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The rapid development of human genome editing (HGE) techniques evokes an urgent need for forward-looking deliberation regarding the aims, processes, and governance of research. The framework of anticipatory governance (AG) may serve this need. This article reviews scholarly discourse about HGE through an AG lens, aiming to identify gaps in discussion and practice and suggest how AG efforts may fill them. Discourse on HGE has insufficiently reckoned with the institutional and systemic contexts, inputs, and implications of HGE work, to the detriment of its ability to prepare for a variety of possible futures and pursue socially desirable ones. More broadly framed and inclusive efforts in foresight and public engagement, focused not only upon the in-principle permissibility of HGE activities but upon the contexts of such work, may permit improved identification of public values relevant to HGE and of actions by which researchers, funders, policymakers, and publics may promote them.

Nature Biotechnology asks a selection of leaders from across biotech to look at the future of the sector and make some predictions for the coming years.

Scholarship on human germline editing has centered on the risks to the genetically-modified child. However, far less emphasis is focused on women who will become pregnant with a genetically modified embryo as part of human subject research or the families that raise children whose genomes were modified as an embryo. The lack of attention on women and families places these key stakeholders in genomic technologies at significant medical, ethical, and personal harm as research rapidly moves forward to advance the science of genomic modification. Now is the time to address how the interests of women and families should be represented in the ethical and scientific frameworks of human genomic modification, with specific considerations for Institutional Review Boards who review protocols for rigorous human subject protections and scientists who develop scientific methodologies that dictate the potential risks conferred to research participants. In this paper, we examine the implications of genomic modification of human embryo for women, children, and families to explore how to review a first-in-human clinical protocol of human genomic officiation responsibly.

Background For gravely ill patients who have no treatment options and who are ineligible for clinical trials, the US Food and Drug Administration (FDA) established the Expanded Access Program (EAP). Motivated by efforts to weaken FDA regulation and sold as providing greater access to experimental drugs, the federal Right to Try Act (RTT) was passed in 2017. It reduces FDA oversight by not requiring physicians to report safety data and foregoes approval of protocols by local institutional review boards. Methods This study explored the views of 17 neuro-oncologists from 15 different academic medical centers with varying experience with EAP and RTT using convenience sampling. We conducted semi-structured interviews and qualitative analysis to identify emerging themes. Results Most oncologists were confused between the two pathways, had little familiarity with RTT, and had little knowledge about experimental medicine available through either pathway. Oncologists reported a preference of enrolling patients in clinical trials over off-trial preapproval pathways with scant data. As a result, oncologists revealed concerns over properly evaluating risks for their patients. Conclusion Our findings suggest that neuro-oncologists need better resources and clearer mechanisms at their institutions to help navigate EAP and RTT in order to counsel patients interested in experimental medicine.

Hank Greely’s (2021) target article, “Human Brain Surrogates Research: The Onrushing Ethical Dilemma” reviews the manifold scientific and ethical questions surrounding models of human brains used i...

The seminal 1984 Warnock Report1 established that research on human embryos should be limited to the first 14 days of development (Box 1). Since that time, the rule has been broadly adopted and adhered to across the research community. With the introduction of new methodologies into human embryology, however, our ability to culture human embryos in vitro has progressed rapidly, to the point where we now are reaching the 14-day Rubicon. In August 2016, two groups in the UK and in the US reported experiments on human embryos that were sustained in culture for 12–13 days after fertilization2,3. To comply with British law, the UK lab destroyed its embryo on the 13th day. In the following article, Nature Biotechnology brings together a group of experts to discuss whether, in the light of these advances, it is now time to reassess the 14-day rule.

Although women are inextricably involved in the study of germline editing, their interests have not been significantly represented in debates about the evolution of genome editing technology. Discussions have taken place about effects of germline editing on women as parents and members of families, but key discussions about women's health and well-being as patients and subjects are lacking. This neglect is due in part to restrictions on uterine transfer of modified human embryos, a boundary that has now been crossed. As a result, only scant discussion has taken place about safeguards needed to ensure that women who participate in germline modification research are not exposed to disproportionate risk in exchange for benefits they might expect for future offspring. This omission sets the stage for serious ethical implications for women and their families.

