Officials yank special rules for gene therapy experiments

U.S. health officials are eliminating special regulations for gene therapy experiments, saying that what was once exotic science is quickly becoming an established form of medical care with no extraordinary risks.

A special National Institutes of Health oversight panel will no longer review all gene therapy applications and will instead take on a broader advisory role, according to changes proposed Wednesday. The Food and Drug Administration will vet gene therapy experiments and products as it does with other treatments and drugs.

It’s an extraordinary milestone for a field that has produced only a few approved treatments, and not all experts agree that it doesn’t need special precautions.

With gene editing and other frontiers looming, “this is not the right time to be making any moves based on the idea that we know what the risks are,” said Stanford bioethicist Mildred Cho.

Gene therapy aims to attack the root cause of a problem by deleting, adding or altering DNA, the chemical code of life, rather than just treating symptoms that result from a genetic flaw.

It’s time to let the FDA review gene therapy proposals on its own without duplicating regulatory efforts, the NIH’s director, Dr. Francis Collins, and FDA chief Dr. Scott Gottlieb wrote Wednesday in the New England Journal of Medicine. The proposed changes will go into effect after a public comment period.

More than 700 proposals for gene therapy are pending now, and “it seems reasonable to envision a day when gene therapy will be a mainstay of treatment for many diseases,” they wrote. “The tools we use to address other areas of science are now well suited to gene therapy.”