Leducq Transatlantic Network of Excellence to Cure Phospholamban-Induced Cardiomyopathy (CURE-PLaN).

The deletion of Arginine 14 of the phosholamban gene (PLN p.R14del) is associated with the pathogenesis of an inherited form of cardiomyopathy with prominent arrhythmias. Patients carrying the PLN R14del mutation are at risk of developing dilated cardiomyopathy or arrhythmogenic right ventricular cardiomyopathy. Although the genetic etiology is well defined, the molecular mechanism underlying the pathogenesis of PLN R14del-cardiomyopathy is unknown. Our CURE PLaN network, funded by the Foundation Leducq, will bring together leading scientists, clinicians, and patients to elucidate the genotype-phenotype relationships in R14del cardiomyopathy with the ultimate goal of developing innovative disease-specific therapeutic modalities. With the generous support of the Leducq Foundation, our Transatlantic Network of Excellence consortium to cure Phospholamban (PLN)-induced cardiomyopathy (CURE-PLaN) unites 6 leading centers to address the current challenges associated with arrhythmogenic right ventricular cardiomyopathy/dilated cardiomyopathy (DCM) with an initial focus on PLN and development of effective treatments. The Network is led by Evangelia (Litsa) Kranias (University of Cincinnati) in the United States and Pieter A. Doevendans (Netherlands Heart Institute/UMC Utrecht NL) in Europe. The other US project leaders are Kevin Costa (Cardiovascular Research Center, Icahn School of Medicine at Mount Sinai, New York) and Mark Mercola and Ioannis Karakikes (Stanford University), who are focusing on induced pluripotent stem cell (iPSC)-based disease models, tissue engineering, gene therapy, and drug discovery. On the European side, the project leaders are Despina Sanoudou (Biomedical Research Foundation of the Academy of Athens) analyzing the PLN interactome and Stephan Lehnart (University of Gottingen) addressing the subcellular and disrupted protein interactions affected in PLN-mutant cardiomyocytes. Other key members within the Netherlands Heart Institute are Peter van Tintelen on PLN genetics, Folkert Asselbergs on epigenetics and Rudolf de Boer on clinical trials. We are also privileged to get support from Arthur Wilde (University of Amsterdam), Sakthivel Sadayappan (University of Cincinnati), and Roger Hajjar (Phospholamban Foundation), who have had a long-standing interest in cardiac physiology and pathophysiology with emphasis on underlying pathways and potential therapeutic targets. The consortium is also fortunate to embrace a patient advocate, Pieter Glijnis, incorporating the voice of the patients to research in every step. Our goal is to build and share a platform of patient data coupled with in vitro and in vivo models to promote scientific discovery and advance novel treatments. Phospholamban is a small phosphoprotein in the cardiac sarcoplasmic reticulum, and it is the major regulator of SERCA2a activity and calcium (Ca)-cycling. Chronic inhibition of SERCA2a by PLN has been implicated in the aberrant Ca-cycling of failing hearts. Studies in HF models have shown that decreasing PLN activity may rescue cardiac remodeling and dysfunction. Several human PLN mutations, leading to inhibition of Ca-uptake into the sarcoplasmic reticulum, are linked to inherited DCM.

Foundation-Based Cleft Care in Developing Countries.

BACKGROUND: Cleft deformities of the lip and palate affect nearly one in 500 to 700 births, and lead to increased morbidity and mortality if untreated. Nevertheless, significant global disparities in access to timely and appropriate care still exist. The relatively basic infrastructure required to surgically correct these deformities and large unmet disease burden have resulted in a significant number of foundation-based cleft care initiatives focused on developing countries. In this study, the authors evaluate the peer-reviewed literature generated by these foundations in an attempt to assess their clinical, scientific, educational, and economic impact. METHODS: A comprehensive review of the literature was performed using key search terms, and the level of evidence of identified articles was determined. Data were then analyzed to determine the different models of foundation-based cleft care in developing countries, and their clinical, scientific, educational, and economic impact. RESULTS: A total of 244 articles were identified through the authors' search and reviewed. Foundation-based cleft care initiatives in developing countries have significantly contributed to a better understanding of disease epidemiology, barriers to care, safety considerations, complications and outcomes, and international and local cleft surgery education. The cleft care center model is more cost-effective than the surgical mission model and provides more sustainable care. CONCLUSIONS: Foundation-based cleft care prevents significant morbidity in developing countries and has provided valuable resources for capacity building. The surgical mission model should be considered as a transitory conduit for establishing the more effective and sustainable cleft care center model of care.

