Even if gene therapy conquers these challenges, will it ever overcome its negative reputation? Some scientists maintain that it has never been that unsafe, relatively speaking. “If you compare the safety profile of gene drugs in development versus the traditional small-molecule pharmaceutical drugs, there’s no evidence that gene therapy is any more dangerous,” says Savio Woo, an oncologist at Mount Sinai Hospital in New York City. Thousands of patients have been treated, and only a few adverse events have been reported, he states; the leukemia that developed in three “bubble boy” patients may have been a side effect specific to the therapeutic gene, which stimulates immune cell growth. “Any time a few cells divide a lot, you always worry about secondary genetic changes, which is how cancers form,” notes Mark Kay, a geneticist at Stanford University.
As the field continues to evolve and improve, scientists hope that the public’s perceptions of it will, too.
“We clearly have had clinical successes, and now we’re on the threshold of achieving many more,” says ASGT president Nienhuis. “I think we’re going to hear a lot about them in the next several years.”
This article was originally published with the title Regaining Lost Luster.