Negative Publicity Puts Gene Therapy at a Crossroads

From the WebMD Archives

March 24, 2000 (Washington) -- Gene therapy studies hold great hope for treating disease and for enhancing lives. In the long run, it may someday cure cancer and enable the heart to grow new vessels to replace clogged ones.

But there have been frightening stories about gene therapy research since the death last year of 18-year-old patient Jesse Gelsinger in a study conducted at the University of Pennsylvania. Gene therapy studies at some major health institutions have been suspended while the government studies the safety of patients enrolled in the trials.

For the vast majority of the public, this is their first exposure to gene therapy research, and it's not a good one. Medical researchers are worried that the public will lose confidence in gene therapy. At risk is funding for studies, but more importantly, there is concern that patients will be unwilling to enroll in the trials.

It's certain that patients in some genetic therapy studies have died. But experimental gene therapy is being tried, for the most part, in patients who are very sick, usually terminally ill.

Based on what has been reported, at least so far, it appears that there are four main issues surrounding the therapy:

  • Have the researchers reported all the adverse events to the government promptly, as required by government rules?
  • Have patients in the studies been properly informed about the risks of participating in the studies?
  • How much information about adverse events that occurred during the gene therapy studies should be reported publicly, while the studies are taking place?
  • How safe are these techniques, as currently practiced?

The answers to some of these questions are emerging:

  • Researchers have not done an admirable job of reporting adverse events. Many say they reported properly to the FDA but not to the National Institutes of Health (NIH). Some say they did not fully understand the reporting requirements. Improvement is needed.
  • Some patients may not have been properly informed of the risks. That's one of the primary concerns in the death of Gelsinger. Improvement is needed.
  • A consensus is developing that serious adverse events from gene therapy studies should be made available while the studies are taking place. This is different from what is required for other clinical studies, in which adverse events are not reported until the study is published or made public. But the government believes that it is in the public's interest to know about gene therapy trials as they are taking place. All indications are that the research community and the companies supporting research agree.
  • As with any new therapy, there are issues of safety. Safety needs to be closely monitored and regularly reevaluated. Increased attention is being paid to this issue.

Both the NIH and the FDA are establishing new rules to assure that these needed improvements will be made. Health officials in Washington believe that more oversight is needed of gene therapy studies to assure that patients in studies are adequately protected. Even President Clinton has spoken on this matter.

So, if you are considering participating in a gene therapy study, be sure that you fit into the medical criteria of the study. (Every study has criteria that must be met by every patient who enrolls.) Most importantly, you should fully understand the risks and the potential benefits before agreeing to participate. If the informed consent form does not do a good enough job of explaining everything you are agreeing to, ask questions.

Gene therapy holds great hope, not only to help seriously ill patients enrolled in studies, but also in the long-term development of new and better medicines.

Such research, however, ultimately will succeed only if public confidence in gene therapy is restored and retained. Gene therapy is clearly at a crossroads. Hopefully, better public understanding of the real issues will allow research to move ahead with public and patient support.



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