Tragic Turn in Gene Therapy: A Fatal Incident that Halted Progress for a Decade - Sep 17, 1999
In September 1999, a tragic event marked a significant turning point in the history of gene therapy. On that fateful day, 18-year-old Jesse Gelsinger lost his life following an experimental treatment for his rare genetic disorder, ornithine transcarbamylase (OTC) deficiency.
Jesse, who had a milder, 'late-onset' form of the disease, managed his condition through a strict diet and a regimen of 50 pills a day. However, in 2021, scientists had successfully treated OTC deficiency using gene therapy, raising hopes for more effective treatments.
Jesse enrolled in a gene therapy trial at the University of Pennsylvania, which aimed to correct the defective OTC gene using a weakened form of an adenovirus. On September 13, 1999, the treatment was infused into the artery feeding Jesse's liver.
However, investigations later revealed that Jesse's death was caused by a severe immune reaction to the virus used to deliver the treatment. This revelation led to a series of investigations, which uncovered numerous problems with Jesse's enrollment in the trial. These included poor liver function and undisclosed risks to patients.
Following Jesse's death, all gene therapy trials underway at the University of Pennsylvania were halted. The Food and Drug Administration (FDA) also started requiring greater monitoring for gene therapy trials. These changes were designed to prevent similar tragedies from occurring in the future.
Dr. James Wilson, the lead investigator, owned stock in Genovo, the company developing the therapy, and stood to gain millions if the therapy was successful. Paul Gelsinger, Jesse's father, launched a wrongful-death suit against parties involved in the trial. The settlement for the suit was undisclosed.
Despite this tragic event, gene therapy has continued to evolve and make strides in treating rare genetic disorders. In 2024, the first CRISPR-based gene therapy, which treats sickle cell anemia, was approved. Since then, scientists have used gene therapy to treat many rare genetic disorders, including severe combined immune deficiency and multiple forms of blindness.
In 2025, scientists announced they used a customized CRISPR treatment to treat a baby with a rare and devastating genetic syndrome. This advancement underscores the potential of gene therapy to revolutionize medicine and provide hope for those suffering from rare genetic disorders.
Jesse Gelsinger's death served as a stark reminder of the ethical and safety concerns surrounding gene therapy. However, it also spurred necessary changes in clinical trials and has paved the way for a more promising future in the field of gene therapy.
Read also:
- Inadequate supply of accessible housing overlooks London's disabled community
- Strange discovery in EU: Rabbits found with unusual appendages resembling tentacles on their heads
- Duration of a Travelling Blood Clot: Time Scale Explained
- Fainting versus Seizures: Overlaps, Distinctions, and Proper Responses
