Data-Gouv-ML/pcri-informations-sur-les-projets-laureats huggingface dataset:
Towards clinical translation of new gene targeting technologies for correcting inherited mutations and empowering adoptive immunotherapy of cancer (SUPERSIST)

Framework: FP7 — Cooperation
Programme: Health
Destination: Horizontal topics for collaborative projects relevant for the whole of theme health
Action type: Collaborative project
Countries involved: Italy,Netherlands,Germany,United Kingdom
Call:  (FP7-HEALTH-2013-INNOVATION-2, 2013)
Start date: 2013-05-01
End date: 2016-10-31
Duration (months): 42.0
Total project cost (EUR): 7892920.36
EU funding (EUR): 5999997.0
Participants: 5
Project status: CLOSED

Substantial evidence supports the therapeutic potential of ex vivo gene therapy based on Hematopoietic Stem Cell (HSC) or T lymphocytes to treat inherited diseases or cancer. Yet, the intrinsic limitations of current gene replacement approaches based on semi-randomly integrating vectors, and the occurrence in some clinical trials of severe adverse events related to gene transfer, prevent safe deployment and broad application of gene therapy. This project aims to overcome these limits by exploiting the results of an earlier highly successful integrated project (PERSIST), which demonstrated the potential of gene targeting technologies based on engineered nucleases to provide radical new solutions to these hurdles. By homology-driven repair of a nuclease-targeted dysfunctional gene, we will insert a functional copy downstream its own endogenous promoter in HSC, thus restoring both function and physiological expression control. For adoptive T-cell therapy, we will combine nuclease-mediated disruption of the endogenous TCR genes with transfer of tumor-specific TCR, thus editing T-cell specificity at the genetic level. We will develop these innovative approaches into robust, scalable and clinically ready processes for safe ex vivo genetic modification of hematopoietic cells, and apply them to the treatment of paradigmatic diseases that provide a favourable risk-benefit ratio for clinical testing. Because both HSC and T-cell based gene therapy strategies share methodological aspects and technological challenges, we will address them systematically by involving a SME which holds unique know-how and top-level expertise in vector manufacturing and HSC and T-cell processing, as already established for our previous and ongoing gene therapy trials. Our findings will lead to the design of new clinical trials to provide durable benefits or even a cure to patients suffering from severe and otherwise fatal diseases, and representing a template for broaden application in medicine.

Keywords: new therapies (gene therapy,cell therapy,stem cells,new technologies for gene targeting) cancer immunotherapy pre-clinical/clinical development,gmp manufacturing genetic disease and leukemia
Human cystic Echinococcosis ReseArch in CentraL and Eastern Societies (HERACLES)

Framework: FP7 — Cooperation
Programme: Health
Destination: Translating research for human health
Action type: Collaborative project
Countries involved: Italy,Italy,Spain,Türkiye,Romania,Bulgaria,Spain
Call:  (FP7-HEALTH-2013-INNOVATION-1, 2013)
Start date: 2013-10-01
End date: 2018-09-30
Duration (months): 60.0
Total project cost (EUR): 3922487.76
EU funding (EUR): 2861500.0
Participants: 10
Project status: CLOSED

Human cystic echinococcosis (CE), one of the most widespread helminthic zoonoses, is a chronic disease caused by infection with the larval stage of the tapeworm Echinococcus granulosus. This disease is highly endemic in some southern (Spain and Italy), eastern (Bulgaria and Romania) European countries and associated (Turkey) countries, where it still represents a major health and economic problem. In endemic areas the annual incidence of CE ranges from 1 to 200 per 100,000 inhabitants and 2-3 million cases are globally estimated. Case series and small clinical trials show a mortality rate of 2-4% for CE, but this increases markedly with substandard treatment and care. The diagnosis of human CE is mainly based on imaging techniques and serological confirmation. Clinical decision making is difficult and the natural history of the cyst is still poorly known. CE is chronic, complex and still neglected. For these reasons the main objectives of HERACLES are: • Identify by ultrasound screening the population affected by CE in endemic rural areas of CEE countries; • Create CEE national registries for surveillance of CE; • Establish a representative bio-bank of genetic Egc isolates and blood/serum/plasma samples; • Validate new molecular-based POC-LOC kits for immunological surveillance, diagnosis and follow-up; • Identify factor/s associated with CE response to therapy or lack thereof through investigation of host-parasite interplay (parasite virulence vs human immunity); • Increase drug bioavailability in an in vivo model, decreasing the length of antiparasitic (ABZ based) treatment of CE and synthesize a new enantiomeric drug based on ABZ. The project stakeholders, especially the rural populations in which CE is endemic, as well as SMEs, will be engaged as an integral part of the project. Special events to increase general awareness of CE and training activities for local health care providers will be organized.

