Life as patient zero for medical discovery
What is it like to volunteer as patient zero for a groundbreaking clinical trial for Friedreich’s Ataxia? Quartz have published a fascinating interview with 19-year-old Josh, who two years ago became the first patient to enrol in an ambitious study to treat his condition using gene therapy.
“Friedreich’s Ataxia is like being drunk all the time except without the fun,” says Josh. “You are tired all the time and constantly feel off balance.”
“FA is a genetic condition in which the body fails to produce enough of a protein called frataxin. The lack of frataxin causes damage to the nervous system and weakens the heart. First the reflexes go; then the feet and legs slowly stop responding to messages from the brain.”
— Darlena Cunha, Quartz
Researchers from the University of Florida have developed a viral vector that they hope will deliver a normal copy of the human frataxin gene around Josh’s body.
You can read the rest of the interview here!