Gene Therapy: The Future Is Now
April 27, 2000 -- For the longest time, there has been talk about how gene
therapy holds great promise for the future of medicine. Well, it looks like the
future is now. Gene therapy is finally showing signs of being a promise kept,
thanks to some French researchers.
In the April 28 issue of Science, the researchers detail their
success in using gene therapy to treat children whose faulty immune systems
otherwise would have forced them to spend their lives in a sterile
"bubble." In the wake of the death last fall of an Arizona teen-ager
during a University of Pennsylvania gene-therapy experiment, the latest advance
is leading to renewed hope for the technique.
But what exactly is that hope? A drug-free, permanent solution for many
types of disease, it would seem.
Drugs work from the outside in, and must be continuously re-administered if
the disease is chronic. Gene therapy works from the inside, and should keep
working indefinitely. A key struggle for researchers is finding the right gene
to address the disease in question, and making sure that gene is delivered to
the right place in the body.
Which brings us to the recent success. The disease the French researchers
may have cured is well known, but rare. Does gene therapy hold promise for the
big daddies of disease: cancer, diabetes, heart disease? You bet, and then
W. French Anderson, MD, is credited with performing the first gene treatment
in 1990 (for the same type of disease treated by the French researchers). He
tells WebMD that the recent gene therapy successes are analogous to man's first
landing on the moon.
"We started off with a big burst of enthusiasm, just like [what]
happened when [President] Kennedy made the announcement we're going to beat the
Russians to the moon. And then there's all this high hope, and everybody rushes
out to watch the rockets go up, and they blow up," Anderson says, making
the comparison to the death in the Pennsylvania trial. "Things got really
bad for a while, then all the little parts started working. ... We made
it." Anderson, who wrote a commentary about the French research in
Science, is director of the gene therapy laboratories at the University
of Southern California in Los Angeles.
The disease involved in the French research, and in Anderson's early work,
is called severe combined immunodeficiency disease (SCID), and is very rare.
Genetically speaking, it affects a single gene, so it is more basic than many
But the proof is now in the pudding, and there's more to come.
Already, advances in cancer, heart disease, and others are close enough to
The new research "establishes as a fact that if you can get enough
gene-corrected cells into the body, you can correct the disease," Anderson
tells WebMD. "So it's been done first with SCID; now it's a matter of
working out the conditions for other diseases. In fact, hemophilia [treatment],
looks like that's going to be successful; growing new blood vessels for
cardiovascular disease looks successful; some of these gene-based vaccines for
cancer ... those look very promising." Using gene therapy to treat the AIDS
virus also shows huge potential, he says.