First Gene Therapy Approved in US

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Calling its endorsement an "historic action", the U.S. Food and Drug Administration on Wednesday approved the world's first genetically engineered immune therapy, Novartis Pharmaceuticals' T-cell treatment for pediatric leukemia.

David Mitchell, a multiple myeloma patient and president of the advocacy group, described the drug's price tag as "excessive". "New technologies such as gene and cell therapies hold out the potential to transform medicine and create an inflection point in our ability to treat and even cure many intractable illnesses". It uses a patient's own immune cells, called T-cells, along with white blood cells.

The treatment is approved for patients up to 25 years of age with B-cell acute lymphoblastic leukemia that doesn't respond to treatment or that returns after initial treatment - as happens in up to 20 percent of patients, according to the FDA. Grupp is Emily Whitehead's doctor - she was the first patient to receive the cell therapy, and though she was as close to 48 hours from organ failure when first enrolled in the experimental treatment, Whitehead has now been cancer-free for five years.

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.

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First developed by the University of Pennsylvania, Kymriah uses the 4-1BB costimulatory domain in its vehicle to enhance cellular responses as well as persistence of the treatment after it is infused into the patient, which may be associated with long-lasting remissions in patients.

Kymriah will carry a boxed warning because of the treatment's potential to cause deadly side effects, including neurological complications and what's known as cytokine release syndrome, a systemic reaction triggered by the destruction of the cancer cells.

In three trials involving about 150 people, the remission rates were 69%, 83%, and 95%. The approval is based on data demonstrating that in clinical trials of vehicle T-cell therapy, patients who received tocilizumab had complete resolution of CRS within 2 weeks following 1 or 2 doses of the treatment. Novartis did not give any specifics as to what the price range might be. "Those T cells are growing very rapidly, and they produce a lot of inflammatory proteins".

"There will be no charge for the therapy if the patient doesn't respond within the first month after treatment", Novartis CEO Joseph Jimenez said during a media call.

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Although the manufacturing and delivery of the living drug poses huge challenges, Novartis' global clinical trial proved the feat can be achieved. Once the cells are modified, they are infused back into the patient to kill the cancer cells.

While the drug marks a watershed moment in the treatment of cancer, it will cost about $450,000 for a round of treatment.

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