US clears breakthrough gene therapy for childhood leukemia

In this July 9, 2015, photo, provided by Novartis Pharmaceuticals Corp., human T cells belonging to cancer patients arrive at Novartis Pharmaceuticals Corp.’s Morris Plains, N.J., facility. The Food and Drug Administration (FDA) approved the first treatment that genetically engineers patients’ own blood cells into an army of leukemia-fighting assassins. Manufacturer Novartis will create those turbocharged cells in this facility and ship them back to hospitals to infuse into patients. (Brent Stirton/Novartis Pharmaceuticals Corp. via AP)

U.S. health officials have approved a breakthrough treatment that genetically engineers patients’ own blood cells into an army of assassins to seek and destroy childhood leukemia.

The Food and Drug Administration calls the approval historic, the first gene therapy to hit the U.S. market. Made from scratch for every patient, it’s one of a wave of “living drugs” under development to fight additional blood cancers and other tumors, too.

Novartis Pharmaceuticals has set the price for its one-time infusion of so-called “CAR-T cells” at $475,000, but says there would be no charge for patients who didn’t show a response within a month.

This photo provided by the Children’s Hospital of Philadelphia, taken in May 2017, shows Emily Whitehead five years after she became the first pediatric patient in the world to receive an experimental therapy at the hospital that has put her leukemia into long-term remission. Opening a new era in cancer care, the Food and Drug Administration (FDA) approved the first treatment that genetically engineers patients’ own blood cells into an army of assassins to seek and destroy childhood leukemia. (Children’s Hospital of Philadelphia via AP)

This undated image made available by Novartis on Wednesday, Aug. 31, 2017 shows an IV bag of their drug Kymriah. On Wednesday, the Food and Drug Administration approved this first treatment that genetically engineers patients’ own blood cells to seek and destroy childhood leukemia. (Novartis via AP)

This photo provided by the Children’s Hospital of Philadelphia, taken in May 2017, shows Dr. Stephan Grupp who led a key study of a newly approved treatment for childhood leukemia. Opening a new era in cancer care, the Food and Drug Administration (FDA) approved the first treatment that genetically engineers patients’ own blood cells into an army of assassins to seek and destroy cancer. (Children’s Hospital of Philadelphia via AP)


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Novel leukemia treatment could be first US gene therapy (Update)

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