One of the most striking themes at the European Society of Gene and Cell Therapy was the extent to which continental European researchers conceptualize first-in-human gene transfer experiments as therapeutic interventions rather than research protocols.
Perhaps the most extreme and explicit expression of this was view was presented by Bonn internest Thomas Heinemann (he also studied philosophy and serves on several ethics committees in Germany). Heinemann advanced the notion of the “controlled individual therapeutic attempt,” for which the primary objective is therapeutic gain; the scientific dimensions of such studies (e.g. collecting safety data) are necessarily secondary. As he put it, research is only justified “ex post facto.”
I found this argument intriguing for several reasons. First, Heinemann justified this claim largely on grounds of autonomy and instrumentalization of desperately ill patients. In contrast, North American bioethicists typically use autonomy and instrumentalization to argue the opposite: that research is primarily intended to serve the ends of others, hence the paramount importance of obtaining consent from volunteers and their guardians, hence the need to be extremely cautious going into a desperately ill population, where autonomy might be compromised.
Second, I was impressed by the speaker’s conviction that first-in-human trials have therapeutic warrant. After almost twenty years of painstaking and at times discouraging research, we seem to have learned two things: first, that first-in-human trials rarely go as expected, and second, that such studies often yield important insights about new interventions. I might have expected a more cautious and seasoned view about the therapeutic merits of first-in-human attempts: does it really enhance the autonomy of volunteers to offer so little by means of therapy, but to foreseeably get so much in terms of social good? (photo credit: virtualais //www.77click.it, Brugge, 2008)
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Jonathan Kimmelman. "In Brugge: The Cure" Web blog post. STREAM research. 24 Nov 2008. Web. 11 Feb 2025. <https://www.translationalethics.com/2008/11/24/in-brugge-the-cure/>
APA
Jonathan Kimmelman. (2008, Nov 24). In Brugge: The Cure [Web log post]. Retrieved from https://www.translationalethics.com/2008/11/24/in-brugge-the-cure/
I‘ve just returned from the annual European Society of Gene and Cell Therapy meeting in Belgium. Lots of great material for upcoming posts. For now, I want to follow on the last posting on the leukemias in the X-SCID study. A warning: those lacking a stomach for science geek-talk might want to skip this posting.
In the previous posting, I stated that a recent paper provided evidence that retroviral integration in the genome (“insertional mutagensis”) had triggered leukemias in the X-SCID study rather than over-expression of the corrective gene (“transgene”), the gamma c-chain (hereafter, “gc”). This was on the basis of data in the graphic above, which used cell sorting to show that levels of gc on the surface of T-cells was within a normal range. In Belgium, Adrian Thrasher presented similar data for the fifth leukemia.
When I first encountered this figure, it bothered me: why did the authors measure gc expression by cell surface markers (a technique called “FACS”) rather than Western or Northern blotting, or quantitative PCR, or something along these lines? It seemed a very indirect way of seeing whether gc expression levels are in fact normal. Here are two possibilities that this figure fails to rule out: 1- gc is expressed at very high levels, but not packaged and presented on the surface of T-cells, perhaps because of insufficiency of other receptor components; 2- some gc transgene is aberrantly spliced, such that surface levels are normal, but intracellular concentrations of the alternate splicing product are abnormal.
A few years back, one team of researchers presented data indicating that gc transgene overexpression contributes to T-cell transformation. Another team claimed it was unable to reproduce this. The jury seems to still be out on whether the gc product contributed to the X-SCID leukemias, and I’m not yet convinced that the latest round of data fully exonerates the gc chain. (Graphic: figure from Salima Hacein-Bey-Abina et al, J Clinical Investigation 2008; 108: 3132-42).
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MLA
Jonathan Kimmelman. "Just the FACS: Reprise on Insertional Mutagenesis" Web blog post. STREAM research. 18 Nov 2008. Web. 11 Feb 2025. <https://www.translationalethics.com/2008/11/18/just-the-facs-reprise-on-insertional-mutagenesis/>
APA
Jonathan Kimmelman. (2008, Nov 18). Just the FACS: Reprise on Insertional Mutagenesis [Web log post]. Retrieved from https://www.translationalethics.com/2008/11/18/just-the-facs-reprise-on-insertional-mutagenesis/
Among the greatest traumas for gene transfer was the development of leukemias in several children participating in trials using retroviral vectors against X-linked Severe Combined Immune Deficiency (X-SCID– also known as “bubble boy syndrome”). About 20 or so children have had their immune systems fully restored by this gene transfer strategy. Tragically, however, five children in two X-SCID studies (one in Paris, the other, London) developed T-cell leukemias that were causally linked to the gene transfer approach.
