrangements” for Kymriah and other potentially life-saving treatments.

In a news release, Novartis, the company that makes Kymriah, said it was collaborating with CMS on an outcomes-based approach to pricing, which would mean that the company would only be reimbursed if a patient responds to the therapy by the end of the first month of treatment.

“Certainly, it’s far and above the expense that we typically see for drugs,” Carraway says. But current treatments can also run into the low six figures, sometimes with little success. The number of relapsed acute lymphoblastic leukemia patients with  is small, “and the options for them in their young lives are pretty limited.”

 

Is Kymriah A Breakthrough Or Another Heartbreak

Cancer is a fierce and intractable disease. Billions of dollars spent, but in fact, we are only marginally better off today than when we started. Is Kymriah the magic bullet? Much more testing is necessary before we have a definitive answer.

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