FDA advisers review data on potential first US gene therapy

Cancer experts who advise government regulators are reviewing what could be the first gene therapy approved in the U.S.

The Food and Drug Administration panel is holding a hearing Wednesday to discuss the treatment developed by the University of Pennsylvania and Novartis Corp. The drugmaker is seeking approval to use the one-time treatment for children and young adults with advanced leukemia.

The therapy could be the first of a wave of cancer treatments custom-made to target a patient’s cancer. Called CAR-T, it involves removing immune cells from a patients’ blood, reprogramming them to create an army of attack cells and putting them back into the patient.

The panel is reviewing the safety, effectiveness and production of the treatment. It will vote on whether to recommend FDA approval.

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