When will therapies from human gene transfer research be available?
Human gene transfer products, like all medical products, must be studied in several phases before the FDA can approve them for use by the public. Most gene transfer trials registered with the NIH have been phase I studies that investigate safety. Only thirteen percent have been phase II studies, which assess both safety and effectiveness and generally involve more subjects. Less than one percent of the trials (four) have progressed to phase III studies, which test for effectiveness using large numbers of subjects. No human gene transfer product has yet been approved for medical use by the FDA. Given the research that must yet be done, gene transfer products are not expected to be available to patients in the immediate future. What’s next? Gene transfer is a relatively new technology that still faces many technical challenges. The body’s immune system, for example, can make it difficult to deliver a foreign substance into cells, a major technical hurdle. Researchers are investigating wa
