Our research team was disheartened by The Post's interpretation of our motivations and actions in the design and implementation of our gene therapy clinical trial for ornithine transcarbamylase (OTC) deficiency -- a rare, and often fatal, inherited liver disorder ["Hasty Decisions in Race to a Cure?" front page, Nov. 21]. In addition to myself, The Post article named researchers Steven Raper and Mark Batshaw.
The OTC study has been through exhaustive and ongoing review by all appropriate oversight bodies (including the Recombinant DNA Advisory Committee of the National Institutes of Health, the Food and Drug Administration and relevant human subjects review boards), beginning two years before the trial began in April 1997.
All data from other published studies as well as from our own work in animal models were incorporated into the protocol design to further ensure patient safety. Seventeen volunteers participated in the OTC trial before Jesse Gelsinger; the patient who immediately preceded him received the same dose of the vector yet showed no unexpected adverse reactions.
The alleged lure of potential financial gain played no role in any clinical decisions. The decision to use adults in the protocol was based on the collective input and recommendations from the University of Pennsylvania's own bioethicists, as well as from families of diseased children and other metabolic disease experts not associated with the study. The informed-consent document signed by Jesse Gelsinger and all participants in the OTC trial accurately reflected risks, including death.
We have been pained and truly humbled by Jesse Gelsinger's unexpected and tragic death. As we continue to search for answers about the cause or causes of his death, we are sustained by the knowledge that our motives and actions have been consistent with the ideal of advancing our understanding of this devastating disease so that an effective treatment may be developed.
JAMES M. WILSON
Institute for Human Gene Therapy
University of Pennsylvania