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Gene Therapy Trial Halted After Death, FDA Investigates
July 30, 2007
A gene therapy that may have killed a clinical trial participant last
week drew the concern of an expert review panel four years ago, reports
the Washington Post.
The latest trial, sponsored by Seattle's Targeted Genetics, was
designed to test a treatment for inflammatory arthritis. The people
involved had a virus injected directly into their joints, where it
delivered genes that blocked the production of tumor necrosis factor,
the substance underlying the most crippling forms of arthritis.
In 2003, NIH reviewers asked the company why "such a novel and possibly
risky approach was to be offered to patients who were not especially
ill, including some who had not even tried standard treatments.
They also pointed out that animal studies had shown a limited benefit,
that the therapy's risks hadn't been adequately documented, and that
the company hadn't clearly told patients "that the study was unlikely
to help them and was designed merely to test the new approach's
The company reportedly remedied these flaws, and the trial went ahead
on 32 people, none of whom suffered serious side effects. The recent
trial involved 100 people, one of whom became ill and died. Details
about the death are unavailable at this time. It is possible that the
death was coincidental.
According to the Associated Press, 28 other ongoing gene therapy trials
involve the same virus, called adeno-associated virus or AAV. The FDA
shut down the Targeted Genetics trial and is reviewing the others.
At this point, it's too soon to jump to conclusions
about the therapy or the death. More on the facts as they become clear.
There are, however, some broad lessons to be drawn -- foremost among
them that, at this point, gene therapy should only be used to treat
critical conditions, and shouldn't be tested in people when it doesn't
Last week we posted on what individuals should think
about when considering taking part in a gene therapy trial. Please
share this with anyone you know who could use it.
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