In the September 2008 issue of Journal of Clinical Investigation, Salima Hacein-Bey Abina and 28 other authors characterize the molecular nature of four of the adverse events, and report the outcome. Before this article, it was known that one of the children died. This article now reports that the other three children have “sustained remission” after chemotherapy.
The authors report that the vector inserted itself at the same genetic locus (LMO2) in 3 of the 4 cases. In one case, vector inserted at a different genetic locus (CCND2); in another, vector inserted itself at LMO2 as well as a second locus, BMI1. This suggests that LMO2 disruption is not the only path to causing cancer for this vector. One other finding stood out. Since the first leukemia was detected, many have speculated that the cancer was partly caused by the gene (rather than just the vector). However, the authors present evidence that the gene was expressed at normal levels in the children who developed leukemia. This lends support to the theory that the leukemias were not caused by the gene, but rather by some combination of the vector, the cell types used, and perhaps some characteristic of the underlying disease. (photo credit: concretecandy, boy in the bubbles, 2006)
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MLA
Jonathan Kimmelman. "Burst Bubbles" Web blog post. STREAM research. 06 Nov 2008. Web. 11 Feb 2025. <https://www.translationalethics.com/2008/11/06/burst-bubbles/>
APA
Jonathan Kimmelman. (2008, Nov 06). Burst Bubbles [Web log post]. Retrieved from https://www.translationalethics.com/2008/11/06/burst-bubbles/
Hope has been a consistent theme in Barack Obama’s campaign, which thankfully came to a glorious end (many of us can now “hope” to actually get some work done after weeks of checking fivethirtyeight.com every ten minutes). His book was titled The Audacity of Hope. In the close of his victory speech, he stated “Let us keep that promise, that American promise, and in the words of scripture hold firmly, without wavering, to the hope that we confess.” Newspaper headlines proclaim “Elections Unleash Flood of Hope Worldwide (NYTimes) and “Time to Hope Again” (Washington Post).
Hope is also a central theme in translational research, driving research advocates (“Hope for a Cure”), perseverance at the lab bench, and for better and for worse, the participation of gravely ill patients in trials that offer the slimmest prospect of serious medical benefit. Hope in many settings– particularly in the political– is an unalloyed good.
But in the context of enrolling patients in early phase trials, hope becomes morally ambiguous. Or at least, so Penn philosopher Adrienne M. Martin would seem to suggest in her critical analysis of hope (“Hope and Exploitation,” Sept / Oct issue of Hastings Center Report).
Martin begins her essay with a set of consensus observations about hope: 1- it involves a desire for an outcome; 2- it involves imaginative engagement with a desired outcome– like praying or fantasy; 3- it often frames how people interpret and use information. She then goes on to explore the various ways that hope can lead to exploitation in clinical research.
Much has been written about “hope” in bioethics, and much of it is drivel. I have a number of reservations about Martin’s article. For my money, however, this is the most compelling analysis of the phenomenon that I know of. I highly recommend this article to anyone who takes seriously the moral dimensions of how translational research engages hope. (photo credit: San Diego Shooter, Tattered Hope, 2008)
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MLA
Jonathan Kimmelman. "Keeping Alive with Hope" Web blog post. STREAM research. 06 Nov 2008. Web. 11 Feb 2025. <https://www.translationalethics.com/2008/11/06/keeping-alive-with-hope/>
APA
Jonathan Kimmelman. (2008, Nov 06). Keeping Alive with Hope [Web log post]. Retrieved from https://www.translationalethics.com/2008/11/06/keeping-alive-with-hope/
Think you’re the only one stashing your financial statements in a filing cabinet without first opening them? How do you think biotechnology companies feel? Today’s New York Times ran a story by Andrew Pollack (“Broader Financial Turmoil Threatens Biotech’s Innovation and Cash”) describing the impact of the economic downturn on the biotechnology sector. Among the observations:
– Of 344 companies that NASDAQ is considering de-listing because share prices have fallen to less than a dollar, 25% are biotechnology companies.
– 113 biotech companies now have less than a year’s cash at current spending rates. This is up from 68 in the first quarter.
– One company with major problems is DeCode- the Icelandic genetic research company. This might have particular symbolic significance, given the company’s profile.
– companies are being pressured by investors to cut their research and focus energy on lead products only.
