As Novartis, Juno Therapeutics and Kite Pharma race to launch what may be the most effective treatments ever seen for leukemia and other blood cancers, they are grappling with how to make them widely available in a reliable and cost-efficient way.
The new therapies, known as CAR T cells, are made by extracting immune system T cells from an individual patient, altering their DNA to sharpen their ability to spot and kill cancer cells, and infusing them back into the same patient. In some early-stage clinical trials, the treatments eliminated all trace of leukemia and lymphoma in 40 percent to 90 percent of patients who had run out of other options.
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"We feel confident we can scale up to thousands of patients a year with a true global facility," said Usman Azam, Novartis' head of cell and gene therapies. That would be enough to satisfy commercial demand for at least a few years after the treatment is first approved, he said, adding that a second plant could be built if necessary. Azam said the company's treatment, called CTL019, is "potentially curative."
Novartis plans to seek U.S. approval next year for CTL019 in children with acute lymphoblastic leukemia. In 2017, Novartis will aim for a far bigger market: patients with the most common form of non-Hodgkins lymphoma, called diffuse large B-cell lymphoma