Keywords: echinococcus granulosus,cystic echinococcosis,ultrasound screening,genomics,treatment efficacy,diagnostic and follow up tools.
Collaborative European NeuroTrauma Effectiveness Research in TBI (CENTER-TBI)

Framework: FP7 — Cooperation
Programme: Health
Destination: Translating research for human health
Action type: Collaborative project
Countries involved: Belgium,United Kingdom,Sweden,Netherlands,Germany,Slovakia,Hungary,Finland,Belgium,France,United Kingdom,United Kingdom,Germany,United Kingdom,Belgium,Germany,Germany,United Kingdom,Denmark,Norway,United Kingdom,United Kingdom,Italy,United States,Australia,New Zealand,China,Germany,Germany,United Kingdom,Austria
Call:  (FP7-HEALTH-2013-INNOVATION-1, 2013)
Start date: 2013-10-01
End date: 2020-03-31
Duration (months): 78.0
Total project cost (EUR): 39467963.09
EU funding (EUR): 29998903.0
Participants: 43
Project status: CLOSED

Traumatic Brain Injury (TBI) is a major cause of death and disability, leading to great personal suffering to victim and relatives, as well as huge direct and indirect costs to society. Strong ethical, medical, social and health economic reasons therefore exist for improving treatment. The CENTER-TBI project will collect a prospective, contemporary, highly granular, observational dataset of 5400 patients, which will be used for better characterization of TBI and for Comparative Effectiveness Research (CER). The generalisability of our results will be reinforced by a contemporaneous registry level data collection in 15-25,000 patients. Our conceptual approach is to exploit the heterogeneity in biology, care, and outcome of TBI, to discover novel pathophysiology, refine disease characterization, and identify effective clinical interventions. Key elements are the use of emerging technologies (biomarkers, genomics and advanced MR imaging) in large numbers of patients, across the entire course of TBI (from injury to late outcome) and across all severities of injury (mild to severe). Improved characterization with these tools will aid Precision Medicine, a concept recently advocated by the US National Academy of Science, facilitating targeted management for individual patients. Our consortium includes leading experts and will bring outstanding biostatistical and neuroinformatics expertise to the project. Collaborations with external partners, other FP7 consortia, and international links within InTBIR, will greatly augment scientific resources and broaden the global scope of our research. We anticipate that the project could revolutionize our view of TBI, leading to more effective and efficient therapy, thus improving outcome and reducing costs. These outcomes reflect the goals of CER to assist consumers, clinicians, health care purchasers, and policy makers to make informed decisions, and will improve healthcare at both individual and population levels.

Keywords: traumatic brain injury,comparative effectiveness research,precision medicine,pathophysiology,healthcare,health utility,outcome,guidelines,quality assessment,genomics,biomarkers,neuroimaging,prognosis
ATMP GMP Open Access Research Alliance - AGORA (AGORA)

Framework: FP7 — Cooperation
Programme: Health
Destination: Other Actions across the Health Theme
Action type: Coordination and support actions
Countries involved: United Kingdom,Germany,Netherlands,Germany,United Kingdom,Germany,Netherlands,Netherlands
Call:  (FP7-HEALTH-2013-INNOVATION-1, 2013)
Start date: 2013-09-01
End date: 2015-10-31
Duration (months): 26.0
Total project cost (EUR): 554776.05
EU funding (EUR): 497432.0
Participants: 8
Project status: CLOSED

