FDA approves breakthrough treatment for dire cancers

Novartis won FDA approval for its CAR-T drug Kymriah on Wednesday

The United States Food and Drug Administration (FDA) on Wednesday announced that it has approved a cell-based gene therapy in the United States. Each dose is a customized treatment created with a patient's own T-cells, which are collected and sent to a manufacturing center where they are genetically modified to include a new gene that contains a specific protein-a chimeric antigen receptor or CAR-that directs the T-cells to target and kill leukemia cells that have a specific antigen (CD19) on the surface.

"We're excited and proud to have moved this auto therapy, in collaboration with Novartis and CHOP, through all phases of development and clinical trials, established its efficacy, and now extended its reach to children across the country under this FDA approval", he added. The patient was nearing death, but has now been cancer-free for five years.

"While both external and Novartis' quantitative assessments of these values indicate that a cost-effective price could be $600,000 to $750,000, we recognize the importance of this paradigm-shifting therapy and are setting the price at $475,000 for this one-time treatment", Dana Cooper, a spokesman for Novartis, said in an interview with OncLive.

To make sure patient safety is the priority, the FDA is requiring Novartis to offer the treatment only through medical centers that have been specially trained and certified.

The drug's maker, Novartis, also pledged that it wouldn't charge any patient who didn't respond to the treatment after a month.

Treatment with Kymriah has the potential to cause severe side effects.

"The therapy", STAT reports, "is made by harvesting patients' white blood cells and rewiring them to home in on tumors". A clinical trial found 83 percent of patients who underwent the treatment went into remission within three months.

The FDA has recognized these dangers in approving the Kymriah treatment, noting that the approval is only directed at patients that have not responded to other, more common, initial treatments.

Until recently, the use of vehicle T-cell therapy has been restricted to small clinical trials, largely in patients with advanced blood cancers.

Dana-Farber/Boston Children's has recently participated in a similar clinical trial of vehicle T-cells - led by Silverman and pediatric oncologist Steven Margossian, MD, PhD - that evaluated the efficacy of auto T-cell therapy (short for chimeric antigen receptor T-cell therapy) in treating relapsed or treatment-resistant B-cell ALL. About half of the patients in the Kymriah study got cytokine release syndrome, a response to the reprogrammed cells running loose around the body. However, ultimately, the cell therapy could go a long way in saving hundreds of young people's lives each year.

Shares of Gilead Sciences Inc, which this week announced an $11.9 billion deal to buy Kite Pharma, were up 6.2 percent to $80.47 on Wednesday afternoon, as Kite is widely expected to receive the next US approval of a CAR-T therapy for a different blood cancer. This allows the cells to replicate quickly and zero in on cancer cells, fighting the disease for years. Penn and Novartis are also investigating the next generation of auto therapies for multiple myeloma, and for solid tumors, through trials in glioblastoma, mesothelioma, and ovarian and pancreatic cancer.

There could be side effects. And a year ago, Juno said five people in its clinical trials had died, all from cerebral edema.



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