Seeking justice for my son

Ten years ago today, my 18-year-old son, Jesse Gelsinger, died at the University of Pennsylvania in a gene-therapy trial. Who is responsible? Could his life have been saved? Are other patients at risk? These questions have yet to be fully answered.

Jesse lived with a rare metabolic disease. The point of the research trial was to see if an adenovirus (a cousin of the cold virus) could safely deliver corrective genes to Jesse's liver. Instead, the adenovirus killed Jesse.

Jesse became the poster child for what not to do in human-subjects research. Neither he nor I was warned that, as the Washington Post later reported, monkeys had died in a prior trial. And the Wall Street Journal reported that the researcher who developed the adenovirus, James Wilson, had a seven-figure financial interest in the trial's outcome, as did Penn.

I stood by Penn, Wilson, and the two principal investigators, Mark Batshaw and Steven Raper, until I understood the extent of Wilson's financial ties and the extent to which Jesse was misled about the risks and efficacy of the therapy. I eventually sued and ultimately settled. The amount of the settlement was sealed; the documents were not.

The federal government also sued, claiming that the researchers blew through clinical stop signs in conducting the trial, ignoring evidence that Jesse was not well enough to receive the adenovirus and failing to sufficiently alert the Food and Drug Administration about severe incidents involving prior patients. The researchers disagreed, saying they had the discretion to proceed despite Jesse's test results, and that they had alerted the FDA. The government also reached a settlement, which restricted the researchers' activities for a period of years.

Before the government settled, I urged federal officials to make the documents they collected public. They refused, saying this is simply not done.

I believe a better understanding of what happened to Jesse could improve practices. So I gave the documents collected for my lawsuit to a law professor, Robin Wilson, who teaches at Washington and Lee University in Lexington, Va. They appear in her new book, Health Law and Bioethics: Cases in Context.

While they are incomplete, the documents show a chilling pattern. They show that a lot of good people inside Penn raised alarms about Wilson's financial ties from the get-go, but that Penn approved the deal anyway. The documents also show misleading disclosures to Jesse about the risks posed to him. For example, although animals died in prior trials, we were told that "animals have not shown toxic effects ... at the dosage of virus that is needed to transport the gene in this study."

I thought the lawsuits brought by me and the government would change research practices and the rules governing research. When I settled, real reform seemed likely. The Senate had held hearings, the FDA was investigating whether mistakes were made in the trial, and influential medical bodies such as the Association of American Medical Colleges had begun to examine disclosure practices and financial ties.

But, sadly, we have not yet learned enough from Jesse's death. The shroud of secrecy that envelops legal settlements has helped hinder reform. No one has publicly accounted for the mistakes that led to Jesse's death.

We don't know whether the FDA was misled or dropped the ball. We don't know whether the researchers' claims of efficacy had any basis in fact or were just wishful thinking. We don't know why Penn approved the deal despite warnings. And we don't know whether the researchers' decision to administer the virus to Jesse was reasonable or reckless.

Ten years ago today, my son died in a science experiment. A complete record of what the researchers and FDA regulators knew is the best precaution against future tragedies like Jesse's death.

I am asking that the University of Pennsylvania and the FDA finally do the right thing and release their records. If they did nothing wrong, let us see the proof. If they made a mistake, let us all learn from it and do better in the future. We owe it to Jesse to make his life and death mean something.