Gelsinger case may spark new regulations

A proposed Food and Drug Administration "sunshine rule" has resurrected the spectre of the Jesse Gelsinger tragedy, while forcing the biotechnology industry to mobilize for a regulatory war. Next Wednesday -- the close of a federally-mandated public comment period on the new rule -- marks the next major step toward greater federal oversight of gene therapy trials, an effort spurred on largely by the 1999 death of 18-year-old Gelsinger in a Penn-sponsored study. Some involved in the Gelsinger case believe the regulation would prevent such tragedies from happening again, and they have expressed their views to the FDA. "If this regulation is quashed, as a similar effort was in 1995, then we will have more innocents like my son dying needlessly," Paul Gelsinger, Jesse's father, wrote in a letter to the FDA. The Tucson, Ariz., native claimed that the FDA's proposed "Disclosure Rule for Gene Therapy and Xenotransplantion Clinical Trials" would have prevented Penn researcher James M. Wilson from continuing the research that led to Jesse's death. The proposed FDA regulation, announced on January 17, "would provide public access to most of the study design and safety information about" gene therapy trials and interspecies organ transplants, according to an FDA press release. "Both of these technologies hold great promise, but they may also pose a remote, but unique risk to the individuals who have volunteered to participate in these types of studies," Jane Henney, then-FDA commissioner, said in January. In September 1999, Jesse volunteered to participate in Wilson's study at Penn's Institute for Human Gene Therapy. Wilson, director of the IHGT, was testing a new therapy which had patents held by Genovo, Inc., a company in which Wilson had a financial stake. On September 17, Jesse died suddenly after his body reacted to a protein on the surface of a virus he was given as part of the therapy. In the ensuing investigation, Wilson's team was implicated in a series of mishaps, most notably failing to adequately inform the FDA of the death of primates who had been given a similar treatment. Wilson, who was one of the most respected gene therapy researchers in the nation prior to the Gelsinger case, is currently the target of FDA proceedings aimed at disqualifying him from ever again participating in human drug trials of any kind. "In my personal experience, the researchers who advised my son and me unwittingly misled us into believing that gene transfer was working and safe," Gelsinger wrote. According to Alan Milstein, attorney for the Gelsinger family, the biotechnology industry has hired a team of professional lobbyists to stop the proposed FDA rule. "The industry is still attempting to keep material closer to the vest," Milstein said, explaining that, in his view, biotechnology companies want to keep adverse events quiet so as not to give an advantage to another company. "If one entity spends $10 million along a certain path and fails, the industry doesn't want the next company to skip that [step]," Milstein said. "The industry says in essence that Penn or Genovo would have a proprietary interest in the facts surrounding Jesse Gelsinger's death." But Mike Warner, a board member of the Biotechnology Industry Organization -- which is lobbying for nearly 850 companies and academic institutions -- denied any attempt to defeat the FDA rule. "We have no intention of derailing anything," Warner said. "It is critically important to balance the need for public disclosure and the need for information with a [research] sponsor's need to maintain its commercial confidentiality and trade-secret advantage." Despite Warner's denial of a defensive stance on the part of BIO toward the new FDA rule, a statement from the Seattle-based Targeted Genetics, indicates their displeasure with the regulation. "We believe that the release of raw data to the public in real-time, as proposed by this new rule, will hinder rather than enhance public understanding of the potential benefits and risks of participating in gene transfer clinical trials," the statement read. Targeted Genetics bought out Genovo last year. Its CEO, Stewart Parker, is a board member of BIO. In opposition to the biotech industry, there are some -- such as Milstein -- who believe Jesse Gelsinger's death could have been prevented by the FDA rule. According to Milstein, a top FDA official has told his client that Jesse Gelsinger would be alive today had the regulation been in place a decade ago. "That's a pretty horrific thought," Milstein said.

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