Jesse Gelsinger

Jesse Gelsinger (June 18, 1981 - September 17, 1999) was the first person publicly identified as having died in a clinical trial for gene therapy. He was 18 years old. Gelsinger suffered from ornithine transcarbamylase deficiency, an X-linked genetic disease of the liver, the symptoms of which include an inability to metabolize ammonia - a byproduct of protein breakdown. The disease is usually fatal at birth, but Gelsinger had not inherited the disease; in his case it was the result of a genetic mutation and as such was not as severe - some of his cells were normal which enabled him to survive on a restricted diet and special medications.

Gelsinger joined a clinical trial run by the University of Pennsylvania that aimed at developing a treatment for infants born with severe disease. On September 13, 1999, Gelsinger was injected with an adenoviral vector carrying a corrected gene to test the safety of the procedure. He died four days later, September 17, at 2:30 pm, apparently having suffered a massive immune response triggered by the use of the viral vector used to transport the gene into his cells, leading to multiple organ failure and brain death.

A Food and Drug Administration (FDA) investigation concluded that the scientists involved in the trial, including the lead researcher Dr. James M. Wilson (U Penn), broke several rules of conduct:

• Inclusion of Gelsinger as a substitute for another volunteer who dropped out, despite having high ammonia levels that should have led to his exclusion from the trial
• Failure by the university to report that two patients had experienced serious side effects from the gene therapy
• Failure to disclose, in the informed-consent documentation, the deaths of monkeys given a similar treatment.

The University of Pennsylvania later issued a rebuttal^[1] but paid the parents an undisclosed amount in settlement. Both Wilson and the University are reported to have had financial stakes in the research.^[2][3] The Gelsinger case was a severe setback for scientists working in the field.^{[citation needed]}

Notes

1. ^ "Institute for Human Gene Therapy Responds to FDA - Almanac Between Issues". Upenn.edu. 2/14/2000. http://www.upenn.edu/almanac/between/FDAresponse.html. Retrieved 2010-11-16. 
2. ^ Greenberg, Daniel S. "Science for Sale. The Perils, Rewards, and Delusions of Campus Capitalism". Chicago: U. Chicago Press, 2007, 324pp., pages 104-106.
3. ^ "Don't Compromise Ethics in Human Experiments, Bioethics Expert Says". Law.virginia.edu. 2008-04-18. http://www.law.virginia.edu/html/news/2008_spr/milstein.htm. Retrieved 2010-11-16. 

External links

• Another Chance For Gene Therapy?: http://www.wired.com/news/technology/0,1282,31613,00.html
• Paul Gelsinger, Jesse's father, tells of Jesse's death: http://www.guineapigzero.com/jesse.html
• On gene therapy and informed consent (2008 debate): http://blog.bioethics.net/2008/01/on-gene-therapy-and-informed-consent/#comments
• BBC Horizon Trial and error - the rise and fall of genome therapy: http://www.bbc.co.uk/science/horizon/2003/trialerror.shtml

Persondata
Name	Gelsinger, Jesse
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Date of birth	June 18, 1981
Place of birth
Date of death	September 17, 1999
Place of death