An experimental gene therapy that turns a patient’s own blood cells into cancer killers worked in a major study, with more than one-third of very sick lymphoma patients showing no sign of disease six months after a single treatment…
In all, 82 percent of patients had their cancer shrink at least by half at some point in the study.
Its sponsor, California-based Kite Pharma, is racing Novartis AG to become the first to win approval of the treatment, called CAR-T cell therapy, in the U.S. It could become the nation’s first approved gene therapy.
A hopeful sign: the number in complete remission at six months — 36 percent — is barely changed from partial results released after three months, suggesting this one-time treatment might give lasting benefits for those who do respond well.
The therapy is not without risk. Three of the 101 patients in the study died of causes unrelated to worsening of their cancer, and two of those deaths were deemed due to the treatment.
It was developed at the government’s National Cancer Institute and then licensed to Kite. The Leukemia and Lymphoma Society helped sponsor the study.
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