Success is not built on success. It's built on failure. It's built on frustration. Sometimes its built on catastrophe. ~ Sumner Redstone
Gene Therapy Emerges From Disgrace to Be the Next Big Thing, Again
BY CARL ZIMMER
Courtesy of University of Pennsylvania Gene Therapy Program/NIH Laboratory of Structural Biology Research
Rarely does a whole life’s work crumble in a single week, but james wilson’s did. The first glimmer of impending ruin came on a Tuesday morning—September 14, 1999—as he sat in his office at the University of Pennsylvania. In his role as founder and director of Penn’s Institute for Human Gene Therapy, Wilson was one of the most prominent researchers in the nascent field, which sought to put genes into patients to repair their faulty DNA.
Wilson and his colleagues were adding the final patients to a two-year clinical trial, the ultimate goal of which was to treat a rare but devastating disorder. Called OTCD, or ornithine transcarbamylase deficiency, the genetic disorder renders its victims unable to process nitrogen in their blood. Nitrogen is created when protein is broken down, so the blood of OTCD sufferers becomes poisoned when they eat protein-rich foods: One bite of a hot dog can bring on a coma. As a result, just half of children born with OTCD—estimated at roughly one in 80,000 babies in the US, or 50 per year—live to the age of 5. Wilson and his colleagues hoped to treat this disease by giving sufferers a working copy of the defective gene they carry. To accomplish this, they engineered a virus carrying the functional gene; after successful trials of the virus in mice, they launched a clinical trial to test its safety in humans suffering from OTCD.
That Tuesday morning, Wilson received a call about one of the new patients in the trial: Jesse Gelsinger, an 18-year-old from Arizona. After Gelsinger received his dose of the virus on Monday, his temperature quickly climbed to 104.5 degrees—an unsurprising fact, given that the 17 previous patients had each experienced flulike symptoms after their treatment. But the following morning, there was worse news: His blood tests showed abnormally high levels of coagulation factors. It looked as if the young man’s body was seized with inflammation. “That was the first sign,” Wilson recalls, in a tone of composed regret, “that things were headed in a different direction.”
Keep reading: Gene Therapy Emerges From Disgrace to Be the Next Big Thing, Again – Wired Science.
