Douglas Sipp

Monitoring and Regulating Offshore Stem Cell Clinics

), butthere are many advertisements for medicalprocedures that have never been provenefficacious in appropriately designedclinical trials. To date, proven thera-peutic applications for stem cellshave been mainly for blood andimmunological disorders. Thescientific community and ad-vocacy groups have begun torespond by formulating guide-lines for physicians andscientists engaged in theclinical translation of stemcell research (

Setting Global Standards for Stem Cell Research and Clinical Translation: The 2016 ISSCR Guidelines

The International Society for Stem Cell Research (ISSCR) presents its 2016 Guidelines for Stem Cell Research and Clinical Translation (ISSCR, 2016). The 2016 guidelines reflect the revision and extension of two past sets of guidelines (ISSCR, 2006, ISSCR, 2008) to address new and emerging areas of stem cell discovery and application and evolving ethical, social, and policy challenges. These guidelines provide an integrated set of principles and best practices to drive progress in basic, translational, and clinical research. The guidelines demand rigor, oversight, and transparency in all aspects of practice, providing confidence to practitioners and public alike that stem cell science can proceed efficiently and remain responsive to public and patient interests. Here, we highlight key elements and recommendations in the guidelines and summarize the recommendations and deliberations behind them.

Tissue interactions in neural crest cell development and disease.

Neural Crest Development The vertebrate neural crest is characterized by a migratory population of multipotent cells that spread out from the dorsal side of the neural tube. Many different cell types and tissues originate here, including cells of the peripheral nervous system, the adrenal medulla, melanocytes, and some skeletal cells. Dysregulation of neural crest cells can lead to defects in cell differentiation and the cell cycle, as well as to the formation of ectopic tissue, which can result in various human diseases. Takahashi et al. (p. 860) review the normal development of neural crest cells, highlighting important associations of this cell population with local environments to influence tissue interactions and function, and describe pathogenesis that results when developmental events go awry. The neural crest is a transient population of migratory cells in the embryo that gives rise to a wide variety of different cell types, including those of the peripheral nervous system. Dysfunction of neural crest cells (NCCs) is associated with multiple diseases, such as neuroblastoma and Hirschsprung disease. Recent studies have identified NCC behaviors during their migration and differentiation, with implications for their contributions to development and disease. Here, we describe how interactions between cells of the neural crest and lineages such as the vascular system, as well as those involving environmental signals and microbial pathogens, are critically important in determining the roles played by these cells.

Global Distribution of Businesses Marketing Stem Cell-Based Interventions.

A structured search reveals that online marketing of stem-cell-based interventions is skewed toward developed economies including the United States, Ireland, Australia, and Germany. Websites made broad, imprecise therapeutic claims and frequently failed to detail procedures. Widespread marketing poses challenges to regulators, bioethicists, and those seeking realistic hope from therapies.

Confronting stem cell hype

Against hyperbole, distortion, and overselling The way science is represented to the public can influence understanding and expectations, frame policy debates, and affect the implementation and use of emerging technologies. Inaccurate representations of research may, for example, lead to public confusion about the readiness of a technology for clinical application. As a result, the issue of science “hype”—in which the state of scientific progress, the degree of certainty in models or bench results, or the potential applications of research are exaggerated—is receiving increased attention from the popular press, the research community, and scientific societies (1). In newly issued guidelines on the ethical conduct of human pluripotent stem cell research and clinical translation (2), the International Society for Stem Cell Research (ISSCR) explicitly recognizes and confronts the issue of science hype. By placing a clear obligation on researchers, the ISSCR hopes to make balance in public representations of research a norm associated with scientific integrity. The focus on public communication, which is new to this version of the guidelines, is the result of both specific concerns regarding how stem cell research has been portrayed in the public sphere and the growing recognition that researchers play an important role in the science communication process.

Patients Beware: Commercialized Stem Cell Treatments on the Web

A report by the International Society for Stem Cell Research (ISSCR)s Task Force on Unproven Stem Cell Treatments outlines development of resources for patients, their families, and physicians seeking information on stem cell treatments.

Points to consider in the development of seed stocks of pluripotent stem cells for clinical applications: International Stem Cell Banking Initiative (ISCBI)

In 2009 the International Stem Cell Banking Initiative (ISCBI) contributors and the Ethics Working Party of the International Stem Cell Forum published a consensus on principles of best practice for the procurement, cell banking, testing and distribution of human embryonic stem cell (hESC) lines for research purposes [1], which was broadly also applicable to human induced pluripotent stem cell (hiPSC) lines. Here, we revisit this guidance to consider what the requirements would be for delivery of the early seed stocks of stem cell lines intended for clinical applications. The term ‘seed stock’ is used here to describe those cryopreserved stocks of cells established early in the passage history of a pluripotent stem cell line in the lab that derived the line or a stem cell bank, hereafter called the ‘repository’.

U.S. Regulation of Stem Cells as Medical Products

Premarketing approval by the federal government should ensure not only safety, but efficacy as well. A recent decision by a U.S. District Court judge could have profound implications for the increasing number of U.S. clinics that advertise putative “stem cell treatments” for a wide range of clinical, rejuvenation, and aesthetic applications. In United States v. Regenerative Sciences LLC et al., the court upheld the authority of the Food and Drug Administration (FDA) to require the premarketing approval of human stem cell–derived products that meet any of several broad criteria (1). The court concluded that the cultured autologous mesenchymal stem cell–based product at issue in the case is subject to FDA regulatory oversight, as it meets criteria for classification as a biological drug, and its manufacture, distribution, and sale constitute interstate commerce. Although the court described the decision, which has been appealed, as “a close question,” this new ruling reaffirming FDA authority has prompted heightened interest in the regulation of stem cells as medical products.

Marketing of unproven stem cell–based interventions: A call to action

Commercial promotion of unsupported therapeutic uses of stem cells is a global problem that should be addressed by coordinated approaches at the national and international levels. Commercial promotion of unsupported therapeutic uses of stem cells is a global problem that has proven resistant to regulatory efforts. Here, we suggest a coordinated approach at the national and international levels focused on engagement, harmonization, and enforcement to reduce the risks associated with direct-to-consumer marketing of unproven stem cell treatments.

Conditional Approval: Japan Lowers the Bar for Regenerative Medicine Products

A new system for conditional approval of regenerative medicine products will allow products of undetermined efficacy to enter the Japanese market. The potential scientific, economic, and ethical implications of this program highlight the need for further discussion and refinement.