Philip Morris International-funded 'Foundation for a Smoke-Free World': analysing its claims of independence.

The Foundation for a Smoke-Free World was launched in September 2017 with an announced 12-year funding commitment of $1 billion from Philip Morris International (PMI). The Foundation claims that its governing documents (certificate of incorporation, bylaws and a pledge agreement) ensure that it has an independent research agenda and stringent protections from conflicts of interest. We analysed the text of these governing documents. Their provisions have multiple loopholes, particularly regarding conflicts of interest. Further, these documents cannot substitute for other important documentation such as information about PMI's internal business case for investing $1 billion in the Foundation, an unwaivable conflict of interest policy, annual disclosure statements, copies of pre-Foundation establishment correspondence between key individuals, all signed contracts or salary information, none of which, as of July 2018, the Foundation has made publicly available. Even if these were released, however, it is problematic that the Foundation's fundamental purpose was decided on and its leader selected following a tobacco company-paid, privately negotiated arrangement with the Foundation's president. It cannot be regarded as independent.

The Effectiveness of an International Cleft Mission Model in Asia: An Update.

BACKGROUND: Cleft lip/palate is a congenital craniofacial anomaly affecting patients physically and psychosocially and has contributed to the global burden of surgical disease, especially in underprivileged areas. For 20 years, Noordhoff Craniofacial Foundation (NCF) and the Chang Gung Craniofacial Center (CGCFC) have carried out missions to these areas. Rather than implementing short-term missions that lack proper follow-up care, the team has provided an effective, long-term, and multidisciplinary approach for the treatment of patients with cleft lip/palate. In this study, we evaluate the sustainability and effectiveness of the cleft mission model implemented by NCF and CGCFC. METHODS: Data from the years 1998-2017 were retrieved from the NCF database. All local centers were evaluated by a 3-stage categorization, levels 1 to 3, based on 4 criteria: (1) capacity to carry out independent missions, (2) diversity of cleft-care professionals, (3) diversity of surgical service offered, and (4) collaboration with local hospitals. Support and training of personnel were provided based on deficiency in these criteria. Noordhoff Craniofacial Foundation made close collaborations and partnerships with several organizations that shared its mission for comprehensive cleft care in developing countries. RESULTS: In all, 19 partner cleft teams in 9 different countries were established. In coordination with these teams, NCF and CGCFC have treated 1846 patients across 78 mission trips. To date, 158 personnel from 19 different countries have been successfully trained to provide cleft care in local centers. Most partner cleft teams centers have progressively reached category level 3, including those in the Philippines, Cambodia, and Mongolia. CONCLUSIONS: In order to establish and maintain sustainable cleft care in developing regions, commitment and compassion toward those who lack essential resources are necessary. Noordhoff Craniofacial Foundation and CGCFC have achieved a successful and practicable model through seeding medical personnel in order to provide effective and sustainable cleft care to the regions in need.

A descriptive study of patients receiving foundational financial assistance through local specialty pharmacies.

