Sept. 20, 2000 (Washington) -- Almost exactly a year after 18-year old Jesse Gelsinger's death during a gene-therapy experiment, Gelsinger's family has filed a lawsuit against the University of Pennsylvania, where the treatment took place. Gelsinger was the first person known to have died from a gene-therapy treatment.
Also named in the 15-page complaint, filed Monday in Philadelphia, are James Wilson, MD, the lead researcher in the case, and Arthur Caplan, PhD, director of the bioethics program at Penn.
Gelsinger suffered from ornithine transcarbamylase deficiency, a rare liver disorder, and according to the lawsuit, wasn't fully told of the risks of the experimental procedure. Patients with ornithine transcarbamylase deficiency can experience a buildup of a ammonia in the blood, which can be fatal if the levels get too high. During the experiment, Gelsinger's doctors injected the Arizona teen with a cold virus that had been altered to carry a gene they hoped would control the condition.
The lawsuit asks for unspecified damages because of what it describes as carelessness and gross negligence on the part of the doctors. After an investigation of the gene-therapy program at Penn, the FDA shut down the program, citing numerous safety violations -- including a failure to report that other patients had experienced liver problems from the treatment.
"The FDA has made a number of findings that we would hope the jury to make," Alan Milstein, the Gelsinger family's attorney, tells WebMD. He describes the case as a unique example of "scientific malpractice."
Although Penn officials declined to comment directly on the lawsuit, they issued a statement that said Gelsinger's care "met the highest standards," while conceding there were some "weaknesses" in monitoring gene-therapy experiments. University officials say they are already negotiating the case, and promise to respond to the suit "in due course."
Another unusual aspect of the case is the involvement of high-profile ethicist Caplan as a defendant. The suit says he helped to write the informed-consent document for the treatment -- a form that is supposed to inform participants about a therapy's potential risks and benefits -- and consulted with Wilson about other ethical aspects of the experiment.
Caplan says he can't comment directly on the case, but he fears the suit may lead to fallout for those who give ethical advice.
"I'm nervous that even the appearance of a suit ... could have a chilling effect on bioethics conversation at institutions," says Caplan, who tells WebMD he stands by his original guidance to Wilson. That advice, according to the lawsuit, was to avoid enrolling terminally ill infants in the study, as the researchers had originally considered. Instead, Caplan reportedly told the doctors to perform the treatment on adults with a milder version of the affliction, who could give their consent.
The lawsuit also alleges that Wilson stood to benefit financially from the Gelsinger experiment because of his interest in a company doing gene-transfer research. It charges that the university, as well as a former dean who is also a defendant, had a stake in the business as well.
The lawsuit further says Gelsinger's ammonia levels were too high to safely undergo the procedure.
After receiving the treatment on Sept. 13, 1999, Gelsinger's condition began to deteriorate rapidly. His fever climbed to 104 degrees within hours, and he slipped into a coma the next day. By the time he died, many of his organs had failed and he was so swollen that he was unrecognizable.