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Bumps
in the Road
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"This has to be allowed to be rigorously tested," insists
Isner. "Cooler heads will prevail."
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But Insner has little more than "hints" to go on. In 1989,
after he injected the first genetically engineered human cells
into the first human patient, W. Anderson French told the
Washington Post, "We were ready for every problem except the
patient dying. That would have set the whole field back."
Ten
years later, Anderson's nightmare scenario played itself out
when a patient enrolled in a University of Pennsylvania gene
therapy trial died. Eighteen-year-old Jesse Gelsinger suffered
from a liver disorder that allowed poisonous levels of ammonia
to build up in his body. His death on September 17th, 1999
prompted the FDA to investigate and eventually halt all human
genetic experiments by the Penn researcher in charge of Gelsinger's
trial.
A
few months later, following the death of two of his 45 patients
and conflict-of-interest allegations, the FDA shut down Inser's
work as well. Inser maintained the patients died from the
end-stage heart disease that brought them to the study in
the first place. Despite not being able to collect data from
those trials, Isner saw some promising results, promising
enough for him to fight for the right to continue his trial.
"This has to be allowed to be rigorously tested," he insists.
"Over the last year, there has been a lot of concern about
the field of gene therapy in the wake of what was often inaccurate
reporting in the media. But, cooler heads will prevail. Gene
therapy is getting a second look right now."
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Today,
Isner patient Lillian Cooper is going strong, but the
future of the gene therapy remains uncertain. |
Studies similar to Isner's work with the VEGF gene are currently
underway at Cornell University and in Europe. Isner's optimistic
that this research will ultimately lead to the gene therapy
holy grail- learning how to mend the genetic typos that cause
heritable disease. "While our form of gene therapy does not
directly teach us about how to treat inherited disorders,"
Isner forecasts, "it makes sense that clinical experience
with it will give us tips and hints about how to do that one
day." 
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Scientific
American Frontiers is sad to report the death of Dr. Jeffrey
Isner. More on his life's work

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