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    The Potential of Gene Therapy Threatened by Early Tragedies


    Caplan tells WebMD that even though some researchers have financial interests in gene treatments, money is not the main issue, as some news coverage suggests. "Ambition, arrogance, laziness, hubris are all much more important, and so is the drive to succeed and win -- be the first," says Caplan.

    Some of the reform efforts, which typically involve more regulations and more federal oversight, no matter how well intended, are also off the mark, says Caplan. "Oddly enough, it's the ethics of the scientist that count, [and] you can't be there in the lab -- you can't be there with every patient," says Caplan.

    One major issue for scientists is the multiple layers of regulation. Before human trials can begin, the investigators must win approval from local institutional review boards (IRBs), the safety boards that monitor human trials at individual institutions, then get an approval from the FDA, and in many cases, the National Institutes of Health.

    And that's just the tip of the iceberg. "There are four reviews that go on for every study before [an IRB] ever sees the trial, and then it's reviewed by 35 [IRBs] around the country at each of our sites before a human subject ever enters a trial," Robert Schooley, MD, head of infectious diseases at the University of Colorado School of Medicine, tells WebMD of his AIDS research.

    Schooley says it takes thousands of hours to assemble the paperwork necessary to get a major study off the ground. Just last year, Schooley says the FDA shut down the entire research program at the University of Colorado for about 5 months over some alleged discrepancies in paperwork. Thousands of studies were halted, and investigators could not enroll new subjects until the matter was resolved.

    "Patients with HIV infection in Colorado who wanted access to state-of-the-art clinical trials had no access for 6 to 8 months by the time all the studies were rereviewed," says Schooley. "It's bad for patients and bad for progress." A spokeswoman for the FDA declined to comment on the case.

    One solution, says Schooley, is the creation of data safety monitoring boards, which are capable of gathering and reviewing data from different sites all over the country. The board would theoretically be able to spot problems that isolated review boards might miss.

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