Fortifying Genetic Research

By Britt Bailey

This past summer, I was bemused to learn Orrin Hatch, one of the Senate's staunchest conservatives, had loosened his grip on protecting human embryos. After much soul searching, Hatch said he now favored use of federal funds for research involving discarded or unused human embryos. "There is a difference," Hatch said, "between an embryo in a test tube and a baby in the womb."

In his newly found zeal to re-save disabled persons with embryonic cells and parts, Hatch is not only a beat ahead of the research community, but he may be missing the biggest story of all: embryos, children, prisoners and just plain old people like you and me are being conscripted into human research programs without long established safeguards. Leading research universities throughout the nation, institutions such as the University of Pennsylvania, Johns Hopkins, University of California at San Francisco, and Stanford, are conducting human research at an unrelenting pace in the search to extract the benefits from new genetic knowledge. The drive has left many participants in human studies stranded without the protections normally afforded by human subject committees which oversee the research conduct. Some are dying while our strongest ethical protections against research abuse are being neglected. Behind the debates surrounding the societal impact of new genetic developments, and nestled against the core of our most cherished values are standards which assure the protection of research subjects from infringement and exploitation. Where the existing regulations ( see 45CFR46) would normally protect folks participating in research from injury, we are finding instead a sequence of mis-steps and errors that have sometimes led to tragic endpoints. In some cases, the research has ended in the death of people like you and me: young adults on university campuses looking for a little extra money, or those generously donating to furthering the understanding of a particular disease. A case in point was the previously healthy Ellen Roche, a lab tech at Johns Hopkins University who died in early June from complications experienced after involvement in research. Her death followed that of Jesse Gelsinger who participated out of sheer good heartedness in order to advance the understanding of his particular disease so that others might be better treated.

These cases highlight the urgency of re-instituting better research protections as we enter an age of heightened public expectations for benefits from our costly success in cracking the human genome. There has most likely been no greater time in history when we should be assuring that the protections afforded human subjects are painstakingly thorough. To be sure, the explosive availability of new genetic information pushes us towards the brink of significant medical progress. But, that progress requires a hefty wave of research involving people. Without better safeguards, many will be at risk for hastily conceived or poorly executed experiments that neglect human rights.

There has always been controversy concerning the use of human subjects. Long ago, promulgators of standards of justice have held we should not use persons as a means to another's end. More than a half a century ago, a judgment at the Nuremberg "Doctor's Trial" set in motion rules we take for granted as adequate to protect persons enlisted for human research experiments. The center piece of those rules is the process of informed consent. Obtaining consent is a twofold process. Ideally, a person must be able to decide, free from persuasion and duress, to be involved in the research trial. The second element of the process involves not only acknowledging a subject's autonomy, but assuring that researchers are free from bias. The form should describe any commercial motives and conflicts of interest, the risks and benefits involved in the research itself, and all related pertinent information a participant may deem useful in making a voluntary decision.

But currently there exist loopholes in the protections. In the case of Jesse Gelsinger's death, neither the Food and Drug Administration nor Gelsinger's family had been notified of previous adverse reactions from the gene therapy technique involved. Ellen Roche, a Johns Hopkins lab worker, was not informed that she might be risking her life with an unapproved drug when she decided to participate in a clinical trial. Nor was she informed that the chemical compound she inhaled as part of the asthma research had been altered from the one cleared for the experiment by the Institutional Review Board. Had either of the participants been fully informed, they may have made different decisions regarding their involvement, decisions which may have saved their lives.

These examples also illuminate the lack of checks and reviews that ensue once a trial is stamped for ethical approval. Research institutions operate virtually free of oversight and scrutiny, unless and until someone is hurt. Typical consent forms signed by Gelsinger and Roche address the issue of potential benefits by simply stating, "There will be no direct benefit to you from allowing your tissue to be used for research. However, we hope we will learn something that will help in the treatment of future patients." And, as we have already seen, the risks were discounted or down-played in direct violation of the Hippocratic injunction not to harm.

While politicians, like Henry Hyde, swap sides of the bed and attempt to untie the ethical knots inherent in exploiting the much ballyhooed genetic techniques and treatments, others might do well to attend to the fortification of the research protections which will make any new medical progress possible. Before Christopher Reeves or Michael J. Fox promote the liberation of genetic science, perhaps they should look into the extraordinary level of disdain shown for ethical regulations by the research institutions at issue. Many others will be conducting trials to develop the science so facilely endorsed by our Senators and Congress persons. Before we begin these new adventures in genetic science, we would do well to remember that the upper levels of scientific wisdom cannot be reached without the basic floor of care and protection of persons necessary to make the research possible.

Dedicated to Ellen Roche, Jesse Gelsinger, and others injured or
violated during the course of participating in human research.