Wednesday, June 26, 2024

Jesse Gelsinger Case, 1999 

17-year-old Jesse Gelsinger suffered from the genetic metabolic disease called orinthe transcarbamylase OTC deficiency this prevents the body from breaking down ammonia a metabolic waste product unless a person's diet is immediately adjusted and monitored throughout their life they will die soon after birth.

Although gel singer had controlled his OTC well he volunteered for a genetic therapy experiment in hopes of helping infants with a more severe form of the metabolic disorder he had a negative reaction to the treatment caused by severe immune response to the virus Vector used to deliver the corrective gene and died.

Three ethical issues with regard to gene therapy emerge from the Gelsinger case one should a relatively healthy adult volunteer with OTC have been used as a volunteer rather than newborns that were very sick too Gelsinger was told that the treatment was relatively safe however it emerged that past research subjects and experimental animals had become sick from the treatment was there adequate informed consent with regard to the risks three the lead doctor and institution had financial interest in the development of the treatment that was used if it had been successful they would have been well positioned to make a lot of money should researchers and research Institutes have a stake in clinical trials?

Wikipedia provides context on 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. 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 a milder form of the disease, in which the ornithine transcarbamylase gene is mutated in only part of the patient's cells, a condition known as somatic mosaicism. As his deficiency was partial, Gelsinger managed to survive on a restricted diet and special medications.

No comments:

Post a Comment