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. <br/><br/>The treatment transforms genes into what scientists call a living drug that powerfully bolsters the immune system to shut down the disease.<br/><br/>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.<br/><br/>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. <br/><br/>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. <br/><br/>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. <br/><br/>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.
News On AIR | July 14, 2017 7:36 AM
FDA panel recommends approval for gene-altering leukemia treatment