First gene therapy to treat deadly cancer approved in US

First gene therapy to treat deadly cancer approved in US

First gene therapy to treat deadly cancer approved in US

US regulators approved a revolutionary new cancer treatment from Novartis AG on Wednesday, the first in a group of coming therapies that offer breakthrough cures for dire cases as well as stratospheric costs.

The FDA has approved the therapy for relapsing B-cell acute lymphoblastic leukemia (ALL) in children and young adults, who account for 60% of cases. ACGT provided an additional $800,000 of funding in 2008.

CAR-T therapy was pioneered by Dr. Carl June at the University of Pennsylvania, who has been working on the treatment for nearly three decades. The FDA itself called the approval a "historic action".

A groundbreaking gene therapy has been found to cure patients battling cancer by at least 79 percent. "The lessons learned from the clinical trials of vehicle T-cell operations will assist us in being able to provide to the larger population". Salvage therapies for children with relapsed ALL are highly toxic and only work in about 50 percent of relapsed patients.

Each dose of Kymriah is a customized treatment created using the patient's own T-cells.

The new leukemia drug reprograms a patient's own cells to attack the deadly cancer, possibly in a single treatment. It involves removing disease-fighting T cells from a patient, genetically modifying them to better recognize and attack cancer, and then replacing them, where they can circulate for years seeking out the disease.

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.

But there are drawbacks - the main one being the price tag.

The high cost of the therapy reflects the fact that the CAR-T procedure is fairly intensive.

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The approval of Novartis' CAR-T therapy comes just two days after Gilead Sciences Inc. announced an agreement to spend $11.9 billion and buy Kite Pharma Inc., which is developing its own CAR-T therapy scheduled for an FDA ruling by November 29.

Kymriah treats cancer in an entirely new way.

Most patients with ALL recover through other treatments such as radiation, chemotherapy and stem cells.

Of more than 60 ALL-suffering subjects in a clinical trial for Kymriah, 83 percent were cancer-free within three months.

His current research is focused on similar reprogramming of cells but for the detection of lung cancer cells. About half of the patients in the Kymriah study got cytokine release syndrome, a response to the reprogrammed cells running loose around the body.

Bruno Strigini has pointed out that current therapies against leukemia, such as bone marrow transplant, can cost up to $ 800 000 in the United States.

Another group, the Campaign for Sustainable Rx Pricing said in a statement that while the "science behind Kymriah is revolutionary, the business decision to price it at almost half of a million dollars per treatment is not".

"We're at the very beginning of what's going to be a big new field of medicine", David Epstein, who helped license Kymriah in from the University of Pennsylvania while at Novartis.

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