The article goes on to explain that the main problem for biotech companies is NOT credit (apparently, even banks considered most biotech’s too risky to lend to). Instead, the problem is an epidemic of risk aversion among investors, and hedge funds selling biotech stocks to recoup losses elsewhere in their portfolios. The article does note that companies like Amgen, which have widely used products, are thriving compared with the general market. As for the others, one analyst, Andrew Baum, describes the sector as a plane “for which the financial crisis just tipped the nose straight down.” The implications for translational research are obvious. (photo credit: Hawk914, Koga’s Zero- 1942, 2007)
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MLA
Jonathan Kimmelman. "Cash Crash" Web blog post. STREAM research. 29 Oct 2008. Web. 11 Feb 2025. <https://www.translationalethics.com/2008/10/29/cash-crash/>
APA
Jonathan Kimmelman. (2008, Oct 29). Cash Crash [Web log post]. Retrieved from https://www.translationalethics.com/2008/10/29/cash-crash/
The October 2008 issue of Nature Reviews–Drug Discovery contains a very informative perspective piece on how drug regulators negotiate uncertainty, risk, and benefit when making approval decisions (“Balancing early market access to new drugs with the need for benefit/risk data: a mounting dilemma”). I have long argued that novel biologics like gene transfer will require creative approaches from regulators, because on the one hand many types of adverse events might be latent and unpredictable, while on the other hand, many novel biologics will target highly morbid or lethal conditions like primary immunodeficiencies.
The authors (Hans-Georg Eichler et al) are all employees of drug regulatory agencies in Europe. Not surprisingly, then, the article is balanced and presents drug agencies as making appropriate trade-offs between patient access and public safety. The article studiously avoids any criticism of pharmaceutical companies. And it makes some questionable claims. For example, in several passages it suggests that drug regulation is increasingly “risk averse” (it seems to me the opposite, but who knows?). Another is that the article contains ample evidence that premature approval has had important costs in terms of health and economics. Nowhere does it provide clear evidence or anecdotes that delay of approval, or restrictive evidentiary standards have had important public health or economic impacts (it might, but if you are going to suggest that the balance is appropriately struck, one needs a clear picture of the benefit side of the equation).
The article contains a number of observations and policy approaches that cry out for careful ethical analysis. Here are two:
1- Drug regulatory agencies accept greater uncertainty about safety and efficacy when new drugs address serious, unmet health needs. Thus, new cancer drugs can be approved on a weaker evidentiary base than new acid reflux drugs (in several instances, new drugs were approved on the basis of uncontrolled, surrogate endpoints (e.g. gefitinib). Why should evidentiary standards be relaxed in this way? And to what degree? If the disease is serious enough and standard care non-existent, then what is the basis for any drug regulation?
2- The article states that “rare drug reactions will continue to be identified only after wider use in the market,” and that more sophisticated approaches to drug safety will increasingly “blur the line between pre-marketing and post-marketing activities.” How will this affect ethics review and oversight? How will privacy protections be maintained in this gulf-stream of flowing health data? How will trial registries absorb and respond to post-marketing studies?
This article contains multitudes– I highly recommend it to anyone interested, as I am, in the problem of uncertainty, risk, and drug regulation (photo credit: Alincolnt, schedule 5, 2006)
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MLA
Jonathan Kimmelman. "The Future of Pharmaceutical Regulation" Web blog post. STREAM research. 28 Oct 2008. Web. 11 Feb 2025. <https://www.translationalethics.com/2008/10/28/the-future-of-pharmaceutical-regulation/>
APA
Jonathan Kimmelman. (2008, Oct 28). The Future of Pharmaceutical Regulation [Web log post]. Retrieved from https://www.translationalethics.com/2008/10/28/the-future-of-pharmaceutical-regulation/
In the October 20, 2008 issue of Journal of Clinical Oncology, oncologist Zeba Aziz describes morning rounds in a cancer ward in Lahore, Pakistan. The first patient earns $20 a month and requires a combination therapy costing $10K. In the second case, a father can only pay 15% of the treatment needed by his daughter. The third case involves a single mother, who showed a significant tumor response on the first cycle of treatment but can not afford subsequent rounds.
According to Aziz, only 2% of Pakistan’s population has health insurance; 95% earn $30-100 / month. Aziz’s hospital serves 10K cancer patients annually, and has a medicines budget of around $80K each year. Treatments that wipe out the immune system are especially tricky, because medicines for infection might not be affordable.
Low and middle-income countries (LMICs) are undergoing an epidemiological transition in which chronic diseases replace infection as the main drivers of mortality. According to Franco Cavalli (“The World Cancer Declaration: A Roadmap for Change,” Lancet Oncology, September 2008), cancer kills more people worldwide than AIDS, tuberculosis, and malaria combined. Cavalli describes a “World Cancer Declaration” issued by the Geneva based International Union Against Cancer that calls for improvements in the prevention, detection, reporting, and treatment of cancer in LMICs.