With an approval likely at the EMA, ex vivo gene therapy in hematopoietic stem cells appears poised for prime time.

The Broad Institute's recent licensing of its gene editing patent portfolio demonstrates how licenses can be used to restrict controversial applications of emerging technologies while society deliberates their implications.

A review of the key issues associated with the commercialization of biobanks Timothy Caulfield∗, Sarah Burningham, Yann Joly, ZubinMaster, Mahsa Shabani, Pascal Borry, Allan Becker, Michael Burgess, Kathryn Calder, Christine Critchley, Kelly Edwards, Stephanie M. Fullerton, Herbert Gottweis, Robyn Hyde-Lay, Judy Illes, Rosario Isasi, Kazuto Kato, Jane Kaye, Bartha Knoppers, John Lynch, AmyMcGuire, Eric Meslin, Dianne Nicol, Kieran O’Doherty, Ubaka Ogbogu, Margaret Otlowski, Daryl Pullman, Nola Ries, Chris Scott, Malcolm Sears, HelenWallace andMa’n H. Zawati†

While the major scientific discoveries that would extend the length and health of human lives are not yet here, the research that could create them is already underway. As prospects for a world in which extended and improved lives inches closer into reality, the discourse about what to consider as we move forward grows richer, with corporate executives, ideologues, scientists, theologians, ethicists, investigative journalists, and philosophers taking part in imagining and anticipating the rich array of humanity's possible futures. Drawing from in-depth interviews with key stakeholders (n = 22), we offer empirical insights into key values and beliefs animating the “longevity movement,” including what constitutes an ideal human state, the imperative to intervene, and the role of individual liberty and concerns for equality. Emerging from these interviews are common concerns about reducing suffering, preserving diversity in visions of successful aging and how best to promote access to a future that may not remain hypothetical for long.

Background: Looking through the lens of the media reveals much about how new biotechnologies are portrayed and how these reports influence public policy and opinion. The roles of patients, scientists, and the government loom large in this equation. These stakeholders tell us what is important to them and why. But little research exists comparing representations of new clinical technologies in the mainstream media across time. What has changed in newspaper accounts of new biotechnologies since the 1970s? How are the stakeholders portrayed, and what do these individuals have to say? Here, we seek to answer these questions by examining media reporting of Laetrile and stem cell transplants during the time of their clinical advent. Methods : Using a qualitative coding framework, we obtained frequencies of relevant excerpts and analyzed the data using the Mann–Whitney U test. Results: We saw significant differences in how stakeholders talk about their support and criticism of unproven treatments between the two...

For the field of pluripotent stem cell biology to realize its promising future, current researchers will need to pass the torch to new generations. We asked a group of successful scientists in this area, “What advice would you give a young person considering a career in stem cell research?”

More nations are joining the human embryonic stem cell (hESC) "race" by aggressively publishing in the peer-reviewed journals. Here we present data on the international use and distribution of hESC using a dataset taken from the primary research literature. We extracted these papers from a comprehensive dataset of articles using hESC and human induced pluripotent stem cells (hiPSC). We find that the rate of publication by US-based authors is slowing in comparison to international labs, and then declines over the final year of the period 2008-2010. Non-US authors published more frequently and at a significantly higher rate, significantly increasing the number of their papers. In addition, international labs use a more diverse set of hESC lines and Obama-era additions are used more in non-US locations. Even considering the flood of new lines in the US and abroad, we see that researchers continue to rely on a few lines derived before the turn of the century. These data suggest "embargo" effects from restrictive policies on the US stem cell field. Over time, non-US labs have freely used lines on the US registries, while federally funded US scientists have been limited to using those lines approved by the NIH.

As the healthcare industry moves from a twentieth century approach of providing treatments of last resort to a future of individualized medicine, biobanks will play a pivotal role in this transition. Yet at the cutting edge of biobanking research are new ethical, social and policy challenges beyond those familiar to basic biomedical research.

