Wilson lays out plan in response to FDA

The FDA charged Penn's gene therapy program with violating several federal regulations. In a conciliatory letter to federal regulators on Friday, the embattled director of Penn's gene therapy program laid out the corrective steps his institute is taking to comply with federal research protocol that it allegedly violated. Most of the steps outlined in the letter from James M. Wilson, director of the Institute for Human Gene Therapy, deal with proper oversight for gene therapy clinical trials. Two major changes, both scheduled to begin in April, include hiring an outside consultant for IHGT researchers and implementing formal standard operating procedures. Wilson also announced the creation of a new IHGT position -- a scientific chief operating officer -- responsible for overseeing several aspects of research, including clinical trials. The letter to the U.S. Food and Drug Administration, which was made public yesterday, came in response to the FDA's scathing March 3 warning letter upholding its January decision to suspend all gene therapy experiments at Penn. In that letter, FDA officials criticized Wilson's monitoring of clinical trials at IHGT and accused him of violating research protocol while conducting the trial that cost 18-year-old Jesse Gelsinger his life last September. Wilson's latest letter did not attempt to dispute the accusations made in the warning letter. "IHGT does not agree with all those statements," Wilson wrote, "but it will not enumerate areas of disagreement in this response." Meanwhile, a committee of external scientists appointed by the University to review the institute's monitoring systems has already visited campus and is expected to release its findings in April. The charges levied against IHGT include that subjects were not properly informed about the dangers posed by the study, that the FDA was not notified of the deaths of lab animals undergoing treatment regimens similar to the human trial and that many patients were ineligible to participate. FDA spokesman Lawrence Bachorik would not comment yesterday on what the agency's next step would be. "The FDA will pay careful attention to the response and will factor it into our overall evaluation of the situation there," he said. Wilson was not available for comment yesterday. University spokesman Ken Wildes said IHGT is planning to hire Parexel International Corporation -- a consulting firm specializing in providing compliance services -- as a contract research organization to advise researchers on following regulations. "It just helps you maintain your perspective," he explained. "If you're so close to the research? sometimes it's hard to do some of the administrative work." Wildes said IHGT hopes to sign a contract with Parexel in April. But Arthur Caplan, the director of Penn's Center for Bioethics, emphasized that companies like Parexel act purely as consultants and do not assume responsibility for their client's studies. "Principal investigators are responsible for their research," he noted. "It's your study at the end of the day." Another major emphasis of Wilson's letter was the implementation of standard operating procedures -- sets of guidelines all researchers would follow. The new regulations are expected to go into effect on April 6. Among those being developed are guidelines for submitting information and possible study changes to the FDA, the National Institutes of Health and Penn's Institutional Review Board, which must approve all research studies. Other areas that will receive particular attention in the procedures are confirming patient eligibility and obtaining informed consent -- both of which were questioned by the FDA in Wilson's study. Under the new procedures, informed consent forms must be submitted in advance to the IRB. In addition, patient eligibility must be confirmed by the investigators as well as the quality assurance unit at IHGT -- the division that monitors protocol compliance. None of the procedures outlined in the letter are uncommon to an academic research institution, Caplan said.