What’s the difference between testing a typical small molecule drug, and testing a novel cell therapy strategy? And where might the latter raise ethical challenges that the former doesn’t? These questions are extensively discussed in my book, and given human drama in a recent story by Jennifer Couzin-Frankel in the Feb 12, 2010 issue of… Continue reading Transplanting Autoimmune Research
Author: Jonathan Kimmelman
Cooperation and Medical Research
Why do patients cooperate with medical researchers? So asks sociologists Mary Dixon-Woods and Carolyn Grant in a study analysis appearing in the June 2009 issue of Social Science and Medicine. You might think the answer is simple: they think they will benefit; they want to contribute to medical knowledge; or, they trust researchers who invite… Continue reading Cooperation and Medical Research
Hypothesis Generator
Is good medical research directed at testing hypotheses? Or is there a competing model of good medical research that sees hypothesis generating research as a valuable end? In an intriguing essay appearing in the August 21, 2009 issue of Cell, Maureen O’Malley and co-authors show how current funding mechanisms at agencies like NIH and NSF… Continue reading Hypothesis Generator
Annus Mirabilis for Gene Transfer
Time to review the year 2009 for cutting edge clinical research. For the field of gene transfer, it has been an annus mirabilis: a year that has seen very encouraging results in a wide variety of human clinical studies, as well as preclinical studies. Indeed, I regret that this blog has only been able to… Continue reading Annus Mirabilis for Gene Transfer
Finding Skew: Informed Consent and Bias in Clinical Trials
Clinical researchers have long claimed that patients who enter clinical trials are better off medically than those who don’t. I’m open to the notion that patients might derive personal meaning from trial participation, but I’ve always been dubious of the suggestion that trial participation in itself is therapeutically beneficial–above and beyond drugs received– in part… Continue reading Finding Skew: Informed Consent and Bias in Clinical Trials
Expectation is a Vascular Condition: Thoughts on Media Coverage of "Liberation Procedures" for Multiple Sclerosis
Disclaimer to all readers: I am not expert in multiple sclerosis. I am not intimately familiar with recent research findings on a novel surgical treatment (“liberation procedure”) for multiple sclerosis that have received wide coverage in the Canadian media. Now here are my “claimers:” recent media accounts of this novel approach border on the irresponsible,… Continue reading Expectation is a Vascular Condition: Thoughts on Media Coverage of "Liberation Procedures" for Multiple Sclerosis
More on Lenti’s, Gene Transfer and Adrenoleukodystrophy
(…continued from the previous post). There are several features that make the recent Adrenoleukodystrophy (ALD) gene transfer study noteworthy. 1- A New Viral Vector Debuts: this is the first successful application of HIV-derived viruses in gene transfer (lentiviruses). These vectors have various advantages over retroviruses used in other protocols. One is that, in theory, at… Continue reading More on Lenti’s, Gene Transfer and Adrenoleukodystrophy
Gene Transfer and Adrenoleukodystrophy: There Will Always Be Paris
Last week’s Science magazine reported what seems likely to count as one of gene transfer’s greatest clinical successes to date: stabilization of adrenoleukodystrophy in two boys receiving genetically modified blood stem cells. Preliminary results of this study had been presented at this summer’s American Society of Gene and Cell Therapy meeting. Adrenoleukodystrophy (ALD) is a… Continue reading Gene Transfer and Adrenoleukodystrophy: There Will Always Be Paris
California Dreamin: CIRM Announces New Stem Cell Awards
California’s Institute for Regenerative Medicine just announced a series of large funding awards to fund translational research initiatives involving (mostly) stem cells. The projects funded are telling with respect to what was funded, and what they will attempt to achieve. First, notwithstanding a press release containing the words “bringing stem cell therapies to the clinic,”… Continue reading California Dreamin: CIRM Announces New Stem Cell Awards
The Need for Speed: GAO Reports on Accelerated Approval
Several blog posts ago, I wrote about the policy of accelerated approval (briefly, a mechanism whereby new drugs can be approved for sale by the FDA before definitive evidence of efficacy and safety are available). In that post, I reported on a recent paper where the authors claimed that, all things considered, accelerated approval enabled… Continue reading The Need for Speed: GAO Reports on Accelerated Approval