Officials remove special rules for 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 this week. 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 so far, and not all experts agree that it doesn’t still 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.

When it was first proposed, there were so many safety worries and scientific unknowns that the NIH created a panel of independent scientists, called the Recombinant DNA Advisory Committeeto assess each experiment and potential risks to patients. The risks were underscored in 1999, when a teen’s death in a gene experiment put a chill on the field.

Since then, much has been learned about safety, and last year the FDA approved the nation’s first gene therapies, for cancer and an inherited form of blindness.

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.