OBJECTIVES: To describe the population of patients who received financial assistance from the Good Days Foundation (GDF) as facilitated by Walgreens local specialty pharmacies (LSPs). STUDY DESIGN: This was a retrospective descriptive study. METHODS: This study used a joint foundational and pharmacy claim database between January 1, 2014, and December 31, 2016. RESULTS: Among 1572 eligible patients who received GDF financial assistance as facilitated by Walgreens LSPs, 1524 had disease state information and 14 of these patients receveived financial assistance for 2 disease states (patient count denominator, 1538). The top 3 disease states by patient count were oncology (1403; 91.2%), multiple sclerosis (49; 3.2%), and hepatitis C (39; 2.5%). Of the 777 patients who had complete data and disease state information, 2 received finanical assistance for 2 disease states (denominator, 779); oncology remained the disease with the highest patient count (724; 92.9%). The mean annual financial assistance per patient was highest for hepatitis C ($4156), followed by oncology ($3603) and miscellaneous/rare disease ($1829), which covered 98.8%, 99.3%, and 99.6% of these patients' total co-pay requirements, respectively. In addition to prescription co-pay assistance, 21 patients received travel assistance of $554 per year per patient from GDF. The mean persistence of oncology patients was 170.7 days without a 30-day gap over 1 year of observation time. CONCLUSIONS: The facilitation of treatment by GDF and Walgreens LSPs may be the key to many patients receiving their treatment and maintaining medication persistence. GDF co-pay assistance helped cover most out-of-pocket costs associated with medications and aided with travel expenses for patients, especially in the area of oncology. For many patients, this meant reducing the significant financial barriers to accessing care and facilitating the necessary treatment for their chronic or life-altering disease. Without this assistance, many patients would simply not have been able to meet the expected medication persistence and thus would have received suboptimal treatment.

The atlas network: a "strategic ally" of the tobacco industry.

Amid growing academic and policy interest in the influence of think tanks in public policy processes, this article demonstrates the extent of tobacco industry partnerships with think tanks in the USA, and analyzes how collaborating with a network of think tanks facilitated tobacco industry influence in public health policy. Through analysis of documents from tobacco companies and think tanks, we demonstrate that the Atlas Economic Research Foundation, a network of 449 free market think tanks, acted as a strategic ally to the tobacco industry throughout the 1990s. Atlas headquarters, while receiving donations from the industry, also channeled funding from tobacco corporations to think tank actors to produce publications supportive of industry positions. Thirty-seven per cent of Atlas partner think tanks in the USA received funding from the tobacco industry; the majority of which were also listed as collaborators on public relations strategies or as allies in countering tobacco control efforts. By funding multiple think tanks, within a shared network, the industry was able to generate a conversation among independent policy experts, which reflected its position in tobacco control debates. This demonstrates a coherent strategy by the tobacco industry to work with Atlas to influence public health policies from multiple directions. There is a need for critical analysis of the influence of think tanks in tobacco control and other health policy sectors, as well as greater transparency of their funding and other links to vested interests. © 2016 The Authors The International Journal of Health Planning and Management Published by John Wiley & Sons Ltd.

[From the national competence network for viral hepatitis (HepNet) emerged the German Liver Foundation (Deutsche Leberstiftung)]. / Vom Kompetenznetz Hepatitis (HepNet) zur Deutschen Leberstiftung.

The competence network for viral hepatitis (HepNet) was founded in 2002 with funding from the German government and has influenced the research on viral hepatitis in Germany. HepNet collaborator sites have been involved in numerous national and international investigator-initiated, as well as industry-sponsored, phase 1-3 studies. Within the HepNet Study-House, many groundbreaking investor-initiated trials have been completed and are still ongoing. For example, the acute hepatitis C trials and trials on chronic hepatitis D (delta), which led to therapy optimization. Continuation of the competence network on viral hepatitis has been achieved by the foundation of the German Liver Foundation, which has been an external cooperation partner of the German Center for Infection Research (DZIF) for two years. The well-established HepNet Study-House acts here as the clinical trial platform for all DZIF hepatitis trials.

MesobanK UK: an international mesothelioma bioresource.

Malignant pleural mesothelioma causes the greatest societal burden of all the asbestos-related diseases. Progress in better understanding tumour biology will be facilitated by the availability of quality-assured annotated tissue. MesobanK has been created to establish a bioresource of pleural mesothelioma tissue linked to detailed anonymised clinical data. When complete, the bioresource will comprise a 750-patient tissue microarray and prospectively collected tissue, blood and pleural fluid from 300 patients with mesothelioma. Twenty-six new cell lines have also been developed. MesobanK meets all appropriate ethical and regulatory procedures and has recently opened to requests for tissue and data.