Gene therapy: An Interview with an Unfortunate Pioneer

From Scientific American:

Gene-therapy-an-interview_1 Ten years ago this month the promise of using normal genes to cure hereditary defects crashed and burned, as Jesse Gelsinger, an 18-year-old from Tucson, Ariz., succumbed to multiorgan failure during a gene therapy trial at the University of Pennsylvania. Today the boardroom of the Translational Research Lab at the university is filled with artifacts reminiscent of the trial. Books such as Building Public Trust and Biosafety in the Laboratory sit on the shelves, and “IL-6” and “TNF-α” are scribbled on the whiteboard—abbreviations representing some of the very immune factors that fatally spiraled out of control in Gelsinger’s body.

These allusions to the past aren’t surprising considering how drastically the clinical trial changed gene therapy and, in particular, the career of James M. Wilson, the medical geneticist who headed Penn’s Institute for Human Gene Therapy, where the test took place. The U.S. Food and Drug Administration banned it from conducting human trials, and Wilson left his post at the now defunct institute (but he continued doing research at Penn). He disappeared from the public spotlight until 2005, when the agency announced he could begin clinical trials with a designated monitor but could not lead trials for five years and asked him to write an article about the lessons he has learned. He published it in Molecular Genetics and Metabolism this past April. Since then, he has begun giving university lectures about the importance of exercising caution as a clinical scientist, especially when it comes to stem cells, which today have the cachet once held by gene therapy.

More here.