But a serious response to cancer in LMICs will require changes in translational research as well. Clearly, the vast majority of cancer treatment research is directed toward markets in high-income countries. These treatments have properties– costs, side-effect profiles, demand for ancillary services– that make their application in LMIC populations improbable. If, as argued by many commentators, we in HICs have a duty to persons in LMICs, translational researchers and funding agencies should give some thought and effort to the development and testing of treatments that, while perhaps not adding as many weeks of extra median survival, are deployable in settings like Aziz’s clinic. (photo credit: CasaDeQueso 2008)
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MLA
Jonathan Kimmelman. "Cancer, Low and Middle-Income Countries, and Translational Research" Web blog post. STREAM research. 21 Oct 2008. Web. 11 Feb 2025. <https://www.translationalethics.com/2008/10/21/cancer-low-and-middle-income-countries-and-translational-research/>
APA
Jonathan Kimmelman. (2008, Oct 21). Cancer, Low and Middle-Income Countries, and Translational Research [Web log post]. Retrieved from https://www.translationalethics.com/2008/10/21/cancer-low-and-middle-income-countries-and-translational-research/
Last night, Obama and McCain confronted each other in the final Presidential debate. A flagging economy and two wars have left little room in the two campaigns for discussion of science, policy, and human research. Yet last night’s debate touched on two themes: embryonic stem cell (hES) research, and biomedical research funding.
Obama accused McCain of opposing embryonic stem cell research. From what I can tell, McCain actually supported the use of embryonic tissue for research and opposed Bush’s ban and vetoes. But the logic of McCain’s attacks on Obama, of late, are that personal associations tell us something about who a person is and where they stand. And McCain pals around with embryo research opponents like his running mate.
Contrast the two candidates’ statements on hES research from Sciencedebate 2008– a group that invited McCain and Obama to declare positions on various science policy issues. McCain stated “While I support federal funding for embryonic stem cell research, I believe clear lines should be drawn….” The remainder of his response qualifies his support. On his own website, McCain stops short of declaring support–or opposition– for hES research, and talks more about what he would oppose than what he would support. Obama’s support is more full-throated at Sciencedebate 2008: “As president, I will lift the current administration’s ban on federal funding of research on embryonic stem cell lines… embryonic stem cells remain the ‘gold standard,’ and studies of all types of stem cells should continue in parallel for the foreseeable future.”
Elsewhere at Sciencedebate 2008, Obama’s campaign singled out gene transfer in a statement on genetics: “As a result [of safety issues involving ‘gene therapy’], the NIH established the Recombinant DNA Advisory Committee…. Until we are equipped to ascertain the safety of such methods, I will continue to support the activities and recommendations of the Recombinant DNA Advisory Committee.” [Note: Harold Varmus chairs a science advisory committee for the Obama campaign. Varmus reorganized RAC when he was the director of the NIH under the Clinton administration]
What about research– specifically translational research? Just as they do for Joe the plumber, both candidates support NIH research. According to a report in Science (“Scientists Strive for a Seat at the Table of Each Campaign,” Jeffrey Mervis, 26 Sept), Obama pledged to double the NIH budget in five years. Elsewhere, his campaign said 10 years. Maybe the latter figure is inflation adjusted? Obama’s statement on Science and Innovation singles out “rapid translation of medical research.”
I am not aware of any clear statements on translational research from McCain, though he favors greater funding for NIH, and based on his debate and website, he seems to have a soft spot for autism research. As on other issues, McCain is less willing to commit to a timetable on NIH budget doubling. (photo credit: Thomas Hawk, Wordle of McCain and Obama convention speeches, 2008)
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MLA
Jonathan Kimmelman. "From Bench to Ringside: The Presidential Debate" Web blog post. STREAM research. 16 Oct 2008. Web. 11 Feb 2025. <https://www.translationalethics.com/2008/10/16/from-bench-to-ringside-the-presidential-debate/>
APA
Jonathan Kimmelman. (2008, Oct 16). From Bench to Ringside: The Presidential Debate [Web log post]. Retrieved from https://www.translationalethics.com/2008/10/16/from-bench-to-ringside-the-presidential-debate/
“Since 2005, we’ve started seeing the big 20 pharma corporations making investments [in gene therapy],” says a deputy head in European Medicine Agency (EMEA) in the October 2008 issue of Nature Biotechnology (“Ark floats gene therapy’s boat, for now,” by Randy Osborne). “When you want to know what season is there and when the weather will change, you have to see which birds are flying.”
