One year ago yesterday, 18-year-old Jesse Gelsinger died of complications from the gene therapy treatment he was receiving at the Hospital of the University of Pennsylvania under the direction of the Institute for Human Gene Therapy. The year since has been marked by a great contrast: rapid advances in the fields of genomics and gene therapy, and national embarrassment for Penn as the IHGT reeled from allegations of improper conduct in its gene therapy trials. Though still unproven, gene therapy holds great promise as a powerful weapon in the war against human disease. And with its investments in the biomedical sciences -- including state-of-the-art facilities and world-class faculty -- Penn should work to return to its former status at the vanguard of the revolution in genomics-based medicine. But it cannot do so with James M. Wilson, the IHGT's embattled director and lead researcher in the Gelsinger trial, affiliated with this institution. In the wake of the gene therapy fiasco, the University stripped the IHGT of its authority to conduct human gene therapy tests, rendering it a hollow shell of an institute. Instead, testing will be done by individual departments and schools. Penn should not try to shore up this decentralized -- but utterly unsatisfying -- structure. To remain a leader in the field, it needs a body like the IHGT to provide University researchers with a single primary source of guidance and support. Further, the resuscitated institute needs to be run with the same honor and integrity that is the hallmark of academic research at Penn. After the initial allegations against the IHGT, we gave Wilson the benefit of the doubt and an opportunity to mount a plausible defense and restore his credibility. One year later, he has failed to do so. The defenses proffered against many of the charges filed against Wilson and the IHGT is that many such lapses are common at other universities as well. So, to administrators and researchers alike, know that "everybody does it" is not a valid excuse. And failing to notify patients of the risks of their therapy -- among other charges against Penn -- is never trivial, and we suspect not very common. Recent successes in gene therapy elsewhere and the cracking of the human genetic code point to great potential for the field of gene therapy. And it is a field in which a tarnished Penn can again be a leader -- but only by making a bold statement by asking Wilson to step down.
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