Between the Local and the Global: Evaluating European regulation of stem cell regenerative medicine

Aims Bone marrow-derived mononuclear cell (BM-MNC) therapy may improve myocardial recovery in patients following acute myocardial infarction (AMI), though existing trial results are inconsistent. Methods and results Originally an open-label, multicentre Phase III trial, BAMI was designed to demonstrate the safety and efficacy of intracoronary infusion of BM-MNCs in reducing the time to all-cause mortality in patients with reduced left ventricular ejection fraction (LVEF, ≤45%) after primary angioplasty (PPCI) for ST-elevation AMI. Unexpectedly low recruitment means the trial no longer qualifies as a hypothesis-testing trial, but is instead an observational study with no definitive conclusions possible from statistical analysis. In total, 375 patients were recruited: 185 patients were randomized to the treatment arm (intracoronary infusion of BM-MNCs 2–8 days after PPCI) and 190 patients to the control arm (optimal medical therapy). All-cause mortality at 2 years was 3.26% [6 deaths; 95% confidence interval (CI): 1.48–7.12%] in the BM-MNC group and 3.82% (7 deaths; 95% CI: 1.84–7.84%) in the control group. Five patients (2.7%, 95% CI: 1.0–5.9%) in the BM-MNC group and 15 patients (8.1%, CI : 4.7–12.5%) in the control group were hospitalized for heart failure during 2 years of follow-up. Neither adverse events nor serious adverse events differed between the two groups. There were no patients hospitalized for stroke in the control group and 4 (2.2%) patients hospitalized for stroke in the BM-MNC group. Conclusions Although BAMI is the largest trial of autologous cell-based therapy in the treatment of AMI, unexpectedly low recruitment and event rates preclude any meaningful group comparisons and interpretation of the observed results.

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“…Although ultimately the logistical issues were solved, the cost in time and money significantly impacted the success of the trial. 19 …”

Section: Discussionmentioning

confidence: 99%

The effect of intracoronary infusion of bone marrow-derived mononuclear cells on all-cause mortality in acute myocardial infarction: the BAMI trial

Mathur

Fernández‐Avilés

Bartúnek

et al. 2020

European Heart Journal

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“…Nearly a third of publications (29%; n = 25) mentioned financial costsand in particular the out-of-pocket expenses-associated with DTC products and services. Many highlighted the significant financial investment required to obtain DTC stem cell treatments [49,[70][71][72], which can cost tens of thousands of dollars [46,53,73]. Concerns were also raised about the potential financial burden of DTC testing such as cardiac screening and p-tau blood testing [69,74].…”

Section: Individual-related Ethical Concernsmentioning

confidence: 99%

Ethical issues in direct-to-consumer healthcare: A scoping review

Nagappan,

Kalokairinou,

Wexler

2024

PLOS Digit Health

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An increasing number of health products and services are being offered on a direct-to-consumer (DTC) basis. To date, however, scholarship on DTC healthcare products and services has largely proceeded in a domain-specific fashion, with discussions of relevant ethical challenges occurring within specific medical specialties. The present study therefore aimed to provide a scoping review of ethical issues raised in the academic literature across types of DTC healthcare products and services. A systematic search for relevant publications between 2011–2021 was conducted on PubMed and Google Scholar using iteratively developed search terms. The final sample included 86 publications that discussed ethical issues related to DTC healthcare products and services. All publications were coded for ethical issues mentioned, primary DTC product or service discussed, type of study, year of publication, and geographical context. We found that the types of DTC healthcare products and services mentioned in our sample spanned six categories: neurotechnology (34%), testing (20%), in-person services (17%), digital health tools (14%), telemedicine (13%), and physical interventions (2%). Ethical arguments in favor of DTC healthcare included improved access (e.g., financial, geographical; 31%), increased autonomy (29%), and enhanced convenience (16%). Commonly raised ethical concerns included insufficient regulation (72%), questionable efficacy and quality (70%), safety and physical harms (66%), misleading advertising claims (56%), and privacy (34%). Other frequently occurring ethical concerns pertained to financial costs, targeting vulnerable groups, informed consent, and potential burdens on healthcare providers, the healthcare system, and society. Our findings offer insights into the cross-cutting ethical issues associated with DTC healthcare and underscore the need for increased interdisciplinary communication to address the challenges they raise.

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“…Auf Basis dessen lässt sich fragen, welche Formen und Kriterien für die Sicherheit und Wirksamkeit der klinischen Translation der Organoidforschung angemessen und welche Hürden absehbar sind. Im Zusammenhang damit sind einerseits die regulatorischen Herausforderungen des anvisierten Überganges organoid-basierter biomedizinischer Innovation in die Klinik vor dem Hintergrund vielfältiger Formen epistemischer Unsicherheit von Interesse, die bereits die klinische Translation der Stammzellforschung erschweren (Hauskeller 2018), andererseits sind die von Haddad (2019) in Bezug auf Stammzellen thematisierten Formen, Probleme und Herausforderungen post-pharmazeutischer Modelle der Gesundheitsforschung und -versorgung auch für Organoide relevant.…”

Section: 14unclassified