Penn, Feds Revise Gene Therapy Plans

In the 8 months since a teenager died in a gene therapy experiment at the University of Pennsylvania, clinical researchers have been on tenterhooks to learn the consequences for Penn--and for national policy. Last week, they began to find out. Several federal agencies put forward proposals for sharply increasing the surveillance of human trials. And Penn's Institute for Human Gene Therapy (IHGT) is getting out of the clinical trials business.

Penn president Judith Rodin announced 24 May that the IHGT will focus on basic science and animal studies. The institute's six active clinical protocols had already been put on hold by the Food and Drug Administration (FDA); their fate is unresolved. However, 12 externally funded gene therapy trials under way elsewhere at Penn will continue.

Responding to congressional concern, federal agencies also announced plans to boost surveillance of clinical research. On 22 May, Health and Human Services (HHS) Secretary Donna Shalala sketched out a strategy that would elevate research safety concerns at HHS. Three days later, Shalala's chief scientific adviser, William Raub, testified before the Senate's public health subcommittee, issuing a long list of promises. HHS, he said, will ask grantee institutions to make sure that research subjects have given proper consent. Clinicians will now be asked to inform participants about any serious adverse events in related studies and "reconfirm" their consent in writing.

Meanwhile, the National Institutes of Health intends to ask clinicians to submit plans for monitoring the safety of smaller Phase I and Phase II trials. New reporting requirements are being considered that would require sharing more data from ongoing trials with local review boards.

One intrepid hearing witness--Savio Woo, president of the American Society of Human Gene Therapy--offered a contrary view. He warned that rushing forward with some new regulations could have damaging consequences. By requiring even small clinical trials to be managed by someone other than the principal investigator, Woo said, the government could impose "excessive costs that will stifle academic clinical research." Innovative and experimental projects would be hit hardest, he added.