The evolution and ongoing challenge of unproven cell-based interventions.

Unproven cell-based interventions (CBIs) emerged early in the 2000s as a particularly problematic form of unproven therapy and remain a vexing policy problem to this day. These unproven interventions can harm patients both physically and financially and can complicate the process of developing a rigorous evidence base to support the translation of novel stem cell or other cell therapies. In this concise review, we examine the emergence of unproven CBIs and the various policy approaches that have been pursued or proposed to address this problem.

International Society for Cell & Gene Therapy Position Paper: Key considerations to support evidence-based cell and gene therapies and oppose marketing of unproven products.

The field of regenerative medicine, including cellular immunotherapies, is on a remarkable growth trajectory. Dozens of cell-, tissue- and gene-based products have received marketing authorization worldwide while hundreds-to-thousands are either in preclinical development or under clinical investigation in phased clinical trials. However, the promise of regenerative therapies has also given rise to a global industry of direct-to-consumer offerings of prematurely commercialized cell and cell-based products with unknown safety and efficacy profiles.

An International Society for Cell & Gene Therapy working group short report on the future of expanded access to unapproved cell and gene therapies.

Patient interest in non-trial access pathways to investigational cell-and gene-based interventions, such as expanded access in the USA, is increasing, while the regulatory and business environments for non-trial access in the cell and gene therapy field are shifting. Against this background, in 2022 the International Society for Cell & Gene Therapy (ISCT) established a Working Group on Expanded Access to identify practical, ethical, and regulatory issues emerging from the use (and possible misuse) of the expanded access pathway in the cell and gene therapy field. In this Short Report, the Working Group sets the stage for its future activities by analyzing the history of expanded access and identifying three examples of questions that we anticipate arising as uses of expanded access for investigational cell and gene-based interventions increase and evolve.

Navigating ethical challenges in the development and translation of biomaterials research.

Biomaterials--from implanted iron teeth in the second century to intraocular lenses, artificial joints, and stents today--have long been used clinically. Today, biomaterials researchers and biomedical engineers are pushing beyond these inert synthetic alternatives and incorporating complex multifunctional materials to control biological interactions and direct physiological processes. These advances are leading to novel strategies for targeted drug delivery, drug screening, diagnostics and imaging, gene therapy, tissue regeneration, and cell transplantation.

Ethical issues and public communication in the development of cell-based treatments for COVID-19: Lessons from the pandemic.

The significant morbidity and mortality of coronavirus disease 19 (COVID-19) prompted a global race to develop new therapies. These include interventions using cell- or cell-derived products, several of which are being tested in well-designed, properly controlled clinical trials. Yet, the search for cell-based COVID-19 treatments has also been fraught with hyperbolic claims; flouting of crucial regulatory, scientific, and ethical norms; and distorted communication of research findings.

Decadal climate variability in the tropical Pacific: Characteristics, causes, predictability, and prospects.

Climate variability in the tropical Pacific affects global climate on a wide range of time scales. On interannual time scales, the tropical Pacific is home to the El Niño–Southern Oscillation (ENSO). Decadal variations and changes in the tropical Pacific, referred to here collectively as tropical Pacific decadal variability (TPDV), also profoundly affect the climate system.

The Impact of Upcoming Treatments in Huntington's Disease: Resource Capacity Limitations and Access to Care Implications.

Background: The most advanced disease-modifying therapies (DMTs) in development for Huntington's disease (HD) require intrathecal (IT) administration, which may create or exacerbate bottlenecks in resource capacity.

Objective: To understand the readiness of healthcare systems for intrathecally administered HD DMTs in terms of resource capacity dynamics and implications for patients' access to treatment.

Methods: Forty HD centres across 12 countries were included.

Recurrent heart failure hospitalizations increase the risk of cardiovascular and all-cause mortality in patients with heart failure in Sweden: a real-world study.

Aims: Heart failure (HF) is a leading cause of hospitalization and is associated with high morbidity and mortality. We examined the impact of recurrent HF hospitalizations (HFHs) on cardiovascular (CV) mortality among patients with HF in Sweden.

Methods And Results: Adults with incident HF were identified from linked national health registers and electronic medical records from 01 January 2005 to 31 December 2013 for Uppsala and until 31 December 2014 for Västerbotten.

A Bibliometric Measure of Translational Science.

Science funders are increasingly requiring evidence of the broader impacts of even basic research. Initiatives such as NIH's CTSA program are designed to shift the research focus toward more translational research. However, tracking the effectiveness of such programs depends on developing indicators that can track the degree to which basic research is influencing clinical research.

Burden of Treatment-Induced Peripheral Neuropathy in Patients with Multiple Myeloma in Sweden.