Human embryonic stem cell (hESC) research has sparked incredible scientific and public excitement as well as significant controversy. Because they are pluripotent, hESCs can in theory be differentiated into any type of cell found in the human body. Thus they evoke great enthusiasm about potential clinical applications. They are controversial because the method used to derive hESC lines destroys a 2-4 day old human embryo. Research and discoveries using human pluripotent stem cells are simultaneously cutting edge contributions to fundamental understanding and potentially invaluable sources of new treatments for some our most devastating diseases and injuries. Stem cell science represents an important case of “use-inspired basic research,” a class of scientific work that Donald Stokes (1997) compellingly argued could be used to reframe the increasingly fragile “contract” between science and society (Guston & Kenniston 1994). In this case, however, federal funding restrictions, legal challenges, and public controversy imposed on the field's development. Thus, hESC research also offers a “laboratory” for examining the effects high level science policy decisions have on the trajectory of an emerging scientific field. Today, nearly fifteen years after the discoveries that made human pluripotent stem cell science feasible, continued federal funding for this research is highly uncertain. We believe that federal funding for human pluripotent stem cell science should be expanded and stabilized through legislation. Explaining why requires that we begin with a simplified, schematic history of the field.

How has the development of human induced pluripotent stem cells (hiPSCs) modified the trajectory of stem cell research? Here, coauthorship networks of stem cell research articles and analysis of cell lines used in stem cell research indicate that hiPSCs are not replacing human embryonic stem cells, but instead, the two cell types are complementary, interdependent research tools. Thus, we conclude that a ban on funding for embryonic stem cell research could have unexpected negative ramifications on the nascent field of hiPSCs.

Electronic registries for clinical trials present an opportunity for miscommunication of benefits and risks to potential subjects. We explored early phase clinical trials registered on the National Library of Medicine's ClinicalTrials.gov for inappropriate language patterns (terms and statements) that could convey therapeutic intent. We sampled early-phase heart disease clinical trial records involving stem cells registered on Clincaltrials.gov between April 24, 2004, and September 18, 2008, and randomly selected heart disease drug trials from the same period for comparison. Trial sponsorship and location were coded. Textual data were extracted from titles, purpose, outcome measures, and detailed description statements and coded for readability and the presence of therapeutic language. Content and multiple correspondence analyses were employed to explore language patterns suggestive of therapeutic intent. We found statistically significant differences in therapeutic language use between stem cell (n = 72,...

This paper explores the notions of hope and how individual patient autonomy can trump carefully reasoned ethical concerns and policies intended to regulate stem cell transplants. We argue that the same limits of knowledge that inform arguments to restrain and regulate unproven treatments might also undermine our ability to comprehensively dismiss or condemn them. Incautiously or indiscriminately reasoned policies and attitudes may drive critical information and data underground, impel patients away from working with clinical researchers, and tread needlessly on hope, the essential motivator of patients, advocates and researchers alike. We offer recommendations to clinicians and health care providers to help balance the discourse with individuals seeking treatment while guarding against fraud, misconception, and patient harm.

Technology transfer offices are ubiquitous institutions within American universities. Most are underfunded and understaffed, will never turn a profit, drain limited university resources and potentially hinder innovation and knowledge transfer. Yet, inexplicably, new offices continue to be established. This article suggests that the continued hype over rare but lucrative blockbuster patents fuels much of this unnecessary expansion. These new offices, essentially mandated to discover the next big one, tend to make poor patenting and licencing decisions that effectively impinge on university knowledge transfer capabilities. This article suggests that although these offices may be necessary for some major research institutions, the bulk of academic research universities ought to share regional transfer offices that can benefit from economies of scale and experienced technology transfer officers. This article further suggests that the current status quo is the result of the somewhat arbitrary granting of patent rights to universities by the Carter era Bayh–Dole Act. Instead, this article proposes, in addition to a general overhaul in the methodology for transferring university knowledge, that a hochschullehrerprivileg or ‘professor’s privilege’ be established: academic inventors, not their bureaucratically bogged-down universities, should retain the patent rights that can then be licenced through the regional technology transfer offices without the, often ineffectual, university intervention. In addition to streamlining the current technology transfer process, such a system will also create strong incentives for research scientists to transfer knowledge and become more entrepreneurial. Although an overhaul of the American structure, while necessary, seems unlikely, such a system can nevertheless be implemented in both developed and developing countries that are futilely seeking to reproduce American successes through mimicking the current American Bayh–Dole arrangement.