We propose an academic & industrial partnership to facilitate safe and effective delivery of new advanced therapy medicines within the framework of the relevant EU regulations which our previous FP7 funded project have identified as creating unanticipated barriers to development of this field. Over 80% of advanced therapy medicines (ATMPs) trials in the EU are academic investigator led and sponsored. Many ATMPs are not making it to first-in-man trials due to the difficulties of academic manufacture to GMP compliance and the variation of application of the Directives between EU MS. We will deliver a supporting programme of resources to academic trialists to streamline their development programmes, enhance compliance with GMP requirements, develop industrial partnerships for improved manufacture and scale-up and establish the first university certified training programmes in cell/tissue engineering to address the critical shortage in scientific/technical/regulatory staff in this newly expanding field across the EU. The future supply of successful ATMPs to EU citizens will require partnerships between academic GMP facilities, contract ATMP manufacturers (CMOs) and the biopharma industry and this project will deliver the necessary support and training structure to create this partnership. The need for this project has been identified from the impact assessment part of our current FP7 project, “Academic GMP” and the culmination of the project will be a scientific analysis of the effect of the programme on the impact of the legislation relevant to ATMP development and provision across the EU.

Keywords: atmp gmp clinical trials commercial supply partnership
Credits-based, people-centric approach for the adoption of healthy life-styles and balanced Mediterranean diet in the frame of social participation and innovation for health promotion. (CREDITS4HEALTH)

Framework: FP7 — Cooperation
Programme: Health
Destination: Optimising the delivery of healthcare to European citizens
Action type: Collaborative project
Countries involved: Italy,Spain,Germany,Greece,Germany,Germany,Italy,United Kingdom
Call:  (FP7-HEALTH-2013-INNOVATION-1, 2013)
Start date: 2013-09-01
End date: 2016-08-31
Duration (months): 36.0
Total project cost (EUR): 7597317.0
EU funding (EUR): 5996863.0
Participants: 16
Project status: CLOSED

The CREDITS4HEALTH project’s main goal is to develop a person centric approach based on the “credits for health” concept to reduce sedentary behavior and enhance the level of physical activity and healthy dietary styles in people living in Euro-Mediterranean Countries. The concept of credits is simple and effective: this means that each participant will earn credits from his local, regional or national Health Authority on the basis of his/her participation and involvement in the achievement of an healthy life-style, and his/her attitude to sponsor this philosophy in his community. The CREDITS4HEALTH vision is therefore to have people directly acting for their health and well being, operating on three fundamental levers to enhance the quality of their lives, through the reduction of sedentary behavior, the active participation to the social life and the adoption of an healthier diet. The CREDITS4HEALTH approach is virtuous and straightforward: 1)First, we will define personalised algorithms containing dietary and physical activity prescriptions, taking into account the medical, psychological, social, and economic background of each person; 2) Once screened by the local Credits4Health Committee, the participant receives an electronic card, which enables him to track his compliance to the regime through a dedicated web based platform; 3)With his electronic card, the participant also gets access to a full range of suppliers which offer him goods for fulfilling his health-related objectives;4)Suppliers must be certified by the local authority, according to a specified set of requirements and they must certify the products they intend to commercialise for this initiative;5)Participants gain credits as far as they comply with their daily, weekly and monthly goals; they are also subject to a six monthly mandatory assessment performed by the Credits4Health Committee, in which the algorithm can be modified and fine-tuned to the needs of the participant.

Keywords: social innovation,active life-styles,people empowerment,stakeholder approach
Optimizing Exercise Training in Prevention and Treatment of Diastolic Heart Failure (OPTIMEX)

Framework: FP7 — Cooperation
Programme: Health
Destination: Translating research for human health
Action type: Collaborative project
Countries involved: Norway,Germany,Germany,Belgium,Austria,Germany
Call:  (FP7-HEALTH-2013-INNOVATION-1, 2013)
Start date: 2013-10-01
End date: 2017-09-30
Duration (months): 48.0
Total project cost (EUR): 3848561.17
EU funding (EUR): 2999995.7
Participants: 7
Project status: CLOSED

