KHN Morning Briefing

Summaries of health policy coverage from major news organizations

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Breakthrough Therapy That Uses Kids’ Own Immune Cells To Fight Leukemia Approved By FDA

“We’re entering a new frontier in medical innovation with the ability to reprogram a patient’s own cells to attack a deadly cancer,” Food and Drug Administration Commissioner Scott Gottlieb said. The price tag on the treatment is $475,000.

The Associated Press: US Clears Breakthrough Gene Therapy For Childhood Leukemia
Opening a new era in cancer care, U.S. health officials on Wednesday 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 called 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. (Neergaard, 8/30)

The New York Times: F.D.A. Approves First Gene-Altering Leukemia Treatment, Costing $475,000
The new therapy turns a patient’s cells into a “living drug,” and trains them to recognize and attack the disease. It is part of the rapidly growing field of immunotherapy that bolsters the immune system through drugs and other therapies and has, in some cases, led to long remissions and possibly even cures. The therapy, marketed as Kymriah and made by Novartis, was approved for children and young adults for an aggressive type of leukemia — B-cell acute lymphoblastic leukemia — that has resisted standard treatment or relapsed. The F.D.A. called the disease “devastating and deadly” and said the new treatment fills an “unmet need.” (Grady, 8/30)

The Washington Post: FDA Clears First Gene-Altering Therapy — ‘A Living Drug’ — For Childhood Leukemia
“We’re entering a new frontier in medical innovation with the ability to reprogram a patient’s own cells to attack a deadly cancer,” FDA Commissioner Scott Gottlieb said. “New technologies such as gene and cell therapies hold out the potential to transform medicine” and cure intractable illnesses. He said companies are pursuing hundreds of experimental treatments involving gene therapy products. (McGinley and Johnson, 8/30)

NPR: CAR-T Therapy For Leukemia Wins FDA Approval
The treatment, which is also called CTL019, produced remission within three months in 83 percent of 63 pediatric and young adult patients. The patients had failed to respond to standard treatments or had suffered relapses. Based on those results, an FDA advisory panel recommended the approval in July. (Stein, 8/30)

Kaiser Health News: Pioneering Cancer Gene Therapy Gets Green Light — And $475,000 Price Tag
Novartis said that it priced its drug based on several considerations. British health authorities have said a price of $649,000 for a one-time treatment would be cost-effective given Kymriah’s significant benefits. Novartis also considered the cost of bone-marrow transplants, which are currently given to many leukemia patients whose cancer relapses. Those transplants can cost up to $800,000, Novartis said. (Szabo, 8/30)

Bloomberg: Novartis's $475,000 Cancer Breakthrough Heralds New Era Of Cures 
The Basel, Switzerland-based company is wading into a new arena of performance-based pricing, said Sam Fazeli, a Bloomberg Intelligence analyst. “We’re not used to drugs curing people” of major diseases like cancer, he said. “It’s a paradigm shift.” Shares of Novartis rose 0.8 percent to 79.85 Swiss francs as of 10:07 a.m. in Zurich trading. The stock has advanced 7.7 percent in the past year. (Edney, Paton and Chen, 8/30)

Detroit Free Press: FDA Gives New Hope To Kids With Leukemia In Approving Gene Therapy
Offering the treatment at a large medical center like U-M is essential, said Dr. Rajen Mody, a pediatric oncologist at Mott, because of the severity of potential side effects. "It can cause serious side effects, especially within the first 21 days," said Mody, who is Mott's director of pediatric oncology. "Patients can have high fevers, bleeding complications, trouble breathing, infections. ... That’s why a hospital like the University of Michigan is the ideal place. ... Patients who undergo this treatment are usually so sick after an infusion of the CAR-T cells, that they can't be safely treated at smaller hospitals." (Shamus, 8/31)

Chicago Tribune: Chicago Hospitals Gearing Up To Offer Breakthrough Treatment For Pediatric Cancer 
University of Chicago Medical Center and Lurie Children's Hospital could be among the first hospitals in the country to offer a new, breakthrough pediatric cancer treatment approved Wednesday by the U.S. Food and Drug Administration. The treatment, which uses a patient's own modified cells to battle a form of acute lymphoblastic leukemia, is the first gene therapy approved for use in the U.S. Acute lymphoblastic leukemia is the most common childhood cancer. (Schencker, 8/30)

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