Newer methods may boost gene therapy's use for more diseases

Jordan Janz knew his gamble on an experimental gene therapy for his rare disease might be paying off when he returned to work and a friend sniffed him.

“He said, ‘you have a normal smell, you smell good,’” Janz recalled. “And I’m like, ‘that’s probably the nicest thing you’ve ever said.’”

The 22-year-old Canadian man's previous treatment required 40 to 60 pills a day and left him smelling like rotten eggs or stinky cheese. He was born with a flawed gene that left him unable to make a protein needed by virtually every organ in the body. Kids with this disease can throw up a dozen times a day, need eye drops every hour to prevent blindness and often kidney transplants before they’re adults.

Now, Janz and a growing number of others with rare diseases have hope of not just better treatments, but perhaps a cure. Gene therapy increasingly is being used to attack the root cause of their problems by supplying the DNA they lack.

Janz was the first person in one such study at the University of California, San Diego. He and two other participants no longer need the eye drops and pills that only delayed progression of their disease and left them smelling bad. The company that developed their therapy is testing it for several other diseases by simply adjusting what gene is supplied.

Other companies are doing the same, and U.S. health officials are working on guidance to encourage the trend.

“I am very excited for the field because I feel like we’re beginning to get to a critical mass,” where a single method or product can be deemed safe and then adapted for many uses, said Dr. Peter Marks, head of the U.S. Food and Drug Administration center that oversees gene therapies. “You’re essentially using the same rocket ship to put stuff...