All this should be very encouraging for the field. Yet the story also describes the many travails of companies seeking regulatory approval for their gene transfer products. One is Introgen, which sought approval for its adenovirus-p53 cancer product Advexin. FDA returned Introgen’s submission with a “refuse to file” letter indicating that the materials were incomplete. The study on which this filing was based was presented at the American Society of Gene Therapy meeting last Spring. As I indicated in my June 10 posting, a major concern with this study was that its efficacy claims were based on a subgroup analysis of genetic profiles. Others, like TheStreet.com columnist Adam Feuerstein, have been far less guarded in their assessment of Advexin; Feuerstein called Introgen “a living textbook for what investors need to be wary of when considering a biotech investment.”
The second product profiled in the Nature Biotechnology story is Ark’s Cerepro, which uses herpes simplex virus vectors to deliver a gene, thymidine kinase, to the tumor bed of patients with malignant glioma (a highly aggressive form of brain cancer); the gene then converts a pro-drug, ganciclovir, into a toxin that kills tumor tissue. Ark is reportedly planning to file a license application to EMEA. Preliminary data in a randomized controlled trial against standard of care vs. standard of care + Cerepro indicate a median extension of survival by 42 days (with serious side effects).
One last product profiled briefly is Amsterdam Molecular Therapeutic’s product, Glybera, for a very rare genetic disease lipoprotein lipase deficiency. Amsterdam intends to file for licensure “later this year.”
The fortunes of clinical gene transfer might indeed be flying North. But with some lead products showing such incremental gains in survival and dependence on combination with standard care, clinical application seems less likely to arrive with a bang than with a modest honk. (photo credit: denis collette, 2007)
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MLA
Jonathan Kimmelman. "Northward Migration?" Web blog post. STREAM research. 14 Oct 2008. Web. 11 Feb 2025. <https://www.translationalethics.com/2008/10/14/northward-migration/>
APA
Jonathan Kimmelman. (2008, Oct 14). Northward Migration? [Web log post]. Retrieved from https://www.translationalethics.com/2008/10/14/northward-migration/
Chicago in plastic and balsa. If only animal models were as convincing as the one pictured above from the Museum of Science and Industry.
The August 7 issue of Nature ran a fascinating feature on how many scientists are reassessing the value of animal models used in neurodegenerative preclinical research (“Standard Model,” by Jim Schnabel).
The story centers on the striking failure to translate promising preclinical findings to treatments for various neurodegenerative diseases. In one instance, a highly promising drug, minocycline, actually worsened symptoms in patients with ALS. In other instances, impressive results in mice have not been reproducible. According to the article, a cluster of patient advocacy groups, including organizations like Prize4Life and a non-profit biotechnology company ALS TDI, are spearheading a critical look at standard preclinical models and methodologies.
Much of the report is about limitations of mouse models. Scientists from the Jackson Laboratories (perhaps the world’s largest supplier of research mice) warn that many mouse strains are genetically heterogenous; others develop new mutations on breeding. Other problems described in the article: infections that spread in mouse colonies, problems matching sex or litter membership in experimental and control groups, and small sample sizes. The result is Metallica-like levels of noise in preclinical studies. Combined with nonpublication of negative studies, and the result is many false positives.
The article bristles with interesting tidbits. One that struck me is the organizational challenges of changing the culture of model system use. According to the article, many academic researchers and grant referees have yet to warm to criticisms of models, and some scientists and advocates are asking for leadership from the NIH. Another striking point in the piece-alluded to in the article’s closing-is a fragmentation of animal models that mirrors personalized medicine.
“Drugs into bodies.” That’s the mantra of translational research. It is an understandable sentiment, but also pernicious if it means more poorly conceived experiments on dying patients. What is needed is a way to make animal models- and guidelines pertaining to them- as alluring as supermodels. (photo credit: Celikens 2008)
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MLA
Jonathan Kimmelman. "The Problem with Models" Web blog post. STREAM research. 10 Oct 2008. Web. 11 Feb 2025. <https://www.translationalethics.com/2008/10/10/the-problem-with-models/>
APA
Jonathan Kimmelman. (2008, Oct 10). The Problem with Models [Web log post]. Retrieved from https://www.translationalethics.com/2008/10/10/the-problem-with-models/
One of the most striking themes at the European Society of Gene and Cell Therapy was the extent to which continental European researchers conceptualize first-in-human gene transfer experiments as therapeutic interventions rather than research protocols.
