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?
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Gimme (and ’em) More
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
Verma Wins Vilcek Prize
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
Inflation, Health Care Reform, and Translational Research
“It’s Health Care Costs, Stupid.” So says Ezekiel Emanuel, paraphasing James Carville, in a Feb 29, 2008 commentary in JAMA. Emanuel argues that expanding health care coverage to the uninsured will be impossible and unsustainable unless the health care system gets a handle on cost inflation. What does this have to do with translational research?… Continue reading Inflation, Health Care Reform, and Translational Research
Masks and Random Thoughts on Preclinical Research Validity
Epidemiologists and biostatisticians have evolved numerous ways of reducing bias in clinical trials. Randomization of patients, and masking them to their treatment allocation are two. Another is masking clinicians who assess their outcomes. Why are these simple measures so rarely used in preclinical animal studies? And do animal studies show exaggerated effects as a consequence… Continue reading Masks and Random Thoughts on Preclinical Research Validity
siRNA: Caveat Emptor for Preclinical Studies?
Small interfering RNA molecules (siRNA) are generating a lot of excitement in biomedical research for their ability to “knock down” specific genes– say, those of an invading virus or a tumor cell. But because viruses tend to produce small pieces of RNA, the body often “interprets” siRNA as a viral infection and launches an immune… Continue reading siRNA: Caveat Emptor for Preclinical Studies?
Still Life with Duchenne Muscular Dystrophy
In the February 20th edition of the New York Times, Reed Abelson reports a “New Tack on a Muscle Disease.” The “new tack” is, in fact, a scaling back of ambition in muscular dystrophy (MD) research. Since identification of various genetic mutations that cause muscular dystrophies, gene transfer has been a logical goal. But because… Continue reading Still Life with Duchenne Muscular Dystrophy
Still Life with Duchenne Muscular Dystrophy
In the February 20th edition of the New York Times, Reed Abelson reports a “New Tack on a Muscle Disease.” The “new tack” is, in fact, a scaling back of ambition in muscular dystrophy (MD) research. Since identification of various genetic mutations that cause muscular dystrophies, gene transfer has been a logical goal. But because… Continue reading Still Life with Duchenne Muscular Dystrophy
Can You Keep A Secret?
People often enter drug studies in order to access a promising new drug. But clinical trials take place over a set time period. So is it ethical to withhold from prospective trial subjects preliminary data on safety or efficacy that have been gathered from initial volunteers? In the Feb 1, 2008 issue of Journal of… Continue reading Can You Keep A Secret?
Vector Calculus is Out: Nature Reviews—Genetics Article
As per usual when an article has moved into the post-galley stage, on Monday I woke to a string of friendly reprint requests mainly from Eastern European researches lacking good library facilities. I guess that means my review on the ethics of gene transfer has been published in Nature Reviews—Genetics. In it (the editors rejected… Continue reading Vector Calculus is Out: Nature Reviews—Genetics Article