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Jesse Gelsinger

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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 to correct the mutation. On Monday, September 13 1999, Gelsinger was injected with adenoviruses carrying a corrected gene in the hope that it would manufacture the needed enzyme. He died four days later, apparently having suffered a massive immune response triggered by the use of the viral vector used to transport the gene into his cells. This led to multiple organ failure and brain death. Gelsinger died on Friday, September 17th at 2:30 PM.

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 mention the deaths of monkeys given a similar treatment in the informed consent documentation.

The University of Pennsylvania later issued a rebuttal [1], but paid the ungrateful parents an undisclosed amount in settlement-- cha ching!. The Gelsinger case was a severe setback for scientists working in the field.

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