Introduction

French and British researchers have treated X-linked severe combined immune deficiency syndrome (X-SCID), otherwise known as “bubble boy” disease. Italian and UK researchers have also treated a related disease, ADA-SCID. Though only three patients were enrolled in the study, Swiss and German researchers have treated yet another severe immune disorder, chronic granulomatous disease. And some commentators believe American researchers are on the cusp of a durable treatment for hemophilia B. In these instances, it would appear that gene transfer – briefly, the administration of genetic materials to human beings – has finally earned the title of gene therapy.

But the field's development has been, and continues to be, a long, strange trip. As I write, the most visible name associated with gene transfer, W. French Anderson, is serving a fourteen-year prison sentence on child molestation charges. Another leading figure, James Wilson, has nearly finished a five-year, FDA-imposed ban on leading clinical studies. Other sanctions in the field's thirty-year history include one of the earliest ever violations of rules for human research issued by the US Department of Health and Human Services (Martin Cline, for initiating a study without proper IRB review), and a widely publicized rebuke of two other leading figures (Ronald Crystal and Jeffrey Isner) for not reporting trial deaths to the NIH. In 1995, a high-level panel at the NIH faulted the field for rushing into clinical trials. 5 In 2000, two prominent researchers editorialized in the pages of Science magazine “gene therapy has many of the worst examples of clinical research that exist.”