Gene Therapy Experts Seek Safer Studies
Specifically, FDA will now require researchers to submit their safety plans routinely to the agency, including information about who is actually monitoring the experiments. Anderson estimates that might add $80,000 to the cost of a single safety trial. In addition, the NIH and FDA are initiating a series of public symposia on critical patient safety issues in gene research. The first such discussion involving safer methods of replacing genes in the body is underway at this RAC meeting.
Typically, a virus, often an adenovirus similar to one that might cause a cold, is used to transport the gene treatment to an exact location in the body where it will hopefully crank out a normal protein. However, in Gelsinger's case, the virus itself may have triggered the reaction that led to his death.
One alternative now being considered by researchers is a "gutless" adenovirus from which most of the potentially toxic portions have been removed. The approach has been tested successfully in animals and may be first used in humans to replace a gene that prevents bleeding in hemophiliacs. If the gutless virus succeeds, it may eventually replace the earlier, more dangerous methods of delivering genes.
However, long-time critic of gene therapy Jeremy Rifkin, president of the Foundation on Economic Trends, isn't impressed by attempts to make the treatment safer. On Friday, he plans to ask the RAC for an "immediate moratorium" on gene experiments "except where the protocol can legitimately be considered a treatment of last resort for a life threatening illness."
"I don't think there's any serious concern on anybody's part that a moratorium is necessary or useful, but I think it's important that the issue be discussed," says Anderson.