(Circulation. 2000;101:e9023.)
© 2000 American Heart Association, Inc.
Cardiovascular News |
Human Gene Therapy
Science Under Fire
When 18-year-old Jesse Gelsinger died September 17, 1999, while participating in a trial of gene therapy at the University of Pennsylvania Institute for Human Gene Therapy, his body responded to the introduction of a gene with an adenoviral vector in a totally unexpected and devastating way. However, the resulting firestorm of controversy, regulatory action, and headlines could have been predicted.
The university halted the study voluntarily, although Mr Gelsinger was the next-to-last patient scheduled to undergo the treatment for the rare metabolic disease caused by a defect in the gene that causes cells to make ornithine transcarbamylase, an enzyme necessary to normal metabolism of protein. In January, the US Food and Drug Administration (FDA) put a hold on the trial, which was a controversial study that used people who were not deathly ill to determine if a gene therapy for the disease was possible. Mr Gelsinger’s inclusion in the study was also questionable because of his youth and because he suffered from a less severe form of the disease that seemed to have resulted from a different gene mutation than the disease that puts infants into a coma 72 hours after birth and usually kills them soon after.
Until Jesse Gelsinger died, the most potent criticism of gene therapy
was that it had proved of so little benefit thus far. Even the first
trial in 2 girls who suffered from a form of severe combined immune
deficiency was tainted because the pair continued to receive injections
of peg-ADA, the synthesized