Introduction: Treatment-induced peripheral neuropathy (TIPN) is a complication of multiple myeloma (MM) treatment.

Objective: This real-world, retrospective study used electronic medical record (EMR) data from 3 Swedish clinics to assess the occurrence and economic burden of TIPN in patients with MM.

Methods: Eligible patients had an MM diagnosis in the Swedish Cancer Registry between 2006 and 2015 and initiated treatment during that period.

Epidemiology of Pulmonary Fibrosis: A Cohort Study Using Healthcare Data in Sweden.

Introduction: Data on the epidemiology of idiopathic pulmonary fibrosis (IPF) in Sweden are lacking. This study estimates the incidence and prevalence of IPF in Sweden, and describes the demographic and clinical characteristics and the overall survival of patients with IPF.

Methods: Two cohorts were studied: a national cohort of 17,247 patients with pulmonary fibrosis (ICD-10 code J84.

A multiscale simulation framework for the manufacturing facility and supply chain of autologous cell therapies.

Background Aims: Autologous cell therapy (AuCT) is an emerging therapeutic treatment that is undergoing transformation from laboratory- to industry-scale manufacturing with recent regulatory approvals. Various challenges facing the complex AuCT manufacturing and supply chain process hinder the scale out and broader application of this highly potent treatment.

Methods: We present a multiscale logistics simulation framework, AuCT-Sim, that integrates novel supply chain system modeling algorithms, methods, and tools.

Patient journey and treatment patterns in adults with IPF based on health care data in Sweden from 2001 to 2015.

Background: For patients with idiopathic pulmonary fibrosis (IPF), there is limited real-world data on patient journey and treatment patterns.

Aim: To explore predictors of early diagnosis and treatment initiation, and treatment patterns in IPF patients using linked data from Swedish registers and electronic medical records (EMRs).

Population: A national cohort (C1) of 17,247 pulmonary fibrosis patients (ICD-10 code J84.

Epidemiology of heart failure and trends in diagnostic work-up: a retrospective, population-based cohort study in Sweden.

Purpose: The purpose of this study was to examine the trends in heart failure (HF) epidemiology and diagnostic work-up in Sweden.

Methods: Adults with incident HF (≥2 ICD-10 diagnostic codes) were identified from linked national health registers (cohort 1, 2005-2013) and electronic medical records (cohort 2, 2010-2015; primary/secondary care patients from Uppsala and Västerbotten). Trends in annual HF incidence rate and prevalence, risk of all-cause and cardiovascular disease (CVD)-related 1-year mortality and use of diagnostic tests 6 months before and after first HF diagnosis (cohort 2) were assessed.

Healthcare Resource Use, Cost, and Sick Leave Following Kidney Transplantation in Sweden: A Population-Based, 5-Year, Retrospective Study of Outcomes: COIN.

BACKGROUND Improved understanding of the impact of kidney transplantation on healthcare resource use/costs and loss of productivity could aid decision making about funding allocation and resources needed for the treatment of chronic kidney disease in stage 5. MATERIAL AND METHODS This was a retrospective study utilizing data from Swedish national health registers of patients undergoing kidney transplantation. Primary outcomes were renal disease-related healthcare resource utilization and costs during the 5 years after transplantation.

Differences in the utilization of gestational surrogacy between states in the U.S.

Gestational surrogacy policy in the USA varies by state, but information on state differences is lacking. This study used data from the National Assisted Reproductive Technology Surveillance System from 2010 to 2014 to calculate state differences in gestational carrier cycle characteristics. Of the 662,165 in-vitro fertilization cycles in the USA between 2010 and 2014, 16,148 (2.

State performance in pluripotent and adult stem cell research, 2009-2016.

Aim: To examine how the geographic distribution of pluripotent and adult stem cell research publications within the USA differs from other areas of biomedical research.

Materials & Methods: Publication count data for pluripotent stem cell research, adult stem cell research and a comparison group representative of biomedical research more broadly were collected and analyzed for each US state from 2009 to 2016.

Results: The distribution of pluripotent stem cell research differed from the other fields with overperformance in pluripotent stem cell research observed in California, as well as Wisconsin, Massachusetts, Maryland and Connecticut.

Medical societies, patient education initiatives, public debate and marketing of unproven stem cell interventions.

Businesses marketing unproven stem cell interventions proliferate within the U.S. and in the larger global marketplace.

Erratum: Revisiting the Warnock rule.

This corrects the article DOI: 10.1038/nbt.4015.

Assessing the use of assisted reproductive technology in the United States by non-United States residents.

Objective: To study cross-border reproductive care (CBRC) by assessing the frequency and nature of assisted reproductive technology (ART) care that non-U.S. residents receive in the United States.