A Times Editorial

Scrutinizing gene therapy

© St. Petersburg Times, published February 7, 2000

Gene-therapy experiments hold enormous promise for eliminating inherited illnesses, replacing damaged tissue and ultimately curing diseases such as cancer, AIDS and diabetes. When not abused, this revolutionary science, involving the transplant of properly coded genes to replace defective ones, has the potential to enhance -- and even extend -- human life. Yet its application also raises ethical dilemmas that deserve far more public debate than they have received thus far.

Officials at the National Institutes of Health deserve credit for advancing this medical debate and for proposing rules that promise to make gene therapy safer for patients. In a recent letter to Congress, NIH Acting Director Ruth L. Kirschstein wrote candidly about the dangers associated with genetic research, including the federal government's past problems overseeing gene therapy experiments.

Kirschstein admitted the agency has done a poor job of tracking patients involved in gene-therapy experiments and has failed to enforce rules requiring scientists to report information about harmful side effects. To remedy the problem, the institute has begun immediate inspections of laboratories that conduct genetic research. It also will expand education of scientists about the rules for reporting gene-therapy mishaps.

Gene therapy has grown explosively from its advent in 1990. However, despite the initiation of hundreds of studies and a growing financial investment by biotechnology companies hoping to profit from the new procedures, the field has yet to document its first success. In fact, gene therapy has come under increasing criticism in recent years by some scientists and ethicists who argue it is moving ahead too quickly, jeopardizing patients.

In one particularly troubling case, the federal government was forced to halt all human gene-therapy experiments involving a prominent researcher at the University of Pennsylvania. An investigation into the September death of a teenager there found the school's prestigious program in disarray.

Investigators at the U.S. Food and Drug Administration said they found numerous violations of federal research regulations and shortcomings in the protection of human subjects at the university's Institute for Human Gene Therapy. The university couldn't provide investigators with proof that volunteers in the study had been eligible to participate or had been adequately warned of the risks.

With so much at stake in gene-therapy experiments, such abuses simply cannot be tolerated. The NIH has performed a public service by stepping up its scrutiny of gene-therapy research and by demanding full and immediate disclosure of all potential problems associated with such treatments. Rigorous oversight by the federal government is the only way to prevent another tragedy such as the one that occurred at the University of Pennsylvania.

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