Kite Pharma’s gene therapy for cancer shows hopeful results

❝ 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 United States. 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 worry has been how long Kite’s treatment would last; there’s also been concern about its side effects, which…seem manageable in the study. Follow-up beyond six months is still needed to see if the benefit wanes…

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.

❝ The treatment involves filtering a patient’s blood to remove key immune system soldiers called T-cells, altering them in the lab to contain a gene that targets cancer, and giving them back intravenously. Doctors call it a “living drug” — permanently altered cells that multiply in the body into an army to fight the disease.

First opportunity for peer review will be at an April meeting of the American Association for Cancer Research. Fascinating stuff. Methodology that holds promise for any number of difficult deadly diseases.

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