The field of induced pluripotent stem cells (iPSCs) will be subject to a wide range of laws and research ethics policies, many of which exist as a result of the controversies associated with research on human embryonic stem cells. Understanding this potentially complex regulatory environment will help iPSC research move forward and will inform future policy.

Given the explosion of research on induced pluripotent stem (iPS) cells, it is timely to consider the various ethical, legal, and social issues engaged by this fast-moving field. Here, we review issues associated with the procurement, basic research, and clinical translation of iPS cells., iPS細胞研究を進めるための社会的課題と展望 －国際幹細胞学会でのクローズド・ワークショップの議論を基に－. 京都大学プレスリリース. 2009-12-11.

Document S1. Supplemental Findings, Supplemental Methods, Supplemental References, and One TablexDownload (.06 MB ) Document S1. Supplemental Findings, Supplemental Methods, Supplemental References, and One Table

The controversies surrounding embryonic stem cell research have prompted scientists to invent beyond restrictive national policy and moral concerns. The impetus behind these reports comes from different sources, including individually held moral beliefs, societal pressures and resource constraints, both biological and financial. Along with other contributions to public policy such as advocacy or public testimony, experimentation and scientific curiosity are perhaps more natural responses scientists use to surmount impediments to research. In a research context, we review the history of the first stem cell discoveries, and describe scientific efforts leading up to recent reports of pluripotent lines made without the use of human embryos and eggs. We argue that despite the promise of these new lines, we must not lose sight of fundamental questions remaining at the frontiers of embryology and early human development. The answers to these questions will impact studies of genetics, cell biology and diseases such as cancer, autoimmunity and disorders of development. Human embryonic stem cell research is barely a decade old. The recent pace of discovery--in spite of federal restrictions--is testament to the potential of these cells to uncover some of biology's most intractable mysteries.

The migration of researchers across geographic borders, or “brain drain” as it is commonly called, remains an important issue for governments around the world as loss or gain of highly qualified personnel in research can have substantial social, economic and political consequences. In the present study we seek to examine the forces that drive international professional migration of stem cell (SC) researchers, for which variation of SC policy in different jurisdictions has previously been implicated as a driving force. Structured interviews were carried out with a purposive sample of SC researchers in the professoriate who had made international moves after postdoctoral work between the years 2001–2014, or were actively anticipating a future move. Participants were asked to rank motivators of international movement on a 5-point Likert scale and prompted to elaborate on their answers. The results suggest that career considerations, availability of research funding, and personal considerations are of high importance to the participants when considering an international move, while the permissiveness or restrictiveness SC research policy is of comparably lower importance. Participants also expressed that international movements are beneficial to scientific careers overall. The findings have important implications for policy and strategies to attract and retain members of the SC research community.

Under the newly passed Leahy-Smith America Invents Act (AIA), the U.S. Patent and Trademark Office may hear new challenges to stem cell patents. Here, we explore how the new law affects challenges to stem cell patents, focusing on two recent cases, and discuss the future of stem cell patent disputes.

Traditional methods of instruction can fail to produce enduring ways of learning, especially in rapidly changing disciplines in the life sciences. Educators and funding agencies are thus calling for new, integrated teaching approaches to address the life sciences. Hierarchical frameworks are being proposed as ways to tackle curricula with large numbers of concepts. Comparing lecture-based and interactive formats by measuring performance with pre- and post-tests indicated significantly higher learning gains and better conceptual understanding in the more interactive course. Other work has examined how the history of biology can be used to infuse relevance into the biology curriculum. In this paper, I describe a project at Stanford to create, implement, and evaluate an integrated curriculum in undergraduate stem cell education comprised of scientific, laboratory, and ELSI topics.

Just as human-animal chimeras are beginning to provide tantalizing insights into fundamental scientific questions about human disease and consciousness, conservative US legislators and President Bush are launching a blistering moral attack. Christopher Thomas Scott investigates.

A decade of sound science and aggressive deal making has given Sangamo Biosciences a stranglehold on zinc finger technologies. Now, academic labs that helped build Sangamo's empire want in on the action. Are the ingredients ripe for a revolt that could break the company's monopoly? Christopher Thomas Scott investigates.

