Accusing the Health and Human Services department of moving too slowly to protect patients in clinical trials, senators said Thursday that they were considering legislation to make experimental gene therapy safer.
Tennessee Republican Senator William Frist, who led a recent investigation of gene therapy in Congress, said he was frustrated by a lack of action by HHS to ensure that patients know the risks of taking part in clinical trials.
"It's no action, no action, no action, no action," Frist told a subcommittee hearing, holding up a checklist of recommendations made in 1998 by HHS Deputy Inspector General George Grob. He said HHS had acted on only three of 17 proposals.
Grob's report, issued after a series of abuses were made public, found that patients volunteering for trials often did not know what they were in for and, for example, often did not understand that they may not personally benefit from the experimental treatment being studied.
The issue came up again in September 1999 with the death of Jesse Gelsinger, 18, the first patient to die as a direct result of a gene therapy experiment. An inquest into gene therapy found researchers were not following many rules, including reporting "adverse events" such as the death of a patient to the National Institutes of Health.
Gelsinger's father Paul later told Congress he felt he had not been fully informed of the risks his son was taking.
The center where Gelsinger was treated at the University of Pennsylvania said on Tuesday it was stopping all human experiments and would instead focus on animal and laboratory research. Its work involving people has been suspended by the U.S. Food and Drug Administration.
On Wednesday HHS said it would tighten the so-called informed consent process, in which patients are told of the risks of volunteering for a clinical trial.
It said it would ask Congress for legislation calling for civil fines of between $250,000 and $1 million for violating regulations. HHS also said it would issue new guidelines, including one making scientists tell each patient about any unexpected death or serious adverse health event related to their clinical trial and get their consent all over again.
But Frist, chairman of the Senate subcommittee on public health, said HHS had moved too slowly. "Do we need legislation to ensure that two years from now or six months from now we don't have a repeat of that?" he asked.
"Actually, action has been taken on most of the points," HHS Secretary Donna Shalala responded in an interview. But she said she would not resist legislation.
"I indicated to Senator Frist in a telephone conversation yesterday that we would work with him," she said.
Senator Arlen Specter, a Pennsylvania Republican who has taken an interest in experimental medical issues, said he would support Shalala's proposals to impose fines on researchers who do not follow the rules.
"I think legislation is necessary," Specter said.
Health officials admitted there was work to do but said they were pressing ahead.
"It is true that we are discovering problems as they move forward and they will be addressed," said Kathryn Zoon, director of the Center for Biologics Evaluation and Research at the FDA. "Although we are nowhere near where we want to be, we have made considerable progress."
William Raub, deputy assistant secretary at HHS, said a better job could be done of letting patients know about conflicts of interest that might influence a researcher.
He said researchers and often universities had set up or owned stock in companies that stood to profit from the research. "There is nothing inherently wrong in this," Raub said, but added that regulators needed to keep an eye on it.
"We can move the ball considerably down the field by working within the framework of current regulations," he added.
Dr. Savio Woo, a gene therapist at the Mount Sinai School of Medicine in New York and president of the American Society of Gene Therapy, said HHS, the NIH, and the FDA should be given a chance to make changes. "We must give the administration an opportunity to implement them now that the senator has voiced his concern so powerfully," Woo said.
"If it turns out that progress is not satisfactory, then I don't see any alternative."
Woo said gene therapy researchers had taken the matter seriously.
Industry officials urged against legislation. "Policies and procedures that affect an evolving technology such as gene therapy need to be flexible enough to evolve with the science," Lisa Raines, senior vice president of Genzyme Corp., told Thursday's hearing.
