Search

The International Society for Stem Cell Research has updated its Guidelines for Stem Cell Research and Clinical Translation in order to address advances in stem cell science and other relevant fields, together with the associated ethical, social, and policy issues that have arisen since the last update in 2016. While growing to encompass the evolving science, clinical applications of stem cells, and the increasingly complex implications of stem cell research for society, the basic principles underlying the Guidelines remain unchanged, and they will continue to serve as the standard for the field and as a resource for scientists, regulators, funders, physicians, and members of the public, including patients. A summary of the key updates and issues is presented here.

The field of regenerative medicine is widely viewed as having the potential to improve treatment options for a broad range of conditions. Stem cell research in particular has been celebrated for its considerable clinical promise. Although measured enthusiasm surrounding this area of research is warranted, it must be balanced by patience and set in the context of a long-term perspective that is cognizant of the many steps required to bring safe and efficacious therapies to market. Creating therapeutic applications of stem cell technologies is an intricate process involving complex biology. It will require careful scientific investigation and evaluation under responsible ethical frameworks and regulatory standards in order to safely maximize their potential. Alongside the many promising avenues of responsible research currently underway in countries throughout the world, a global market has emerged where a wide range of putative stem cell-based interventions are sold on a direct-to-consumer basis and marketed over the internet.1,2,3,4

Repair and regeneration of a diseased lung using stem cells or bioengineered tissues is an exciting therapeutic approach for a variety of lung diseases and critical illnesses. Over the past decade, increasing evidence from preclinical models suggests that mesenchymal stromal cells, which are not normally resident in the lung, can be used to modulate immune responses after injury, but there have been challenges in translating these promising findings to the clinic. In parallel, there has been a surge in bioengineering studies investigating the use of artificial and acellular lung matrices as scaffolds for three-dimensional lung or airway regeneration, with some recent attempts of transplantation in large animal models. The combination of these studies with those involving stem cells, induced pluripotent stem cell derivatives, and/or cell therapies is a promising and rapidly developing research area. These studies have been further paralleled by significant increases in our understanding of the molecular and cellular events by which endogenous lung stem and/or progenitor cells arise during lung development and participate in normal and pathological remodeling after lung injury. For the 2023 Stem Cells, Cell Therapies, and Bioengineering in Lung Biology and Diseases Conference, scientific symposia were chosen to reflect the most cutting-edge advances in these fields. Sessions focused on the integration of "omics" technologies with function, the influence of immune cells on regeneration, and the role of the extracellular matrix in regeneration. The necessity for basic science studies to enhance fundamental understanding of lung regeneration and to design innovative translational studies was reinforced throughout the conference. [ABSTRACT FROM AUTHOR]

Patients with chronic pain face significant barriers in finding clinicians to manage long-term opioid therapy (LTOT). For patients on LTOT, it is increasingly common to have them sign opioid treatment agreements (OTAs). OTAs enumerate the risks of opioids, as informed consent documents would, but also the requirements that patients must meet to receive LTOT. While there has been an ongoing scholarly discussion about the practical and ethical implications of OTA use in the abstract, little is known about how clinicians use them and if OTAs themselves modify clinician prescribing practices. To determine how clinicians use OTAs and the potential impacts of OTAs on opioid prescribing. We conducted qualitative analysis of four focus groups of clinicians from a large Midwestern academic medical center. Groups were organized according to self-identified prescribing patterns: two groups for clinicians who identified as prescribers of LTOT, and two who did not. 17 clinicians from General Internal Medicine, Family Medicine, and Palliative Care were recruited using purposive, convenience sampling. Discussions were recorded, transcribed, and analyzed for themes using reflexive thematic analysis by a multidisciplinary team. Our analysis identified three main themes: (1) OTAs did not influence clinicians' decisions whether to use LTOT generally but did shape clinical decision-making for individual patients; (2) clinicians feel OTAs intensify the power they have over patients, though this was not uniformly judged as harmful; (3) there is a potential misalignment between the intended purposes of OTAs and their implementation. This study reveals a complicated relationship between OTAs and access to pain management. While OTAs seem not to impact the clinicians' decisions about whether to use LTOT generally, they do sometimes influence prescribing decisions for individual patients. Clinicians shared complex views about OTAs' purposes, which shows the need for more clarity about how OTAs could be used to promote shared decision-making, joint accountability, informed consent, and patient education. [ABSTRACT FROM AUTHOR]

In several controversial drug approval decisions in recent years, the Food & Drug Administration ("FDA") has publicly justified its decision partly on the ground that approving the drugs in question would support innovation in those fields going forward. To some observers, these arguments were surprising, as the Agency's determination whether a drug is "safe" and "effective" does not seem to depend on whether its approval also supports innovation. But FDA'suse of these innovation arguments in drug approval decisions is just one example of the ways in which the Agency has come to make many innovation-related judgments as part of its regulation of drugs. In this Article, we investigate the broad set of innovation-related judgments that FDA has been making and argue that there are serious concerns with the major innovation role FDA has been playing, at least as the Agency is currently constituted. We conclude that FDA should not separately weigh innovation in decisions about a product's safety and effectiveness. In other areas, health policymakers could reasonably decide that FDA should have either a larger or a smaller role than it currently does in shaping the development of novel drugs. But policymakers should do so while thoughtfully considering both the opportunities and challenges of FDA actively considering innovation incentives in its decisions; those challenges have been rarely considered in the literature and policy discourse. Further, we argue that whether policymakers aim to bolster or limit the ways that FDA considers innovation in its regulatory decisions, changes are needed to the Agency's structure to support its ability to make reasoned judgments based on relevant expertise. [ABSTRACT FROM AUTHOR]

INTRODUCTION 1401 I. CRISPR SCIENCE AND APPLICATIONS 1409 A. CRISPR Science 1409 B. Target Cells 1410 C. Human Applications 1411 II. THE USE OF CRISPR BY DIY BIOLOGISTS 1415 A. [...], Although scientists have been manipulating genomes since the 1970s, the recent discovery of Clustered Regularly Interspaced Short Palindromic Repeats ("CRISPR") has expanded the possibilities not only for what gene editing might accomplish but also who might accomplish it. Because CRISPR is relatively easy, efficient, and inexpensive, it is accessible to individuals--known as "citizen scientists"--who work in nontraditional laboratory settings and may not have formal scientific training. Prompted by concerns about human applications of CRISPR, the United States is cohosting a series of international summits on human gene editing, while organizations around the world race to issue their own reports and recommendations. For the most part, however, these efforts have focused on the use of CRISPR by professional scientists working in institutional settings who are already subject to layers of formal and informal oversight. They have largely overlooked the do-it-yourself ("DIY") use of CRISPR by citizen scientists--even as instances of self-experimentation with CRISPR are being reported and raising unique concerns. Drawing on qualitative interviews with almost forty citizen scientists and their supporters, critics, and observers, this Article provides a critical analysis of the practice and governance of DIY CRISPR in the United States. It concludes that existing laws and regulations potentially reach a number of DIY CRISPR activities, although their application to citizen-science contexts is thus far untested. Meanwhile, DIY communities have developed mechanisms of self-regulation that appear to be working reasonably well thus far in discouraging potentially dangerous human applications of CRISPR by citizen scientists. However, we are concerned about the possibility that, as lay understanding of and proficiency with the technology increases, there will be an uptick in risky (if not illegal) human experimentation in the future. Therefore, this Article concludes with suggestions for shoring up the oversight readiness and capacities of regulatory bodies and DIY communities.