Using donated human embryos for scientific research raises ethical questions about the donation process. We describe a two-stage consent process designed to help couples make informed decisions about embryo disposition. This consent methodology minimizes conflict of interest, respects patient choice, and provides a much-needed resource to patients and the research community.

Summary Geron Corporation is a publically traded company that launched a phase I clinical trial of a human embryonic stem cell-based therapy for spinal cord injury. The company enrolled the first patient in October 2010 and stopped the trial 1 year later. The fifth patient had been enrolled but not transplanted when the company announced the trial's end. After discussions with clinical staff and family, an agreement was reached to add her to the cohort and proceed with the transplant. Two and half years later, the research is still waiting to restart. With this background in mind, we discuss the major ethical and social questions raised by the Geron case. We offer recommendations for institutional review boards and clinical sites as they deliberate approvals of early-phase trials in frontier medicine.

Too often, biopharmaceutical companies stop their clinical trials solely for financial reasons. In this chapter, we discuss this phenomenon against the backdrop of a 2011 decision by Geron Corporation to abandon its stem cell clinical trial for spinal cord injury (SCI), the preliminary results of which were released in May 2014. We argue that the resultant harms are widespread and are different in nature from the consequences of stopping trials for scientific or medical reasons. We examine the ethical and social effects that arise from such decisions and discuss them in light of ethical frameworks, including duties of individual stakeholders and corporate sponsors. We offer ways that sponsors and clinical sites can ensure that trials are responsibly started, and once started adequately protect the interests of participants. We conclude with recommendations that industry sponsors of clinical trials should adopt in order to advance a collective and patient-centered research ethic.

New applications of iPSC technology to research on complex idiopathic conditions raise several important ethical and social considerations for potential research participants and their families. In this short review, we examine these issues through the lens of emerging research on autism spectrum disorder (ASD). We begin by describing the current state of iPSC technology in research on ASD. Then we discuss how the social history of and current controversies in autism research combined with the emergence of autism-specific iPSC biobanks indicate an urgent need for researchers to clearly communicate the limitations and possibilities of iPSC research to ensure research participants have the ability to provide fully informed, voluntary consent. We conclude by offering recommendations to bolster informed consent for research involving iPSC biobanks, both in the specific context of ASD and more broadly.

An examination of three patents in the fast-moving iPS space may help determine their ultimate value.

Years of tinkering with the mouse immune system genes has finally produced a winner, with the approval of the first fully human monoclonal antibody made in a mouse. A real breakthrough or an incremental improvement? Christopher Thomas Scott investigates.

In April 2015 a team of Chinese scientists reported in a little known journal, Protein & Cell, the use of CRISPR/Cas9 to cleave and then repair the HBB gene in nonviable human embryos (Liang et al....

Too often, biopharmaceutical companies stop their clinical trials solely for financial reasons. In this chapter, we discuss this phenomenon against the backdrop of a 2011 decision by Geron Corporation to abandon its stem cell clinical trial for spinal cord injury (SCI), the preliminary results of which were released in May 2014. We argue that the resultant harms are widespread and are different in nature from the consequences of stopping trials for scientific or medical reasons. We examine the ethical and social effects that arise from such decisions and discuss them in light of ethical frameworks, including duties of individual stakeholders and corporate sponsors. We offer ways that sponsors and clinical sites can ensure that trials are responsibly started, and once started adequately protect the interests of participants. We conclude with recommendations that industry sponsors of clinical trials should adopt in order to advance a collective and patient-centered research ethic.

Industry hopes to build on recent successes of monoclonal antibodies in oncology and inflammatory disease. But evidence is mounting that the exquisite selectivity and binding capacity of these therapeutics can have unwanted side effects, particularly in autoimmune disease. Christopher Thomas Scott investigates.

The nature of compensation for women who donate eggs (oocytes) for research remains a contentious issue internationally. This position paper lays out the arguments for, and discusses the arrangements in which, a modest payment might be ethically justifiable.

Why did Geron—corporate standard bearer for regenerative medicine—fail, whereas technology pioneers in other areas persist?