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US faces ethical issues after gene therapy death

BMJ 2000; 320 doi: (Published 04 March 2000) Cite this as: BMJ 2000;320:602
  1. Janice Hopkins Tanne
  1. New York

    Gene therapy is no different from other kinds of risky medical research, said Dr Arthur Caplan, professor and director of the Center for Bioethics at the University of Pennsylvania in Philadelphia, speaking at the New York Academy of Sciences earlier this week. Better regulation of all research is needed.

    A gene therapy experiment at the University of Pennsylvania led to the death of Jesse Gelsinger, an 18 year old man with an inherited metabolic disorder. Dr Caplan, a well known ethicist and author, was not involved in the experiment.

    Ethical issues about genetics have been discussed for years, but, said Dr Caplan, “the American culture has a hard time getting political consensus on what to do. We believe in freedom. The British, the Australians, the Dutch all have a much more regulated view than we do, but they don't have the same commitment to privacy that we do,” Dr Caplan said.

    Privacy of medical information is important in the United States because knowledge of genetic disorders may make the person “uninsurable, unemployable, potentially even unmarriageable,” according to Dr Caplan. The problems in gene therapy are really problems in review and oversight of clinical research.

    The press asked why a healthy young man had entered the trial. Dr Caplan said the man had not been healthy. He had had to take 40 pills a day to control his metabolic disorder and had four life threatening crises a year. No one with his disorder had lived past 27.

    Patients cannot be entered into a clinical research trial unless they may benefit. They must be healthy enough for the trial to show whether the treatment benefits them. If they are near death, the researchers cannot determine whether death resulted from the treatment or from the disease.

    They must also be able to give informed consent, which usually ruled out children or babies.

    The press hinted that researchers' ties to biotechnology companies might compromise studies. Dr Caplan pointed out that such ties had been encouraged by federal law for 20 years, to bring research efforts to the market place and benefit patients. Almost all leading researchers had such ties.

    “The area is rife with hypocrisy,” said Dr Caplan, who enrolled in a clinical trial himself and saw problems with incomplete information and coercion of subjects. “The system is broken. It's been broken for a long time. It took gene therapy to make it evident. Monitoring is non-existent. Adverse event reporting is a joke.”

    To repair the system, Dr Caplan recommended tougher monitoring, debriefing of at least 5% of study subjects by institutional review boards, random audits of institutions conducting trials, and better coordination between data safety and monitoring boards and institutional review boards to share information on adverse outcomes.

    Finally, he suggested a “no fault” insurance fund to compensate for medically related injuries instead of the current system of malpractice lawsuits.

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