Penn hit by gene therapy lawsuit

The family of Jesse Gelsinger filed a wrongful death suit against the University and others involved with Penn's Institute for Human Gene Therapy yesterday, a year and a day after the teenager died while participating in a Penn gene therapy research program.<P> The <a href="http://www.sskrplaw.com/links/healthcare2.html" target="_new"> complaint, </a> filed in Philadelphia Common Pleas Court, claims Gelsinger's death was a direct result of negligence by Penn, IHGT Director James M. Wilson and the two other scientists who ran the experiment in which Gelsinger was enrolled.<P> In addition, the suit alleges that Wilson and then-Health System CEO William Kelley owned several gene therapy patents and stood to gain financially from a successful outcome to the trial, and thus their judgement was compromised. <P> The Children's Hospital of Philadelphia, the Children's National Medical Center in Washington, D.C., IHGT investor Genovo, Inc. -- founded by Wilson -- and Arthur Caplan, the renowned director of Penn's Center for Bioethics, are also named in the suit.<P> The suit lists six causes for action, including wrongful death, fraud, emotional distress and assault and battery. It asks for $50,000 for each count, as well as punitive damages.<P> Filed on behalf of both Gelsinger's estate and his father, Paul, the suit echoes the violations that the U.S. Food and Drug Administration uncovered last year after a lengthy investigation of the IHGT that ended in a federal suspension of clinical gene therapy trials at Penn and a later decision by the University to end all human testing at the Institute.<P> None of the defendants named in the suit would comment last night, though Penn released a statement defending itself against the "one-sided" lawsuit and expressing sorrow over Gelsinger's death. <P> "Penn has readily acknowledged weaknesses in the IHGT's monitoring and oversight of the clinical trials. At the same time, the University continues to believe that these weaknesses did not contribute to Jesse's death," the statement read.<P> "We continue to regret deeply the death of Jesse Gelsinger and will do everything we can to work with Mr. Gelsinger's lawyer," University President Judith Rodin said last night. <P> Jesse Gelsinger was born with a mild form of ornithine transcarbamylase deficiency, or OTC -- a disease which affects the liver's ability to break down ammonia, a by-product of protein digestion. Most OTC sufferers die as infants, but Jesse's disease could be regulated with medication and a low-protein diet.<P> According to the lawsuit, Gelsinger agreed to participate in the gene therapy study at IHGT in the hopes of helping others with more serious forms of OTC, although the treatment would not benefit him personally.<P> On September 13, 1999, the lawsuit claims, Jesse was injected with an experimental vector. Over the next four days, Gelsinger showed increasingly serious symptoms, slipped into a coma and passed away on the afternoon of September 17.<P> One year later, his father is still facing the tragedy. "I experienced the pain on Saturday and for the whole week before that. I relived the whole experience," Paul Gelsinger told the Associated Press yesterday. "This should never have happened."<P> Though Gelsinger had originally defended Penn researchers, the complaint alleges that, while Jesse and Paul were trying to decide whether Jesse was a good candidate for the gene transfer trial, researchers Steven Raper and Mark Batshaw withheld important information about the risks involved in the trials.<P> Raper and Batshaw allegedly failed to mention that other patients in the trials had suffered serious adverse side effects, and that prior to Jesse's enrollment, monkeys injected with the same virus either became ill or died -- suffering many of the same symptoms that affected the 18-year-old during the days following his gene transfer.<P> In the suit, Paul Gelsinger claims he and his son were unaware that vectors had been stored in the lab for 25 months before they were used in animal experiments, which Paul Gelsinger now feels "may have resulted in an underestimation of the vectors' potency" during Jesse's trial.<P> Not only had the vectors in the Gelsinger trial been stored for only two months, the lawsuit alleges, the animals had been given "a dose of vector from 52.2 to 65.3 percent below the vector dose specified for humans."<P> The suit also claims Wilson and his staff were financially motivated in their research.<P> Wilson was the founder of the biotech company Genovo. By 1999, Genovo had agreed to fund the IHGT's research, giving them more than $4 million over five years to conduct genetic research. The University was given shares of the company, and Wilson, Batshaw, Raper and Kelley all owned shares.<P> In return, Genovo was given licenses for any gene therapy technologies developed under Wilson. The lawsuit alleges that this conflict of interest led the researchers to compromise patient safety.<P> In its statement, Penn denied that financial matters played any role in the mistakes that were made.<P> "The University categorically rejects the notion expressed in the complaint that financial gain played any part in any aspect of the OTC trial," it reads.<P> Originally, the study was designed for terminally ill infants with OTC. But according to the lawsuit, Caplan -- considered an authority on bioethics -- said parents of such infants could not give informed consent. Instead, he suggested the research be conducted on healthy adults with only mild forms of OTC, like Jesse.<P> The Children's Hospital of Philadelphia is also being held accountable since its Internal Review Board allegedly approved the protocol in the gene transfer experiment.