Search

Summary We assessed the extent to which the publication of clinical trial results of innovative cell-based interventions reflects International Society for Stem Cell Research best practice guidelines. We assessed: (1) characteristics and time to publication of completed trials; (2) quality of reported trials; and (3) results of published trials. We identified and analyzed publications from 1,052 novel stem cell clinical trials: 179 (45.4%) of 393 completed trials had published results; 48 trials were registered by known stem cell tourism clinics, none of which reported results. Completed non-industry-sponsored trials initially published more rapidly, but differences with industry-sponsored trials decreased over time. Most publications reported safety, and 67.3% (mainly early-stage trials) reported positive outcomes. A higher proportion of industry trials reported positive efficacy. Heightened patient expectations for stem cell therapies give rise to ethical obligations for the transparent conduct of clinical trials. Reporting guidelines need to be developed that are specific to early-phase clinical trials., Highlights • We assessed clinical trial publications of innovative cell-based interventions • Only 54.5% of completed trials reported results • Private clinics known to provide unproven stem cell therapies register “trials” • Trial reporting guidelines are needed, specific to early-phase clinical trials, Fung and colleagues assessed publication patterns of clinical trials of novel stem cell therapies. Trial results remain underreported and stem cell tourism clinics are promoting their clinic activities on trial registries. Clinical trial reporting guidelines specific to early-phase clinical trials are needed.

Bio-repositories and databases for biomedical research enable the efficient community-wide sharing of reagents and data. These archives play an increasingly prominent role in the generation and dissemination of bioresources and data essential for fundamental and translational research. Evidence suggests, however, that current funding and governance models, generally short-term and nationally focused, do not adequately support the role of archives in long-term, transnational endeavours to make and share high-impact resources. Our qualitative case study of the International Knockout Mouse Consortium and the International Mouse Phenotyping Consortium examines new governance mechanisms for archive sustainability. Funders and archive managers highlight in interviews that archives need stable public funding and new revenue-generation models to be sustainable. Sustainability also requires archives, journal publishers, and funders to implement appropriate incentives, associated metrics, and enforcement mechanisms to ensure that researchers use archives to deposit reagents and data to make them publicly accessible for academia and industry alike.

Whereas biological materials were once transferred freely, there has been a marked shift in the formalisation of exchanges involving these materials, primarily through the use of Material Transfer Agreements (MTAs). This paper considers how risk aversion dominates MTA negotiations and the impact it may have on scientific progress. Risk aversion is often based on unwarranted fears of incurring liability through the use of a material or loss of control or missing out on commercialisation opportunities. Evidence to date has suggested that complexity tends to permeate even straightforward transactions despite extensive efforts to implement simple, standard MTAs. We argue that in most cases, MTAs need do little more than establish provenance, and any attempt to extend MTAs beyond this simple function constitutes stifling behaviour. Drawing on available examples of favourable practice, we point to a number of strategies that may usefully be employed to reduce risk-averse tendencies, including the promotion of simplicity, education of those engaged in the MTA process, and achieving a cultural shift in the way in which technology transfer office (TTO) success is measured in institutions employing MTAs.

Natural resource development is accelerating in the circumpolar region, raising questions about the balance between potential economic benefits for northern indigenous peoples with the risks of increasing long-standing health inequities. New communication tools are needed to document and synthesize complex and diverse evidence of health impacts in a context relevant for natural resource management. Our research examines the perspectives of circumpolar experts on Geographic Information Systems (GIS) as a platform to synthesize and communicate scientific and indigenous knowledge. Thirty policy makers, academic researchers, and community-level practitioners participated in semi-structured interviews examining indigenous community health, natural resource management, and the use of GIS within Arctic and subarctic communities. Qualitative analysis of the interview transcripts for key themes indicated that the majority of circumpolar experts supported employing GIS to facilitate more extensive collaboration with indigenous communities and to produce higher quality data outputs for decision-making forums. To ensure GIS enables natural resource development decision-making to improve the social and economic welfare of indigenous people, experts recommended increasing communities’ access to technology and training, community stewardship of data, utilizing data for ongoing monitoring of development impacts, and coordinating cumulative impact monitoring within regions. Findings focus on circumpolar natural resource management in jurisdictions with a mandate for public participation, with relevance to improving accountability to indigenous populations in other regions.

Purpose: Ocular gene transfer clinical trials are raising patient hopes for the treatment of choroideremia – a blinding degenerative retinopathy. Phase I choroideremia gene transfer trials necessitate communicating about the risks of harm and potential benefits with patients while avoiding the sensationalism that has historically undermined this field of translational medicine. Methods: We conducted interviews between June 2011 and June 2012 with 6 choroideremia patient advocates, 20 patients, and 15 clinicians about their hopes for benefits, perceived risks of harm, and hopes for the time frame of clinical implementation of choroideremia gene transfer. Results: Despite the safety focus of phase I trials, participants hoped for direct visual benefits with evident discrepancies between stakeholder perspectives about the degree of visual benefit. Clinicians and patient advocates were concerned by limited patient attention to risks of harm. Interviews revealed confusion about the time frames for the clinical implementation of choroideremia gene transfer and patient urgency to access gene transfer within a limited therapeutic window. Conclusion: Differences in stakeholder perspectives about choroideremia gene transfer necessitate strategies that promote responsible communications about choroideremia gene transfer and aid in its translation. Strategies should counter historical sensationalism associated with gene transfer, promote informed consent, and honor patient hope while grounding communications in current clinical realities.

Intellectual property occupies a central position in the biotechnology innovation system, the expected source of new medicines, foods and bio-energy. An international and inter-disciplinary research team has convened for the last seven years in an attempt to better understand the mechanisms of intellectual property in biotechnology innovation, and to suggest improvements to the role of intellectual property in that system. This report represents the research team's core findings and recommendations.