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FDA panel recommends approval for gene-altering leukemia treatment


A US Food and Drug Administration panel opened a new era in medicine, unanimously recommending that the agency approve the first-ever treatment that genetically alters a patient’s own cells to fight cancer.

The treatment transforms genes into what scientists call a living drug that powerfully bolsters the immune system to shut down the disease.

If the FDA accepts the recommendation, which is likely, the treatment will be the first gene therapy to reach the market. Novartis is poised to be the first with a treatment for a type of leukemia. It is working on similar types of treatments for another form of the disease, as well as multiple myeloma and an aggressive brain tumor.

A single dose of the resulting product has brought long remissions, and possibly cures, to scores of patients in studies who were facing death because every other treatment had failed.

The panel recommended approving the treatment for B-cell acute lymphoblastic leukemia that has resisted treatment, or relapsed, in children and young adults aged three to 25.

The main evidence that Novartis presented to the FDA came from a study of 63 patients who received the treatment from April 2015 to August 2016. Fifty-two of them, or 82.5 per cent, went into remission – a high rate for such a severe disease.

The panel raised concerns about potentially life-threatening side effects like those Emily experienced, and longterm worries about whether the infused cells could cause secondary cancers or other problems.

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