Stem cell-based innovation is one pathway to clinical translation that stands in contrast to clinical research and medical treatment. After reviewing recently issued guidelines for responsible innovation, this article examines the potential benefits and harms of using this pathway as well as practical barriers and conceptual concerns regarding it.

Criticisms of the traditional clinical research pathway and its extensive oversight often focus on proposals for deregulation or assert that as in clinical treatment, clinical research should always offer benefit to patient-subjects. Proponents of medical innovation take a different, middle path, arguing that innovation is distinguishable from both research and treatment. This article considers this third pathway by examining stem cell-based innovation.Stem cell-based medical innovation is one pathway toward clinical translation. In fact, such an approach was taken in developing umbilical (...) cord blood transplantation, which is now widely viewed as a safe and effective means for treating a range of malignant and non-malignant conditions. While the Guidelines for the Clinical Translation of Stem Cells created by the International Society for Clinical Research provide an approach to clinical translation that involves “responsible innovation” as an alternative to clinical research, there are at least four sets of unanswered questions regarding the use of an innovation pathway in this context that warrant careful attention. (shrink)

Demands for improved access to innovative therapies have prompted a discourse that claims patients have rights to access treatments that may be of benefit, even if evidence that demonstrates safety and efficacy is lacking. This rights-based discourse is grounded in accounts of autonomy and assertions claiming that the state ought to not interfere with the free choices of patients and clinical decision-making. In this essay, we scrutinise these arguments to defend the ethical and legal permissibility of interference in contexts where (...) the uncertainty of benefit and potential for harm creates vulnerabilities that undermine patient capacity for self-determination. In support of this argument, we draw on two theoretical approaches to explore the limits of autonomy in innovative contexts and analyse the legal bases of the rights-based discourse. We then apply this analysis to the case example of stem cell transplantation as an innovative treatment for multiple sclerosis. (shrink)

This paper discusses exceptional circumstances under which patients outside of clinical trials are likely to receive innovative stem cell-based interventions. These circumstances involve: (1) stem cell interventions not initially amenable to a clinical trials approach; (2) expanded access to investigational stem cell products (“compassionate use”); and (3) off-label uses of FDA approved stem cell products. This paper proposes a new approach to regulating these exceptional cases.

Armed with expanded federal funding for human embryonic stem cell research and new methods for deriving pluripotent stem cells, stem cell researchers in the U.S. are poised to proceed with unprecedented speed toward the development of new clinical therapies. Staring into the new dawn of regenerative medicine, many observers may assume that the only responsible route to the clinic, both scientifically and ethically, is through FDA-approved clinical trials processes. Conventional wisdom dictates that, like pharmaceutical drugs and the use of biological (...) therapeutics, stem cell-based therapies will have to pass through a series of controlled studies — initially to assess safety and toxicity, then efficacy and comparative efficacy — before they can be justifiably administered to patients outside a clinical trials context. (shrink)

Innovative practice occurs when a clinician provides something new, untested, or nonstandard to a patient in the course of clinical care, rather than as part of a research study. Commentators have noted that patients engaged in innovative practice are at significant risk of suffering harm, exploitation, or autonomy violations. By creating a pathway for harmful or nonbeneficial interventions to spread within medical practice without being subjected to rigorous scientific evaluation, innovative practice poses similar risks to the wider community of patients (...) and society as a whole. Given these concerns, how should we control and oversee innovative practice, and in particular, how should we coordinate innovative practice and clinical research? In this article, I argue that an ethical approach to overseeing innovative practice must encourage the early transition to rigorous clinical research without delaying or deferring the development of beneficial innovations or violating the autonomy rights of clinicians and their patients. (shrink)

Controversy swept the U.K. in January of 2000 over public disclosure of the fact that a Scottish surgeon named Robert Smith had amputated the limbs of two able-bodied individuals who reportedly suffered from a condition known as apotemnophilia. The patients, both of whom had sought and consented to the surgery, claimed they had desperately desired for years to live as amputees and had been unable, despite considerable efforts, to reconcile themselves psychologically to living with the bodies with which they were (...) born. Both surgeries were successful, and both patients, who had undergone psychiatric evaluation prior to the amputations, subsequently reported having no regrets. In the wake of a wave of sensationalistic stories in the media, the hospital at which the surgeries had been performed, the Falkirk and District Royal Infirmary, banned any future surgeries of the kind. Outraged local politicians promptly announced their intention to pass laws banning the procedure outright. One member of Scottish Parliament declared the surgery obscene and asserted that the whole thing is repugnant and legislation needs to be brought in now to outlaw this. (shrink)

Controversy swept the U.K. in January of 2000 over public disclosure of the fact that a Scottish surgeon named Robert Smith had amputated the limbs of two able-bodied individuals who reportedly suffered from a condition known as apotemnophilia. The patients, both of whom had sought and consented to the surgery, claimed they had desperately desired for years to live as amputees and had been unable, despite considerable efforts, to reconcile themselves psychologically to living with the bodies with which they were (...) born. Both surgeries were successful, and both patients, who had undergone psychiatric evaluation prior to the amputations, subsequently reported having no regrets. In the wake of a wave of sensationalistic stories in the media, the hospital at which the surgeries had been performed, the Falkirk and District Royal Infirmary, banned any future surgeries of the kind. Outraged local politicians promptly announced their intention to pass laws banning the procedure outright. One member of Scottish Parliament declared the surgery obscene and asserted that the whole thing is repugnant and legislation needs to be brought in now to outlaw this. (shrink)