General interest piece, FWIW...
Sunday November 7 2:09 PM ET
Experts: Gene Therapy More Hopeful In Heart Disease
By Maggie Fox, Health and Science Correspondent
ATLANTA (Reuters) - Using gene therapy to treat patients with heart and artery disease -- often
helping them literally grow their own bypasses -- looks more hopeful than ever, researchers said
Sunday.
They also took the opportunity to defend their experimental research against reports alleging they
had failed to report deaths to the proper authorities, saying the deaths were reported and were not
due to the gene therapy itself.
Several teams of researchers are now experimenting with the idea of introducing genes into the
body to help it grow new blood vessels. They are testing these techniques in patients who cannot
benefit from standard therapies, including bypass surgery, to treat clogged arteries.
The very first experiment, reported at the American Heart Association's annual meeting three years
ago, was performed by Dr. Jeffrey Isner of St. Elizabeth's Hospital in Boston and colleagues. He
used gene therapy to help patients grow new blood vessels in badly blocked legs.
Researchers have reported solid progress since, Dr. Valentin Fuster, director of the cardiovascular
institute at Mt. Sinai Medical Center in New York, told a news conference at the current meeting
of the Association, which opened in Atlanta Sunday.
''Today we can say it is almost clinically applicable,'' Fuster said, meaning the technique is almost
ready to use in patients outside experimental research.
Isner and others have also reported good results using gene therapy in patients with blockages to
their hearts.
''I think there is an awful lot of promise in gene therapy,'' Dr. Lynn Smaha, president of the
American Heart Association, said in an interview. ''We are on the verge of uncovering what
possibilities there are.''
As many as half of the more than 500,000 bypasses performed each year in the United States fail,
and doctors are keen for an alternative.
But the treatments have become controversial in recent weeks, with the news that an 18-year-old
patient receiving experimental gene therapy for another condition -- completely unrelated to heart
disease -- died from the treatment itself in Pittsburgh.
Newspapers reported that Isner and another doctor working in the field, Dr. Ron Crystal of Tufts
University, had failed to report deaths among their patients to the National Institutes of Health
(NIH), although they did report them to the Food and Drug Administration (FDA).
Both agencies play roles in regulating gene therapy.
Isner, currently working on three separate trials involving 72 patients, denied he had failed to
report anything. ''There have been a total of two deaths,'' he said at the news conference. ''Both
were promptly reported to the FDA. In both cases the FDA agreed ... there was no need to stop
the trial.''
Considering that he was working with very ill heart disease patients, this was a low death rate,
Isner said.
Dr. Todd Rosengart, chief of cardiothoracic surgery at Evanston Hospital outside Chicago, is
working with Crystal on one of the other trials. He said he and other gene therapy experts are very
concerned about safety.
''We had a meeting yesterday ... (among) all the investigators in the field,'' Rosengart said. ''It is
obviously very critical and very important to me to do what is safe,'' he added. ''We looked at
every case.''
The researchers noted that patients chosen for their trials are chosen because they are so ill, and
the experimental therapy is their last resort.
''These are very sick patients. The reason we were offering them therapies that are research
therapies is because they have, quote, no option,'' Rosengart said.
The researchers denied reports that they had declined to report the deaths to the NIH. ''We are
happy to go along with whatever the federal agencies decide is appropriate, so long as it is clear
and consistent,'' Isner said.
He added that he also reported the two deaths to the NIH's Office of Recombinant DNA Activity,
which oversees gene therapy trials. |
