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Gene therapy researchers fined over trial participant's death

11 February 2005
By BioNews
Appeared in BioNews 295

Two US institutions that ran a gene therapy trial in which a teenager died in 1999 agreed to pay more than $1 million in a civil settlement last week. The government had alleged that the researchers, based at the University of Pennsylvania and the Children's National Medical Center, had failed to protect patients who volunteered for the experimental treatment. At the age of 18, Jesse Gelsinger took part in a gene therapy trial at the University of Pennsylvania for an inherited liver disorder called ornithine transcarboxylase deficiency (OTCD), but he died from multiple organ failure four days after beginning the treatment.

The allegations included charges of failure to halt the experiment when serious side effects first arose, failure to flag up the potential dangers of the study to participants, and falsely suggesting that earlier patients had benefited from the therapy. Under the terms of the settlement, the University of Pennsylvania team, lead by James Wilson, do not admit any wrongdoing. However, the University will pay a fine of $517, 496 to the government, while the Children's National Medical Center will pay $514,622. Wilson is barred from carrying out further experiments on humans until 2010, a ban which has been in place since 2000. His colleagues Mark Batshaw and Steven Raper will also have restrictions placed on their work.

Gelsinger's death was thought to have been caused by an immune response to the adenovirus used to deliver the therapeutic gene to his body. At the time, the Food and Drug Administration (FDA) immediately halted all gene therapy trials at the university, while an investigation was carried out. It revealed that the trial in which Gelsinger took part had not been properly supervised or monitored. The resulting public and scientific outcry triggered a reappraisal of the way in which 'adverse events' in gene therapy trials are reported in the US, and the National Institutes of Health called for gene therapy researchers to report all their past failures as well as successes. By February 2000, this request had brought to light 692 'serious adverse events' in gene therapy trials, only 39 of which had been reported previously.

It is not known how many of these failed experiments, which involved either serious deterioration or, in a few cases, death, were caused by the gene therapy itself. Patients enrolling on gene therapy trials are often terminally ill, and have not responded to any other form of treatment. However, some researchers feared that poor results were sometimes being kept under wraps because of the potential financial damage they might cause the funding companies. In March 2000, the FDA announced its new gene therapy clinical trial monitoring plan, designed to increase the level of scrutiny of gene therapy experiments. And new regulations unveiled in January 2001 include the requirement that researchers must publish details of any potential risks on the FDA's website.

Paul Gelsinger, Jesse's father, said that the rule changes for research involving humans that followed his son's death offered some consolation. But he told the Washington Post that he thought the government's decision not to pursue criminal charges or impose stricter penalties was disappointing. 'I wanted some accountability, and that's not going to happen', he said. He is also upset that none of the documents relating to the case are being released to the public, a move which he said would have ensured that 'real changes could be made in the way human research is conducted in this country'. David Hoffman, a prosecutor for the US Attorney's office, said that the fine would serve as a warning to researchers to comply with government regulations. 'The money is significant money', he said, adding ' but most important to me, quite frankly, is ensuring that research participant safety is number one'.

$1 Million to Be Paid Over Teen's Death in Gene Therapy Study
LA Times |  10 February 2005
$1M Settlement in Pa. Gene Study Death
Yahoo Daily News |  9 February 2005
Gene Therapy Settlement: Experts: Compared to other schools, Penn got off easy
Daily Pennsylvanian |  11 February 2005
U.S., Researchers Reach Deal in '99 Gene Therapy Case
The Washington Post |  10 February 2005
20 August 2007 - by Ailsa Stevens 
Last month a gene therapy trial for arthritis, carried out by US company Targeted Genetics, was halted when following the death of 36-year-old participant Jolee Mohr. Although the exact cause of her death is still unknown, the usually mild fungal infection found throughout Mrs Mohr's body...
22 January 2001 - by BioNews 
The USA's Food and Drug Administration (FDA) has proposed new regulations for gene therapy researchers, aimed at addressing public concern over the safety of patient trials. Sparked by the outrage that followed the death of teenager Jesse Gelsinger during a gene therapy trial in 1999, the new rules say that...
30 May 2000 - by BioNews 
The Institute for Human Gene Therapy at the University of Pennsylvania will no longer conduct gene therapy trials on patients, officials announced at a news conference last week. Dr Judith Rodin, president of the university, said it will also strengthen oversight of gene therapy research and other clinical trials, and...
7 February 2000 - by BioNews 
Hundreds of failed gene therapy experiments, including a number of deaths, came to light in the US this week. The reports flooded in to the US National Institutes of Health (NIH), after they asked researchers to provide information that might explain the death of Jesse Gelsinger during a gene therapy...
31 January 2000 - by BioNews 
The US Food and Drug Administration (FDA) shut down eight gene therapy trials at the University of Pennsylvania last week as a result of an ongoing investigation. All the trials have been halted 'indefinitely' after FDA findings of 'serious deficiencies' in the supervision and monitoring of one gene therapy trial...
4 October 1999 - by Juliet Tizzard 
Jesse Gelsinger's parents are no doubt still reeling from the news of their son's death. But as they try to get on with the rest of their lives, a public and professional debate about why he died is raging in the United States. Jesse, who was 18 years old, died...
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