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
5 - Succor or suckers? Benefit, risk, and the therapeutic misconception
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
Patients with undiagnosed glioblastoma multiforme – an advanced form of brain cancer – typically arrive at the doctor's office complaining of headaches and weakness; a good many might also have experienced a seizure. Within a year, approximately half will be dead.
Progress against glioblastoma has been halting, and even modest improvements in treatment, like a median extension of survival of two and a half months, are greeted as breakthroughs. And so it was that, in 1992, a 51-year-old woman, whose husband was a prominent lawyer and chair of the board of the San Diego Cancer Center, was diagnosed with glioblastoma. Conventional therapies were unsuccessful in controlling her disease, and the woman sought enrollment in a gene-transfer study being planned at the San Diego Cancer Center. The problem was that the protocol had not yet been reviewed by the RAC, which met every three months; the patient was expected to live no more than two.
A former client of the woman's husband knew Iowa's Senator Harkin, and offered to contact him to see whether political pressure might be brought to bear. Shortly thereafter, Harkin forwarded a letter to NIH head Bernadine Healy requesting that she waive normal RAC review, though the developer of the intervention, Ivor Royston, had previously stated that he was “not optimistic that this therapy will work for his patient.”
RAC members bristled at Healy's involvement; they furthermore considered the supporting preclinical data insufficient to justify the protocol.
- Type
- Chapter
- Information
- Gene Transfer and the Ethics of First-in-Human ResearchLost in Translation, pp. 72 - 88Publisher: Cambridge University PressPrint publication year: 2009