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FDA OKs 1st Gene Therapy for Use in U.S.

It transforms a patient's immune system cells to fight a form of leukemia

genetic helix

WEDNESDAY, Aug. 30, 2017 (HealthDay News) -- The U.S. Food and Drug Administration broke new ground in cancer care Wednesday by approving the first gene therapy for patients in the United States.

Kymriah (tisagenlecleucel) genetically tweaks a patient's own immune system cells into what scientists call "a living drug" to battle a form of acute lymphoblastic leukemia (ALL).

The immunotherapy now can be used in children and young adults with B-cell ALL that will not respond to other therapies, the FDA announced.

Wednesday's announcement will "change the face of modern medicine and drug development," FDA Commissioner Dr. Scott Gottlieb said at a news briefing.

"Gene therapy products are now being studied in many diseases and conditions, including genetic disorders, autoimmune diseases, heart disease, cancer, diabetes and HIV/AIDS," Gottlieb added.

ALL is a cancer of the bone marrow and blood in which the body makes abnormal lymphocytes, a type of white blood cell. According to the U.S. National Cancer Institute, about 3,100 patients aged 20 and younger are diagnosed with ALL each year.

About 15 percent to 20 percent of patients with B-cell ALL have cancer that either did not respond to treatment or has recurred, the FDA says. It's these patients for whom Kymriah is intended.

In the treatment, doctors collect the patient's own T-cells -- one of the immune system's main cell types -- and genetically reprogram them to target and attack leukemia cells. They are then reintroduced back into the patient to do battle against the tumor.

Dr. Kenneth Anderson is president of the American Society of Hematology, which focuses on blood cancers. In a statement, he said that Wednesday's approval "marks an important shift in the blood cancer treatment paradigm. We now have proof that it is possible to eradicate cancer by harnessing the power of a patient's own immune system. This is a potentially curative therapy in patients whose leukemia is unresponsive to other treatments."

Dr. Otis Brawley, chief medical officer at the American Cancer Society, agreed.

"This is big. Big," he said. "I'm going to predict this technology will spread to other diseases over the next few years. It's a new method of treating cancer that I think we will successfully further exploit over the next decade."

However, Anderson stressed that "this approval only pertains to a small population of children [with ALL]."

"More research is needed to make this therapy more effective for a broader population, to reduce the severe side effects that patients experience during treatment, and ultimately to find a broader application beyond blood cancers," he said.

The FDA based its decision on a clinical trial involving 63 patients with B-cell ALL. After three months of treatment, 83 percent of the patients remained cancer-free.

Gottlieb said the FDA approval was expedited, coming just seven months after the agency received the initial application.

However, Kymriah comes with the potential for severe side effects. The worst is cytokine release syndrome, a common immunotherapy complication that causes potentially life-threatening fever and flu-like symptoms. The therapy also can cause neurological events, serious infections, low blood pressure and acute kidney injury.

Because of these safety concerns, the FDA will require that hospitals receive special certification to use Kymriah, the agency said.

And then there's the issue of cost. Kymriah's maker, Novartis, hasn't provided a price for the drug. But speaking earlier this year to The New York Times, drug industry analysts estimated that individualized therapies could cost more than $300,000.

Other genetic therapies for cancer are also in the research pipeline. In June, a clinical trial in China showed promise in treating another blood cancer, multiple myeloma, using reprogrammed T-cells in much the same way Kymriah does.

According to Gottlieb, the FDA has already granted more than 550 active investigational new drug applications related to gene therapy products, including 76 active investigational new drug applications related to CAR-T cell products like Kymriah.

And while the path to safe and effective gene therapies has been a long one, "Today, a pivotal leg in that journey is complete," Gottlieb said.

"The science has reached a point of superiority, where enough of the components of these endeavors have worked out," he explained. "We can deliver effective therapies to patients. We can deliver on the original promise."

More information

For more on childhood acute lymphoblastic leukemia, visit the U.S. National Cancer Institute.

SOURCES: Scott Gottlieb, M.D., commissioner, U.S. Food and Drug Administration, news briefing, Aug. 30, 2017; Otis Brawley, M.D., chief medical officer, American Cancer Society; American Society of Hematology, news release, Aug. 30, 2017; The New York Times
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