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It’s coming up on ten years since Jesse Gelsinger died in a gene transfer study investigating a rare hereditary liver disorder, OTC deficiency. Gelsinger’s death In two recent articles, James Wilson, who led that study, has gone public with regrets, lessons learned, and warnings. The first consists of a confessional 1- The clinical protocol is… Continue reading Confessions of James Wilson

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

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?

…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

…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?

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

…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

What are the benefits of participating in a clinical trial? Ethicists have tended to divide benefits into three categories: direct, collateral, and aspirational. The first involves medical benefits flowing from the study intervention; the second, from increased medical monitoring. The third refers to the benefits that flow to society from the knowledge gained. But are… Continue reading Gimme (and ’em) More

Inder Verma has won the Vilcek Foundation annual award for, among other things, his work in gene transfer (for those of you who, like me, enjoy contemporary music, this year’s other recipient was Oswaldo Golijov– an eclectic Argentine-born composer). As a former editor of Molecular Therapy and in other writings, Verma has often offered thoughtful… Continue reading Verma Wins Vilcek Prize