In the May 1, 2008 issue of Nature, Alison Abbott reports on fraud allegations against Austrian researcher Hannes Strasser for performing an adult stem cell trial for urinary incontinence without having his protocol reviewed by an ethics committee. According to the story, the volunteers paid approximately $17K U.S. to enter the study. They also were… Continue reading Bladder Trouble at the Frontier
Author: Jonathan Kimmelman
Goodbye, Helsinki
According to recent news reports, the U.S. FDA recently decided to abandon its endorsement of the Declaration of Helsinki when sponsors submit clinical trial data obtained overseas. The Declaration of Helsinki (DoH)- which was first adopted in 1964- is the World Medical Association’s statement on ethical requirements for human experimentation. According to an editorial in… Continue reading Goodbye, Helsinki
Kid Time for LCA gene transfer?
(…continued from previous posts). The promising results, and the surge of expectation they generated, will be used by the investigators and many advocates for the blind to defend pursuing the remainder of the study in children. After all, children stand a better chance of benefiting, because their retinas have not yet degenerated. Traditionally, bioethicists have… Continue reading Kid Time for LCA gene transfer?
Imprinting Expectation
(…continued from previous post) Whatever the ambiguity and severe limitations in terms of sample size and interpretability, the NEJM studies have unleashed a torrent of expectation. Accoding to the U.K. Guardian, “an 18-year-old man… has amazed doctors by navigating a maze…” and the technique “represented a ‘huge advance.’” The Washington Post’s Rick Weiss was harshly… Continue reading Imprinting Expectation
Seeing Light?
…continued from previous post: So did the researchers make the blind see? Any suggestion that vision has been restored is premature. The authors of both reports acknowledge that measures of vision are subjective and visual improvements might reflect a placebo effect. What can be said– at least of the U.S. study– is that the pupil… Continue reading Seeing Light?
Safety First: Two Gene Transfer Studies Against Blindness
…continued from previous post: The preclinical studies supporting these human trials are about as good as it ever gets in translational research: numerous large animals (dogs) were treated effectively, and their vision in treated eyes seems to have been restored out to as much as eight years. The NEJM papers report delivering nearly identical vectors… Continue reading Safety First: Two Gene Transfer Studies Against Blindness
Envisioning Gene Transfer: Report on Congenital Blindness Studies
This week, the New England Journal of Medicine published two preliminary trial reports using gene transfer against a rare form of congenital blindness– Leber’s Congenital Amaurosis type 2. Since Jean Bennett and coworkers at the University of Pennsylvania reported in 2001 successfully correcting visual deficiencies in dogs afflicted with a nearly identical genetic condition, the… Continue reading Envisioning Gene Transfer: Report on Congenital Blindness Studies
More Grey Matter: Parkinson’s Disease Gene Transfer
Ceregene published long awaited results of its phase 1 gene transfer study of a Parkinson’s disease treatment in the May issue of Lancet Neurology. The open-label study involved two dose levels. After one year of observation, the authors report no major adverse events relating to the study product, CERE-120 (importantly, no neutralizing antibodies against the… Continue reading More Grey Matter: Parkinson’s Disease Gene Transfer
Teddy’s Frowning
This blog primarily covers the ethics of translational clinical trials. Products tested in such studies, should they progress towards licensure, will eventually fall under the oversight of drug regulatory agencies like the FDA. How prepared will the FDA be to protect the public once these highly complex interventions are introduced to clinical practice? And when… Continue reading Teddy’s Frowning
Is Embryonic Stem Cell Research Fully Developed?
As reported by Monya Baker in the April 10 issue of Nature (and Alicia Mundy in Wall Street Journal, April 11), FDA is convening a public hearing on the safety of therapies derived from embryonic stem cells as I write this blog entry. (Note: for info on the meeting, visit http://www.fda.gov/OHRMS/ DOCKETS/98fr/E7-24629.htm) As yet, (reputable)… Continue reading Is Embryonic Stem Cell Research Fully Developed?