Jesse Gelsinger

From Wikipedia, the free encyclopedia
Jump to: navigation, search

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 apparently the result of a spontaneous genetic mutation after conception and as such was not as severe – some of his cells were normal, enabling 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 co-investigator Dr. James M. Wilson (Director of the Institute for Human Gene Therapy), broke several rules of conduct:

  • Inclusion of Gelsinger as a substitute for another volunteer who dropped out, despite Gelsinger's 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.[4]

Notes[edit]

  1. ^ "Institute for Human Gene Therapy Responds to FDA – Almanac Between Issues". Upenn.edu. 2000-02-14. 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. Retrieved 2010-11-16. 
  4. ^ "A History Lesson for Stem Cells". Sciencemag.org. 2009-05-08. Retrieved 2012-02-29. 

External links[edit]