These People Risked their Lives to Advance Medicine and Cure Disease

These People Risked their Lives to Advance Medicine and Cure Disease

Larry Holzwarth - April 19, 2020

These People Risked their Lives to Advance Medicine and Cure Disease
Jesse Gelsinger died after volunteering for a study in gene therapy to develop a cure for a rare disease. Science History

19. Jesse Gelsinger

Jesse Gelsinger suffered from a relatively rare genetic disease of the liver, usually fatal at birth. His milder form of the illness allowed him to survive, based on a strictly controlled diet and a regimen of medications. Those suffering from the disease are unable to metabolize ammonia, which is produced by the body when breaking down proteins. Jesse was diagnosed with the disease at the age of two, leading to his growing up while taking more than four dozen different pills per day. Despite occasional severe health problems as a result of his illness, Jesse attended elementary and high school, developed a love for motorcycles, and worked a part-time job. In 1999, at the age of 18, he learned of a clinical trial for a potential treatment for the disease (known as OTCD) being held at the University of Pennsylvania in Philadelphia.

Jesse flew from his home in Tucson to take part in the study as a volunteer. The study included the addition of an enzyme to the OTC gene, preventing the buildup of ammonia to lethal levels in the body. Recipients of the virus normally developed mild flu-like symptoms. Jesse, the 18th person injected with the virus, developed symptoms including jaundice, liver failure, kidney failure, and lung failure. He was placed on life support within hours. Jesse died four days after being injected, after being declared brain dead. His death was a blow to the developing promise of gene therapy as a miracle cure for a variety of illnesses and conditions. Later investigators determined that Jesse had not been informed of several of the risks involved with the treatment when he volunteered to participate in the study.

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