Picking Nits or Learning Lessons?

The full story of 36-year-old Jolee Mohr's recent death in a gene therapy clinical trial for rheumatoid arthritis is still unfolding. The study, sponsored by Seattle-based Targeted Genetics, remains on hold. A team of 20 doctors and scientists at the University of Chicago Medical Center has combed through autopsy samples. The FDA has yet to announce the direct cause of death, and the Recombinant DNA Advisory Committee (RAC) will discuss the case at its September 17 meeting.

There's a lot we don't yet know. But there's a fair amount we do know, and much of it is troubling. Gene therapy's high public profile has led journalists to investigate information released by Mohr's family that raises questions about the study's design, inclusion criteria, and conduct. Some are specific to the Targeted Genetics study while others are broader. Even if no one had died, a number of these issues would still be quite relevant given gene therapy's record to date: inconclusive at best, and certainly disappointing given the high hopes repeated over two decades.

One key concern is whether people whose...