More than 14 million Europeans suffer from heart failure (HF), of which more than 50 % have HF with preserved left ventricular (LV) ejection fraction (EF) (HFPEF, “diastolic heart failure”). HFPEF is the only cardiovascular disease with increasing prevalence and incidence, affecting 10-20% of the elderly and contributing substantially to hospitalizations of elderly HF patients. Currently, no medical treatment has been shown to be effective and the economic, social and personal burden of HFPEF is enormous; this disease constitutes one of the most pressing unmet clinical needs. A cardinal feature of HFPEF is exercise intolerance. The pathophysiology of exercise intolerance in HFPEF depends on multiple factors in heart, endothelium and skeletal muscles. From a pathophysiological point of view, exercise could by far outweigh any pharmacological intervention in this heterogeneous syndrome, since lifestyle dependent risk factor, physical inactivity, and physical deconditioning underlay and contribute to HFPEF. OptimEx will focus on the cardiovascular effects of exercise training as primary and secondary prevention of HFPEF. We will combine in vivo and in vitro studies in man and rats in serial experiments that will advance our understanding of fundamental cellular and molecular mechanisms underpinning dose-dependent exercise-induced changes in the heart, blood vessels and skeletal muscles. This research is aimed to tackle one of the major health problems the developed world faces with its ageing societies and increasing prevalence of the HFPEF and will support sustainable health systems in EU member states through improvements in the clinical management of a common and disabling disease. The project is therefore highly relevant to improve the health of European citizens and important to promote healthy ageing and preventing disease.

Keywords: exercise is medicine,aerobic interval training,cardiovascular health
A Gateway to European Kidney Research Resources - KidneyConnect (KIDNEYCONNECT)

Framework: FP7 — Cooperation
Programme: Health
Destination: Other Actions across the Health Theme
Action type: Coordination and support actions
Countries involved: Ireland,Italy,Austria,Germany,Finland,Germany,Greece,Spain,United Kingdom,Italy,Sweden,France,United Kingdom,Switzerland
Call:  (FP7-HEALTH-2013-INNOVATION-1, 2013)
Start date: 2013-08-01
End date: 2016-07-31
Duration (months): 36.0
Total project cost (EUR): 554662.39
EU funding (EUR): 499978.0
Participants: 18
Project status: CLOSED

Diabetes, Cardiovascular and Chronic kidney (DCC) -diseases are relentlessly increasing globally, causing enormous human suffering, premature deaths and unsustainable costs. Leading European research has indisputably pointed that the kidney filtration barrier and its epithelial cell, the podocyte, is a common denominator for the DCC-diseases. However, European excellence and expertise have remained uncoordinated in separate pockets and, consequently, underutilised for full societal benefits and capacity creation to combat the challenges of diabetic, hypertensive and primary kidney diseases. Notably, these diseases are of major healthcare interest and of key importance for discovery intensive biopharma industry. KidneyConnect brings together teams of excellence to underpin nationally funded programs under a) Discovery and Future Technologies b) New Research Platforms c) Translational and d) Clinical Podocyte Research to create connected capacities, access to well trained talents and to optimize strategies for industry-academia cowork. In addition to resource maps, KidneyConnect supports international congresses, training courses, talent coaching, special seminars and builds systematically relations to key stakeholders. Due to the limited funds available, main aims are to provide roadmaps for future efforts, outlines for shared data -and sample repositories, targeted training, societal outreach and, as a result, competitive European funded programs. Our events are arranged as satellites of established meetings and supported by in-kind contribution from partners. The goal is to establish faster translation from discovery to clinical practices by creating dynamic networks, sustainable capacities and outlines for improved kidney disease management. High cohesion and shared resources together with the most prominent European authorities included will guarantee optimized resource usage. Substantial benefits and competitiveness in the huge global markets are to be expected

Keywords: diabetes,kidney,translation,clinical,training,patients,industry,startup,road maps
Biomarker Development for Postoperative Cognitive Impairment in the Elderly (BIOCOG)

Framework: FP7 — Cooperation
Programme: Health
Destination: Translating research for human health
Action type: Collaborative project
Countries involved: Germany,Netherlands,United Kingdom,Italy,Luxembourg,Germany,Germany,Germany,Germany
Call:  (FP7-HEALTH-2013-INNOVATION-1, 2013)
Start date: 2014-02-01
End date: 2019-01-31
Duration (months): 60.0
Total project cost (EUR): 7793733.05
EU funding (EUR): 5982400.0
Participants: 12
Project status: CLOSED

