October 20, 1995
Web posted at: 2:05 a.m. EDT
From Medical Correspondent Jeff Levine
BETHESDA, Maryland (CNN) -- Just five years ago, 14-year-old Cynthia Cutshall's outlook was bleak. She suffers from a rare inherited disease called ADA that devastates her immune system.
But Cynthia and another young girl have made medical history as the first to receive gene therapy. Both are doing well. "It showed the treatment worked," says Dr. R. Michael Blase, of the National Institutes of Health. "What's even more exciting is the fact that they've (gone) almost three years without additional treatment."
The treatment involves substituting a healthy gene for a sick one. What doctors at the National Institutes of Health were able to do is mix a key part of the patient's immune system with a weakened virus.
The result is a solution that spreads the good gene through the body like a blood transfusion. The hope is that this could become a permanent fix. "We think that there will be a one-shot treatment with gene therapy for ADA deficiency," says Dr. Kenneth Culver, also with the National Institutes for Health.
That news comes as great comfort to children with ADA and their families. The disease often proves fatal before adulthood.
Zach Riggins has the disorder, which is often referred to as the "bubble boy" disease, because patients live in virtual isolation. "Gene therapy is such a breakthrough for medicine and for everybody, not just for (our family)," says Zach's father Rich, "But there's so many others that will benefit from it." (180K AIFF sound or 180K WAV sound)
The successful ADA treatment has raised the hopes of researchers at the National Institutes of Health. In theory, gene therapy could be a treatment for some 4,000 diseases, from cancer to AIDS.
The ADA patients received about a dozen infusions with only mild side effects.
But researchers stress that this treatment and other potential applications are highly experimental and probably won't be on the market for years. "I'm really quite optimistic that we're going to have some useful treatments. What gene therapy gives us is a new box of tools that we've never had before," says Dr. Blase. (198K AIFF sound or 198K WAV sound)
Skeptics complain that gene therapy has been overhyped, and it's taken years to get only modest results. But doctors at the National Institutes of Health say that their two gene therapy children were worth the wait.
Copyright © 1995 Cable News Network, Inc.
ALL RIGHTS RESERVED.
External sites are not necessarily endorsed by CNN Interactive.