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
9 - Great Expectations and Hard Times: expectation management in gene transfer
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
In 2001, a report in Nature Genetics raised the possibility that a rare, hereditary form of blindness, Leber's congenital amaurosis (LCA), might soon have a cure. A team of researchers led by Jean Bennett of the University of Pennsylvania's Scheie Eye Institute had successfully restored vision to three dogs with LCA. According to news stories, the report “electrified” families with the disease; said one mother of an LCA child, “we are bursting at the seams.” Word spread like “wildfire,” according to Bennett, who received hundreds of inquiries from expectant parents.
The next year, the Alliance for Eye and Vision Research and the Foundation Fighting Blindness dispatched a team of researchers to meet with lawmakers during hearings on NIH appropriations. Traveling with them was Lancelot, one of the Briard mix dogs whose blindness had been partially corrected. One member of the research team, Cornell's Gustavo Aguirre, directed lawmakers to note the dog's posture: Lancelot always stood to one side – he favored his right eye because his left had served as the uncorrected experimental control. “If any of the investigators in the study had not received funding from the National Eye Institute/NIH,” Aguirre stated, “this amazing breakthrough would never have come to pass. Without increased funding, much of this promise will languish on the laboratory bench.”
The hearings followed one year on the heels of the first leukemia diagnosis in the Paris X-SCID study, and two years after Jesse Gelsinger's death. In addition to embodying the promise of vision research, Lancelot's eye also projected a favorable image for the embattled field.
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- Gene Transfer and the Ethics of First-in-Human ResearchLost in Translation, pp. 153 - 177Publisher: Cambridge University PressPrint publication year: 2009