Postoperative delirium (POD) is characterized by the progressive deterioration of sensory/cognitive function after surgery with incidences of up to 30-80%. It is frequently followed by postoperative cognitive dysfunction (POCD) which tends to persist over time. In elderly patients, POCD resembles chronic dementia and appears to accelerate the cognitive decline in Alzheimer dementia. POD is strongly associated with subsequent dementia after 3.2 and 5.0 years of follow-up: odds ratio = 12.52 [95% CI, 1.86-84.21] corrected for baseline dementia, severity of illness, age. In an aging society like the EU, the socioeconomic implications of POD/POCD are therefore profound. At present no treatment exists and there are no established molecular or imaging biomarkers that allow risk and clinical outcome prediction. We will establish valid biomarkers panels for risk and clinical outcome prediction of POD/POCD in N=1200 surgical patients according to the regulatory requirements of the European Medicines Agency. Thus, a valuable database will be created not yet existing worldwide. Neuroimaging investigations, which directly provide information on brain structure/function, will include structural magnetic resonance imaging (MRI), diffusion tensor imaging (DTI), arterial spin labelling (ASL), functional magnetic resonance imaging with simultaneous electrophysiology (fMRI/EEG) and glutamate MR-spectroscopy (MRS). These investigations will be complemented by genetic/gene expression analyses (sequencing of cholinergic candidate genes/corresponding transcripts) and analyses of plasma and cerebrospinal fluid markers (inflammatory/metabolic). Supported by bioinformatics approaches, integration of neuroimaging data with knowledge from molecular biomarkers (multivariate expert system) is expected to allow patient stratification. This will greatly support decision-making before surgical intervention (balancing benefits and risks) as well as the development of novel therapies in POD/POCD.

Keywords: postoperative cognitive dysfunction,neuroimaging/molecular biomarker
Integrated DEsign and AnaLysis of small population group trials (IDEAL)

Framework: FP7 — Cooperation
Programme: Health
Destination: Other Actions across the Health Theme
Action type: Collaborative project
Countries involved: Germany,Germany,Austria,France,Luxembourg,Sweden,Poland,Sweden,Belgium
Call:  (FP7-HEALTH-2013-INNOVATION-1, 2013)
Start date: 2013-11-01
End date: 2017-04-30
Duration (months): 42.0
Total project cost (EUR): 3648320.2
EU funding (EUR): 2958449.0
Participants: 10
Project status: CLOSED

There exist more than 7000 rare diseases worldwide and the European Society of Paediatric Oncology stated that 75% of rare diseases affect children and 30% of rare disease patients die before the age of five. Usual statistical methods for proving efficacy and safety of therapies fail to provide cost-efficient and reliable results in small populations. There is a pressing need to integrate a broad range of innovative methodologies improving clinical trials in the setting of small sample population groups (SPG). The objective of this research is to produce methods of general applicability irrespective of indication by Integrated DEsign and AnaLysis of clinical trials in SPG (IDEAL) through a multidisciplinary closely collaborating consortium of researchers from European universities, research institutes and industry. The consortium will work in 10 WPs, focussed on assessment of randomization procedures, extrapolating dose-response information, investigation of adaptive designs, optimal designs in mixed models, pharmacogenetic designs, simulation of clinical trials, genetic factors influencing the response, decision analysis and biomarker surrogate endpoints as well as WPs on project management and dissemination of results. Relevant stakeholder concerns (patient needs, regulatory issues, reimbursement, clinical feasibility) will be monitored by a Clinical Scientific Advisory Board. Because of its integrative structure, this research program extends previous approaches, which focus on a certain methodology only. In its totality, the WPs constitute a logically coherent set of methodologies that is of sufficient breadth to tackle these important, multidisciplinary challenges. By combining, enhancing and developing different statistical methodologies and assessment methods, this research program will impact the scientific discussion in promoting efficient statistical methodology for clinical trials in SPG, also in view of existing regulatory guidance in the EU.

Keywords: biostatistics,clinical trial,design of experiments,pharmacogenetics