What is “special” about the ethics of gene transfer trials? To many, the answer is “nothing.” Indeed, many gene transfer researchers resent what they perceive as an unusually high bar for initiating human studies of gene transfer. The March 2008 issue of Molecular Therapy contains an excellent article by ethicist Nancy King and gene transfer… Continue reading Unique Forms of Discontinuity
Author: Jonathan Kimmelman
Seeing Red
In the next issue of the journal Haemophilia, two researchers, Katherine Ponder and Alok Srivistava, take me to task for an article I recently published on the ethics of hemophilia gene transfer trials. My article discusses the little noticed phenomenon of researchers at elite medical centers in the U.S. recruiting trial subjects in Brazil and… Continue reading Seeing Red
For Whom the Cell Tolls?
A month ago or so, I wrote about a new gene-based strategy against disease: RNAi. Recall that in 2006, Andrew Fire and Craig Mello won Nobel Prizes in Medicine for the discovery of RNAi. The idea of using RNAi in therapeutic applications would be to administer these small genetic sequences to “knock down” the activity… Continue reading For Whom the Cell Tolls?
Low Summit: Setbacks in HIV Vaccine Research
March 26 NYTimes and Washington Post ran stories about a “summit meeting” of HIV vaccine researchers at the NIH. According to the reports, the meeting was prompted by a widely publicized study in which a highly promising Merck HIV vaccine candidate proved ineffective in a large placebo controlled study. Even more disturbing is an analysis… Continue reading Low Summit: Setbacks in HIV Vaccine Research
Misfolding Proteins, MisLede-ing Headlines?
On March 14th, the Guardian reported on a recent advance using gene transfer against a degenerative brain condition called spinalcerebellar ataxia. The condition belongs to a class of diseases that also includes Huntington’s disease, and as the report indicates, the study could prove useful for many other degenerative brain disorders. The lede begins “Scientists are… Continue reading Misfolding Proteins, MisLede-ing Headlines?
Little Orphan Ending
…At any rate, the NEJM article describes four bills as having been introduced in the U.S. Congress in 2007 to expedite approval of follow-on protein products. None reached the floor. The article cheerfully concludes “despite failures of the bills… [they] collectively represent important first steps that should help stimulate further discourse… and signal an end… Continue reading Little Orphan Ending
A Fruity Defense?
…Another reason why drugs like Cerezyme are so expensive is because it is very difficult for generic drug companies to license follow-on (that is, generic) drugs under existing drug regulations. In the March 17 issue of New England Journal of Medicine, two Boston-based researchers review the legal and regulatory environment for generic protein-drugs. Under the… Continue reading A Fruity Defense?
The Cost of Orphan Upkeep
Why is Cerezyme so expensive? One reason is that it targets an ultra-rare disease. Drug companies generally avoid developing products for such “orphan diseases” because there is little consumer demand. To spur development of orphan drugs, the U.S. and other countries have enacted legislation granting market exclusivity (in the U.S., seven years) for orphan drug… Continue reading The Cost of Orphan Upkeep
Wardens of the Orphanage
…the other NYTimes article on Cerezyme concerned Genzyme’s cultivation of relationships with the small Gaucher’s disease population. The article described Genzyme as employing a staff of 50 to help patients negotiate insurance coverage for their products. Genzyme has also established treatment centers and built a stable of Gaucher’s specialists. Said one Gaucher’s specialist, Genzyme has… Continue reading Wardens of the Orphanage
Cutting Recombinant Protein Pills?
On March 16, the NYTimes ran two stories on Cerezyme, an expensive protein-based drug used to treat a rare genetic disorder, Gaucher’s disease. Both articles covered issues that might be emblematic of those that will be encountered when gene transfer applications are commercialized. One article described uncertainties clinicians face in establishing the appropriate dose for… Continue reading Cutting Recombinant Protein Pills?