COLUMN: A death on the road to destiny

From Binyamin Appelbaum's, "Carving Marble," Fall '00 From Binyamin Appelbaum's, "Carving Marble," Fall '00Somewhere in France, a pair of 18-month-old boys are fast approaching their second birthdays. Born without the ability to produce two kinds of cells central to the human body's ability to fight infections, the two boys should be dead. Somewhere in Arizona, the Gelsinger family is mourning the loss of their 18-year-old son, Jesse. Jesse Gelsinger was born without the ability to properly process ammonia, a poisonous by-product of protein digestion. His was a mild case; thanks to dietary controls and medication, he was in no imminent danger from the often-fatal deficiency. But Gelsinger agreed to participate in a Penn-sponsored gene therapy study seeking a cure for his condition, called OTC -- ornithine transcarbamylase -- deficiency. Four days after receiving an infusion of healthy genes, he died of lung failure. · The theory behind gene therapy is simultaneously breathtaking and elegantly simple. It proposes nothing more or less than to replace flawed genes with healthy ones. To rewrite the very code that makes us who we are. The problem is that gene therapy isn't therapeutic. In over 300 trials involving some 6,000 patients, gene therapy has not yet been shown to cure anything. Even the genes infused into the two French boys may have taken up residence in short-lived cells. Or they may simply cease to function at some future point. We just don't know. But what if. What if we could cure Jesse Gelsingers and little French boys. What if, for that matter, we could cure cancer or cystic fibrosis. Those are what-ifs with a lot of money riding on them -- money that has been invested in places like Penn's Institute for Human Gene Therapy. · According to the FDA, the Penn team that treated Jesse Gelsinger broke just about every rule in the book. The researchers -- led by James M. Wilson -- failed to obtain proper informed consent from any of their patients. They experimented on patients who were too sick to meet the study's standards. And they failed to stop the experiment after four successive volunteers experienced severe and unexpected side effects. The fifth was Jesse Gelsinger. He died on September 17, 1999. · Who knows why James Wilson decided he didn't have to play by the rules. Newspapers have been quick to point out that Wilson had a substantial financial interest in the outcome of the study through his stake in Genovo, a gene therapy company that provided funding for his institute at Penn. But this is not the story of a drug pushed too quickly to market. Any financial rewards lay in the distant future at best. And Wilson, a man who commanded the near-universal respect of his colleagues, was also past president of the American Society for Gene Therapy. Perhaps Wilson simply believed in his work, believed in his ability to cure the sick so completely that he pushed ahead against the odds. Perhaps he believed so deeply that he was willing to risk the lives of others on his own cognizance. And perhaps he must now live with the reality that a life has ended and he is no closer to preventing other lives from ending. He is no closer to creating a cure. · Would Jesse Gelsinger have volunteered to participate in Wilson's study if he had known that previous patients had experienced complications? Maybe. And if not Jesse Gelsinger, then perhaps someone more desperate than he. And if not this study, then some other study. Gelsinger's story is the tale of the march of progress trampling an innocent sacrifice underfoot. The predictable outcry. The call for more regulation. The certainty that other lives will be put at risk in future gene therapy experiments. The possibility that a treatment for a previously incurable disease will result. Actors, to your places. The march must go on. Not because we do not fear the results; not because we are always so certain of our place in this brave new world; but because progress truly is inevitable. There is, after all, a lot of money riding on it.