The victim's father will testify tomorrow during a Senate hearing. Penn's Institute for Human Gene Therapy will respond within days to the federal government's ban on all gene therapy research at Penn, University spokesman Ken Wildes said yesterday. In their response, the IHGT must address each of the 18 specific violations found by the U.S. Food and Drug Administration on the part of researchers involved in a study that left an 18-year-old dead last fall, sparking intense controversy over the Penn team's conduct and the entire field of gene therapy. Meanwhile, the father of Jesse Gelsinger, the subject who died in September, will testify tomorrow before a U.S. Senate committee examining federal oversight in gene therapy -- a field which seeks to cure diseases by essentially changing a patient's DNA. Paul Gelsinger, who until now has been a staunch supporter of IHGT Director James M. Wilson -- the study's leader -- said through his lawyer that he hopes the hearing yields information about the extent of possible lapses in FDA oversight in the field of gene therapy. The hearing will examine federal oversight of gene therapy as a whole, not specifically the Penn case. U.S. Sen. Bill Frist (R-Tenn.), chairman of the Senate subcommittee on Public Health that will hold tomorrow's hearing, said in a written statement that he hopes the proceedings will "provide a careful review of the oversight mechanisms in place for gene therapy trials." The hearing comes just five days after the FDA took the shocking step of suspending Penn's eight gene therapy studies. The purpose of Frist's investigation is to look into over The agency accused the IHGT in a two-page letter Friday of "numerous serious deficiencies" in the study in which Gelsinger was enrolled, and said that the Institute had not yet taken steps to correct those problems. The FDA's two-month investigation into the study raised "substantial concerns" about the safety of patients enrolled in the Institute's other studies. "We have to have assurance that [the IHGT] can conduct a clinical trial in the proper way [before the ban is removed]," an FDA spokeswoman said yesterday. Among the most serious of the violations alleged against Wilson and his research team are that researchers lied to patients about the risks involved, changed research protocols without notifying the FDA, included in the study patients like Gelsinger who should not have been eligible to participate and mishandled important documentation. Alan Milstein, the attorney representing Gelsinger's father, explained that his client thinks the FDA's action is appropriate. "The FDA report speaks volumes as to mistakes that were made," Milstein said. "Many of the observations made by the FDA suggest that Jesse should not have taken part in the experiment at that time." Milstein said his client is now in the process of evaluating all the information that is being released. He would not comment on the possibility of a lawsuit against the University. "Given what the FDA said about Penn's mistakes, he still has concerns about the FDA's oversight," said Milstein about his client. "It's too easy just to blame Penn." University President Judith Rodin directed Provost Robert Barchi on Friday to form an external committee of scientists to perform an institutional review of the IHGT. The committee will report directly to Rodin. But Wildes said he doubts the committee will make its final recommendations this semester. The ban on gene therapy can only be lifted after the University responds to the letter and the 18 allegations, and the FDA deems the IHGT's revised studies compliant with federal regulations. The process will likely consist of a back and forth exchange between IHGT scientists and FDA investigators, Wildes said, with the scientists submitting a reply and any proof that they were complying with FDA regulations for each allegation. At that point, FDA regulators would decide whether to accept each individual response. "Once we respond, the ball is in their side of the court," Wildes said. "We have pledged our cooperation." The FDA's ban drew national media attention to the University and its nationally renowned gene therapy program. It also increased the scrutiny on Wilson, an acclaimed researcher who once served as president of the American Society for Gene Therapy. Though Wilson remained unavailable for comment, he has consistently defended his work and maintained that there was nothing he or his colleagues could have done to prevent Gelsinger's death. Gelsinger, 18, who suffered from an inherited liver disorder known as ornithine transcarbamylase deficiency (OTC) died on September 17 after IHGT scientists, led by Wilson, infused genetically altered cold viruses into his liver. Wilson and his investigators later determined that Gelsinger died of multiple organ failure induced by the gene therapy.
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