Book contents
- Frontmatter
- Contents
- Acknowledgments
- List of abbreviations
- 1 Introduction: gene transfer lost in translation
- 2 What is gene transfer?
- 3 Safety, values, and legitimacy: the protean nature of risk in translational trials
- 4 Taming uncertainty: risk and gene-transfer clinical research
- 5 Succor or suckers? Benefit, risk, and the therapeutic misconception
- 6 Looking backward: a model of value for translational trials
- 7 The chasm: the ethics of initiating first-in-human clinical trials
- 8 Tropic of cancers: gene transfer in resource-poor settings
- 9 Great Expectations and Hard Times: expectation management in gene transfer
- 10 Something in the sight adjusts itself: conclusions
- Epilogue
- Index
1 - Introduction: gene transfer lost in translation
Published online by Cambridge University Press: 28 January 2010
- Frontmatter
- Contents
- Acknowledgments
- List of abbreviations
- 1 Introduction: gene transfer lost in translation
- 2 What is gene transfer?
- 3 Safety, values, and legitimacy: the protean nature of risk in translational trials
- 4 Taming uncertainty: risk and gene-transfer clinical research
- 5 Succor or suckers? Benefit, risk, and the therapeutic misconception
- 6 Looking backward: a model of value for translational trials
- 7 The chasm: the ethics of initiating first-in-human clinical trials
- 8 Tropic of cancers: gene transfer in resource-poor settings
- 9 Great Expectations and Hard Times: expectation management in gene transfer
- 10 Something in the sight adjusts itself: conclusions
- Epilogue
- Index
Summary
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.”
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- Chapter
- Information
- Gene Transfer and the Ethics of First-in-Human ResearchLost in Translation, pp. 1 - 7Publisher: Cambridge University PressPrint publication year: 2009